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

• Register: ClinicalTrials.gov
• Last refreshed on: 21 March 2022
• 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, Tolerability, and Efficacy of Subcutaneous Injections of Elamipretide (MTP-131) in Subjects With Genetically Confirmed Barth Syndrome Followed by an Open-Label Treatment Extension
• Date of first enrolment: May 1, 2017
• Target sample size: 12
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/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

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 at screening

- Ambulatory and impaired during the 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 occurrance 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

Primary Outcome(s)

Change in distance walked during the 6-minute walk test (6MWT) [Time Frame: 12 weeks]

Secondary Outcome(s)

Change in 2-D and 3-D echocardiographic measurements [Time Frame: 12 weeks]

Change in muscle strength as measured by handheld dynamometry [Time Frame: 12 weeks]

Change in Patient Reported Outcomes [Time Frame: 12 weeks]

Change in five times sit-to-stand test [Time Frame: 12 weeks]

Change in SWAY application balance assessments [Time Frame: 12 weeks]

Change in biomarkers [Time Frame: 12 weeks]

Change in Clinician Global Impression [Time Frame: 12 weeks]

Change in accelerometry counts [Time Frame: 12 weeks]

Number of participants with treatment related adverse events [Time Frame: 12 weeks]

Secondary ID(s)

SPIBA-201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors