[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Idorsia Pharmaceuticals Ltd.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":108},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,41,55,79],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":4},"100652855","phase-3-a-study-to-learn-how-well-lucerastat-works-and-how-safe-it-is-in-untreated-adult-male-participants-with-fabry-disease-100652855",false,"NCT07778667","A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease","A Multicenter, Open-label, Single-arm, Baseline-controlled Trial to Assess the Efficacy and Safety of Lucerastat in Treatment-naïve\u002FPseudo-naïve Adult Male Participants With Fabry Disease","Fab-Klear","Inclusion Criteria:\n\n* Confirmed diagnosis of Fabry disease:\n\n  * Plasma and\u002For leukocyte α-galactosidase A (α-GalA) \\\u003C 1% mean normal levels or\n  * Known \"pathogenic\" or \"likely pathogenic\" Gene coding for α-galactosidase A (GLA) variant with a low level (i.e., \\\u003C 30% mean normal levels) of plasma and\u002For leukocyte α-GalA.\n* History of at least one of the following clinical manifestations of Fabry disease:\n\n  * Neuropathic pain\n  * Cornea verticillata\n  * Angiokeratoma\n* Treatment-naïve or pseudo-naïve i.e. without prior treatment with an approved or any investigational therapy for Fabry disease within at least 6 months prior to screening.\n* Plasma globotriaosylsphingosine ≥ 20 ng\u002Fml (as assessed centrally).\n* Screening eGFR (central laboratory) ≥ 45 mL\u002Fmin\u002F1.73 m2.\n\nExclusion Criteria:\n\n* Any intercurrent condition or concomitant therapy considered a contraindication for kidney biopsy, as per local standard of care, or in the investigator's opinion may preclude accurate interpretation of trial data.\n* Urine albumin-to-creatinine ratio \\> 300 mg\u002Fg at screening (central laboratory) unless treated with background therapy, such as Angiotensin-converting enzyme inhibitors, Angiotensin receptor blocker or Sodium-glucose cotransporter 2 inhibitors, as per local practice.\n* Inherited or acquired coagulopathy, uncorrected bleeding disorders, international normalized ratio \\> 1.5, platelet count \\\u003C 50,000\u002FμL or inability to safely hold anticoagulants or antiplatelet therapy as applicable per local practice (usually 1-2 days for anticoagulants and 3-7 days for antiplatelets).\n* Hemoglobin level \\\u003C 9.0 g\u002FdL at screening.\n* History of acute kidney injury within 12 months prior to screening visit.\n* Documented poorly controlled diabetes mellitus (i.e., Hemoglobin A1c \\> 8.0% at screening as reported by the central laboratory).\n* History of cerebrovascular event (e.g. stroke, transient ischemic attack), cardiovascular event (e.g., myocardial infarction, unstable angina), cardiac surgery (e.g., coronary artery bypass graft, valvular repair\u002Freplacement) or percutaneous coronary intervention within 6 months prior to screening.\n* Congestive heart failure New York Heart Association class IV or hospitalization for heart failure within 3 months prior to screening.\n* Implementation of cardiac device (e.g., pacemaker, implantable cardioverter defibrillator, cardiac resynchronization therapy device) or hospitalization for arrhythmia within 6 weeks prior to screening.\n* Any other known factor or disease that might interfere with treatment compliance, trial conduct, or interpretation of the results, such as drug or alcohol dependence or psychiatric disease including severe depression or suicidal ideation at screening or history of suicide attempt or behavior within 6 months prior to screening visit.\n* Previous exposure to gene or cell therapy.\n* Use of cationic amphiphilic drugs, such as amiodarone or hydroxychloroquine that may preclude accurate interpretation of kidney biopsy data within 6 months prior to screening.","MALE","18 Years","60 Years",{"count":21,"type":22},16,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3","The purpose of this clinical trial is to learn how well lucerastat works and how safe it is in untreated adult male participants with Fabry disease.