[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Otsuka Pharmaceutical Development & Commercialization, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":386},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,15,0,[8,40,67,92,114,138,159,190,213,233,256,283,317,341,363],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":4},"100652123","phase-3-trial-of-sibeprenlimab-in-children-and-adolescents-with-immunoglobulin-a-nephropathy-igan-100652123",false,"NCT07770815","Trial of Sibeprenlimab in Children and Adolescents With Immunoglobulin A Nephropathy (IgAN)","A Phase 3, Open-label, Single-arm, Repeat-dose Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Sibeprenlimab Administered Subcutaneously in Children and Adolescents With Immunoglobulin A Nephropathy","1. Source-verified, biopsy-confirmed IgA nephropathy (IgAN).\n2. Receiving a stable and maximally tolerated dose of authorized angiotensin converting enzyme inhibitors (ACEIs) and\u002For angiotensin receptor blockers (ARBs) for at least 12 weeks.\n3. Participants receiving a stable dose of sodium-glucose cotransporter-2 inhibitor (SGLT2i) therapy for IgAN, if available, in addition to ACEIs and\u002For ARBs, must have initiated SGLT2i treatment for at least 12 weeks.\n4. Participants must have a urine protein\u002Fcreatinine ratio (uPCR) ≥ 0.75 grams per gram (g\u002Fg), as measured from the geometric mean of 3 first void spot urine samples (collected within the screening window prior to Dose 1).\n5. Estimated glomerular filtration rate (eGFR) ≥ 30 milliliters per minute per 1.73 square meters (mL\u002Fmin\u002F1.73 m\\^2).\n6. No evidence of coexisting chronic kidney disease other than IgAN.\n7. Serum immunoglobulin G (IgG) ≥ 600 mg\u002FdL at screening.\n8. Kidney biopsy findings not demonstrating additional pathological features inconsistent with IgAN.","ALL","2 Years","17 Years",{"count":20,"type":21},24,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","This Phase 3, open-label study will evaluate the efficacy, safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of sibeprenlimab administered subcutaneously every 4 weeks in pediatric participants aged 2 to 17 years with IgA nephropathy (IgAN). Sibeprenlimab will be administered as an add-on to standard of care therapy consisting of angiotensin converting enzyme inhibitors (ACEIs) and\u002For angiotensin receptor blockers (ARBs).",[27],"IgA Nephropathy","NOT_YET_RECRUITING","2026-08-13",{"date":31,"type":32},"2026-08-18","ACTUAL",{"date":34,"type":21},"2026-09-30",{"date":36,"type":21},"2030-02-23",{"name":38,"class":39},"Otsuka Pharmaceutical Development & Commercialization, Inc.","INDUSTRY",{"id":41,"slug":42,"hasResults":11,"nctId":43,"briefTitle":44,"officialTitle":45,"acronym":4,"eligibilityCriteria":46,"healthyVolunteers":11,"sex":16,"minAge":47,"maxAge":4,"enrollmentInfo":48,"targetDuration":4,"studyType":22,"phases":50,"briefSummary":51,"conditions":52,"keywords":54,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":66},"100652018","phase-3-a-trial-of-the-efficacy-and-safety-of-a-fixed-dose-of-sep-363856-in-adults-with-generalized-anxiety-disorder-100652018","NCT07767903","A Trial of the Efficacy and Safety of a Fixed Dose of SEP-363856 in Adults With Generalized Anxiety Disorder","A Phase 3, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled, Multicenter Trial to Compare the Efficacy and Safety of a Fixed Dose of SEP-363856 to Placebo in the Treatment of Adults With Generalized Anxiety Disorder","Key Inclusion Criteria\n\n1. Male or female participants at least 18 years of age at the time of informed consent\n2. Primary diagnosis of GAD per Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5), confirmed by Mini-International Neuropsychiatric Interview (MINI) and clinical psychiatric evaluation\n3. Body Mass Index (BMI) 18-40 kilograms per square meter (kg\u002Fm\\^2)\n4. Participant is willing and able to comply with study procedures and visit schedule\n5. Medically stable as determined by medical history, physical exam, vital signs, and laboratory tests and able to comply with study procedures\n6. Willing and able to discontinue prohibited medications\u002Ftherapies per protocol\n7. (i ) Females of childbearing potential: negative pregnancy test and agreement to use acceptable contraception (ii) Males: agreement to follow protocol-defined contraception and reproductive restrictions\n8. Willing and able to discontinue prohibited medications\u002Ftherapies per protocol\n\nKey Exclusion Criteria\n\n1. Current DSM-5 diagnosis of major depressive episode, post traumatic stress disorder (PTSD), or any psychiatric disorder other than GAD that was the primary focus of treatment within 12 months prior to screening\n2. Substance use disorder (excluding nicotine\u002Fcaffeine) within 12 months prior to screening\n3. Lifetime history of certain psychiatric disorders (e.g., schizophrenia spectrum disorders, bipolar disorder, obsessive-compulsive disorder, borderline or antisocial personality disorder)\n4. Significant suicide risk e.g., recent suicidal behavior\u002Fideation per Columbia-Suicide Severity Rating Scale \\[C-SSRS\\] or MADRS)\n5. Use of prohibited medications\u002Ftherapies within protocol-defined timeframes (e.g., certain antipsychotics, mood stabilizers, benzodiazepines, investigational treatments)\n6. Prior exposure to restricted treatments\n7. Pregnant or breastfeeding females\n8. Participation in another investigational study within 180 days (or more than \\[\\>\\] 1 trial in last year)\n9. Known history of human immunodeficiency virus seropositivity, hepatitis B or C","18 Years",{"count":49,"type":21},384,[24],"This is a global, phase 3, randomized, double-blind, parallel-group, 2-arm, placebo controlled, multicenter trial designed to compare the efficacy, safety and tolerability of a fixed dose of SEP-363856 (75 millligrams per day \\[mg\u002Fday\\]) to placebo over an 8-week double-blind treatment period in adults with generalized anxiety disorder (GAD).",[53],"Generalized Anxiety Disorder",[53,55,56],"GAD","Anxiety","RECRUITING","2026-08-11",{"date":60,"type":32},"2026-08-17",{"date":62,"type":32},"2026-08-07",{"date":64,"type":21},"2028-11-01",{"name":38,"class":39},2,{"id":68,"slug":69,"hasResults":11,"nctId":70,"briefTitle":71,"officialTitle":72,"acronym":4,"eligibilityCriteria":73,"healthyVolunteers":74,"sex":16,"minAge":47,"maxAge":75,"enrollmentInfo":76,"targetDuration":4,"studyType":22,"phases":78,"briefSummary":80,"conditions":81,"keywords":4,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":83,"lastUpdatePostDateStruct":84,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":90,"locationsCount":91},"100647863","phase-1-a-trial-to-examine-if-repinatrabit-is-processed-differently-in-adults-with-reduced-liver-or-kidney-function-compared-to-adults-with-normal-liver-and-kidney-function-100647863","NCT07713758","A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function","A Phase 1, Open-label, Single-dose, Parallel-group Study Evaluating the Pharmacokinetics of Oral Repinatrabit (JNT-517) Immediate Release Tablet in Hepatic or Renal Impaired Adult Participants Matched to Adult Participants With Normal Hepatic and Renal Function","Inclusion criteria:\n\n1. Body mass index (BMI) from 18.0 to 38.0 kilograms per square meter (kg\u002Fm\\^2) (inclusive).\n2. Able to provide written, informed consent.\n3. Male participants and female participants of childbearing potential (POCBP) must agree to remain fully abstinent from intercourse where pregnancy can occur OR practice 2 different contraceptive methods, where at least one must be highly effective.\n4. Otherwise be in generally good health.\n5. Additional criteria for Group 1:\n\n   i. Have moderate hepatic impairment determined using Child-Pugh classification (Class B).