[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"countryName\":\"Romania\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":697},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,443,0,25,[9,51,89,119,144,171,199,230,258,279,303,330,353,375,400,424,451,472,499,527,547,589,616,643,665],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":16,"eligibilityCriteria":17,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":20,"enrollmentInfo":21,"targetDuration":24,"studyType":25,"phases":4,"briefSummary":26,"conditions":27,"keywords":31,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":39,"lastUpdatePostDateStruct":40,"startDateStruct":43,"completionDateStruct":45,"leadSponsor":47,"locationsCount":50},"100627153","validation-of-sudden-cardiac-arrest-risk-factors-in-patients-with-cad-100627153",false,"NCT07444931","Validation of Sudden Cardiac Arrest Risk Factors in Patients With CAD","Validation of Sudden Cardiac Arrest Risk Factors in Patients With CAD - CVDLINK Clinical Validation Study.","SCAR","Inclusion Criteria:\n\n* Age ≥ 18 years old\n* Ability to give informed consent\n* Imaging confirmed diagnosis within the past 12 months (coronary artery angiography or contrast enhanced coronary computed tomography)\n\n  •\\>50% of stenosis and\u002For fractional flow-reserve \\\u003C0.8 verified by selective coronary angiography or by contrast enhanced coronary computed tomography (CT) or type I MI with atherosclerotic origin (despite stenosis percentage)\n* Patients may be recruited during index event visit (out-patient clinic visit, invasive procedure, hospitalization) (or if logistically possible recall patients previously diagnosed within 12 months)\n\nExclusion Criteria:\n\n* Age \\> 75 years of age\n* Clinically significant previously treated valvular heart disease or requiring operative (surgical or endovascular) treatment within following three months\n* Diagnosed severe neurodegenerative disease (ALS, myositis, multiple sclerosis, or Parkinson's disease) or known impaired cognitive function (MMSE \\\u003C23)\n* Known developmental disability impairing legal ability to give written consent\n* Serious\u002Factive malignancy with possibly reduced life expectancy of \\\u003C1 year (estimated by a physician)\n* Inability to give written consent for some other reason\n* Other significant cardiac condition severely linked to the risk of fatal ventricular arrhythmia (for example ARVCD, non-ischemic DCM, HCM or genetic long or short QT syndrome)\n* Other cardiac disease with \\\u003C1 years of life expectancy\n* Do-Not-Resuscitate (DNR) order made due to any reason\n* Previously done or planned cardiac, renal, or liver transplant\n* Participation in another clinical trial where the active treatment","ALL","18 Years","75 Years",{"count":22,"type":23},1500,"ESTIMATED","1 Year","OBSERVATIONAL","Long-term sudden cardiac death (abbreviation: SCAR) focuses on improving the predictability of sudden cardiac death (SCD) in patients diagnosed with coronary artery disease. The aim of the study is to determine the predictive value of measurable biological variables (including genetic factors, cardiac electrical activity, biological markers measured from circulation, and coronary artery anatomy) as well as the patients' psychosocial factors in predicting SCDs.\n\nThe purpose of this study is the identification of a subgroup of coronary artery disease patients at sufficiently high risk in whom it may be possible to prevent sudden cardiac arrests and subsequent deaths using implantable cardioverter-defibrillators. The study is intended to establish a clear foundation for future interventional studies targeting high-risk coronary artery disease patients.\n\nThe primary endpoint of the study is SCD\u002Fsudden cardiac arrest (SCA) or a comparable malignant arrhythmic event (i.e., resuscitation). Secondary endpoints include other major cardiovascular events occurring during the follow-up period (such as cerebrovascular events, myocardial infarctions, revascularizations, and new arrhythmias like atrial fibrillation following procedures or after the patient has been discharged following recruitment) or the occurrence and mortality of other significant life-threatening diseases (such as cancer). Secondary endpoints also include poor success in secondary prevention, which can be assessed through completed medication purchases and the achievement of secondary prevention goals.\n\nThis observational, prospective study includes collecting multimodal data from hospitals in Finland (TAUH), Israel (HYMC), Moldova (IMSP) and Romania (UMFCD). Each participating institution has followed a process structured by Tampere Heart Hospital (TAUH) for securing permissions in line with EU and national regulations.",[28,29,30],"Coronary Arterial Disease (CAD)","Sudden Cardiac Arrest","Sudden Cardiac Death",[32,33,34,35,36,37],"Coronary artery disease","Sudden cardiac arrest","Sudden cardiac death","post operative atrial fibrillation","genetics","risk factors","RECRUITING","2026-08-24",{"date":41,"type":42},"2026-08-25","ACTUAL",{"date":44,"type":42},"2025-01-05",{"date":46,"type":23},"2027-12-31",{"name":48,"class":49},"Tampere Heart Hospital","OTHER",3,{"id":52,"slug":53,"hasResults":12,"nctId":54,"briefTitle":55,"officialTitle":56,"acronym":4,"eligibilityCriteria":57,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":58,"targetDuration":4,"studyType":60,"phases":61,"briefSummary":64,"conditions":65,"keywords":67,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":39,"lastUpdatePostDateStruct":80,"startDateStruct":81,"completionDateStruct":83,"leadSponsor":85,"locationsCount":88},"100600637","phase-2-study-of-izalontamab-brengitecan-bms-986507-versus-platinum-pemetrexed-for-egfr-mutated-non-small-cell-lung-cancer-after-failure-of-egfr-tki-therapy-izabright-lung01-100600637","NCT07100080","Study of Izalontamab Brengitecan (BMS-986507) Versus Platinum-Pemetrexed for EGFR-mutated Non-small Cell Lung Cancer After Failure of EGFR TKI Therapy (IZABRIGHT-Lung01)","IZABRIGHT-Lung01: A Randomized, Open-label, Phase 2\u002F3 Study of Izalontamab Brengitecan (BMS-986507) Versus Platinum-based Chemotherapy in Patients With EGFR-mutated Non-small Cell Lung Cancer and Disease Progression on EGFR Tyrosine Kinase Inhibitor Therapy","Inclusion Criteria:\n\n* Non-squamous NSCLC, not amenable to treatment in curative intent.\n* Documented evidence of EGFR mutation (exon 19 deletion, L858R mutation).\n* Progressive disease on a 3rd-generation (such as osimertinib, furmonertinib, lazertinib,...) EGFR-TKI-based mono- or combination therapy regimen as the most recent line of therapy in an adjuvant, locally advanced, or metastatic treatment setting.\n* Eligible to receive a platinum-based doublet chemotherapy regimen (either cisplatin or carboplatin in combination with pemetrexed).\n\nExclusion criteria:\n\n* Inadequate organ function and\u002For bone marrow reserve.\n* Leptomeningeal metastases or spinal cord compression.\n* Poorly controlled systemic medical conditions.\n* Other protocol-defined inclusion\u002Fexclusion criteria apply.",{"count":59,"type":23},500,"INTERVENTIONAL",[62,63],"PHASE2","PHASE3","A Study of Izalontamab Brengitecan (BMS-986507) versus Platinum-Pemetrexed for EGFR-mutated Non-small Cell Lung Cancer after failure of EGFR TKI Therapy",[66],"Non-Small Cell Lung Cancer",[68,69,70,71,72,73,74,75,76,77,78,79],"Epidermal Growth Factor Receptor","Tyrosine Kinase Inhibitors","Osimertinib","Standard of Care","Lung Neoplasms","Antineoplastic Agents","Izalontamab brengitecan","Iza-bren","BL-B01D1","Carboplatin","Cisplatin","Pemetrexed",{"date":41,"type":42},{"date":82,"type":42},"2025-11-05",{"date":84,"type":23},"2030-11-15",{"name":86,"class":87},"Bristol-Myers Squibb","INDUSTRY",186,{"id":90,"slug":91,"hasResults":12,"nctId":92,"briefTitle":93,"officialTitle":94,"acronym":95,"eligibilityCriteria":96,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":97,"targetDuration":4,"studyType":60,"phases":99,"briefSummary":100,"conditions":101,"keywords":103,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":39,"lastUpdatePostDateStruct":112,"startDateStruct":113,"completionDateStruct":115,"leadSponsor":117,"locationsCount":118},"100597842","a-study-to-compare-the-combination-of-navlimetostat-bms-986504-with-pembrolizumab-and-chemotherapy-versus-placebo-plus-pembrolizumab-and-chemotherapy-in-first-line-metastatic-non-small-cell-lung-cancer-participants-with-homozygous-mtap-deletion-100597842","NCT07063745","A Study to Compare the Combination of Navlimetostat (BMS-986504) With Pembrolizumab and Chemotherapy Versus Placebo Plus Pembrolizumab and Chemotherapy in First-line Metastatic Non-small Cell Lung Cancer Participants With Homozygous MTAP Deletion","A Randomized Phase 2\u002F3 Study of Navlimetostat (BMS-986504) in Combination With Pembrolizumab and Chemotherapy Versus Placebo Plus Pembrolizumab and Chemotherapy in First-line Metastatic Non-small Cell Lung Cancer Participants With Homozygous MTAP Deletion","MountainTAP-29","Inclusion Criteria\n\n* Participants must have Metastatic (Stage IV or recurrent) non-small cell lung cancer (NSCLC) (as defined by the American Joint Committee on Cancer, Ninth Edition) with no prior systemic anti-cancer therapy for metastatic disease.\n* Participants must have histologically confirmed diagnosis of NSCLC and homozygous methylthioadenosine phosphorylase (MTAP) deletion or MTAP loss.\n* Participants must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0-1.\n* Participants must have at least 1 measurable lesion as per RECIST v1.1.\n\nExclusion Criteria\n\n* Nonsquamous participants must not have documented targetable oncogenic mutation or actionable genetic alterations (AGAs) for which there is a standard of care (SoC) available as first-line (1L) therapy.\n* Participants must not have symptomatic brain metastases or spinal cord compression.\n* Participants must not have any prior systemic therapy (chemotherapy, immunotherapy, targeted therapy, or biological therapy) for metastatic non-small cell lung cancer (mNSCLC).\n\nNote: One cycle of SoC treatment prior to randomization will be allowed for participants who require immediate treatment if clinically indicated.\n\n* Participants must not have any known or suspected impairment of gastrointestinal function that may prohibit the ability to absorb or swallow an oral medication without chewing or crushing.\n* Other protocol-defined Inclusion\u002FExclusion criteria apply.",{"count":98,"type":23},590,[62,63],"The purpose of this study is to compare the clinical benefit of the combination of Navlimetostat (BMS-986504) (a selective MTA-cooperative inhibitor of PRMT5) plus pembrolizumab and chemotherapy versus placebo plus pembrolizumab and chemotherapy in first-line metastatic non-small cell lung cancer participants with homozygous MTAP deletion",[102],"Metastatic Non-small Cell Lung Cancer With MTAP Deletion",[104,105,106,107,108,109,110,111],"PRMT5","Lung cancer","NSCLC","MTAP","CDKN2A","MRTX1719","First-line","Navlimetostat",{"date":41,"type":42},{"date":114,"type":42},"2026-01-02",{"date":116,"type":23},"2031-08-12",{"name":86,"class":87},320,{"id":120,"slug":121,"hasResults":12,"nctId":122,"briefTitle":123,"officialTitle":124,"acronym":4,"eligibilityCriteria":125,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":126,"enrollmentInfo":127,"targetDuration":4,"studyType":60,"phases":129,"briefSummary":130,"conditions":131,"keywords":133,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":39,"lastUpdatePostDateStruct":137,"startDateStruct":138,"completionDateStruct":140,"leadSponsor":142,"locationsCount":143},"100587506","phase-3-a-study-to-assess-the-long-term-safety-of-karxt-for-the-treatment-of-manic-episodes-in-bipolar-i-disorder-balsam-3-100587506","NCT06929273","A Study to Assess the Long-term Safety of KarXT for the Treatment of Manic Episodes in Bipolar-I Disorder (BALSAM-3)","A Phase 3, Open-label Extension Study to Assess the Long-term Safety of KarXT for the Treatment of Mania or Mania With Mixed Features in Bipolar-I Disorder (BALSAM-3)","Inclusion Criteria:\n\n* Participants who participated in double-blind placebo-controlled study (CN0120036, CN0120037, or CN0120046):\n\n  a. Participants must have completed treatment period of parent study.