\n\nThe main question this clinical trial aims to answer is:\n\n• Does treatment with lucerastat affects the amount of globotriaosylceramide (Gb3), a fatty substance that builds up in the kidneys, in untreated adult men with Fabry disease?\n\nThis is an open-label, single-arm trial, which means that participants will know which trial medication they receive and only one trial medication will be given.\n\nTrial participants will:\n\n* Take lucerastat every day for 18 months\n* Have kidney biopsies at the end and start of the trial\n* Visit the clinic 10 times for check-up and tests\n* Take part in the trial for up to 21 months in total",[28],"Fabry Disease","NOT_YET_RECRUITING","2026-08-18",{"date":32,"type":33},"2026-08-21","ACTUAL",{"date":35,"type":22},"2026-09",{"date":37,"type":22},"2029-03",{"name":39,"class":40},"Idorsia Pharmaceuticals Ltd.","INDUSTRY",{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":4,"acronym":4,"eligibilityCriteria":46,"healthyVolunteers":11,"sex":4,"minAge":4,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":47,"phases":4,"briefSummary":48,"conditions":49,"keywords":4,"overallStatus":50,"whyStopped":4,"lastUpdateSubmitDate":51,"lastUpdatePostDateStruct":52,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":54,"locationsCount":4},"100651659","post-trial-access-to-lucerastat-for-patients-with-fabry-disease-who-participated-in-study-id-069a302-100651659","NCT07762638","Post Trial Access to Lucerastat for Patients With Fabry Disease Who Participated in Study ID-069A302","Inclusion Criteria:\n\n\\-\n\nExclusion Criteria:\n\n\\-","EXPANDED_ACCESS","This is a Post Trial Access program for patients with Fabry Disease who previously participated in the ID-069A302 (MODIFY OLE) trial and who were still enrolled in the study at the time the Sponsor decided to discontinue ID-069A302. The program is designed to provide continued access to lucerastat.",[28],"AVAILABLE","2026-08-07",{"date":53,"type":33},"2026-08-13",{"name":39,"class":40},{"id":56,"slug":57,"hasResults":11,"nctId":58,"briefTitle":59,"officialTitle":60,"acronym":4,"eligibilityCriteria":61,"healthyVolunteers":11,"sex":62,"minAge":18,"maxAge":63,"enrollmentInfo":64,"targetDuration":4,"studyType":23,"phases":66,"briefSummary":68,"conditions":69,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":71,"lastUpdatePostDateStruct":72,"startDateStruct":74,"completionDateStruct":76,"leadSponsor":78,"locationsCount":4},"100644043","phase-2-a-study-to-learn-how-safe-act-777991-is-and-how-well-it-works-in-adults-with-non-segmental-vitiligo-100644043","NCT07667569","A Study to Learn How Safe ACT-777991 is and How Well it Works in Adults With Non-segmental Vitiligo","A Phase 2a, Proof of Concept, Multicenter, Double Blind, Randomized, Placebo Controlled, Parallel Group Trial to Assess the Efficacy and Safety of ACT-777991 in Adults With Non-segmental Vitiligo","Inclusion Criteria:\n\n* Clinical diagnosis of either active or stable non segmental vitiligo for at least 3 months prior to Screening and meet all the following criteria:\n\n  * F-VASI score ≥ 0.3 based on BICR at Screening.\n  * T-VASI score ≥ 5 based on investigator assessment at Screening and Randomization.\n  * Total body surface area (BSA) involvement, including the face, ≤ 50% based on investigator assessment at Screening and Randomization.\n* Participants must agree not to use therapeutic agents and procedures to treat vitiligo from Screening until Participant Last Visit.\n\nExclusion Criteria:\n\n* Clinical diagnosis of other forms of vitiligo (e.g., segmental) or other hypo- or depigmentation disorders (e.g., piebaldism, leukoderma, Vogt-Koyanagi-Harada disease, malignancy-induced hypopigmentation).\n* Any autoimmune disease, except adequately treated thyroid disease.\n* History of systemic immunotherapy treatment, including JAK inhibitors, for any inflammatory disease in the 12 months prior to Randomization.\n* History of topical JAK inhibitors for any inflammatory disease in the 6 weeks prior to Screening.