2025-523837-25-00招募中4 期
4TAZPower: A Phase 3b/4, 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
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 22
- 试验地点
- 5
- 主要终点
- Change from Baseline to Week 72 in a composite normalized score of the following three functional tests: 6MWT, 3TUG, and 5XSST
研究概览
简要总结
Evaluate the efficacy of once daily subcutaneous (SC) injections of elamipretide in subjects who have genetically confirmed Barth Syndrome
入排标准
- 年龄范围
- 0 years 至 65+ years（18-64 Years, 65+ Years, 0-17 Years）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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.
- •Agrees to adhere to the trial requirements for the length of the trial.
- •Must have genetically confirmed Barth syndrome (pathogenic variant in the TAZ gene)
- •Male aged ≥ 5 years at time of the Screening Visit.
- •Left Ventricular Ejection fraction of ≥ 50% by 3-D/2-D Echocardiogram at the Screening Visit.
- •Able to administer IMP or have an appropriate designee who can administer the IMP (i.e., a capable family member or a caregiver).
- •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.
排除标准
- •Unable to perform the 6MWT, 3TUG, or 5XSST functional tests or undergo echocardiography.
- •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.
- •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
- •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.
- •Patients with an implantable cardioverter defibrillator (ICD) and with a known occurrence of ICD discharge in the 3 months prior to the Screening Visit.
- •Current placement on the waiting list for heart transplantation.
- •Hospitalization for heart failure within 6 months prior to the Screening Visit.
- •Active malignancy or any other cancer from which the subject has been cancer-free for < 2 years. Localized squamous or non-invasive basal cell skin carcinomas are allowed, if appropriately treated prior to Screening.
研究组 & 干预措施
Elamipretide injection placebo
Placebo
干预措施: Elamipretide injection placebo (Drug)
Elamipretide
Test
干预措施: Elamipretide (Drug)
结局指标
主要结局
Change from Baseline to Week 72 in a composite normalized score of the following three functional tests: 6MWT, 3TUG, and 5XSST
Change from Baseline to Week 72 in a composite normalized score of the following three functional tests: 6MWT, 3TUG, and 5XSST
次要结局
- Change from Baseline to Week 72 in the 6MWT
- Change from Baseline to Week 72 in the 3TUG
- Change from Baseline to Week 72 in the 5XSST
- Change from Baseline to Week 72 in the Patient Global Impression of Severity Scale (PGI-S)
- Change from Baseline to Week 72 in the Clinician Global Impression of Severity Scale (CGI-S)
- Change from Baseline to Week 72 in knee extensor muscle strength as measured by handheld dynamometry (HHD)
- Change from Baseline to Week 72 in hip flexor muscle strength as measured by HHD
研究者
Chief Clinical Development Officer
Scientific
Stealth Biotherapeutics Inc.
研究点 (5)
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