\n\n   ii. Have relatively stable hepatic function for the duration of the study as determined by the investigator and liver function test at the time of screening or stable hepatic function 1 year prior to screening.\n\n   iii. Have normal renal function. iv. Have a clinical diagnosis of hepatic cirrhosis based on biopsy and\u002For clinical criteria.\n6. Additional criteria for Group 2:\n\n   i. Have estimated glomerular filtration rate based on the Chronic Kidney Disease-Epidemiology Collaboration (eGFRCKD-EPI) lesser than (\\\u003C) 30 milliliters per minute per 1.73 square meters (mL\u002Fmin\u002F1.73 m\\^2) indicating severe renal impairment.\n\nii. Not currently be on dialysis. iii. Have relatively stable renal function as determined by eGFRCKD-EPI. 7) Additional criteria for Group 3: i. eGFRCKD-EPI greater than or equal to (≥) 90 mL\u002Fmin\u002F1.73 m\\^2 indicating normal renal function.\n\nii. Have liver enzymes within normal limits.\n\nExclusion criteria:\n\n1. Participants who are breastfeeding.\n2. Participants who have a positive pregnancy test result prior to receiving the investigational medical product (IMP).\n3. Participants with international normalized ratio (INR) greater than (\\>) 2.3. Participants with hepatic impairment with elevated INR will be excluded based on the investigator's discretion.\n4. Participants with hepatic carcinoma or porto-hepatic shunts that have been surgically created or planted.\n5. A history of difficulty in obtaining intravenous access.\n6. The donation of blood or plasma \\> 250 milliliters (mL) within 30 days prior to screening or received \\> 50 mL of blood or plasma within 30 days prior to screening.\n7. Participants who have taken an investigational drug within 30 days prior to screening.\n8. Exposure to any substances known to stimulate hepatic microsomal enzymes within 30 days prior to screening (eg, occupational exposure to pesticides, organic solvents, etc).\n9. Any participant who, in the opinion of the investigator, should not participate in the trial.\n10. Previous exposure to repinatrabit.\n11. Additional criteria for Group 1: History of serious mental disorder including participants who have greater than Grade 1 encephalopathy as assessed by the Child-Pugh score and\u002For the investigator's judgment.\n12. Additional criteria for Group 2:\n\n    i) Participants that have undergone a renal transplant. ii) Participants expected to require dialysis within 45 days. iii) Clinically significant abnormal findings in the serum chemistry, hematology, or urinalysis results obtained at screening or on Day -1, other than those associated with underlying renal conditions and other stable medical conditions consistent with the disease processes.\n13. Additional criteria for Group 3: History of or current hepatitis, or carriers of hepatitis B surface antigen (HBsAg) and\u002For hepatitis C antibodies (anti-HC).\n\nNote: Other protocol-specified inclusion\u002Fexclusion criteria may apply.",true,"65 Years",{"count":77,"type":21},32,[79],"PHASE1","This trial is intended to understand how the body processes repinatrabit in people with moderate liver impairment and severe kidney impairment and also to compare these results with people of similar age, weight, and sex who have normal liver function.",[82],"Phenylketonuria","2026-08-10",{"date":85,"type":32},"2026-08-12",{"date":87,"type":32},"2026-07-17",{"date":89,"type":21},"2027-02-15",{"name":38,"class":39},1,{"id":93,"slug":94,"hasResults":11,"nctId":95,"briefTitle":96,"officialTitle":97,"acronym":4,"eligibilityCriteria":98,"healthyVolunteers":74,"sex":16,"minAge":47,"maxAge":99,"enrollmentInfo":100,"targetDuration":4,"studyType":22,"phases":102,"briefSummary":103,"conditions":104,"keywords":106,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":83,"lastUpdatePostDateStruct":108,"startDateStruct":109,"completionDateStruct":111,"leadSponsor":113,"locationsCount":91},"100627266","phase-1-a-trial-to-examine-the-interaction-of-repinatrabit-with-ethinyl-estradiolnorethindrone-metformincarbamazepine-rosuvastatin-and-methotrexate-when-administered-together-100627266","NCT07446400","A Trial to Examine the Interaction of Repinatrabit With Ethinyl Estradiol\u002FNorethindrone, Metformin,Carbamazepine, Rosuvastatin, and Methotrexate When Administered Together","A Phase 1, 4-arm, Open-label, Drug-drug Interaction Trial to Evaluate the Pharmacokinetics of Repinatrabit Oral Tablets When Co-administered With Ethinyl Estradiol\u002FNorethindrone, Metformin, Carbamazepine, Rosuvastatin, and Methotrexate in Healthy Participants","Inclusion Criteria:\n\n1. Body mass index (BMI) from 18 to 35 kilograms per square meter (kg\u002Fm\\^2) (inclusive).\n2. Male and female (of non-childbearing potential) assigned at birth, inclusive of all gender identities. Arm 1 will only recruit biological females.\n3. In good health as determined by:\n\n   1. Medical history\n   2. Physical examination\n   3. ECG\n   4. Serum chemistry, urinalysis, hematology, and serology tests\n4. Willing to stay in the clinic for the required period and willing to be contacted for the safety follow-up via telephone contact.\n5. Ability to provide written, informed consent prior to initiation of any trial-related procedures, and ability, in the opinion of the principal investigator, to comply with all the requirements of the trial.\n\nExclusion Criteria:\n\n1. Female participants of childbearing potential.\n2. Female participants who have used HRT within 60 days or hormonal contraceptives within 14 days prior to Day 1 (Arm 1 only).\n3. Clinically significant abnormality in past medical history, or at the screening physical examination, that in the investigator's or sponsor's opinion may place the participant at risk or interfere with outcome variables including absorption, distribution, metabolism, and excretion of drug. This includes, but is not limited to, history of or concurrent cardiac, hepatic, renal, neurologic, endocrine, gastrointestinal, respiratory, hematologic, and immunologic disease or cholecystectomy.\n4. For Arm 3, participants who test positive for HLA haplotypes associated with carbamazepine-induced hypersensitivity (HLA-B\\*15:02, HLA-A\\*31:01, and HLA-B\\*15:11).\n\nNote: Other protocol-specified Inclusion\u002FExclusion criteria may apply.","55 Years",{"count":101,"type":21},48,[79],"The purpose of this study is to assess the drug-drug interaction (DDI) of repinatrabit with ethinyl estradiol\u002Fnorethindrone or norethisterone (EE\u002FNE), metformin, rosuvastatin, carbamazepine, and methotrexate in healthy participants.",[105],"Healthy Volunteers",[82,107],"Drug-Drug Interaction (DDI) study",{"date":85,"type":32},{"date":110,"type":32},"2026-03-31",{"date":112,"type":21},"2026-11-07",{"name":38,"class":39},{"id":115,"slug":116,"hasResults":11,"nctId":117,"briefTitle":118,"officialTitle":119,"acronym":4,"eligibilityCriteria":120,"healthyVolunteers":11,"sex":16,"minAge":47,"maxAge":75,"enrollmentInfo":121,"targetDuration":4,"studyType":22,"phases":123,"briefSummary":124,"conditions":125,"keywords":128,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":131,"lastUpdatePostDateStruct":132,"startDateStruct":133,"completionDateStruct":135,"leadSponsor":137,"locationsCount":4},"100651465","phase-3-a-study-to-test-the-effects-of-centanafadine-in-adults-with-attention-deficithyperactivity-disorder-and-emotional-dysregulation-100651465","NCT07759154","A Study to Test the Effects of Centanafadine in Adults With Attention-deficit\u002FHyperactivity Disorder and Emotional Dysregulation","A Phase 3b, Multicenter, Open-label, Single-arm Trial to Evaluate the Efficacy and Safety of Orally Administered Centanafadine QD XR Capsules in Adults With Attention-deficit\u002FHyperactivity Disorder and Emotional Dysregulation","Inclusion Criteria\n\n1. Primary diagnosis of ADHD per the Adult ADHD Clinical Diagnostic Scale (ACDS).\n2. Adult ADHD Investigator Symptom Rating Scale (AISRS) total score of ≥ 28 at baseline.