\n* De novo participants who did not participate in double-blind placebo-controlled studies:\n\n  1. Participants must have primary diagnosis of Bipolar-I disorder established by a comprehensive psychiatric evaluation based on DSM-5-TR criteria and confirmed by the Mini International Neuropsychiatric Interview (MINI, v7.0.2), with symptoms of mania or mixed mania.\n  2. Participants must have Young Mania Rating Scale (YMRS) score of ≥ 14 at Screening and at baseline.\n  3. Participants must have CGI-BP score of ≥ 3 at Screening and at baseline.\n  4. Participants does not require hospitalization for acute mania.\n\nExclusion Criteria:\n\n* All participants:\n\n  1\\. All participants with a risk for suicidal behavior at baseline as determined by Investigator's clinical assessment or history of suicidal behavior as assessed on C-SSRS.\n* Participants who participated in double-blind placebo-controlled study (CN0120036, CN0120037, or CN0120046):\n\n  1\\. Discontinuation from any KarXT parent studies.\n* De novo participants who did not participate in double-blind placebo-controlled studies:\n\n  1. Participants must not have primary diagnosis of BP-I with rapid cycling (ie, ≥ 4 distinct mood episodes in one year).\n  2. Participants must not have any primary DSM-5-TR disorder other than BP-I with mania or mania with mixed features within 12 months before Screening (confirmed using MINI version 7.0.2 at Screening), including BP-I with depression, (previous 3 months only), Bipolar-II disorder, major depressive disorder, borderline personality disorder, and primary psychotic disorder, with the exception of mild anxiety disorders.\n  3. Individual has a DSM-5-TR diagnosis of moderate to severe substance use disorder (except tobacco use disorder) within the 12 months before Screening (confirmed using MINI version 7.0.2 at Screening), or current use as determined by urine toxicology screen or alcohol test.\n  4. Participants must not have history of irritable bowel syndrome (with or without constipation) or serious constipation requiring treatment within the last 6 months.\n  5. Participants must not have history or high risk of urinary retention, gastric retention, or untreated narrow-angle glaucoma.\n* Other protocol-defined Inclusion\u002FExclusion criteria apply.","65 Years",{"count":128,"type":23},450,[63],"This is a phase 3, open-label extension study to assess the long-term safety of KarXT for the treatment of mania or mania with mixed features in Bipolar-I disorder (BP-I)\n\nThe primary objective of the study is to evaluate the long-term safety and tolerability of KarXT in the treatment of participants with mania or mania with mixed features associated with BP-I.",[132],"Bipolar Disorder Type I With Mania",[134,135,136],"Bipolar-I disorder","Mania","Bipolar-I disorder with Mania",{"date":41,"type":42},{"date":139,"type":42},"2025-07-18",{"date":141,"type":23},"2028-06-13",{"name":86,"class":87},174,{"id":145,"slug":146,"hasResults":12,"nctId":147,"briefTitle":148,"officialTitle":149,"acronym":4,"eligibilityCriteria":150,"healthyVolunteers":12,"sex":18,"minAge":151,"maxAge":4,"enrollmentInfo":152,"targetDuration":4,"studyType":60,"phases":154,"briefSummary":155,"conditions":156,"keywords":158,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":39,"lastUpdatePostDateStruct":163,"startDateStruct":164,"completionDateStruct":166,"leadSponsor":168,"locationsCount":170},"100544252","phase-3-a-study-of-pitolisant-in-patients-with-prader-willi-syndrome-100544252","NCT06366464","A Study of Pitolisant in Patients With Prader-Willi Syndrome","A Phase 3, Randomized, Double-Blind, Placebo-controlled, Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients With Prader-Willi Syndrome","Inclusion Criteria:\n\n* Genetically confirmed diagnosis of PWS\n* Excessive daytime sleepiness\n* Has a consistent parent\u002Fcaregiver (preferably the same person throughout the study) who is willing and able to complete the required study assessments.\n* In the opinion of the Investigator, the patient\u002Fparent(s)\u002Fcaregiver(s)\u002Flegal guardian(s) are capable of understanding and complying with the requirements of the protocol and administration of oral study drug.\n\nExclusion Criteria:\n\n* Has a diagnosis of sleep apnea (OSA, CSA) that is not adequately controlled\n* Has a diagnosis of hypersomnia due to another sleep\u002Fmedical disorder\n* Participation in an interventional research study involving another investigational medication, device, or behavioral treatment within 30 days or 5 half-lives (whichever is longer) of the investigational medication prior to Screening","6 Years",{"count":153,"type":23},134,[63],"This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome.\n\nThe primary objective of this study is to evaluate the efficacy of pitolisant in treating excessive daytime sleepiness (EDS) in patients ≥6 years of age with Prader-Willi syndrome.\n\nSecondary objectives include assessing the impact of pitolisant on:\n\nIrritable and disruptive behaviors Hyperphagia Other behavioral problems including social withdrawal, stereotypic behavior, hyperactivity\u002Fnoncompliance, and inappropriate speech",[157],"Prader-Willi Syndrome",[159,160,161,162],"pitolisant","excessive daytime sleepiness","irritable and disruptive behaviors","Prader-Willi syndrome",{"date":41,"type":42},{"date":165,"type":42},"2024-05-28",{"date":167,"type":23},"2028-04",{"name":169,"class":87},"Harmony Biosciences Management, Inc.",57,{"id":172,"slug":173,"hasResults":12,"nctId":174,"briefTitle":175,"officialTitle":176,"acronym":4,"eligibilityCriteria":177,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":178,"targetDuration":4,"studyType":60,"phases":180,"briefSummary":181,"conditions":182,"keywords":185,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":39,"lastUpdatePostDateStruct":191,"startDateStruct":192,"completionDateStruct":194,"leadSponsor":196,"locationsCount":198},"100409600","phase-2-phase-2-trial-of-adagrasib-monotherapy-and-in-combination-with-pembrolizumab-and-a-phase-3-trial-of-adagrasib-in-combination-in-patients-with-a-kras-g12c-mutation-krystal-7-100409600","NCT04613596","Phase 2 Trial of Adagrasib Monotherapy and in Combination With Pembrolizumab and a Phase 3 Trial of Adagrasib in Combination in Patients With a KRAS G12C Mutation KRYSTAL-7","A Phase 2 Trial of Adagrasib Monotherapy and in Combination With Pembrolizumab and a Phase 3 Trial of Adagrasib in Combination With Pembrolizumab Versus Pembrolizumab in Patients With Advanced Non-Small Cell Lung Cancer With KRAS G12C Mutation","Inclusion Criteria:\n\n* Phase 2: Histologically confirmed diagnosis of unresectable or metastatic NSCLC with KRAS G12C mutation and any PD-L1 TPS\n* Phase 3: Histologically confirmed diagnosis of unresectable or metastatic squamous or nonsquamous NSCLC with KRAS G12C mutation and PD-L1 TPS\\>=50%\n* Phase 3: Presence of measurable disease per RECIST1.1\n* Phase 3: CNS Inclusion - Based on screening brain imaging, patients must have one of the following:\n\n  1. No evidence of brain metastases\n  2. Untreated brain metastases not needing immediate local therapy\n  3. Previously treated brain metastases not needing immediate local therapy\n\nExclusion Criteria:\n\n* Phase 2 and Phase 3: Prior systemic treatment for locally advanced or metastatic NSCLC including chemotherapy, immune checkpoint inhibitor therapy, or a therapy targeting KRAS G12C mutation (e.g., AMG 510).\n* Phase 2: Active brain metastases\n* Phase 3: Patients with known central nervous system (CNS) lesions must not have any of the following:\n\n  1. Any untreated brain lesions \\> 2.0 cm in size\n  2. Any brainstem lesions\n  3. Ongoing use of systemic corticosteroids for control of symptoms of brain lesions at a total daily dose of \\> 10 mg of prednisone (or equivalent) prior to randomization.\n  4. Have poorly controlled (\\> 1\u002Fweek) generalized or complex partial seizures, or manifest neurologic progression due to brain lesions notwithstanding CNS-directed therapy\n* Phase 3: Radiation to the lung \\> 30 Gy within 6 months prior to the first dose of study treatment",{"count":179,"type":23},626,[62,63],"The Phase 2 portion of this study evaluates the efficacy and safety of MRTX849 monotherapy and in combination with pembrolizumab in cohorts of patients with advanced NSCLC with KRAS G12C mutation and any PD-L1 TPS and who are candidates for first-line treatment.\n\nThe Phase 3 portion of the study compares the efficacy of adagrasib in combination with pembrolizumab versus pembrolizumab in patients with unresectable, locally advanced or metastatic squamous or nonsquamous NSCLC with KRAS G12C mutation and PD-L1 TPS \\>=50% and who are candidates for first line treatment.",[183,184],"Advanced Non-Small Cell Lung Cancer","Metastatic Non-Small Cell Lung Cancer",[186,187,106,184,188,189,190],"KRAS G12C","Non-small cell lung cancer","Adagrasib","Krazati","TPS",{"date":41,"type":42},{"date":193,"type":42},"2020-12-02",{"date":195,"type":23},"2029-10-31",{"name":197,"class":87},"Mirati Therapeutics Inc.",770,{"id":200,"slug":201,"hasResults":12,"nctId":202,"briefTitle":203,"officialTitle":204,"acronym":205,"eligibilityCriteria":206,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":207,"enrollmentInfo":208,"targetDuration":4,"studyType":60,"phases":210,"briefSummary":212,"conditions":213,"keywords":215,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":222,"startDateStruct":223,"completionDateStruct":224,"leadSponsor":226,"locationsCount":229},"100653306","cognitive-load-and-inhaler-technique-in-copd-100653306","NCT07786220","Cognitive Load and Inhaler Technique in COPD","Cognitive Load-Induced Inhaler Errors in COPD: Linking Dual-Task Mobility to Treatment Self-Management","DUAL-INH","Inclusion Criteria:\n\nAge ≥18 years. Established diagnosis of chronic obstructive pulmonary disease (COPD). Clinically stable at the time of assessment. Current use of at least one maintenance handheld inhaler (pMDI, SMI, or DPI). Ability to perform the Timed Up and Go test and the study cognitive tasks. Ability to understand the study procedures and provide written informed consent.\n\nExclusion Criteria:\n\nAcute COPD exacerbation or incomplete recovery from a recent exacerbation at the time of assessment.\n\nClinical condition preventing safe completion of the Timed Up and Go test. Inability to complete the cognitive or inhaler assessment procedures required by the protocol.\n\nInability to provide informed consent.","100 Years",{"count":209,"type":23},100,[211],"NA","This study evaluates whether cognitive load affects inhaler technique in adults with chronic obstructive pulmonary disease (COPD). During a single study visit, participants will complete assessments of cognitive function, mobility, and respiratory status. Dual-task mobility will be assessed using the Timed Up and Go test performed alone and while completing a cognitive task. Inhaler technique will then be evaluated in two conditions: usual inhaler use without an additional task and inhaler use while maintaining a short sequence of digits in working memory. Inhaler technique will be scored using a standardized five-step device-specific checklist, and inhaler use time will also be recorded. The study will examine whether inhaler technique worsens under cognitive load and whether this deterioration is associated with dual-task mobility performance, cognitive function, respiratory symptoms, and functional capacity. No inhaler training or correction will be provided between the two inhaler conditions. All assessments are performed during a single visit.",[214],"COPD",[216,217,218,219,220],"Chronic Obstructive Pulmonary Disease","Inhaler Technique","Cognitive Load","Dual-Task Performance","Timed Up and Go","2026-08-21",{"date":41,"type":42},{"date":221,"type":42},{"date":225,"type":23},"2027-11-30",{"name":227,"class":228},"Spitalul Clinic de Boli Infecțioase și Pneumoftiziologie Dr. Victor Babeș Timișoara","OTHER_GOV",1,{"id":231,"slug":232,"hasResults":12,"nctId":233,"briefTitle":234,"officialTitle":235,"acronym":236,"eligibilityCriteria":237,"healthyVolunteers":12,"sex":18,"minAge":238,"maxAge":20,"enrollmentInfo":239,"targetDuration":4,"studyType":60,"phases":241,"briefSummary":242,"conditions":243,"keywords":245,"overallStatus":249,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":250,"startDateStruct":251,"completionDateStruct":253,"leadSponsor":255,"locationsCount":257},"100651359","phase-2-a-study-of-brenipatide-ly3537031-in-adult-participants-with-moderate-to-severe-chronic-obstructive-pulmonary-disease-copd-100651359","NCT07759245","A Study of Brenipatide (LY3537031) in Adult Participants With Moderate-to-Severe Chronic Obstructive Pulmonary Disease (COPD)","A Phase 2, Multicenter, Randomized, Double-Blind, 52-week Study to Investigate the Efficacy and Safety of Brenipatide Compared With Placebo for the Treatment of Adult Participants With Moderate-to-Severe Chronic Obstructive Pulmonary Disease (COPD)","RENEW-COPD","Inclusion Criteria:\n\n* Have a physician diagnosis of Chronic Obstructive Pulmonary Disease (COPD), at least 12 months prior to screening who meet the following criteria:\n\n  * Current or former smokers with a smoking history of greater than or equal to (≥) 10 pack-years\n  * Moderate-to-severe COPD (post-Bronchodilator (BD) Forced Expiratory Volume in 1 Second (FEV₁)\u002Fforced vital capacity (FVC) less than (\\\u003C) 70 percent (%)\n  * Modified Medical Research Council Dyspnea Scale (mMRC-DS) grade ≥2\n  * Exacerbation history of ≥2 moderate or ≥1 severe exacerbations within the year prior to inclusion.