\n* Use of laser or light-based treatment (phototherapy), including tanning beds, in the 8 weeks prior to Screening.\n* eGFR \\\u003C 90 mL\u002Fmin\u002F1.73 m2, defined by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation, at Screening.","ALL","65 Years",{"count":65,"type":22},30,[67],"PHASE2","The purpose of this clinical trial is to learn how well ACT-777991 works, how safe it is and how well it is tolerated by adults with non-segmental vitiligo.\n\nThe main question this clinical trial aims to answer is:\n\n• Can ACT-777991 help return color to the skin of the face of adults with non-segmental vitiligo?\n\nResearchers will compare ACT-777991 to placebo (a look-alike inactive treatment that contains no medicine) to see if ACT-777991 works to treat non-segmental vitiligo.\n\nTrial participants will:\n\n* Take the trial intervention (either ACT-777991 or placebo) daily for 24 weeks.\n* Visit the clinic 7 times for check-up and tests.",[70],"Non-Segmental Vitiligo","2026-06-24",{"date":73,"type":33},"2026-06-25",{"date":75,"type":22},"2026-06",{"date":77,"type":22},"2027-07",{"name":39,"class":40},{"id":80,"slug":81,"hasResults":11,"nctId":82,"briefTitle":83,"officialTitle":84,"acronym":4,"eligibilityCriteria":85,"healthyVolunteers":11,"sex":86,"minAge":87,"maxAge":88,"enrollmentInfo":89,"targetDuration":91,"studyType":92,"phases":4,"briefSummary":93,"conditions":94,"keywords":96,"overallStatus":98,"whyStopped":4,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":100,"startDateStruct":102,"completionDateStruct":104,"leadSponsor":106,"locationsCount":107},"100554364","registry-to-collect-information-on-pregnancy-neonatal-and-infant-outcomes-in-pregnant-women-exposed-to-quviviq-100554364","NCT06498128","Registry to Collect Information on Pregnancy, Neonatal, and Infant Outcomes in Pregnant Women Exposed to QUVIVIQ®","QUVIVIQ® Pregnancy Registry","A) Eligibility criteria for prospective pregnancies:\n\nInclusion Criteria:\n\n1. Diagnosis of insomnia disorder prior to pregnancy.\n2. Pregnancy is ongoing and outcome of pregnancy (i.e., pregnancy loss or live birth) is not known.\n3. One of the following:\n\n   1. Exposure to QUVIVIQ at any time during the current pregnancy or within 5 half-lives prior to conception.\n   2. Exposure to other, non-orexin receptor antagonist medications for insomnia during pregnancy or within 5 half-lives of the respective insomnia medication prior to conception.\n   3. No exposure to any insomnia medication during pregnancy and within 5 half-lives of any insomnia medication taken prior to conception.\n\nExclusion Criteria:\n\n* Exposure to any orexin receptor antagonist other than QUVIVIQ - including BELSOMRA® (suvorexant), DAYVIGO® (lemborexant), any orexin receptor antagonist newly approved during the study period, or any orexin receptor antagonist in pre-market clinical studies - during the current pregnancy or within 5 half-lives of the respective medication prior to conception.\n\nB) Eligibility criteria for retrospective pregnancies:\n\nInclusion criteria:\n\n1. Diagnosis of insomnia disorder prior to pregnancy.\n2. Pregnancy has ended.\n3. Exposure to QUVIVIQ during pregnancy or within 5 half-lives prior to conception.\n\nExclusion criteria:\n\n* Exposure to any orexin receptor antagonist other than QUVIVIQ - including suvorexant, lemborexant, any orexin receptor antagonist newly approved during the study period, or any orexin receptor antagonist in pre-market clinical studies - during pregnancy or within 5 half-lives of the respective medication prior to conception.","FEMALE","15 Years","50 Years",{"count":90,"type":22},785,"21 Months","OBSERVATIONAL","This study will investigate pregnancy, neonatal, and infant outcomes in women exposed to QUVIVIQ during pregnancy compared to women unexposed to QUVIVIQ during pregnancy.",[95],"Insomnia",[97],"Pregnancy","RECRUITING","2025-12-01",{"date":101,"type":33},"2025-12-02",{"date":103,"type":33},"2024-11-21",{"date":105,"type":22},"2033-03",{"name":39,"class":40},7,""]