\n3. Symptoms of ED as determined by the WRAADDS-ED subscale score ≥ 7 at baseline.\n4. CGI-S-ADHD rating ≥ 4 (at least moderate severity) at baseline.\n\nExclusion Criteria\n\n1. Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) criteria for a lifetime diagnosis of bipolar and related disorders, schizophrenia spectrum and other psychotic disorders, borderline and antisocial personality disorders, neurocognitive disorders, autism spectrum disorder, or intellectual disability.\n2. DSM-5 criteria for a current diagnosis of major depressive disorder, post-traumatic stress disorder, any substance use disorder, untreated\u002Funstable obstructive sleep apnea, eating disorders, or obsessive compulsive disorder.\n3. Any other current DSM-5 comorbid psychiatric disorder identified by the Mini International Neuropsychiatric Interview (MINI) or any medical condition that, in the judgment of the investigator, could be expected to require treatment with medications prohibited in this trial that cannot be safely discontinued with completion of an appropriate washout, could confound efficacy or safety assessments, or be the primary driver of ED.\n4. Presence of an unstable or uncontrolled medical condition that, in the opinion of the investigator, could pose a safety risk to the participant or could manifest with psychiatric symptoms (eg, thyroid disease).\n5. In the clinical opinion of the investigator, the participant has not derived significant therapeutic benefit from 2 or more ADHD therapies of 2 different classes (eg, amphetamine and methylphenidate, or amphetamine and atomoxetine) given with an acceptable dose and duration during adulthood (aged 18 years or older).\n6. Current use of prohibited psychotropic medications that cannot be discontinued within 7 to 28 days prior to enrollment.\n7. Any disorder that is the primary focus of treatment other than ADHD.\n8. Evidence of current substance use disorder or history in the past 12 months.\n9. History of any prior exposure to centanafadine.\n10. Participated in other clinical trials involving investigational drugs within 180 days prior to screening or participated in more than 2 interventional clinical trials involving investigational drugs within the past year.\n11. Have an allergy to the IMP or any component of the IMP.\n\nNote: Other protocol-specified inclusion\u002Fexclusion criteria may apply.",{"count":122,"type":21},80,[24],"The primary purpose of this study is to evaluate the efficacy of centanafadine once daily (QD) extended-release (XR) capsules in adults with attention-deficit\u002Fhyperactivity disorder (ADHD) and emotional dysregulation (ED).",[126,127],"Attention-deficit\u002FHyperactivity Disorder","Emotional Dysregulation",[129,130],"ADHD","ED","2026-08-06",{"date":85,"type":32},{"date":134,"type":21},"2026-08-20",{"date":136,"type":21},"2027-05-27",{"name":38,"class":39},{"id":139,"slug":140,"hasResults":11,"nctId":141,"briefTitle":142,"officialTitle":143,"acronym":4,"eligibilityCriteria":144,"healthyVolunteers":11,"sex":16,"minAge":47,"maxAge":4,"enrollmentInfo":145,"targetDuration":4,"studyType":22,"phases":147,"briefSummary":148,"conditions":149,"keywords":150,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":131,"lastUpdatePostDateStruct":152,"startDateStruct":153,"completionDateStruct":155,"leadSponsor":157,"locationsCount":158},"100651378","phase-3-a-trial-of-the-efficacy-and-safety-of-fixed-doses-of-sep-363856-in-adults-with-generalized-anxiety-disorder-100651378","NCT07759128","A Trial of the Efficacy and Safety of Fixed Doses of SEP-363856 in Adults With Generalized Anxiety Disorder","A Phase 3, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled, Multicenter Trial to Compare the Efficacy and Safety of Fixed Doses of SEP-363856 to Placebo in the Treatment of Adults With Generalized Anxiety Disorder","Key Inclusion Criteria:\n\n1. Male or female participants at least 18 years of age at the time of informed consent\n2. Primary diagnosis of Generalized Anxiety Disorder (GAD) per Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5), confirmed by Mini-International Neuropsychiatric Interview (MINI) and clinical psychiatric evaluation\n3. Body Mass Index (BMI) between 18 and 40 kilograms per square meter (kg\u002Fm\\^2) (inclusive)\n4. Participant is willing and able to comply with study procedures and visit schedule\n5. Medically stable as determined by medical history, physical exam, vital signs, and laboratory tests\n6. Willing and able to discontinue prohibited medications\u002Ftherapies per protocol\n7. i) Females of childbearing potential: negative pregnancy test and agreement to use acceptable contraception ii) Males: agreement to follow protocol-defined contraception and reproductive restrictions\n8. Participant is willing and able to safely discontinue all prohibited medications\u002Ftherapies\u002Fherbal supplements in accordance with the protocol-required washouts prior to baseline\u002FDay 1 and during the trial period.\n\nKey Exclusion Criteria:\n\n1. Current DSM-5 diagnosis of major depressive episode, post traumatic stress disorder (PTSD), or any psychiatric disorder other than GAD that was the primary focus of treatment within 12 months prior to screening\n2. Substance use disorder (excluding nicotine\u002Fcaffeine) within 12 months prior to screening\n3. Lifetime history of certain psychiatric disorders (e.g., schizophrenia spectrum disorders, bipolar disorder, obsessive-compulsive disorder, borderline or antisocial personality disorder)\n4. Significant suicide risk (e.g., recent suicidal behavior\u002Fideation per Columbia-Suicide Severity Rating Scale \\[C-SSRS\\] or MADRS)\n5. Use of prohibited medications\u002Ftherapies within protocol-defined timeframes (e.g., certain antipsychotics, mood stabilizers, benzodiazepines, investigational treatments)\n6. Prior exposure to restricted treatments\n7. Pregnant or breastfeeding females\n8. Participation in another investigational study within 180 days (or more than \\[\\>\\] 1 trial in last year)\n9. Known history of human immunodeficiency virus seropositivity, hepatitis B or C",{"count":146,"type":21},576,[24],"This is a global, phase 3, randomized, double-blind, parallel-group, 3-arm, placebo-controlled, multicenter study designed to compare the efficacy, safety, and tolerability of fixed doses of SEP-363856 (50 and 75 milligrams per day \\[mg\u002Fday\\]) to placebo in adults at least 18 years of age with generalized anxiety disorder (GAD) over 8 weeks.",[53],[53,55,151],"anxiety",{"date":85,"type":32},{"date":154,"type":32},"2026-07-28",{"date":156,"type":21},"2028-12-13",{"name":38,"class":39},3,{"id":160,"slug":161,"hasResults":11,"nctId":162,"briefTitle":163,"officialTitle":164,"acronym":4,"eligibilityCriteria":165,"healthyVolunteers":11,"sex":16,"minAge":166,"maxAge":47,"enrollmentInfo":167,"targetDuration":4,"studyType":22,"phases":169,"briefSummary":170,"conditions":171,"keywords":173,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":182,"lastUpdatePostDateStruct":183,"startDateStruct":184,"completionDateStruct":186,"leadSponsor":188,"locationsCount":189},"100422548","phase-3-a-study-to-see-if-tolvaptan-is-safe-in-infants-and-children-who-at-enrollment-are-28-days-to-less-than-18-years-old-with-autosomal-recessive-polycystic-kidney-disease-arpkd-100422548","NCT04782258","A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)","A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 18 Years of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)","Inclusion Criteria:\n\n1. Male or female subjects between 28 days and less than 18 years of age, with clinical features that are consistent with a diagnosis of ARPKD.