\n  * Background double therapy \\[long-acting β₂-agonists (LABA) + long-acting muscarinic antagonists (LAMA)\\] or triple therapy \\[inhaled corticosteroids (ICS) + LABA + LAMA)\\] for 3 months prior to randomization with a stable dose of medication for ≥1 month prior to screening.\n\nExclusion Criteria:\n\n* Have a known pre-existing, clinically important lung condition other than COPD.\n* Have a current or recent acute, active infection before screening and up to randomization.","40 Years",{"count":240,"type":23},606,[62],"The main purpose of this study is to assess if different dose levels of Brenipatide are safe and work the way they are intended to work in participants with moderate-to-severe Chronic Obstructive Pulmonary Disease (COPD), when compared with placebo. The study will last approximately one year.",[244],"Pulmonary Disease, Chronic Obstructive",[246,247,248],"Emphysema","Chronic Bronchitis","Lung Disease","NOT_YET_RECRUITING",{"date":39,"type":42},{"date":252,"type":23},"2026-08",{"date":254,"type":23},"2028-11",{"name":256,"class":87},"Eli Lilly and Company",128,{"id":259,"slug":260,"hasResults":12,"nctId":261,"briefTitle":262,"officialTitle":263,"acronym":4,"eligibilityCriteria":264,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":265,"targetDuration":4,"studyType":60,"phases":267,"briefSummary":268,"conditions":269,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":271,"startDateStruct":272,"completionDateStruct":274,"leadSponsor":276,"locationsCount":278},"100627915","phase-2-study-to-evaluate-switching-to-brelovitug-for-the-treatment-of-chd-in-participants-receiving-bulevirtide-100627915","NCT07454837","Study to Evaluate Switching to Brelovitug for the Treatment of CHD in Participants Receiving Bulevirtide","A Phase 2b\u002F3, Open-Label, Multicenter Trial Evaluating the Efficacy and Safety of Switching to Brelovitug for the Treatment of Chronic Hepatitis Delta Infection in Participants Receiving Bulevirtide (AZURE-3)","Inclusion Criteria:\n\n1. Willing and able to provide written informed consent.\n2. Male or female, ≥18 years of age at Screening.\n3. Taking or willing to take TDF, TAF, or ETV at baseline, and willing to remain on stable treatment for the duration of the study.\n4. Currently taking bulevirtide treatment for CHD for ≥6 months at the time of Screening.\n5. HDV RNA ≥100 IU\u002FmL at Screening.\n\nExclusion Criteria:\n\n1. Evidence of decompensated liver disease (e.g., CTP Class B or C, history of hepatic encephalopathy, clinically significant ascites, or variceal bleeding).\n2. Known history of immune-complex disease.\n3. Active or clinically significant co-infection with hepatitis C virus (HCV) or human immunodeficiency virus (HIV).\n4. Evidence of other significant liver diseases (e.g., autoimmune hepatitis, primary biliary cholangitis, primary sclerosing cholangitis).\n5. History of hepatocellular carcinoma (HCC) or evidence of HCC on screening imaging.",{"count":266,"type":23},120,[62,63],"This is a Phase 2b\u002F3, randomized, open-label, multicenter trial evaluating the efficacy and safety of switching from bulevirtide to brelovitug for the treatment of chronic hepatitis Delta infection (CHD).",[270],"Chronic Hepatitis D",{"date":41,"type":42},{"date":273,"type":42},"2026-02-26",{"date":275,"type":23},"2029-03-30",{"name":277,"class":87},"Mirum Pharmaceuticals, Inc.",41,{"id":280,"slug":281,"hasResults":12,"nctId":282,"briefTitle":283,"officialTitle":284,"acronym":4,"eligibilityCriteria":285,"healthyVolunteers":12,"sex":18,"minAge":286,"maxAge":287,"enrollmentInfo":288,"targetDuration":4,"studyType":60,"phases":290,"briefSummary":291,"conditions":292,"keywords":294,"overallStatus":249,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":296,"startDateStruct":297,"completionDateStruct":299,"leadSponsor":301,"locationsCount":302},"100614915","phase-3-a-study-of-karxt--karx-ec-for-treatment-of-irritability-in-children-and-adolescents-with-autism-spectrum-disorder-100614915","NCT07285798","A Study of KarXT + KarX-EC for Treatment of Irritability in Children and Adolescents With Autism Spectrum Disorder","A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Safety, and Tolerability of KarXT + KarX-EC in Children and Adolescents (5 to 17 Years of Age) With Irritability Associated With Autism Spectrum Disorder","Inclusion Criteria\n\n* Participants must have a confirmed diagnosis of ASD, as defined by the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, Text Revision (DSM-5-TR) criteria, confirmed by the K-SADS-PL and must be experiencing symptoms of irritability.\n* Participants must have an ABC-I ≥18 (Irritability subscale of the ABC) and CGIS specific to irritability ≥4, at screening and baseline (Day 1).\n\nExclusion Criteria\n\n* Participants must not have a current primary DSM-5 diagnosis of bipolar disorder, including bipolar II disorder, schizophrenia, schizoaffective disorder, major depressive episode as determined by clinical instrument, or post-traumatic stress disorder (PTSD).\n* Exception Include: Participants with comorbid ADHD, provided that attention deficit\u002Fhyperactivity disorder (ADHD) is not the primary disorder, the participant is adequately treated and based on the investigator judgment the disorder is clinically stable.\n* Participants must not have history\u002Fpresence of clinically significant disease or disorder that would jeopardize participant safety or validity of study results.\n* Participants must not have a risk for suicidal behavior, and any clinically significant abnormal laboratory test.\n* Other protocol-defined Inclusion\u002FExclusion criteria may apply.","5 Years","17 Years",{"count":289,"type":23},176,[63],"The purpose of this study is to assess KarXT + KarX-EC for the treatment of irritability associated with autism in children and adolescents.",[293],"Irritability Associated With Autism Spectrum Disorder",[295],"Autism Spectrum Disorder",{"date":39,"type":42},{"date":298,"type":23},"2026-09-11",{"date":300,"type":23},"2029-08-06",{"name":86,"class":87},63,{"id":304,"slug":305,"hasResults":12,"nctId":306,"briefTitle":307,"officialTitle":308,"acronym":4,"eligibilityCriteria":309,"healthyVolunteers":12,"sex":18,"minAge":310,"maxAge":4,"enrollmentInfo":311,"targetDuration":4,"studyType":60,"phases":313,"briefSummary":314,"conditions":315,"keywords":318,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":323,"startDateStruct":324,"completionDateStruct":326,"leadSponsor":328,"locationsCount":329},"100611500","phase-3-a-study-of-orforglipron-ly3502970-on-cardiovascular-outcomes-in-adults-with-atherosclerotic-cardiovascular-disease-andor-chronic-kidney-disease-attain-outcomes-100611500","NCT07241390","A Study of Orforglipron (LY3502970) on Cardiovascular Outcomes in Adults With Atherosclerotic Cardiovascular Disease and\u002For Chronic Kidney Disease (ATTAIN-Outcomes)","A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Event-Driven Study to Investigate the Effect of Orforglipron on the Incidence of Major Adverse Cardiovascular Events in Participants With Established Atherosclerotic Cardiovascular Disease and\u002For Chronic Kidney Disease","Inclusion Criteria:\n\n* Have established ASCVD and\u002For CKD\n\nExclusion Criteria:\n\n* Have type 1 diabetes\n* Have had a major heart condition within 60 days prior to screening\n* Have New York Heart Association Functional Classification Class IV heart failure","50 Years",{"count":312,"type":23},7140,[63],"The purpose of this study is to measure cardiovascular outcomes with orforglipron compared with placebo in participants with atherosclerotic cardiovascular disease (ASCVD) and\u002For chronic kidney disease (CKD). Participation in the study will last about 5 years.",[316,317],"Atherosclerosis Cardiovascular Disease","Chronic Kidney Disease",[319,320,321,322],"Heart Disease","Kidney Disease","Outcomes","Stroke",{"date":39,"type":42},{"date":325,"type":42},"2025-12-01",{"date":327,"type":23},"2031-08",{"name":256,"class":87},567,{"id":331,"slug":332,"hasResults":12,"nctId":333,"briefTitle":334,"officialTitle":335,"acronym":4,"eligibilityCriteria":336,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":337,"enrollmentInfo":338,"targetDuration":4,"studyType":60,"phases":340,"briefSummary":341,"conditions":342,"keywords":344,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":346,"startDateStruct":347,"completionDateStruct":349,"leadSponsor":351,"locationsCount":352},"100608388","phase-3-a-trial-evaluating-brelovitug-bjt-778-vs-bulevirtide-for-the-treatment-of-chronic-hepatitis-delta-infection-azure-2-100608388","NCT07200908","A Trial Evaluating Brelovitug (BJT-778) vs Bulevirtide for the Treatment of Chronic Hepatitis Delta Infection (AZURE-2)","A Global, Randomized, Open-label, Multicenter Phase 3 Trial Evaluating BJT-778 vs Bulevirtide for the Treatment of Chronic Hepatitis Delta Infection (AZURE-2)","Key Inclusion Criteria:\n\n1. Willing and able to provide written informed consent\n2. Chronic HDV infection\n3. HDV RNA \\>500 IU\u002FmL at Screening\n4. ALT \\>ULN at Screening\n5. Willing to take or already taking HBV neucleos(t)ide therapy.\n\nKey Exclusion Criteria:\n\n1. Pregnant or nursing females\n2. Unwilling to comply with contraception requirements during the study\n3. Difficulty with blood collection and\u002For poor venous access for the purposes of phlebotomy\n4. Clinical hepatic decompensation (i.e., ascites, encephalopathy variceal hemorrhage).\n5. Solid organ or bone marrow transplantation\n6. Presence of other liver disease(s) (non-HBV\u002FHDV), such as nonalcoholic steatohepatitis (NASH), alcohol associated hepatitis, cholestatic liver disease, hepatocellular carcinoma.\n\nNote - Other protocol-defined Inclusion\u002FExclusion criteria apply.","99 Years",{"count":339,"type":23},172,[63],"This is a Phase 3, global, randomized, open-label, multicenter, trial evaluating brelovitug (BJT-778) vs bulevirtide for the treatment of chronic hepatitis delta infection (CHD). The main goal of this study is to test the effectiveness of brelovitug compared to bulevirtide as a long-term treatment in patients with chronic HDV infection.",[343],"Chronic Hepatitis D Infection",[345],"Hepatitis Delta virus, HDV, Hepatitis D infection; Hepatitis D virus",{"date":41,"type":42},{"date":348,"type":42},"2025-08-27",{"date":350,"type":23},"2029-09-30",{"name":277,"class":87},53,{"id":354,"slug":355,"hasResults":12,"nctId":356,"briefTitle":357,"officialTitle":358,"acronym":4,"eligibilityCriteria":359,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":360,"enrollmentInfo":361,"targetDuration":4,"studyType":60,"phases":363,"briefSummary":364,"conditions":365,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":367,"startDateStruct":368,"completionDateStruct":370,"leadSponsor":372,"locationsCount":374},"100598128","phase-3-a-study-of-orelabrutinib-in-patients-with-primary-progressive-multiple-sclerosis-100598128","NCT07067463","A Study of Orelabrutinib in Patients With Primary Progressive Multiple Sclerosis","A Phase 3, Randomized, Double-blind, Efficacy and Safety Study Comparing Orelabrutinib to Placebo in Patients With Primary Progressive Multiple Sclerosis","Inclusion Criteria:\n\n* 18 to 60 years of age, inclusive\n* Diagnosed with Primary Progressive MS (PPMS) according to 2017 McDonald criteria\n* Participant must have documented evidence of disability progression observed during the 24 months before screening.