A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 22 April 2024
• Main ID: NCT03098797
• Date of registration: 21/03/2017
• Prospective Registration: Yes
• Primary sponsor: Stealth BioTherapeutics Inc.
• Public title: A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome TAZPOWER
• Scientific title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Trial to Evaluate the Safety and Efficacy of Subcutaneous Injections of Elamipretide in Subjects With Genetically Confirmed Barth Syndrome Followed by Open-Label Treatment
• Date of first enrolment: July 5, 2017
• Target sample size: 12
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03098797
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2/Phase 3

Countries of recruitment

United States

Contacts

• Name: Hilary Vernon, MD, PhD
• Affiliation: McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University, Baltimore, MD, USA

Key inclusion & exclusion criteria

Inclusion Criteria:

- Genetically confirmed Barth Syndrome

- Male aged 12 and above

- At the screening visit, eGFR must meet the following:

1. Body weight >30 kg AND eGFR = 90mL/min at screening

2. Body weight >40kg AND eGFR =60 but <90mL/min/ 1.73m² at screening

- Ambulatory and impaired during the baseline 6MWT

- On stable medication for 30 days prior to the baseline visit

Exclusion Criteria:

- Participated in another interventional clinical trial within 30 days of or is
currently enrolled in a non-interventional clinical trial at the baseline visit
potentially confounding with this trial

- Prior or current medical condition that would prevent the subject from safely
participating in the trial

- Undergone any inpatient hospitalizations within 30 days of the baseline visit

- Is undergoing an apparent pubertal growth spurt

- Has uncontrolled hypertension

- History of substance abused within the year before the baseline visit or is likely to
be uncompliant

- History of heart transplantation or current placement on the waiting list for a heart
transplant

- For subjects with an ICD: known occurrence of ICD discharge in the 3 months prior to
the baseline visit

- For subjects without an ICD: expected to undergo an implantation of an ICD during the
conduct of the study

- Currently receiving treatment with chemotherapeutic agents or immunosuppressant agents
or has received prior radiation therapy to the chest

- Recipient of stem cell or gene therapy or is currently being treated by a therapeutic
investigational device



Age minimum: 12 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Barth Syndrome

Intervention(s)

Drug: Elamipretide

Drug: Placebo

Primary Outcome(s)

Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. [Time Frame: Pre-dose, Weeks 1, 4, 8, and 12 (end of treatment)]

Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit [Time Frame: Pre-dose to Week 12 (end of treatment)]

Secondary Outcome(s)

Part 1: Muscle Strength as Measured by HHD by Visit [Time Frame: Pre-dose to Week 12 (end of treatment)]

Part 2: Change From Baseline: CGI Symptom Scale [Time Frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192]

Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit [Time Frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192]

Part 2: Distance Walked During the 6MWT [Time Frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192]

Part 1: Patient Global Impression Scales of Symptoms [Time Frame: Pre-dose, Week 1, Week 12 (end of treatment)]

Part 1: SWAY Application Balance Assessments by Visit [Time Frame: Pre-dose to Week 12 (end of treatment)]

Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit [Time Frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192]

Part 1: 5XSST by Visit [Time Frame: Pre-dose to Week 12 (end of treatment)]

Part 1: Part 1: Clinician Global Impression [Time Frame: Pre-dose to Week 12 (end of treatment)]

Part 2: Change From Baseline: 5X Sit to Stand by Visit [Time Frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192]

Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit [Time Frame: Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192]

Part 2: Change From Baseline: SWAY Application Balance Assessment [Time Frame: Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192]

Secondary ID(s)

SPIBA-201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 16/04/2024
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03098797