\n2. Ability for parent\u002Flegal guardian to provide written, informed consent prior to initiation of any trial-related procedures, and ability, in the opinion of the principal investigator, to comply with all the requirements of the trial. Ability to provide written informed assent from all subjects old enough per local laws to provide assent.\n\nExclusion Criteria:\n\n1. Premature birth (≤ 32 weeks gestational age) for infants 28 days to \\\u003C 12 weeks of age.\n2. Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation.\n3. Evidence of syndromic conditions associated with renal cysts (other than ARPKD).\n4. Abnormal liver function tests including ALT and AST, \\> 1.2 × ULN (upper limit of normal).\n5. Has splenomegaly or portal hypertension (HTN).\n6. Parents with renal cystic disease.\n7. Receiving chronic diuretic that could not be adjusted after tolvaptan initiation.\n8. Cannot be monitored for fluid balance.\n9. Has or at risk of having sodium and potassium electrolyte imbalances, as determined by the investigator.\n10. Has or at risk of having significant hypovolemia as determined by investigator.\n11. Clinically significant anemia, as determined by investigator.\n12. Platelets \\\u003C 50000 µL.\n13. Severe systolic dysfunction defined as ejection fraction \\\u003C 14%.\n14. Serum sodium levels \\\u003C 130 mmol\u002FL or \\>145 mmol\u002FL.\n15. Taking any other experimental medications.\n16. Require ventilator support.\n17. Taking medications known to induce CYP3A4 (CYP = Cytochrome P).\n18. Having an infection including viral that would require therapy disruptive to IMP (Investigational Medicinal Product) dosing.\n19. Females who are breast-feeding or who have a positive pregnancy test result prior to receiving IMP.\n20. Subjects with a history of substance abuse (within the last 6 months).\n21. Subjects who have bladder dysfunction and\u002For difficulty voiding.\n22. Subjects taking a vasopressin agonist (eg, desmopressin).\n23. Subjects with a history of persistent noncompliance with antihypertensive or other important medical therapy.\n24. Subjects taking medications or having concomitant illnesses likely to confound endpoint assessments, including taking approved (ie, marketed) therapies for the purpose of affecting PKD cysts such as tolvaptan, vasopressin antagonists, anti-sense ribonucleic acid (RNA) therapies, rapamycin, sirolimus, everolimus, or somatostatin analogs (ie, octreotide, sandostatin).\n25. Received or are scheduled to receive a liver transplant.\n26. History of cholangitis within the last 6 months.\n27. Has findings consistent with clinically significant portal hypertension (eg, varices, variceal bleeding, hypersplenism indicated by thrombocytopenia).\n28. Subjects who do not agree to remain abstinent or assent to use a combination of 2 of the following highly effective birth control methods for at least 28 days before the first dose of IMP, during the trial (including during IMP dose interruptions), and for at least 30 days after the last dose of IMP:\n\n    * Barrier method of contraception: condoms (male or female) with or without a spermicidal agent, diaphragm or cervical cap with spermicide\n    * Intrauterine device\n    * Hormone-based contraceptives which are associated with inhibition of ovulation.","28 Days",{"count":168,"type":21},20,[24],"To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD",[172],"Autosomal Recessive Polycystic Kidney (ARPKD)",[174,175,176,177,178,179,180,181],"ARPKD","TOLVAPTAN","Polycystic Kidney Disease","Autosomal Recessive Polycystic Kidney Disease","Adolescent","Renal Cysts","Oligohydramnios","Anhydramnios","2026-08-04",{"date":62,"type":32},{"date":185,"type":32},"2023-01-23",{"date":187,"type":21},"2028-08-14",{"name":38,"class":39},23,{"id":191,"slug":192,"hasResults":11,"nctId":193,"briefTitle":194,"officialTitle":195,"acronym":4,"eligibilityCriteria":196,"healthyVolunteers":11,"sex":197,"minAge":47,"maxAge":99,"enrollmentInfo":198,"targetDuration":4,"studyType":22,"phases":199,"briefSummary":200,"conditions":201,"keywords":203,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":205,"lastUpdatePostDateStruct":206,"startDateStruct":208,"completionDateStruct":210,"leadSponsor":212,"locationsCount":66},"100633382","phase-1-a-study-in-adult-males-with-x-linked-congenital-nephrogenic-diabetes-insipidus-to-test-the-effects-of-ndi-5001-given-for-multiple-days-and-to-test-how-ndi-5001-is-tolerated-and-taken-up-in-the-body-100633382","NCT07525960","A Study in Adult Males With X-linked Congenital Nephrogenic Diabetes Insipidus to Test the Effects of NDI-5001 Given for Multiple Days and to Test How NDI-5001 is Tolerated and Taken up in the Body","A Phase 1b, Open-label Trial to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of NDI-5001 in a Capsule Following Daily Oral Doses to Adult Males With X-linked Congenital Nephrogenic Diabetes Insipidus","Inclusion Criteria\n\n1. X-linked congenital NDI with a genotype-confirmed arginine vasopressin receptor 2 (gene) (AVPR2) mutation and no mutation in aquaporin-2 (AQP2).\n2. Urine osmolality of ≤ 200 milliosmoles per kilogram of water (mOsm\u002Fkg) in a spot urine sample obtained from a first morning void upon awakening (at screening, check-in \\[Day -4\\], and Day -1) before consuming any food or drink after awakening.\n\n3\\) BMI between 18.0 to 32.0 kilograms per square meter (kg\u002Fm\\^2), inclusive.\n\nExclusion Criteria\n\n1. Participant who is not willing to follow a low-sodium (\\\u003C=1 gram\u002Fday) diet during the in-clinic portion of the trial.\n2. A positive drug screen for substances of abuse (including THC, cannabidiol) at screening or check-in.\n3. Estimated glomerular filtration rate \\\u003C60 milliliters per minute per 1.73-kilogram square meters by serum creatinine and cystatin C at screening.\n4. Participants who have taken an investigational drug within 30 days prior to screening.\n5. Previous exposure to NDI-5001.\n6. Any history of significant bleeding or hemorrhagic tendencies.\n\nNote: Other protocol-specified inclusion\u002Fexclusion criteria may apply.","MALE",{"count":20,"type":21},[79],"The primary purpose of this trial is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NDI-5001 administered as a capsule following daily oral dosing in adult males with X-linked congenital nephrogenic diabetes insipidus (NDI) due to vasopressin receptor Type 2 (V2R) mutations.",[202],"Diabetes Insipidus",[204],"X-linked Congenital Nephrogenic Diabetes Insipidus","2026-07-29",{"date":207,"type":32},"2026-07-31",{"date":209,"type":32},"2026-07-07",{"date":211,"type":21},"2027-08-25",{"name":38,"class":39},{"id":214,"slug":215,"hasResults":11,"nctId":216,"briefTitle":217,"officialTitle":218,"acronym":4,"eligibilityCriteria":219,"healthyVolunteers":11,"sex":16,"minAge":47,"maxAge":75,"enrollmentInfo":220,"targetDuration":4,"studyType":22,"phases":222,"briefSummary":223,"conditions":224,"keywords":4,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":205,"lastUpdatePostDateStruct":226,"startDateStruct":227,"completionDateStruct":229,"leadSponsor":231,"locationsCount":232},"100584811","phase-3-a-trial-of-the-efficacy-and-safety-of-sep-363856-in-acutely-psychotic-participants-with-schizophrenia-100584811","NCT06894212","A Trial of the Efficacy and Safety of SEP-363856 in Acutely Psychotic Participants With Schizophrenia","A Phase 3, Randomized, Double-blind, Parallel-group, Placebo-controlled, Multicenter Trial to Evaluate the Efficacy and Safety of SEP-363856 in Acutely Psychotic Participants With Schizophrenia","Key Inclusion Criteria:\n\n* Male or female participants between 18 to 65 years of age (inclusive) at the time of consent.\n* Participant has an identified reliable informant (eg, caregiver, relative, friend, case worker, residential treatment staff).\n* Participant is experiencing an acute exacerbation or relapse of symptoms, with onset ≤ 2 months prior to screening\n\n  1. The participant requires hospitalization for this acute exacerbation or relapse of symptoms.