\n* Expanded disability status scale (EDSS) score between 3.0 to 6.5 points, inclusive, at Screening.\n\nExclusion Criteria:\n\n* Diagnosed with relapsing-remitting MS (RRMS) or secondary progressive MS (SPMS)\n* Immunologic disorder other than MS or any other conditions requiring oral, intravenous (IV), intramuscular, or intra-articular corticosteroid therapy.\n* History or current diagnosis of other neurological disorders that may mimic MS\n* History of any other significant active medical condition\n* History of suicidal behavior within 6 months prior to Screening\n* Any prior history of malignancy if no recurrence within 5 years\n* Patients on anticoagulation, or antiplatelet therapy will be excluded\n* Patients took strong\u002Fmoderate CYP3A inhibitors or strong\u002Fmoderate CYP3A inducerswithin 14 days\n* Clinically significant laboratory abnormalities at Screening.\n* Any allergy, contraindication, or inability to tolerate orelabrutinib or any of the excipients in the study intervention\n* Vaccination with live or live-attenuated virus vaccine within 1 month prior to Screening\n* History of alcohol abuse or alcohol use disorder or other drug abuse within 12 months prior to screening.","60 Years",{"count":362,"type":23},705,[63],"Orelabrutinib is a CNS-penetrable BTK inhibitor. This is a phase 3, randomized, double-blind, parallel-group, multicenter study to evaluate the efficacy and safety of orelabrutinib compared with placebo in patients with PPMS. Patients will be treated for approximately 30 to 60 months, with a minimum treatment duration of 12 months. The study will enroll approximately 705 subjects in a 2:1 randomization (orelabrutinib: placebo), globally.",[366],"Multiple Sclerosis (MS) Primary Progressive",{"date":39,"type":42},{"date":369,"type":42},"2026-03-23",{"date":371,"type":23},"2030-07",{"name":373,"class":87},"Zenas BioPharma (USA), LLC",48,{"id":376,"slug":377,"hasResults":12,"nctId":378,"briefTitle":379,"officialTitle":380,"acronym":381,"eligibilityCriteria":382,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":20,"enrollmentInfo":383,"targetDuration":4,"studyType":60,"phases":385,"briefSummary":386,"conditions":387,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":392,"startDateStruct":393,"completionDateStruct":395,"leadSponsor":397,"locationsCount":399},"100593896","phase-2-a-study-of-long-acting-antibodies-alone-and-in-combinations-for-moderate-to-severe-ulcerative-colitis-100593896","NCT07012395","A Study of Long-acting Antibodies Alone and in Combinations for Moderate to Severe Ulcerative Colitis","Phase 2 Platform Trial to Assess the Efficacy and Safety of Long-acting Antibodies as Single Agents and in Combinations for Moderately to Severely Active Ulcerative Colitis","SKYLINE-UC","Inclusion Criteria:\n\n* Diagnosis of UC for ≥3 months before Day 1, confirmed by endoscopy and histology either previously or during Screening\n* Active UC with disease extent of ≥15 cm from the anal verge, as confirmed by Screening endoscopy (up to approximately 15% allowed to have only proctitis)\n* Moderately to severely active disease as defined by a modified Mayo score of 5-9, rectal bleeding subscore of ≥1, and Mayo endoscopic subscore ≥2\n\nExclusion Criteria:\n\n* Current diagnosis of Crohn's disease or Inflammatory Bowel Disease (IBD)-Undefined\n* Confirmed or suspected fulminant colitis, toxic megacolon, bowel perforation and\u002For other conditions that will likely require surgery during induction\n* Failed 4 or more approved or investigational advanced therapy classes",{"count":384,"type":23},645,[62],"This is a Phase 2, multicenter, proof-of-concept platform study in adult participants with moderately to severely active ulcerative colitis (UC). The primary goal of the study is to assess the efficacy and safety of multiple interventions following intravenous (IV) induction and subcutaneous (SC) maintenance treatment.",[388,389,390,391],"Ulcerative Colitis","Inflammatory Bowel Diseases","Colitis","Colitis, Ulcerative",{"date":41,"type":42},{"date":394,"type":42},"2025-05-27",{"date":396,"type":23},"2028-03",{"name":398,"class":87},"Spyre Therapeutics, Inc.",267,{"id":401,"slug":402,"hasResults":12,"nctId":403,"briefTitle":404,"officialTitle":405,"acronym":406,"eligibilityCriteria":407,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":408,"targetDuration":4,"studyType":60,"phases":410,"briefSummary":411,"conditions":412,"keywords":414,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":416,"startDateStruct":417,"completionDateStruct":419,"leadSponsor":421,"locationsCount":423},"100592970","phase-3-a-phase-iii-study-of-azd0780-on-major-adverse-cv-events-in-patients-with-a-history-of-ascvd-events-or-at-high-risk-for-a-first-event-100592970","NCT07000357","A Phase III Study of AZD0780 on Major Adverse CV Events in Patients With a History of ASCVD Events or at High Risk for a First Event","A Phase III, Randomised, Double-blind, Placebo-controlled, Parallel-group Study to Assess the Effect of AZD0780 on Major Adverse Cardiovascular Events in Patients With Established Atherosclerotic Cardiovascular Disease (ASCVD) or at High Risk for a First ASCVD Event","AZURE-Outcomes","Inclusion Criteria:\n\n* Meets one of the following:\n\n  1. Participants with history of an ASCVD event: Participants ≥ 18 years of age at the time of signing the ICF with a history of MI or ischaemic stroke suspected to be due to atherosclerotic vascular disease ≥ 1 month prior to randomisation (presumed lacunar or cardioembolic strokes are not qualifying events), or revascularisation for symptomatic lower limb PAD any time prior to screening\n\n     Additional risk factors based on the level of the LDL-C and timing of MI or stroke:\n\n     o Participants with an LDL-C ≥ 75 mg\u002FdL (≥ 1.9 mmol\u002FL) need to have at least one of the other additional risk factors (i to viii) below.\n\n     ii) T2DM requiring ongoing medical therapy iii) Age ≥ 65 years v) Previous above ankle amputation due to PAD vi) Previous diagnosis of non-end stage CKD\n  2. Participants at increased risk of a first ASCVD event: Male participant ≥ 50 years of age or female participant ≥ 55 years of age at the time of signing the ICF with LDL-C ≥ 100 mg\u002FdL (≥ 2.6 mmol\u002FL), with no prior history of MI, ischaemic stroke due to atherosclerotic disease, or leg revascularisation for symptomatic lower limb PAD, and with diagnostic evidence of at least one of the following disease categories (i, ii, or iii):\n\n  (i) Significant atherosclerotic artery disease (ii) High-risk Type 1 or Type 2 diabetes mellitus with manifestation of at least one of the following end-organ diseases:\n  1. Nephropathy - Persistent (≥ 2 readings) microalbuminuria (urine albumin\u002Fcreatinine ratio ≥ 30 mg\u002Fg) and\u002For persistent eGFR \\\u003C 60 mL\u002Fmin\u002F1.73 m2. At least one reading must come from the medical record within the last 12 months in addition to the reading from screening\n  2. Retinopathy - Treated diabetic retinopathy (surgical intervention or injectable therapy) or prior diagnosis made by a relevant healthcare specialist\n  3. Neuropathy - Treated neuropathy (medical therapy for pain relief or symptom alleviation) or prior diagnosis made by a relevant healthcare specialist\n  4. ABI \\\u003C 0.9 or \\> 1.4 - confirmed either in study during screening or randomisation, or from the medical record within the last 5 years (iii) Documented atherosclerosis of less significance\n\n     For (ii) and (iii), participants need to have at least one of the additional risk factors below:\n\n  \u003C!-- -->\n\n  1. CKD with eGFR x mL\u002Fmin\u002F1.73 m2\n  2. Current tobacco use\n  3. Age ≥ 65\n  4. T2DM (if included on the less significant atherosclerosis criterion iii)\n* Participants should receive a background lipid lowering regimen anticipated to achieve at least a \\~50% reduction in LDL-C. Except in cases of intolerance, the regimen should include a high intensity statin therapy or lower intensity statin therapy in combination with an oral agent with proven outcome benefit (eg, ezetimibe and\u002For bempedoic acid).\n\nParticipants must achieve a stable background lipid lowering therapy \\> 28 days before screening.\n\nExclusion criteria:\n\n* Any underlying known disease, or condition including homozygous familial hypercholesterolaemia, or LDL or plasma apheresis within 12 months prior to randomisation, that, in the opinion of the investigator, might interfere with the interpretation of the clinical study results.\n* Any revascularisation procedure planned within the next 3 months.\n* Available imaging assessment within the last 3 years showing either coronary calcium score of zero, or a coronary computed tomography angiography with no atherosclerosis.\n* Calculated eGFR \\\u003C 15 mL \u002Fmin\u002F1.73 m2 at screening.\n* Any laboratory values with the following deviations at screening:\n\n  * AST or ALT \\> 3 × ULN\n  * TBL \\> 2 × ULN (except for participants with Gilbert's syndrome where TBL 3 × ULN is acceptable provided direct bilirubin \\\u003C 1.5 × ULN)\n  * Fasting triglycerides ≥ 400 mg\u002FdL (≥ 4.52 mmol\u002FL).\n  * Creatine kinase \\> 5 × ULN\n  * Urine albumin\u002Fcreatinine ratio ≥ 500 mg\u002Fg\n* Uncontrolled T2DM defined as HbA1c ≥ 9.5% at screening.\n* Inadequately treated hypothyroidism defined as TSH \\> 1.5 × ULN at screening or participants whose thyroid replacement therapy was initiated or modified within the last 3 months prior to screening.\n* Use of mipomersen or lomitapide (cholesterol-lowering medications) within 12 months of screening or planned use during the study.\n* Use of gemfibrozil within one week prior to the Screening Visit or planned use during the study.\n* Use of PCSK9 inhibitors: evolocumab\u002Falirocumab within 12 weeks of the Screening Visit or planned use during the study, or inclisiran within 18 months of the Screening Visit or planned use during the study, or any other approved PCSK9 inhibitor use within 5 half lives prior to the Screening Visit or planned use during the study.",{"count":409,"type":23},15100,[63],"The purpose of this phase 3, randomized, placebo controlled, event-driven study is to assess the effect of AZD0780, an oral PCSK9 inhibitor, compared with placebo in reducing the risk of MACE-PLUS in patients with established ASCVD or at high risk for a first ASCVD event. The effect of AZD0780 vs placebo on the risk of MACE-PLUS will be evaluated from randomisation until the primary analysis censoring date (PACD). The Study Closure Visit will be scheduled to occur after the PACD and will be the final visit for each participant in the study.",[413],"Cardiovascular Disease",[415],"Atherosclerotic Cardiovascular Disease",{"date":39,"type":42},{"date":418,"type":42},"2025-06-04",{"date":420,"type":23},"2029-10-26",{"name":422,"class":87},"AstraZeneca",1452,{"id":425,"slug":426,"hasResults":12,"nctId":427,"briefTitle":428,"officialTitle":429,"acronym":430,"eligibilityCriteria":431,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":432,"targetDuration":4,"studyType":60,"phases":434,"briefSummary":435,"conditions":436,"keywords":438,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":443,"startDateStruct":444,"completionDateStruct":446,"leadSponsor":448,"locationsCount":450},"100587610","phase-3-a-study-to-assess-the-efficacy-and-safety-of-debio-4126-in-participants-with-acromegaly-previously-treated-with-somatostatin-analogs-100587610","NCT06930625","A Study to Assess the Efficacy and Safety of Debio 4126 in Participants With Acromegaly Previously Treated With Somatostatin Analogs","A Phase 3 Randomized 3-arm Trial (Double-blind Debio 4126, Placebo Control, and Open-label Debio 4126), to Assess the Efficacy and Safety of Debio 4126, a 12-week Octreotide Formulation, in Patients With Acromegaly Previously Treated With Somatostatin Analogs","OXTEND™-03","Inclusion criteria\n\n1. Patients ≥18 years of age\n2. Patients who are receiving octreotide or lanreotide monotherapy for acromegaly for at least 6 months, at a stable dose for the last 12 weeks.