[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Stealth BioTherapeutics Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":81},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,45,60],{"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":29,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":34,"lastUpdatePostDateStruct":35,"startDateStruct":38,"completionDateStruct":40,"leadSponsor":42,"locationsCount":5},"100053968","phase-4-clinical-trial-in-patients-with-barth-syndrome--4tazpower-100053968",false,"NCT07531251","Clinical Trial in Patients With Barth Syndrome- 4TAZPower","Phase 3b\u002F4, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled, Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Patients With Genetically Confirmed Barth Syndrome","4TAZPower","Key Inclusion Criteria:\n\n1. Willing and able to provide signed informed consent form (ICF) prior to participation in any trial-related procedures. If applicable, informed consent in writing from parent(s) or legally-acceptable representative(s) and, informed assent from subject (if age appropriate according to local requirements) should be provided.\n2. Agrees to adhere to the trial requirements for the length of the trial.\n3. Must have genetically confirmed Barth Syndrome (pathogenic variant in the TAZ gene)\n4. Male aged ≥ 5 years at time of the Screening Visit\n5. Left Ventricular Ejection fraction of ≥ 50% by 3-D Echocardiogram at the Screening Visit.\n6. For subjects with a medical history of cardiomyopathy, must be on a stable regimen (unchanged and constant) of background heart failure medications for at least 3 months prior to the Screening Visit.\n7. Able to administer Investigational Medicinal Product (IMP) or have an appropriate designee who can administer the IMP (i.e., a capable family member or a caregiver).\n8. Subjects with female partners of childbearing potential must be willing to use a highly effective method of contraception (e.g., abstinence, dual method of contraception) from the date they sign the ICF until 28 days after the last dose of IMP.\n\nKey Exclusion Criteria:\n\n1. Unable to perform the required functional tests or undergo echocardiography.\n2. History of solid organ transplant, except successful cardiac transplantation \\> 12 months prior to screening, if, in the opinion of the Investigator, there is no evidence of organ rejection and post-transplant pharmacotherapy, is stable, and does not pose additional safety risk to participant.\n3. Patients with an implantable cardioverter defibrillator (ICD) and with a known occurrence of ICD discharge in the 3 months prior to the Screening Visit.\n4. Current placement on the waiting list for heart transplantation.\n5. Hospitalization for heart failure within 6 months prior to the Screening Visit.\n6. Any disease or medical condition that in the opinion of the Investigator would prevent the subject from successfully participating in the trial and reliably completing the assessments or might confound trial results.\n7. Has a history of a systemic eosinophilic illness\n8. Estimated Glomerular Filtration Rate (eGFR) of \\\u003C 30 mL\u002Fmin at the Screening Visit (using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) 2021 formula for subjects \\>16 years of age and the Schwartz 2009 formula for subjects 5-16 years of age).\n9. Active malignancy or any other cancer from which the subject has been cancer-free for \\\u003C 2 years. Localized squamous or non-invasive basal cell skin carcinomas are allowed, if appropriately treated prior to Screening.\n10. Participation in other investigational drug or device clinical trials within 30 days or 5 half-lives (whichever is longer) of Screening; or is currently enrolled in a non-interventional clinical trial that, in the opinion of the Investigator, may be potentially confounding to the results of the current trial.\n11. History of allergic reaction to the IMP or any of its components.\n12. Prior participation in any elamipretide trial or expanded access programs.","MALE","5 Years","55 Years",{"count":21,"type":22},48,"ESTIMATED","INTERVENTIONAL",[25],"PHASE4","Phase 3b\u002F4, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily SC injection of elamipretide in subjects with genetically confirmed BTHS for 72 weeks. The primary trial objective is to confirm the efficacy of elamipretide which is approved in the United States(FORZINITY™) under the accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint.",[28],"Barth Syndrome",[30,31,32],"BTHS","Ultra Rare X-Linked Mitochondrial Disorder","Genetic Defect in TAZ Gene","RECRUITING","2026-07-10",{"date":36,"type":37},"2026-07-13","ACTUAL",{"date":39,"type":37},"2026-07-02",{"date":41,"type":22},"2029-11-30",{"name":43,"class":44},"Stealth BioTherapeutics Inc.","INDUSTRY",{"id":46,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":47,"targetDuration":4,"studyType":23,"phases":48,"briefSummary":26,"conditions":49,"keywords":50,"overallStatus":51,"whyStopped":4,"lastUpdateSubmitDate":52,"lastUpdatePostDateStruct":53,"startDateStruct":55,"completionDateStruct":57,"leadSponsor":58,"locationsCount":59},"100633789",{"count":21,"type":22},[25],[28],[30,31,32],"NOT_YET_RECRUITING","2026-05-04",{"date":54,"type":37},"2026-05-06",{"date":56,"type":22},"2026-06-30",{"date":41,"type":22},{"name":43,"class":44},2,{"id":61,"slug":62,"hasResults":11,"nctId":63,"briefTitle":64,"officialTitle":65,"acronym":4,"eligibilityCriteria":66,"healthyVolunteers":11,"sex":67,"minAge":68,"maxAge":69,"enrollmentInfo":4,"targetDuration":4,"studyType":70,"phases":4,"briefSummary":71,"conditions":72,"keywords":74,"overallStatus":76,"whyStopped":4,"lastUpdateSubmitDate":77,"lastUpdatePostDateStruct":78,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":80,"locationsCount":4},"100415417","an-intermediate-size-expanded-access-protocol-of-elamipretide-100415417","NCT04689360","An Intermediate Size Expanded Access Protocol of Elamipretide","An Intermediate Size Expanded Access Protocol of Elamipretide for Subcutaneous Injection in Patients With Genetically Confirmed Rare Diseases With Known Mitochondrial Dysfunction","Key Inclusion Criteria:\n\n1. ≥1 year and ≤ 80 years of age or ≥12 years for Barth Syndrome in SPIES-007\n2. Patients with genetically confirmed rare primary mitochondrial diseases including Barth Syndrome\n\n   1. observed cardiomyopathy, renal impairment, neuropathic, or ophthalmic manifestation\n   2. where such disease is serious or life-threatening and no comparable or satisfactory alternative therapy options available.\n3. Patients without genetic confirmation of a rare disease with known mitochondrial dysfunction but do exhibit serious or life-threatening clinical manifestations of mitochondrial dysfunction.\n4. Is self-able or has caregiver willing and able to administer SC injection.\n5. Would potentially benefit from treatment with elamipretide and cannot be treated satisfactorily with any approved medicinal product in the opinion of the treating physician.\n\nKey Exclusion Criteria:\n\n1. Known hypersensitivity to elamipretide or any excipients.\n2. Women who are pregnant, are planning on becoming pregnant, or are breast-feeding.\n3. Patients receiving any other investigational agent within 30 days of dosing.\n4. Any active, serious psychiatric, medical, or other conditions\u002Fsituations which, in the treating physician's opinion, could compromise the patient's safety.","ALL","1 Year","80 Years","EXPANDED_ACCESS","Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor to learn more about this program. The treating physician must contact StealthBiotherapeutics using the Expanded Access Program Contacts provided. Elamipretide will only be made available after careful review of an individual request submitted by the treating physician. The initiation and conduct of the treatment with elamipretide for an individual patient, and compliance with this treatment guideline, will be under the full and sole responsibility of the treating physician.",[73,28],"Mitochondrial Diseases",[75],"Barth","AVAILABLE","2025-02-12",{"date":79,"type":37},"2025-02-14",{"name":43,"class":44},""]