\n  2. If already an inpatient at screening, has been hospitalized for less than 2 weeks for the current exacerbation at the time of screening.\n* Participants who are experiencing an acute exacerbation of psychotic symptoms and marked deterioration of usual function as demonstrated by meeting ALL of the following criteria at the screening and baseline visits:\n\n  1. Participant must have a PANSS total score ≥ 80\n\n     AND\n  2. Participant must have a CGI-S score ≥ 4.\n* Participants who have received previous outpatient antipsychotic treatment at an adequate dose (minimal recommended dose for the treatment of schizophrenia according to the manufacturer labeling) for an adequate duration (at least 6 weeks) and who showed a previous good response.\n\nKey Exclusion Criteria:\n\n* Sexually active participants or persons of childbearing potential who do not agree to practice 2 different clinical trial sponsor approved methods of birth control or remain abstinent during the course of the trial and for 30 days after the last dose of study drug.\n* Participant has a current DSM-5 diagnosis or presence of symptoms consistent with a DSM-5 diagnosis other than schizophrenia.\n* Participant has had psychiatric hospitalization(s) for more than 30 days (cumulative) during the 90 days prior to screening.\n* Participant has previously received SEP-363856 or was previously enrolled in a SEP-363856 clinical study",{"count":221,"type":21},522,[24],"Evaluate the efficacy and safety of Ulotaront (SEP-363856) in acutely psychotic subjects with schizophrenia",[225],"Schizophrenia",{"date":207,"type":32},{"date":228,"type":32},"2025-02-28",{"date":230,"type":21},"2026-10-29",{"name":38,"class":39},73,{"id":234,"slug":235,"hasResults":11,"nctId":236,"briefTitle":237,"officialTitle":238,"acronym":4,"eligibilityCriteria":239,"healthyVolunteers":11,"sex":16,"minAge":47,"maxAge":4,"enrollmentInfo":240,"targetDuration":4,"studyType":22,"phases":242,"briefSummary":243,"conditions":244,"keywords":245,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":247,"lastUpdatePostDateStruct":248,"startDateStruct":250,"completionDateStruct":252,"leadSponsor":254,"locationsCount":255},"100590769","phase-3-a-study-of-jnt-517-in-participants-with-phenylketonuria-pku-100590769","NCT06971731","A Study of JNT-517 in Participants With Phenylketonuria (PKU)","A Phase 3, Double-Blind, Randomized, Two-Period, Multicenter, Placebo-Controlled, Efficacy and Safety Study of JNT-517 for the Treatment of Participants With Phenylketonuria","Inclusion Criteria:\n\n1. Males and females ≥18 years of age on Day 1\n2. Clinical diagnosis of PKU\n3. Average of at least 3 plasma Phe levels (after \\>4-hour fast) during Screening period of ≥360 μmol\u002FL\n4. Not on pegvaliase within 4 weeks prior to Screening\n5. If on sapropterin or large neutral amino acids, such as PheBloc®, NeoPhe®, and PreKunil® at Screening, must be on a stable dose 4 weeks prior to Screening and for the entire study duration.\n6. Willing and able to maintain a stable diet in Phe and total protein (intact protein and medical food protein) and able to adjust diet through the duration of the study according to the Dietary Management Guidelines\n7. Body weight \\>40 kilograms (kg)\n8. If biologically female of childbearing potential:\n\n   1. Must have a negative serum pregnancy test at Screening and a negative urine pregnancy test by Day 1\n   2. Must practice sexual abstinence, or if involved in any sexual intercourse that could lead to pregnancy, must agree to use 2 highly effective contraceptive methods from Screening until at least 30 days after the last study drug administration\n   3. If taking estrogen- or progesterone-based oral contraceptives, must agree to use 2 other highly effective methods of contraception or must agree to sexual abstinence during the study\n   4. Must refrain from donating ova during the course of the study and for 30 days after the last dose of the study drug.\n9. If a biologically female not of childbearing potential or postmenopausal, defined as follows:\n\n   1. Has had surgical sterilization (hysterectomy, bilateral oophorectomy, or bilateral salpingectomy)\n   2. Has had amenorrhea for minimum of 1 year with confirmation by levels of follicle stimulating hormone testing\n   3. Has not achieved menarche (has not had first menstrual period). If a female achieves menarche during the study, she will need to follow the contraception requirement for females of childbearing potential\n10. If biologically male, must practice sexual abstinence, or if involved in any sexual intercourse that could lead to pregnancy, must agree to use highly effective contraceptive methods from Day 1 until at least 30 days after the last study drug administration and must refrain from donating sperm during the course of the study and for 30 days after the last dose of the study drug NOTE: No restrictions are required for biological males who have undergone a documented vasectomy at least 4 months prior to Screening. If the vasectomy procedure is not documented or was performed less than 4 months prior to Screening, males must follow the same contraception as for non-vasectomized participants.\n11. Participants with psychiatric illness must be well-controlled for the last 6 months prior to the Screening visit and if on medication, on stable medications for the last 3 months.\n12. Capable of giving signed informed consent or parent\u002Flegal guardian to provide informed consent and the participant to give assent and confirm able to comply with study procedures\n\nExclusion Criteria:\n\n* Exclusion Criteria\n\nParticipants will be excluded from the study if any of the following criteria are met:\n\n1. Any acute or uncontrolled chronic medical condition that would prevent the participant from complying with the procedures or place the participant at risk if they participate in the study\n2. Positive for hepatitis B or C or human immunodeficiency virus\n3. Any history of malignancy of any organ system (other than non-melanoma skin cancer or in situ cervical cancer), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases\n4. Any history of significant liver disease\n5. Any history of cataracts or more than minimal cataracts observed during the Screening ophthalmologic examination. Minimal cataracts are defined as changes similar to lens opacities classification system III (LOCS III), lens grade C1, N1 or P1\n6. Any surgical or medical conditions that may affect study drug absorption, distribution, metabolism, or excretion\n7. Estimated glomerular filtration rate (eGFR) \\\u003C60 mL\u002Fmin\u002F1.73 m2 by 2021 Chronic Kidney Disease Epidemiology Collaboration formula\n8. Participation in another investigational drug trial within 30 days or, if known, 5 half-lives of the investigational drug (whichever is longer). For gene therapy or editing trials, participants must have received the intervention \\>6 months prior to Screening visit and with stable plasma Phe in the past 2 months prior to Screening visit.\n9. Alcohol consumption within 5 days of randomization and\u002For unwilling to limit to 1 alcoholic drink per day until after the 6-month study visit\n10. History of drug\u002Falcohol abuse in the last year\n11. Use of any medications that are inhibitors or inducers of cytochrome P450 (CYP3A4) or inhibitors of the transporter P-glycoprotein (P-gp) within 4 weeks prior to randomization and unwilling and\u002For unable to avoid these medications throughout the treatment duration\n12. Use of any medications that are substrates of breast cancer resistance protein (BCRP), multidrug and toxin extrusion (MATE)1, or MATE2-K within 4 weeks prior to randomization and unwilling and\u002For unable to avoid these medications throughout the treatment duration NOTE: Participants will be permitted to continue with estrogen- or progesterone-based oral contraceptives, but must agree to use 2 other methods of contraception, where at least 1 must be highly effective, or must agree to sexual abstinence during the study.