\n3. IGF-1 at screening ≤1x ULN\n4. Acromegaly diagnosis, defined as per protocol\n5. Adequate bone marrow, hepatic and renal function\n6. To enter Period 2 (Arms A and B): IGF-1 ≤1x ULN at Week 34, or up to Week 48 when treated with rescue medication\n7. Other protocol-defined criteria apply\n\nExclusion criteria\n\n1. Compression of optic chiasm causing visual defects\n2. Symptomatic cholelithiasis or bile duct dilatation\n3. Planned cholecystectomy during the trial duration\n4. Acute or chronic pancreatitis\n5. Pituitary radiotherapy\n6. Uncontrolled hypothyroidism\n7. Uncontrolled diabetes\n8. Pituitary surgery within 6 months before screening or planned on trial\n9. Treatment with pasireotide within 6 months prior to screening, pegvisomant or dopamine agonists within 3 months prior to screening\n10. Recent or ongoing cardiovascular or thromboembolic diseases including heart failure, myocardial infarction, stroke, certain arrythmias, pulmonary embolism\n11. Other protocol-defined criteria apply",{"count":433,"type":23},119,[63],"The primary purpose of this study is to assess the effect of Debio 4126 in the maintenance of the levels of insulin-like growth factor 1 (IGF-1) ≤1x upper limit of normal (ULN) in the double-blind period (Period 1) in comparison to placebo at week 36.",[437],"Acromegaly",[439,440,441,442],"IGF-1","Growth hormone","Pituitary gland","Gigantism",{"date":39,"type":42},{"date":445,"type":42},"2025-11-26",{"date":447,"type":23},"2029-03",{"name":449,"class":87},"Debiopharm International SA",73,{"id":452,"slug":453,"hasResults":12,"nctId":454,"briefTitle":455,"officialTitle":456,"acronym":457,"eligibilityCriteria":458,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":459,"targetDuration":4,"studyType":60,"phases":461,"briefSummary":462,"conditions":463,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":465,"startDateStruct":466,"completionDateStruct":468,"leadSponsor":470,"locationsCount":471},"100584534","phase-3-study-of-olomorasib-ly3537982-in-combination-with-standard-of-care-in-participants-with-resected-or-unresectable-kras-g12c-mutant-non-small-cell-lung-cancer-100584534","NCT06890598","Study of Olomorasib (LY3537982) in Combination With Standard of Care in Participants With Resected or Unresectable KRAS G12C-mutant Non-Small Cell Lung Cancer","A Phase 3, Multicenter, Double-Blind, Placebo-controlled Study Assessing the Efficacy and Safety of Olomorasib in Combination With Standard of Care Immunotherapy in Participants With Resected or Unresectable KRAS G12C-Mutant, Non-Small Cell Lung Cancer - SUNRAY-02","SUNRAY-02","Inclusion Criteria:\n\n* Histological or cytological confirmation of NSCLC.\n\n  * Part A\n\n    1. Clinical Stage II-IIIB (N0, N1, N2) treated with presurgical chemoimmunotherapy, with residual tumor present at time of surgery. Patients with a pathologic complete response are not eligible.\n    2. Pathologic Stage II-IIIB (N0, N1, N2) NSCLC treated with initial upfront resection.\n  * Part B - Clinical Stage III, unresectable NSCLC, without progression on concurrent platinum-based chemoradiotherapy.\n* Must have disease with evidence of KRAS G12C mutation.\n* Must have known programmed death-ligand 1 (PD-L1) expression\n* Must have an ECOG performance status of 0 or 1.\n* Able to swallow oral medication.\n* Must have adequate laboratory parameters.\n* Contraceptive use should be consistent with local regulations for those participating in clinical studies.\n* Women of childbearing potential must\n\n  * Have a negative pregnancy test.\n  * Not be breastfeeding during treatment\n\nExclusion Criteria:\n\n* Have known, actionable changes in the EGFR or ALK genes.\n* Have another type of cancer that is progressing or required active treatment within the past 2 years before screening.\n* Have an active autoimmune disease that required systemic treatment in the past 2 years. Endocrine replacement therapy is allowed.\n* Had any immune-related side effect or allergic reaction (Grade 3 or higher) from a previous immunotherapy medicine, or any immune-related side effect greater than Grade 1 that has not resolved. This does not apply for people with hormone-related diseases who are now on stable hormone replacement therapy.",{"count":460,"type":23},700,[63],"The main purpose of this study is to assess if olomorasib in combination with pembrolizumab is more effective than the pembrolizumab and placebo combination in part A in participants with resected KRAS G12C-mutant NSCLC and to assess if olomorasib in combination with durvalumab is more effective than the durvalumab and placebo combination in part B in participants with unresectable KRAS G12C-mutant non-small cell lung cancer. The study may last up to 3 years for each participant.",[464],"Carcinoma, Non-Small-Cell Lung",{"date":39,"type":42},{"date":467,"type":42},"2025-03-27",{"date":469,"type":23},"2032-02",{"name":256,"class":87},369,{"id":473,"slug":474,"hasResults":12,"nctId":475,"briefTitle":476,"officialTitle":477,"acronym":478,"eligibilityCriteria":479,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":207,"enrollmentInfo":480,"targetDuration":4,"studyType":60,"phases":482,"briefSummary":483,"conditions":484,"keywords":486,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":491,"startDateStruct":492,"completionDateStruct":494,"leadSponsor":496,"locationsCount":498},"100570795","phase-3-phase-iii-extension-study-of-efficacy-and-safety-of-ianalumab-with-or-without-study-treatment-withdrawal-in-participants-with-lupus-nephritis-sirius-ln-extension-100570795","NCT06711887","Phase III Extension Study of Efficacy and Safety of Ianalumab With or Without Study Treatment Withdrawal in Participants With Lupus Nephritis (SIRIUS-LN Extension)","An Open-label Extension Study to Assess the Efficacy and Safety of Ianalumab With or Without Study Treatment Withdrawal in Adult Participants With Lupus Nephritis Who Have Completed Study Treatment in the CVAY736K12301 Core Study (SIRIUS-LN Extension)","SIRIUS-LN ext","Inclusion Criteria:\n\n1. Signed informed consent prior to participation in the extension study.\n2. Participants must have participated in the SIRIUS-LN core study and must have completed the entire treatment up to Week 144 on double-blind or open label study treatment.\n\nExclusion Criteria:\n\n1. Use of prohibited therapies\n2. Pregnant or nursing (lactating) women.",{"count":481,"type":23},348,[63],"The purpose of this up to 6-year extension study is the evaluation of the efficacy and safety\n\n1. after study treatment withdrawal in patients with lupus nephritis (LN) who achieved response (complete renal response \\[CRR\\] or partial renal response \\[PRR\\]) on double-blind treatment at the end of the SIRIUS-LN core study, and\n2. of open-label ianalumab 300 mg treatment in patients who, at the end of the SIRIUS-LN core study, were either already receiving ianalumab open-label treatment or did not meet CRR\u002FPRR criteria on double-blind treatment at the end of the SIRIUS-LN core study.",[485],"Lupus Nephritis",[487,488,489,490],"Lupus Nephritis (LN)","B cell depletion","ianalumab","VAY736",{"date":39,"type":42},{"date":493,"type":42},"2025-05-19",{"date":495,"type":23},"2035-08-01",{"name":497,"class":87},"Novartis Pharmaceuticals",47,{"id":500,"slug":501,"hasResults":12,"nctId":502,"briefTitle":503,"officialTitle":504,"acronym":505,"eligibilityCriteria":506,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":507,"enrollmentInfo":508,"targetDuration":4,"studyType":60,"phases":510,"briefSummary":512,"conditions":513,"keywords":515,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":519,"startDateStruct":520,"completionDateStruct":522,"leadSponsor":524,"locationsCount":526},"100568362","phase-1-phase-1-study-to-evaluate-safety-and-antiviral-activity-of-pbgene-hbv-in-adult-patients-with-chronic-hepatitis-b-100568362","NCT06680232","Phase 1 Study to Evaluate Safety and Antiviral Activity of PBGENE-HBV in Adult Patients With Chronic Hepatitis B","A Phase 1, Open-Label, First-in-Human, Dose Escalation (Part 1) and Expansion (Part 2) Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Antiviral Activity of PBGENE-HBV in Participants With Chronic Hepatitis B (ELIMINATE-B)","ELIMINATE-B","Key Inclusion Criteria:\n\n* Male or women of non-child bearing potential\n* BMI 18.0 to 35.0\n* Good overall health deemed by the study Investigator\n* CHB infection documented at least 12 months prior to screening\n* HBeAg-negative CHB\n* Must be virologically suppressed on current NA treatment\n\nKey Exclusion Criteria:\n\n* No history of cirrhosis of the liver\n* No current infections of Hepatitis A, D, and E, human immunodeficiency virus (type 1 and 2), and no history of or current hepatitis C. In addition, no other active infections deemed clinically relevant.\n* No signs of hepatocellular carcinoma\n* Not received an organ transplant\n* No malignancy within 5 years of screening, except for specific cancers that are cured by surgical resection (e.g., basal cell skin cancer)\n* No investigational agent received within 6 months of screening","70 Years",{"count":509,"type":23},45,[511],"PHASE1","This is a Phase 1, open-label, dose escalation and dose expansion study to evaluate the safety, tolerability, PK, and antiviral activity of PBGENE-HBV in adult participants with chronic hepatitis B.",[514],"HEPATITIS B CHRONIC",[514,516,517,518],"Gene Therapy","Gene Editing","PBGENE-HBV",{"date":39,"type":42},{"date":521,"type":42},"2024-11-14",{"date":523,"type":23},"2027-06",{"name":525,"class":87},"Precision BioSciences, Inc.",7,{"id":528,"slug":529,"hasResults":12,"nctId":530,"briefTitle":531,"officialTitle":532,"acronym":4,"eligibilityCriteria":533,"healthyVolunteers":12,"sex":18,"minAge":534,"maxAge":4,"enrollmentInfo":535,"targetDuration":4,"studyType":60,"phases":537,"briefSummary":538,"conditions":539,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":541,"startDateStruct":542,"completionDateStruct":544,"leadSponsor":545,"locationsCount":546},"100542140","phase-3-a-study-of-lebrikizumab-ly3650150-in-participants-with-chronic-rhinosinusitis-and-nasal-polyps-treated-with-intranasal-corticosteroids-contrast-np-100542140","NCT06338995","A Study of Lebrikizumab (LY3650150) in Participants With Chronic Rhinosinusitis and Nasal Polyps Treated With Intranasal Corticosteroids (CONTRAST-NP)","A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lebrikizumab\u002FLY3650150 in Participants With Chronic Rhinosinusitis With Nasal Polyps on Background Intranasal Corticosteroids","Inclusion Criteria:\n\n* Physician-diagnosed chronic rhinosinusitis (CRS) with bilateral nasal polyps (NP).\n* Prior treatment with systemic corticosteroids (SCS) within the last 2 years (or a medical contraindication or intolerance to SCS), prior surgery for NP, or both.\n* Endoscopic bilateral NPS score of at least 5 out of 8, with a minimum score of 2 in each nasal cavity performed at screening and baseline.\n* Ongoing symptoms for at least 8 weeks prior to study entry (screening), including:\n\n  1. Nasal congestion with moderate or severe symptom severity (score 2 or 3) at screening and a weekly average severity score of at least 1 (range 0 to 3) at randomization, and\n  2. At least one other symptom, such as partial loss of smell (hyposmia), total loss of smell (anosmia), or anterior or posterior rhinorrhea.\n* Have concomitant asthma must be stable in the 3 months prior to screening using permitted regular asthma treatment.\n* Adolescent participants ≥12 to \\\u003C18 years of age and weighing ≥40 kg at time of Visit 1.\n\nExclusion Criteria:\n\n* Have received a dose of lebrikizumab.\n* Have received treatment with any rescue medication and\u002For have the need for surgery for NP during screening and\u002For run-in period.