18+ · Research use only
Elamipretide · Phase 2/3 · Completed
TAZPOWER
In TAZPOWER, a phase 2/3 trial of elamipretide in males aged 12 and over with Barth syndrome (n=12), the double-blind crossover part did not show a statistically significant difference from placebo on either primary endpoint (ClinicalTrials.gov NCT03098797 (TAZPOWER)).
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Updated 24 September 2026
Results
The double-blind crossover part did not show a statistically significant difference from placebo on either primary endpoint.
Side effects reported
TAZPOWER: The extension report names injection site reactions as the most commonly reported adverse event, without rates by arm.
Design
Trial design and sources
- Compound
- Elamipretide
- Phase
- 2/3
- Population
- Males aged 12 and over with Barth syndrome
- Participants randomised
- 12
- Duration
- 28 weeks
- Primary endpoint
- Six-minute walk test distance and the Barth Syndrome Symptom Assessment total fatigue score, in a placebo-controlled crossover of two 12-week periods
- Estimand
- Not stated in the source read
- Read out
- 1 November 2020
Ten of the twelve participants continued into an open-label extension of up to 168 weeks with no placebo arm. The extension report names injection site reactions as the most commonly reported adverse event, without rates by arm.
Questions
TAZPOWER, answered
What did TAZPOWER find?
In TAZPOWER, a phase 2/3 trial of elamipretide in males aged 12 and over with Barth syndrome (n=12), the double-blind crossover part did not show a statistically significant difference from placebo on either primary endpoint (ClinicalTrials.gov NCT03098797 (TAZPOWER)).
What side effects were reported in TAZPOWER?
TAZPOWER: The extension report names injection site reactions as the most commonly reported adverse event, without rates by arm.