\n13. Current, recent, or suspected active viral or bacterial infection within 2 weeks prior to and during the Screening Period\n14. Unable to tolerate oral medication or have a condition that would interfere with the absorption of JNT-517\n15. Allergy to JNT-517 or any component of the investigational product\n16. Any of the following laboratory values at the Screening visit: -Alanine aminotransferase or aspartate aminotransferase values \\>1.5× the upper limit of normal (ULN)-Total bilirubin ˃ULN unless history of Gilbert Syndrome and then total bilirubin \\>4 milligrams per deciliter (mg\u002FdL) is exclusionary-Hemoglobin \\\u003C11.0 grams per deciliter (g\u002FdL) \\[\\\u003C110.0 grams per liter (g\u002FL)\\]-White blood cell count \\>ULN-Platelets \\\u003C150 × 10\\^9\u002FLiter (L) (\\\u003C150,000\u002Fcubic millimeters \\[mm\\^3\\]).",{"count":241,"type":21},120,[24],"The goal of this Phase 3, randomized study is to assess the safety, efficacy, tolerability, and pharmacokinetics (PK) of oral JNT-517 in adults (18 years of age or older) with PKU. Participants will receive either JNT-517 or placebo and will be blinded to their treatment assignment. Participants will have a 2 in 3 (or approximately 67%) chance of receiving JNT-517 during the first part of the study which will last approximately six weeks. During the second part of the study every participant who continues in the study will receive one of two doses of JNT-517 for an additional 46 weeks. The study requires a screening period of up to 35 days to ensure dietary stabilization and amino acid levels required to meet study eligibility. In total, participation in the study could last for up to 400 days.\n\nParticipants will:\n\nTake 75 mg JNT-517 or 150 mg JNT-517, or a placebo BID (2x per day) for approximately 365 days; Visit the clinic or have a mobile health nurse visit your home for checkups and tests; Collect urine sample at home and bring to clinic on specified days; Keep a food diary 3 days before each study visit",[82],[82,246],"PKU","2026-07-06",{"date":249,"type":32},"2026-07-08",{"date":251,"type":32},"2025-10-20",{"date":253,"type":21},"2028-04-01",{"name":38,"class":39},26,{"id":257,"slug":258,"hasResults":11,"nctId":259,"briefTitle":260,"officialTitle":261,"acronym":4,"eligibilityCriteria":262,"healthyVolunteers":74,"sex":16,"minAge":47,"maxAge":263,"enrollmentInfo":264,"targetDuration":4,"studyType":22,"phases":266,"briefSummary":267,"conditions":268,"keywords":273,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":275,"lastUpdatePostDateStruct":276,"startDateStruct":278,"completionDateStruct":280,"leadSponsor":282,"locationsCount":4},"100642293","phase-1-a-trial-in-healthy-adult-participants-and-adults-with-autoimmune-disease-to-test-how-hbm7020-is-tolerated-and-absorbed-in-the-body-100642293","NCT07649265","A Trial in Healthy Adult Participants and Adults With Autoimmune Disease to Test How HBM7020 is Tolerated and Absorbed in the Body","A Phase 1 Open-Label, Multicenter Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of HBM7020 in Healthy Adult Participants and Adults With Seropositive Autoimmune Disease","Key Inclusion Criteria for Healthy Participants (Part 1)\n\n1. Participants who are of non-childbearing potential or are using acceptable contraception.\n2. Body mass index (BMI) and body weight within an acceptable range.\n3. Good general health based on medical history, physical examination, electrocardiogram (ECG), and laboratory assessments.\n\nKey Disease-Agnostic Inclusion Criteria for Patient Participants (Part 1)\n\n1. BMI and body weight within an acceptable range.\n2. Adequate hematologic, renal, hepatic, immunologic, and lymphocyte parameters.\n\nKey Disease-Specific Inclusion Criteria for Patient Participants (Part 1)\n\n1. Confirmed autoimmune disease with appropriate supporting autoantibody findings.\n2. Stable background therapy prior to dosing.\n3. Active moderate to severe disease consistent with protocol-defined disease activity criteria for:\n\n   * Systemic lupus erythematosus (SLE)\n   * Systemic sclerosis (SSc)\n   * Rheumatoid arthritis (RA)\n   * Sjögren's disease (SjD)\n\nKey Inclusion Criteria for Rescreening Participants (Part 2)\n\n1. Meets Part 1 disease-agnostic inclusion criteria.\n2. Stable background autoimmune therapy prior to dosing.\n3. Ongoing active moderate to severe disease based on protocol-defined disease-specific criteria.\n\nKey Exclusion Criteria for Parts 1 and 2\n\n1. Pregnant or breastfeeding participants.\n2. Recent vaccination within protocol-defined timelines.\n3. Clinically significant medical history or abnormal physical examination findings.\n4. Clinically significant cardiovascular abnormalities, including blood pressure, heart rate, syncope, or ECG findings.\n5. Prior or recent therapies or conditions that may interfere with study participation or safety evaluations.\n6. Severe pulmonary, renal, or cardiac disease, or clinically significant pulmonary hypertension.","75 Years",{"count":265,"type":21},63,[79],"This first-in-human study evaluates the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of HBM7020. The study will enroll healthy participants at low doses, followed by participants with moderate to severe autoimmune diseases with predominant B-cell involvement. Eligible participants include patients with systemic lupus erythematosus (SLE), systemic sclerosis (SSc), Sjögren's disease (SjD), and rheumatoid arthritis (RA).",[269,270,271,272],"Rheumatoid Arthritis","Systemic Lupus Erythematosus","Systemic Sclerosis","Sjögren's Disease",[274],"Autoimmune disease","2026-06-11",{"date":277,"type":32},"2026-06-16",{"date":279,"type":21},"2026-09-15",{"date":281,"type":21},"2028-11-20",{"name":38,"class":39},{"id":284,"slug":285,"hasResults":11,"nctId":286,"briefTitle":287,"officialTitle":288,"acronym":289,"eligibilityCriteria":290,"healthyVolunteers":11,"sex":16,"minAge":291,"maxAge":4,"enrollmentInfo":292,"targetDuration":4,"studyType":22,"phases":294,"briefSummary":295,"conditions":296,"keywords":298,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":308,"lastUpdatePostDateStruct":309,"startDateStruct":311,"completionDateStruct":313,"leadSponsor":315,"locationsCount":316},"100609069","phase-3-a-study-of-quabodepistat-containing-regimens-for-the-treatment-of-drug-resistant-pulmonary-tuberculosis-100609069","NCT07209761","A Study of Quabodepistat-containing Regimens for the Treatment of Drug-resistant Pulmonary Tuberculosis","A Phase 3, Randomized, Open-label, Multicenter Trial to Evaluate the Efficacy, Safety, and Tolerability of 4-month and 6-month Quabodepistat-containing Regimens for Rifampicin-resistant\u002FMultidrug-resistant Pulmonary Tuberculosis","QUANTUM-TB","Inclusion Criteria:\n\n1. Age ≥14 years\n2. Body weight ≥30.0 kg\n3. Able to provide written informed consent (if under 18, requires both participant assent and parent\u002Fguardian consent)\n4. Documented pulmonary TB: Mtb confirmed by Xpert MTB\u002FRIF Ultra (semi-quantitative result of 'low', 'medium', or 'high')\n5. Rifampicin resistance confirmed by Xpert MTB\u002FRIF Ultra test\n6. Chest radiograph consistent with active TB disease\n7. Able to provide sputum sample\n8. Participants of childbearing potential must use 2 different approved birth control methods during treatment and for 12 weeks after last dose\n9. Willing to have HIV test (unless previous positive result confirmed)\n10. For HIV-positive participants: On stable antiretroviral regimen (dolutegravir, lamivudine\u002Femtricitabine, tenofovir) for ≥3 months, Viral load \\\u003C200 copies\u002FmL, and CD4 count \\>100 cells\u002FmL\n\nExclusion Criteria:\n\n1. Known\u002Fsuspected resistance to BDQ, PMD, LZD, or QBS\n2. Prior treatment with