\n* Allergen immunotherapy (subcutaneous immunotherapy \\[SCIT\\]\u002Fsublingual immunotherapy \\[SLIT\\]) initiated within 6 months prior to screening, that is not on a stable dose (3 months prior to screening).\n* Has received a biologic treatment approved for use in CRSwNP, asthma, or AD, even if administered to treat a different condition, within 4 months or 5 half-lives, whichever is longer, prior to screening.\n* Have received treatment with any biologic or systemic immunosuppressants for inflammatory disease or autoimmune disease prior to the baseline visit:\n\n  1. B cell-depleting biologics, including rituximab, within 6 months.\n  2. other biologics within 5 half-lives (if known) or 8 weeks, whichever is longer.\n  3. Systemic immunosuppressants within 4 weeks prior to baseline.\n* Have had any sinus intranasal surgery (including nasal polypectomy) within 6 months prior to screening\n* Have had prior sino-nasal surgery or sinus surgery changing lateral wall structure of the nose making it difficult to assess endoscopic NPS\n* Have a presence of any of the following conditions that may impact the assessment of endpoints at screening or baseline:\n\n  1. Nasal septal deviation occluding at least one nostril.\n  2. Antrochoanal polyps.\n  3. Acute sinusitis, acute nasal infection, or acute upper respiratory infection.\n  4. Ongoing rhinitis medicamentosa.\n  5. Presence of another diagnosis associated with NP (ie, eosinophilic granulomatosis with polyangiitis, granulomatosis with polyangiitis, Young's syndrome, primary ciliary dyskinesia, cystic fibrosis). Note: for adolescents, documentation for ruling out cystic fibrosis and primary ciliary dyskinesia is required.\n  6. A nasal cavity tumor (malignant or benign).\n  7. Evidence of fungal rhinosinusitis.\n* Have anosmia from COVID or any reason other than CRSwNP.\n* Participants with forced expiratory volume in 1 second (FEV1) 50% or less (of predicted normal) at screening.\n* Female participant who is pregnant, breastfeeding, or is planning to become pregnant, or to breastfeed during the study.","12 Years",{"count":536,"type":23},510,[63],"The main purpose of this study is to evaluate the efficacy and safety of lebrikizumab in participants with chronic rhinosinusitis and nasal polyps treated with intranasal corticosteroids. The study will last about 18 months.",[540],"Chronic Rhinosinusitis With Nasal Polyps (CRSwNP)",{"date":39,"type":42},{"date":543,"type":42},"2024-04-29",{"date":396,"type":23},{"name":256,"class":87},202,{"id":548,"slug":549,"hasResults":12,"nctId":550,"briefTitle":551,"officialTitle":552,"acronym":553,"eligibilityCriteria":554,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":555,"targetDuration":4,"studyType":60,"phases":557,"briefSummary":558,"conditions":559,"keywords":561,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":582,"startDateStruct":583,"completionDateStruct":585,"leadSponsor":587,"locationsCount":588},"100525272","phase-3-a-study-of-first-line-olomorasib-ly3537982-and-pembrolizumab-with-or-without-chemotherapy-in-patients-with-advanced-kras-g12c-mutant-non-small-cell-lung-cancer-100525272","NCT06119581","A Study of First-Line Olomorasib (LY3537982) and Pembrolizumab With or Without Chemotherapy in Patients With Advanced KRAS G12C-Mutant Non-small Cell Lung Cancer","SUNRAY-01, A Global Pivotal Study in Participants With KRAS G12C-Mutant, Locally Advanced or Metastatic Non-Small Cell Lung Cancer Comparing First-Line Treatment of LY3537982 and Pembrolizumab vs Placebo and Pembrolizumab in Those With PD-L1 Expression ≥50% or LY3537982 and Pembrolizumab, Pemetrexed, Platinum vs Placebo and Pembrolizumab, Pemetrexed, Platinum Regardless of PD-L1 Expression","SUNRAY-01","Inclusion Criteria:\n\n* Histologically or cytologically confirmed NSCLC with Stage IIIB-IIIC or Stage IV disease, not suitable for curative intent radical surgery or radiation therapy.\n* Part B and Safety Lead-In Part B: the histology of the tumor must be predominantly non-squamous (in line with pemetrexed label).\n* Must have disease with evidence of KRAS G12C mutation.\n* Must have known programmed death-ligand 1 (PD-L1) expression\n\n  * Part A: Greater than or equal to (≥)50 percent (%).\n  * Part B: 0% to 100%.\n  * Part C: \\\u003C50%.\n* Must have measurable disease per RECIST v1.1.\n* Must have an ECOG performance status of 0 or 1.\n* Estimated life expectancy ≥12 weeks.\n* Ability to swallow capsules.\n* Must have adequate laboratory parameters.\n* Contraceptive use should be consistent with local regulations for those participating in clinical studies.\n* Women of childbearing potential must\n\n  * Have a negative pregnancy test.\n  * Not be breastfeeding during treatment\n\nExclusion Criteria:\n\n* Have a documented additional validated targetable oncogenic driver mutation or alteration in genes such as epidermal growth factor receptor (EGFR), anaplastic lymphoma kinase (ALK), BRAF (V600E), human epidermal growth factor receptor 2 (HER2), MET (exon 14), ROS1, rearranged during transfection (RET), or neurotrophic tyrosine receptor kinase (NTRK)1\u002F2\u002F3.\n* Have had any of the following prior to randomization:\n\n  \\-- Prior systemic therapy (chemotherapy, immunotherapy, targeted therapy, or biological therapy) for advanced or metastatic NSCLC.\n\n  \\--- 1 cycle of standard-of-care treatment prior to study enrollment will be allowed for cases where immediate treatment is clinically indicated:\n* Have known active central nervous system metastases and\u002For carcinomatous meningitis.\n\nExclusion Criteria for Participants receiving Pemetrexed and Platinum (Part B and Safety Lead-In Part B)\n\n* Have predominantly squamous cell histology for NSCLC\n* Only for participants with mild to moderate renal insufficiency: Unable to avoid aspirin, ibuprofen, or other nonsteroidal anti-inflammatory drugs (NSAIDs) two days before (5 days for long acting NSAIDs), day of, and two days after administration of pemetrexed\n* Is unable or unwilling to take folic acid or vitamin B12 supplementation.",{"count":556,"type":23},1264,[63],"The purpose of this study is to assess if adding LY3537982 (olomorasib) in combination with standard of care anti-cancer drugs is more effective than standard of care in participants with untreated advanced NSCLC. NSCLC must have a change in a gene called KRAS G12C. Study participation, including follow-up, could last up to 3 years, depending on how you and your lung cancer are doing.",[464,560],"Neoplasm Metastasis",[183,562,563,564,565,566,567,568,569,570,571,572,573,560,66,574,73,575,576,577,578,579,580,581,72],"KRAS G12 Lung Cancer","Advanced Lung Cancer","Metastatic Lung Cancer","KRAS G12C inhibitor","KRAS G12C Positive","KRAS Mutation","KRAS G12 Mutation","Lung Cancer Mutation","Olomorasib","Lung Diseases","Neoplastic Processes","Pathologic Processes","Non-Small Cell Lung Cancer (NSCLC)","Respiratory Tract Neoplasms","Thoracic Neoplasms","Neoplasms by Site","Neoplasms","Respiratory Tract Diseases","Carcinoma, Bronchogenic","Bronchial Neoplasms",{"date":39,"type":42},{"date":584,"type":42},"2023-12-21",{"date":586,"type":23},"2031-01",{"name":256,"class":87},418,{"id":590,"slug":591,"hasResults":12,"nctId":592,"briefTitle":593,"officialTitle":594,"acronym":4,"eligibilityCriteria":595,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":596,"targetDuration":4,"studyType":60,"phases":598,"briefSummary":599,"conditions":600,"keywords":603,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":608,"startDateStruct":609,"completionDateStruct":611,"leadSponsor":613,"locationsCount":615},"100323125","phase-3-long-term-safety-and-efficacy-extension-study-for-participants-with-advanced-tumors-who-are-currently-on-treatment-or-in-follow-up-in-a-pembrolizumab-mk-3475-study-mk-3475-587keynote-587-100323125","NCT03486873","Long-term Safety and Efficacy Extension Study for Participants With Advanced Tumors Who Are Currently on Treatment or in Follow-up in a Pembrolizumab (MK-3475) Study (MK-3475-587\u002FKEYNOTE-587)","A Multicenter, Open-label, Phase 3 Study to Evaluate the Long-term Safety and Efficacy in Participants Who Are Currently on Treatment or in Follow-up in Studies That Include Pembrolizumab","Inclusion Criteria:\n\n* Treated on the parent pembrolizumab studies established by the Sponsor as MK-3475-587 ready.\n* Currently receiving pembrolizumab, pembrolizumab based combinations or lenvatinib from parent studies or in a follow-up phase.\n\nAdditional eligibility criteria for participants who enter Second Course Phase once they are enrolled on MK-3475-587:\n\n* Has not received any anticancer systemic treatment since the last dose of pembrolizumab or a pembrolizumab-based combination in First Course Phase.\n* Has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.\n* Demonstrates adequate organ function.\n* Have resolution of any toxic effect(s) of First Course Phase trial treatment with pembrolizumab or a pembrolizumab-based combination to Grade 1 or less (except alopecia) before trial treatment in Second Course Phase is started. If participant received major surgery or radiation therapy of \\>30 Gray (Gy), they must have recovered from the toxicity and\u002For complications of the intervention.\n* A female participant is eligible to enroll if she is not pregnant, not breastfeeding, and ≥1 of the following conditions applies: A woman of childbearing potential (WOCBP) who agrees to use contraception during the study treatment period and for ≥120 days (corresponding to time needed to eliminate any study combination treatment(s) plus 30 days (a menstruation cycle) for study treatments with risk of genotoxicity.\n\nAdditional eligibility criteria for participants who enter dosing with Lenvatinib:\n\n* Adequately controlled blood pressure (BP) to \\\u003C150\u002F90 mmHg, with or without antihypertensive medications.\n* For male agrees to be abstinent from penile-vaginal intercourse OR agrees to use a highly effective contraceptive method while receiving study drug and for 7 days after the last dose of lenvatinib.\n* Is female and not pregnant\u002Fbreastfeeding and at least one of the following applies during the study and for ≥4 days after: is not a woman of childbearing potential (WOCBP), is a WOCBP and uses highly effective contraception (low user dependency method OR a user dependent hormonal method in combination with a barrier method) or is a WOCBP who is abstinent from heterosexual intercourse.\n\nExclusion Criteria:\n\n-There are no exclusion criteria to participate in MK-3475-587.\n\nParticipants are excluded from entering Second Course trial treatment once they are enrolled on MK-3475-587 if any of the following criteria applies:\n\n* Has severe hypersensitivity (≥ Grade 3) to pembrolizumab and\u002For any of its excipients.\n* Has received a live vaccine within 30 days prior to the first dose of Second Course Phase trial treatment.\n* Has a diagnosis of immunodeficiency or is receiving chronic systemic steroid therapy (in dosing exceeding 10 mg daily of prednisone equivalent) or any other form of immunosuppressive therapy within 7 days prior to the Cycle 1 Day 1 of Second Course Phase.\n* Has a known additional malignancy that is progressing or requires active treatment. Exceptions include early stage cancers (carcinoma in situ or Stage 1) treated with curative intent, melanoma (non-ulcerated, thin primary), basal cell carcinoma of the skin, squamous cell carcinoma of the skin, in situ cervical cancer, or in situ breast cancer that has undergone potentially curative therapy.\n* Has known active central nervous system metastases and\u002For carcinomatous meningitis.\n* Has an active autoimmune disease that has required systemic treatment in the past 2 years (i.e., use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (e.g. thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency) is not considered a form of systemic treatment and is allowed.\n* Has a history of (non-infectious) pneumonitis that required steroids or has current pneumonitis. Note: Participants that experienced pneumonitis during First Course that did not meet the criteria for permanent discontinuation are eligible.\n* Non-small cell lung cancer (NSCLC) participants only: Has interstitial lung disease.\n* Has an active infection requiring systemic therapy.\n* Has a known history of human immunodeficiency virus (HIV) infection.