BDQ, PMD, LZD, DLM, QBS, or DprE1 inhibitors for ≥1 month within past 3 months\n3. Severe extrapulmonary TB\n4. Abnormal laboratory values: ALT\u002FAST \\>2.5×ULN, Total bilirubin \\>1.5×ULN, eGFR \\\u003C60 mL\u002Fmin\u002F1.73m², Hemoglobin \\\u003C8 g\u002FdL, Platelets \\\u003C100,000 cells\u002Fmm³, WBC \\\u003C2.0×10⁹\u002FL, ANC \\\u003C1000 cells\u002FμL, and HbA1c \\>9.0%\n5. Pre-existing peripheral neuropathy (≥Grade 1), optic neuritis, or visual impairment\n6. Co-enrollment in other therapeutic trials\n7. QTcF \\>450 msec (males) or \\>470 msec (females)\n8. Clinically significant cardiovascular disorders\n9. Bleeding disorders\n10. Conditions interfering with X-ray or sputum assessment\n11. Drug allergies\u002Fhypersensitivity to study medications\n12. Pregnancy or breastfeeding\n13. Positive drug screen (case-by-case assessment for some substances)\n14. Serious mental disorders\n15. Karnofsky score \\\u003C60\n16. BMI \\\u003C16.0 kg\u002Fm²\n17. Significant comorbidities (metabolic, renal, gastrointestinal, neurological, psychiatric, endocrine, liver)\n18. Pulmonary conditions other than TB (silicosis, fibrosis)\n19. Active SARS-CoV-2 infection\n20. Use of prohibited medications\n21. Blood\u002Fplasma donation within 30 days\n22. Current use of herbal remedies or traditional medicines","14 Years",{"count":293,"type":21},532,[24],"This study aims to assess quabodepistat-based treatment regimens for RR\u002FMDR-TB. The study will enroll adults and adolescents with rifampicin-resistant or multidrug-resistant pulmonary TB. The main goal is to see if a new drug called quabodepistat, when combined with other TB drugs, can shorten treatment duration to 4 months and be as effective and safer than current WHO endorsed treatment regimen given for 6-months. The study will compare different drug combinations in two groups of patients: those whose TB is sensitive to fluoroquinolones and those whose TB is resistant to fluoroquinolones. Participants will be randomly assigned to receive either the new treatment or the standard treatment. The study will last for 16 months for each participant and will measure how well the treatments work and how safe they are.",[297],"Pulmonary Tuberculosis",[297,299,300,301,302,303,304,305,306,307],"Quabodepistat","Bedaquiline","Pretomanid","Linezolid","Moxifloxacin","Fluoroquinolone-sensitive TB","Fluoroquinolone-resistant TB","Drug-resistant TB","Shortened TB treatment","2026-05-04",{"date":310,"type":32},"2026-05-06",{"date":312,"type":32},"2025-10-16",{"date":314,"type":21},"2028-09-29",{"name":38,"class":39},35,{"id":318,"slug":319,"hasResults":11,"nctId":320,"briefTitle":321,"officialTitle":322,"acronym":4,"eligibilityCriteria":323,"healthyVolunteers":11,"sex":16,"minAge":324,"maxAge":4,"enrollmentInfo":325,"targetDuration":4,"studyType":22,"phases":327,"briefSummary":328,"conditions":329,"keywords":331,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":332,"lastUpdatePostDateStruct":333,"startDateStruct":335,"completionDateStruct":337,"leadSponsor":339,"locationsCount":340},"100564357","phase-3-a-long-term-study-of-jnt-517-in-participants-with-phenylketonuria-100564357","NCT06628128","A Long-Term Study of JNT-517 in Participants With Phenylketonuria","An Open-Label Study to Evaluate the Long-Term Safety of JNT-517 in Participants With Phenylketonuria","Key Inclusion Criteria:\n\n1. Diagnosis of phenylketonuria (ie, PAH deficiency) by either molecular testing or biochemical criteria consistent with the applicable regional guidelines.\n2. Participants 4 years of age and older, inclusive, at time of Screening.\n3. Not on pegvaliase within 4 weeks of Screening.\n4. Not on sepiapterin within 2 weeks of Screening.\n5. If on sapropterin or large neutral amino acids at Screening, must be on a stable dose for 4 weeks prior to Screening.\n6. Willing and able to maintain a diet consistent in Phe content from the Screening period through the duration of the study, unless otherwise directed by a dietician as allowed in the protocol.\n7. Body weight ≥ 12.5 kg.\n8. If female of childbearing potential:\n\n   1. Must have a negative serum pregnancy test at Screening and a negative urine pregnancy test by Day 1.\n   2. Must practice sexual abstinence, or if involved in any sexual intercourse that could lead to pregnancy, must agree to use 2 different contraceptive methods, where at least 1 method must be highly effective, from Screening until at least 30 days after the last study drug administration.\n   3. Must refrain from donating ova during the course of the study and for 30 days after the last dose of the study drug.\n9. Is a female not of childbearing potential or postmenopausal, defined as follows:\n\n   1. Has had surgical sterilization (hysterectomy, bilateral oophorectomy, or bilateral salpingectomy).\n   2. Has had amenorrhea for minimum of 1 year with confirmation by levels of follicle stimulating hormone testing.\n   3. Has not achieved menarche (has not had first menstrual period). If a female achieves menarche during the study, she will need to follow the contraception requirement for females of childbearing potential.\n10. If male, must practice sexual abstinence, or if involved in any sexual intercourse that could lead to pregnancy, must agree to use 2 different contraceptive methods, where at least 1 method must be highly effective, from Day 1 until at least 30 days after the last study drug administration and must refrain from donating sperm during the course of the study and for 30 days after the last dose of the study drug.\n\n    Note: No restrictions are required for males who have undergone a documented vasectomy at least 4 months prior to Screening. If the vasectomy procedure is not documented or was performed less than 4 months prior to Screening, males must follow the same contraception as for non-vasectomized participants.\n11. Capable of giving signed informed consent, or parent\u002Flegal guardian to provide informed consent and pediatric participant to give assent, and be able to comply with study procedures.\n12. Participants with psychiatric illness must be well-controlled for the last 3 months prior to screening visit and, if on medication, on stable medications for the last 3 months.\n\nKey Exclusion Criteria:\n\n1. Participation in this study is not considered safe and\u002For feasible in the opinion of the Investigator.\n2. Participants have not completed a previous JNT-517 study and are eligible for another active JNT-517 trial at the site, unless approval is obtained from the medical monitor.\n3. Any acute or chronic medical condition that would prevent the participant from complying with the procedures or place the participant at risk if they participate in the study.\n4. Positive for hepatitis B or C or human immunodeficiency virus.\n5. Any history of significant liver disease.\n6. Any history of cataracts or more than minimal cataracts observed during the Screening ophthalmologic examination.\n7. Any surgical or medical conditions that may affect study drug absorption, distribution, metabolism, or excretion.\n8. Estimated glomerular filtration rate \\\u003C 60 milliliters per minute per 1.73 square meters (mL\u002Fmin\u002F1.73 m\\^2) by 2021 Chronic Kidney Disease Epidemiology Collaboration formula (participants aged 17 years or greater) or by Schwartz formula (participants aged 4 to 16 years of age).\n9. History of drug or alcohol abuse in the last year.\n10. Use of any medication that are inhibitors or inducers of cytochrome P450 (CYP)3A4 or inhibitors of the transporter P glycoprotein (P-gp) within 4 weeks prior to the first dose of study drug and unwilling and\u002For unable to avoid these medications throughout the treatment duration.\n11. Use of any medications that are a substrate of breast cancer resistance protein (BCRP), multidrug and toxin extrusion (MATE)1, MATE2-K, organic anion transporter 3 (OAT3), or CYP3A4 within 4 weeks prior to the first dose of study drug and unwilling and\u002For unable to avoid these medications throughout the treatment duration (Appendix A). CYP3A4 substrates may be allowed if reduction in exposure is not expected to impact safety of the participant after consultation with the Medical Monitor.\n\n    NOTE: Participants of childbearing potential will be permitted to continue with estrogen- or progesterone based oral contraceptives, but must agree to use 2 other methods of contraception, where at least 1 must be highly effective, or must agree to sexual abstinence during the study.\n12. Current, recent, or suspected infection within 2 weeks of Screening of Severe Acute Respiratory Syndrome Coronavirus 2\u002FCoronavirus Disease 2019 (SARS-CoV-2\u002FCOVID-19).\n13. Unable to tolerate oral medication or inability to swallow tablets.\n14. Allergy to JNT-517 or any component of the investigational product.\n15. Any of the following laboratory values at the Screening visit:\n\n    1. Alanine aminotransferase or aspartate aminotransferase values ˃ 1.5 x the upper limit of normal (ULN).\n    2. Total bilirubin ˃ULN unless history of Gilbert Syndrome and then total bilirubin \\>4 milligrams per deciliter \\[mg\u002FdL\\] is exclusionary.\n    3. Hemoglobin ˂10.0 g\u002FdL (˂100.0 grams per liter \\[g\u002FL\\])\n    4. White blood cell count ˃1 x ULN\n    5. Platelet count ˂150 × 109\u002FL (˂150,000\u002Fcubic millimeters\\[mm\\^3\\])\n16. Participation in another investigational drug trial within 30 days (other than for JNT-517) or, if known 5 half-lives of investigational drug (whichever is longer).","4 Years",{"count":326,"type":21},240,[24],"The goal of this Phase 3, open-label study is to evaluate the long-term safety of JNT-517 in pediatric and adult participants with Phenylketonuria (PKU) after completion of either Study JNT517-101 (NCT05781399) or JNT517-201 (NCT06637514) as well as participants who have not participated in a prior JNT-517 study. In this trial, all participants will receive JNT-517 using age- and weight-banded dosing as outlined in the protocol, regardless of any dose received in a previous study.",[330],"Phenylketonuria (PKU)",[82,246],"2026-04-27",{"date":334,"type":32},"2026-05-01",{"date":336,"type":32},"2025-08-11",{"date":338,"type":21},"2028-02-01",{"name":38,"class":39},12,{"id":342,"slug":343,"hasResults":11,"nctId":344,"briefTitle":345,"officialTitle":346,"acronym":4,"eligibilityCriteria":347,"healthyVolunteers":74,"sex":197,"minAge":47,"maxAge":99,"enrollmentInfo":348,"targetDuration":4,"studyType":22,"phases":350,"briefSummary":351,"conditions":352,"keywords":353,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":355,"lastUpdatePostDateStruct":356,"startDateStruct":358,"completionDateStruct":360,"leadSponsor":362,"locationsCount":91},"100627934","phase-1-a-study-to-detect-the-radioactivity-of-14c-sep-380135-in-urine-and-feces-in-healthy-male-adults-100627934","NCT07455084","A Study to Detect the Radioactivity of 14C-SEP-380135 in Urine and Feces in Healthy Male Adults","A Phase 1 Trial to Assess the Mass Balance and Pharmacokinetics of a Single Oral Administration of 14C-SEP-380135 in Healthy Male Adults","Inclusion Criteria:\n\n1. Body mass index (BMI) between 18.0 and 32.0 kilograms per square meter (kg\u002Fm²).\n2. In good overall health, based on:\n\n   1. Medical history\n   2. Physical examination\n   3. Neurological examination\n   4. Vital signs\n   5. Electrocardiogram (ECG)\n   6. Blood and urine tests (serum chemistry, hematology, urinalysis, serology)\n3. Regular daily bowel movements (at least one per day) for 30 days before Day -2.\n4. Ability to provide written informed consent and follow all study instructions.\n5. Has a stable living situation at screening and agrees to return to a similar living arrangement after the in-clinic stay.\n\nExclusion Criteria:\n\n1. Have taken an investigational drug within 30 days or 5 half-lives, if known, (whichever is longer) prior to screening.\n2. Have had prior exposure to SEP-380135.\n3. Are currently participating in another clinical trial.\n4. Attempted suicide within 12 months prior to screening.\n5. A history of sick sinus syndrome, any degree of atrioventricular block, Heart attack (myocardial infarction), heart failure (NYHA Class II-IV), cardiomyopathy, pulmonary congestion, cardiac arrhythmias, prolonged QT interval, congenital long QT syndrome, family history of long QT, or moderate to severe hypokalemia. A participant with non-clinically significant ECG abnormalities at screening and check-in requires approval from the medical monitor and the sponsor's physician.\n6. Are predicted poor metabolizer CYP2D6 phenotypes. Note: Other protocol-specified inclusion\u002Fexclusion criteria may apply.",{"count":349,"type":21},8,[79],"The purpose of the present trial is to obtain information on the absorption, distribution, metabolism, excretion and pharmacokinetics (PK) of 14C-SEP-380135 and its metabolites following a single oral dose.",[225],[354],"Mental Health Conditions, Schizophrenia","2026-03-03",{"date":357,"type":32},"2026-03-06",{"date":359,"type":21},"2026-03-20",{"date":361,"type":21},"2026-05-03",{"name":38,"class":39},{"id":364,"slug":365,"hasResults":11,"nctId":366,"briefTitle":367,"officialTitle":368,"acronym":4,"eligibilityCriteria":369,"healthyVolunteers":11,"sex":16,"minAge":370,"maxAge":4,"enrollmentInfo":371,"targetDuration":4,"studyType":22,"phases":373,"briefSummary":375,"conditions":376,"keywords":4,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":377,"lastUpdatePostDateStruct":378,"startDateStruct":380,"completionDateStruct":382,"leadSponsor":384,"locationsCount":385},"100572996","phase-2-trial-of-the-impact-of-sibeprenlimab-on-immunoglobulin-a-nephropathy-kidney-tissue-100572996","NCT06740526","Trial of the Impact of Sibeprenlimab on Immunoglobulin A Nephropathy Kidney Tissue","A Phase 2b, Multicenter, Open-label, Single-arm Trial to Evaluate the Impact of Sibeprenlimab on Kidney Histopathology Through Repeat Kidney Biopsies in Adolescents and Adults With Immunoglobulin A Nephropathy","Inclusion Criteria:\n\n1. Participants must be at least 16 years of age or older at the time of signing the informed consent\u002Fassent.\n2. Source-verified kidney biopsy confirmed diagnosis of IgAN.\n3. Participant has estimated glomerular filtration rate (eGFR) \\> 45 mL\u002Fmin\u002F1.73 m2 using serum creatinine (Chronic Kidney Disease-Epidemiology Collaboration \\[CKD EPI\\] creatinine eGFR 2021 equation for those 18 years and older; Chronic Kidney Disease in Children under age 25 \\[CKiD U25\\] eGFR equation for those younger than 18 years)\n\nExclusion Criteria:\n\n1. Participants who are breast-feeding and\u002For who have a positive pregnancy test result prior to receiving sibeprenlimab.\n2. Participant has coexisting chronic kidney disease, other than IgAN.\n3. Participant has a serum IgG value \\\u003C600 mg\u002FdL at screening.\n4. Participant is currently receiving or has received within 24 weeks prior to the firstdose of sibeprenlimab, systemic corticosteroids or immunosuppression (note:\n\n   topical, ophthalmic, rectal, intra-articular, inhaled corticosteroids are allowed).\n5. Participant has uncontrolled hypertension (defined as systolic blood pressure \\> 140 mmHg or diastolic blood pressure \\> 90 mmHg).\n6. Participants who would be likely to require prohibited concomitant therapy during the trial.","16 Years",{"count":372,"type":21},25,[374],"PHASE2","This is a phase 2b open-label trial to characterize histopathological biomarkers of disease in immunoglobulin A nephropathy (IgAN) and demonstrate potential changes in response to sibeprenlimab.",[27],"2025-04-04",{"date":379,"type":32},"2025-04-06",{"date":381,"type":32},"2024-11-19",{"date":383,"type":21},"2029-04-17",{"name":38,"class":39},5,""]