\n* Has a known history of or is positive for hepatitis B or hepatitis C. For parent studies where inclusion of participants with hepatitis was permitted, MK-3475-587 will follow the parent study eligibility criteria for hepatitis.\n* Is pregnant or breastfeeding or expecting to conceive or father children within the projected duration of the study, starting with the Second Course Phase eligibility Visit through 120 days after the last dose of study treatment.\n* Has severe cardiovascular disease, i.e., arrhythmias, requiring chronic treatment, congestive heart failure (New York Heart Association Class III or IV) or symptomatic ischemic heart disease.\n* Has hepatic decompensation (Child-Pugh score \\>6 \\[class B and C\\]).\n* Has uncontrolled thyroid dysfunction.\n* Has uncontrolled diabetes mellitus.\n* Has had an allogeneic tissue\u002Fsolid organ transplant.\n* Has a known history of active tuberculosis (TB; Bacillus tuberculosis).\n\nAdditional exclusion criteria for participants who enter dosing with Lenvatinib:\n\n* Has had major surgery within 3 weeks prior to first dose of study intervention(s).\n* Has preexisting ≥Grade 3 gastrointestinal or non-gastrointestinal fistula.\n* Has urine protein ≥1 g\u002F24 hours.\n* Has LVEF below the institutional (or local laboratory) normal range, as determined by multigated acquisition scan (MUGA) or echocardiogram (ECHO).\n* Has radiographic evidence of encasement or invasion of a major blood vessel, or of intratumoral cavitation.\n* Prolongation of QT intervals corrected for heart rate using Fridericia's (cube root) correction (QTcF) interval to \\>480 ms.\n* Has clinically significant cardiovascular disease within 12 months from first dose of study intervention, including New York Heart Association Class III or IV congestive heart failure, unstable angina, myocardial infarction, cerebral vascular accident, or cardiac arrhythmia associated with hemodynamic instability.\n* Gastrointestinal malabsorption or any other condition that might affect the absorption of lenvatinib.\n* Active hemoptysis (bright red blood of at least 0.5 teaspoon) within 3 weeks prior to the first dose of study drug.\n* Has a history of any contraindication or has a severe hypersensitivity to any components of lenvatinib.",{"count":597,"type":23},3500,[63],"The purpose of this study is to evaluate the long-term safety and efficacy of pembrolizumab (MK-3475) in participants from previous Merck pembrolizumab-based parent studies who transition into this extension study.\n\nThis study will consist of three phases: 1) First Course Phase, 2) Survival Follow-up Phase or 3) Second Course Phase. Each participant will transition to this extension study in one of the following three phases, depending on the study phase they were in at the completion of the parent study. Participants who were in the First Course Phase of study treatment with pembrolizumab or lenvatinib in their parent study will enter the First Course Phase of this study and complete up to 35 doses or more every 3 weeks (Q3W) or 17 doses or more every 6 weeks (Q6W) of study treatment with pembrolizumab or a pembrolizumab-based combination or lenvatinib according to arm assignment. Participants who were in the Follow-up Phase in the parent study (post-treatment or Survival Follow-up Phase) will enter the Survival Follow-up Phase of this study. Participants who were in the Second Course Phase in their parent study will enter Second Course Phase of this study and complete up to 17 doses Q3W or 8 doses Q6W of study treatment with pembrolizumab or a pembrolizumab-based combination according to arm assignment.\n\nAny participant originating from a parent trial where crossover to pembrolizumab was permitted upon disease progression may be eligible for 35 doses as Q3W or 17 doses Q6W of pembrolizumab (approximately 2 years), if they progress while on the control arm and pembrolizumab is approved for the indication in the country where the potential eligible crossover participant is being evaluated.",[601,602],"Solid Tumors","Hematologic Malignancies",[604,605,606,607],"PD1","PD-1","PDL1","PD-L1",{"date":41,"type":42},{"date":610,"type":42},"2018-08-21",{"date":612,"type":23},"2043-08-04",{"name":614,"class":87},"Merck Sharp & Dohme LLC",782,{"id":617,"slug":618,"hasResults":12,"nctId":619,"briefTitle":620,"officialTitle":621,"acronym":622,"eligibilityCriteria":623,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":624,"targetDuration":4,"studyType":60,"phases":626,"briefSummary":627,"conditions":628,"keywords":631,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":634,"lastUpdatePostDateStruct":635,"startDateStruct":636,"completionDateStruct":638,"leadSponsor":640,"locationsCount":642},"100639449","phase-3-a-study-to-compare-elritercept-to-placebo-in-adults-with-myelofibrosis-and-anemia-who-are-taking-ruxolitinib-100639449","NCT07623161","A Study to Compare Elritercept to Placebo in Adults With Myelofibrosis and Anemia Who Are Taking Ruxolitinib","A Phase 3, Double-Blind, Randomized Trial Evaluating the Efficacy and Safety of Elritercept (TAK-226) Compared to Placebo in Participants With Myelofibrosis and Anemia on Concurrent Ruxolitinib Therapy","ELRISE MF","Inclusion Criteria:\n\n1. Aged ≥18 years at the time of signing the informed consent form (ICF).\n2. Able to understand the purpose and risks of the trial and voluntarily sign an ICF.\n3. Diagnosed with primary myelofibrosis (PMF), post-essential thrombocythemia (post-ET MF) or post-polycythemia vera (post-PV MF) according to the 2022 WHO criteria (WHO Classification of Tumours Editorial Board 2024), confirmed by local pathology report.\n4. Transfusion status as assessed in the 12 weeks immediately preceding randomization classified as Transfusion Dependent: 3 to 8 RBC units over 12 weeks.\n5. Receiving ruxolitinib (as approved in the country of the trial site) as the standard of care treatment for MF for at least 12 consecutive weeks, and on a stable daily dose for at least the 8 weeks immediately preceding the date of randomization.\n6. Eastern Cooperative Oncology Group score less than or equal to (≤) 2.\n\nExclusion Criteria:\n\n1. Prior treatment with luspatercept, sotatercept, or other transforming growth factor beta inhibitors or activin receptor ligand traps.\n2. Systemic treatment within 28 days before randomization with any of the following:\n\n   1. Androgens (including danazol). Participants on stable androgen dosing for hypogonadism for ≥8 weeks are allowed.\n   2. erythropoiesis-stimulating agents.\n   3. granulocyte colony stimulating factor or granulocyte-macrophage colony stimulating factor.\n   4. High dose corticosteroids. Participants on stable chronic steroid doses of prednisone ≤10 mg\u002Fday or corticosteroid equivalent for ≥4 weeks are allowed. Other treatments for autoimmune diseases may be allowed upon medical monitor review.\n   5. Hydroxyurea.\n   6. Immunomodulatory drugs (for example, thalidomide, pomalidomide, or lenalidomide).\n   7. Interferon.\n   8. Thrombopoietin receptor agonists.\n   9. Any investigational drug, including antihemojuvelin antibody. If the half-life of the investigational product is known, the exclusionary period prior to randomization is equal to 5 half-lives of the investigational product or 28 days, whichever is longer.\n3. Initiation of new iron chelation therapy or dose adjustments to existing iron chelation therapy ≤8 weeks prior to randomization. Participants on stable doses of iron chelation therapy for ≥8 weeks are allowed.\n4. Clinically significant anemia that is due to causes other than MF or Janus kinase (JAK) inhibitor therapy (for example, thalassemia, iron deficiency, vitamin B12 and\u002For folate deficiencies, autoimmune or hemolytic anemia, infections, or any active clinically significant bleeding or sequestration).\n5. Receipt of RBC transfusion for any reason(s) other than underlying MF within 12 weeks before randomization.\n6. Life expectancy \\\u003C12 months per investigator's judgment.\n7. Clinically significant cardiovascular disease, defined as:\n\n   1. New York Heart Association heart disease Class III or IV;\n   2. Fridericia corrected QT interval \\>500 millisecond (ms) during screening;\n   3. Uncontrolled arrhythmia, myocardial infarction, or unstable angina within 6 months before screening.\n8. Uncontrolled hypertension, defined as repeated elevations of systolic blood pressure of ≥160 millimetres of mercury (mmHg) and\u002For diastolic blood pressure ≥100 mmHg despite adequate treatment.\n9. Medical history of thromboembolic events within 6 months before screening, including history of cerebrovascular accident (including ischemic, embolic, and hemorrhagic cerebrovascular accident), transient ischemic attack, deep venous thrombosis (including proximal and distal), pulmonary or arterial embolism, arterial thrombosis, or other venous thrombosis. Participants with prior superficial thrombophlebitis are allowed.\n10. Prior history of malignancies, other than MF. Participants who are free of other malignant disease for ≥2 years and have completed treatment, including maintenance, are allowed. Participants with a history or concurrent diagnosis of the following conditions are allowed if not requiring systemic therapy:\n\n    1. Basal or squamous cell carcinoma of the skin;\n    2. Carcinoma in situ of the cervix;\n    3. Carcinoma in situ of the breast; and\u002For\n    4. Incidental histologic finding of prostate cancer (T1a or T1b using the Tumour, Node, and Metastasis (TNM) staging system);\n    5. Early papillary thyroid cancer (stage I \\[T1-T2, N0, M0\\]).\n11. History of solid organ or bone marrow transplantation.\n12. Active infection requiring intravenous antibiotics within 28 days or oral antibiotics within 7 days before randomization. Prophylactic antibiotics and\u002For antifungals for neutropenia are allowed.\n13. Known positivity for Human Immunodeficiency Virus (HIV), active hepatitis B virus (HBV), or active hepatitis C virus (HCV). Participants without known history of HIV, HBV, and\u002For HCV do not require further testing, unless testing is mandated per local guidelines.\n14. Body mass index ≥40 kilograms per square meter (kg\u002Fm\\^2).\n15. Major surgery within 28 days before randomization.\n16. History of allergy\u002Fanaphylaxis to recombinant proteins, investigational product, or excipients (refer to the current elritercept investigator's brochure for a list of excipients), or ruxolitinib.\n17. Any of the following local laboratory abnormalities:\n\n    1. Absolute neutrophil count \\\u003C500\u002Fmicroliter (μL) (0.5×109\u002F liter (L)).\n    2. Platelet count \\\u003C50,000\u002FμL (50×109\u002FL) or \\>1,000,000\u002FμL (1000×109\u002FL).\n    3. Blasts \\>5% as assessed in peripheral blood at screening or ≥10% in any historical bone marrow assessments. Participants with isolated transient elevations of peripheral blood blasts may be eligible after discussion between the investigator and medical monitor to confirm the blast count is not indicative of disease progression.\n    4. Serum aspartate aminotransferase or alanine aminotransferase ≥3× the upper limit of normal (ULN).\n    5. Total bilirubin ≥2×ULN. Participants with known history of Gilbert syndrome with unconjugated bilirubin less than (\\\u003C) 3×ULN are allowed. Higher levels if attributed to active RBC precursor destruction within the bone marrow (ineffective erythropoiesis) may be allowed upon medical monitor review.\n    6. Estimated glomerular filtration rate \\\u003C30 milliliters per minute per 1.73 square meters (mL\u002Fmin\u002F1.73 m\\^2) as determined by the Chronic Kidney Disease Epidemiology Collaboration equation.\n    7. Ferritin ≤50 micrograms per liter (μg\u002FL).\n    8. Folate ≤2.0 nanograms per milliliter (ng\u002FmL).\n    9. Vitamin B12 ≤200 picograms per milliliter (pg\u002FmL).\n18. Ongoing participation in another interventional clinical trial.\n19. Participant is unwilling or, in the opinion of the investigator, the participant is unable to comply with the requirements of the protocol.\n20. Is a person of childbearing potential but does not agree to use at least 1 form of highly effective contraception from the time of signing the ICF until at least 60 days after the last dose of elritercept or placebo.\n21. Participants of male birth who are fertile and who have partners of childbearing potential, who do not agree to use acceptable barrier contraception, that is, a male condom, during the entire treatment period until at least 60 days after the last dose of elritercept or placebo.\n22. If applicable, participant with a positive serum pregnancy test during the screening period or known to be pregnant or a lactating participant who does not agree to forego breastfeeding during the entire treatment period until at least 60 days after the last dose of elritercept or placebo.\n23. For participants in France: Persons under court protection, persons not affiliated with a social security system, and protected adults.",{"count":625,"type":23},324,[63],"The main aim of this study is to find out how well elritercept works to improve anemia in participants with myelofibrosis (MF) who are taking ruxolitinib when compared to placebo.\n\nOther aims are to learn how elritercept improves anemia compared to placebo; to learn if elritercept reduces tiredness, improves symptoms related to MF, and helps participants do physical activities more easily. The study also aims to find out how elritercept affects the bone marrow, the spleen, and whether participants develop antibodies to the study drug.\n\nThe study will also check how safe elritercept is compared to placebo, and if elritercept stays safe over a long period of time. Participants will receive study treatment for at least 9 months (36 weeks). After this period, participants who received placebo will have the option to switch to elritercept.",[629,630],"Myelofibrosis","Anemia",[632,633],"TAK-226","Drug therapy","2026-08-20",{"date":39,"type":42},{"date":637,"type":23},"2026-09-02",{"date":639,"type":23},"2034-03-30",{"name":641,"class":87},"Takeda",195,{"id":644,"slug":645,"hasResults":12,"nctId":646,"briefTitle":647,"officialTitle":648,"acronym":4,"eligibilityCriteria":649,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":126,"enrollmentInfo":650,"targetDuration":4,"studyType":60,"phases":652,"briefSummary":653,"conditions":654,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":634,"lastUpdatePostDateStruct":658,"startDateStruct":659,"completionDateStruct":661,"leadSponsor":663,"locationsCount":664},"100635042","a-study-of-ly3971297-in-participants-with-heart-failure-100635042","NCT07547540","A Study of LY3971297 in Participants With Heart Failure","A Phase 1, Single-Blinded, Single-Ascending Dose Study to Evaluate the Safety and Tolerability of a Single Dose of LY3971297 in Participants With HFpEF and Participants With HFrEF","Inclusion Criteria:\n\n* Are diagnosed with chronic heart failure with New York Heart Association Class II-III (Heart Failure) HF symptomatology at screening and on guideline-directed HF therapy for at least 6 months prior to screening.\n* Have not changed optimal guideline-directed HF therapy, either medication or medication dose, in the last 4 weeks prior to screening and during screening period, and do not plan to change HF therapy for the next 90 days.\n* Must be on a stable dose of vasodilator therapy for at least 4 weeks prior to screening, with no dose adjustments planned during the study.\n* Have an estimated glomerular filtration rate of greater than or equal to (≥) 30 milliliter per minute per 1.73 square meters (mL\u002FMinute\u002F1.73m²) at screening.\n* Have systolic blood pressure (SBP) greater than (\\>) 110 millimeters of mercury (mmHg) at screening and at enrollment.\n* Have a body mass index within the range of 18.5 to 40 kilograms per square meter (kg\u002Fm²) (inclusive).\n* Are individuals assigned male or female at birth, who are not of childbearing potential.\n* Have venous access sufficient to allow blood sampling.\n* Applicable to heart failure with preserved ejection fraction (HFpEF) participants only\n\n  * Have left ventricular ejection fraction (LVEF) \\>45 percent (%).\n  * Left atrial volume index \\>34 milliliters per square meter (mL\u002Fm²) in participants in sinus rhythm, or \\>40 mL\u002Fm² in participants with atrial fibrillation (AF).\n  * N-terminal pro-B-type natriuretic peptide (NT-proBNP) \\>300 picograms per milliliter (pg\u002FmL) for participants without AF or \\>850 pg\u002FmL for participants with AF.\n  * Have a documented history of signs, symptoms, or both, consistent with HFpEF.\n* Applicable to heart failure with reduced ejection fraction (HFrEF) participants only:\n\n  * Have LVEF \\\u003C40% .\n  * NT-proBNP \\>600 pg\u002FmL for participants without AF or \\>900 pg\u002FmL for participants with AF.\n  * Have a documented history of signs, symptoms, or both, consistent with HFrEF.\n\nExclusion Criteria:\n\n* Have known allergies to related compounds of LY3971297 or any components of the formulation, or a history of significant atopy.\n* Had a myocardial infarction, unstable angina pectoris, coronary artery bypass graft surgery, revascularization or other major cardiovascular surgery, stroke, or transient ischemic attack in the last 90 days prior to screening.\n* Have New York Heart Association (NYHA) Class 4, acute decompensated HF (exacerbation of HF) requiring IV diuretics, IV inotropes, or IV vasodilators, within 30 days prior to screening, and\u002For during screening period until randomization.\n* Have SBP ≥180 mmHg at screening.\n* Have symptomatic hypotension.\n* Have resting heart rate \\>90 beats per minute (bpm) at screening.\n* Have known cardiac amyloidosis, infiltrative myocardial diseases, muscular dystrophies, cardiomyopathy with reversible causes, hypertrophic cardiomyopathy, pericardial constriction, or complex congenital heart disease.\n* Have any history of moderate-to-severe stenosis of the mitral and\u002For aortic valve or severe mitral and\u002For aortic regurgitation.\n* Have any history of moderate-to-severe tricuspid or pulmonic valve stenosis or severe tricuspid or pulmonic regurgitation.\n* Have a history of syncope that, in the opinion of the investigator, may affect the participant's safety.\n* Bioprosthetic valve replacement within 12 months prior to screening or any history of mechanical valve replacement, or planned valve replacement or repair during the study period.\n* Have any history of greater than moderate pulmonary hypertension.\n* Have a pacemaker or implantable cardioverter-defibrillator placement within 90 days prior to screening.\n* Have severe chronic obstructive pulmonary disease (COPD).\n* Have clinically significant or uncontrolled cardiac arrhythmia.\n* Have a significant history of, or presence of, hepatic disease, including any abnormal liver function tests.\n* Have, within 3 years prior to screening, a history of an active or untreated malignancy or are in remission from a clinically significant malignancy (Exceptions: basal or squamous cell skin cancer).\n* For US sites: have donated blood of more than 500 mL within the previous 90 days of screening or intend to donate blood during the course of the study.\n* For Japan sites: have donated any blood within the last 4 weeks, any apheresis (blood components) within the last 2 weeks, at least 400 mL of blood within the last 16 weeks for female participants or 12 weeks for male participants, or at least 800 mL of blood for female participants or 1200 mL of blood for male participants within 12 months.\n* Other sites: Participants who have recently donated blood or blood components, or who intend to donate during the course of the study.\n* Have not been on a stable dose of medications for at least 4 weeks prior to screening, or have planned dose adjustments during the study.\n* Participants must abstain from taking new prescription or nonprescription drugs.\n* Have concurrent use or intend to use phosphodiesterase 5 inhibitor or soluble guanylyl cyclase activators.\n* Have any history of intolerance to vasodilator medications that, in the opinion of the investigator, would put them at risk of not tolerating study drug.\n* Have BP and\u002For pulse rate constituting a risk when taking the Investigational Medicinal Product (IMP).\n* Are diagnosed with orthostatic hypotension.\n* Show evidence of an acute infection with fever or infectious disease at screening.\n* Applicable to HFrEF participants only\n\n  * Have been listed for cardiac transplantation and\u002For anticipated or implanted ventricular assist device.\n  * Have received cardiac resynchronization therapy for less than 6 months.\n* Hospitalization for heart failure within 30 days of screening.",{"count":651,"type":23},90,[511],"The main purpose of this study is to assess how well LY3971297 is tolerated and what side effects may occur in participants with heart failure with preserved ejection fraction (HFpEF) and participants with heart failure with reduced ejection fraction (HFrEF). Blood tests will be performed to investigate how the body processes the study drug and how the study drug affects the body. For each participant, the study will last about 2 months and will include 1 inpatient visit lasting approximately 4 days and 5 outpatient visits.",[655,656,657],"Heart Failure","Heart Failure, Diastolic","Heart Failure, Systolic",{"date":221,"type":42},{"date":660,"type":42},"2026-08-11",{"date":662,"type":23},"2027-11",{"name":256,"class":87},12,{"id":666,"slug":667,"hasResults":12,"nctId":668,"briefTitle":669,"officialTitle":670,"acronym":671,"eligibilityCriteria":672,"healthyVolunteers":12,"sex":18,"minAge":673,"maxAge":287,"enrollmentInfo":674,"targetDuration":4,"studyType":60,"phases":676,"briefSummary":677,"conditions":678,"keywords":680,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":634,"lastUpdatePostDateStruct":690,"startDateStruct":691,"completionDateStruct":693,"leadSponsor":695,"locationsCount":7},"100626918","phase-2-study-to-evaluate-the-efficacy-and-safety-of-bmn-333-versus-vosoritide-in-children-with-achondroplasia-100626918","NCT07441876","Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia","A Multicenter, Randomized, Operationally Seamless Phase 2\u002F3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia","ASPEN","Inclusion Criteria:\n\n1. Participants must be aged ≥ 2 to \\\u003C 11 years (Phase 2) or ≥ 2 to \\\u003C 18 years (Phase 3), at the time of signing the informed consent\n2. Participants must have ACH (confirmed by documented genetic testing) and open epiphyses\n3. Are Tanner Stage I (Phase 2) or any Tanner stage (Phase 3)\n4. Are ambulatory and able to stand without assistance\n\nExclusion Criteria:\n\n1. Have any short stature condition other than ACH (eg, hypochondroplasia, trisomy 21, pseudoachondroplasia, GH deficiency)\n2. Have any of the following disorders: Hypothyroidism or hyperthyroidism, unless treated with evidence of normalized thyroid-stimulating hormone (TSH) levels, diabetes mellitus, unless considered well-controlled, autoimmune inflammatory disease, inflammatory bowel disease, autonomic neuropathy, anemia defined as hemoglobin \\\u003C 10 g\u002FdL, vitamin D deficiency, significant hip pathology.\n3. Have history of any renal insufficiency or cardiac\u002F cardiovascular disease that places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension.\n4. Have had bone fractures of the long bones or spine within 6 months prior to screening.\n5. Have used vosoritide, any other approved product (except GH, as detailed below), investigational product, or investigational medical device for the treatment of ACH or short stature at any time\n6. Have been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start","2 Years",{"count":675,"type":23},160,[62,63],"This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2\u002F3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH). The study consists of a Phase 2 part and a Phase 3 part.",[679],"Achondroplasia",[679,681,682,683,684,685,686,687,688,689],"ACH","Bone Diseases, Developmental Dwarfism","Bone Diseases","Genetic Diseases, Inborn","Musculoskeletal Diseases","Natriuretic Peptide, C-type","Osteochondrodysplasias","Physiological Effects of Drugs","Skeletal Dysplasias",{"date":221,"type":42},{"date":692,"type":42},"2026-04-20",{"date":694,"type":23},"2029-09",{"name":696,"class":87},"BioMarin Pharmaceutical",""]