Study record · Elamipretide
TAZPOWER Barth syndrome crossover trial
Blinded primary endpoints were not significantly different; longer open-label extension produced signals used in the broader rare-disease evidence package.
Key result
Blinded primary endpoints were not significantly different; longer open-label extension produced signals used in the broader rare-disease evidence package.
Study details
Study designRandomized double-blind placebo-controlled crossover study
PhasePhase II/III rare disease
PopulationBarth syndrome
Sample size12
RouteSubcutaneous
Regimen / exposure40 mg once daily
Duration12 weeks per blinded period, followed by extension
ComparatorPlacebo
Primary endpoint / question6-minute walk test and symptom measures
Safety resultInjection-site reactions reported; small programme limits certainty.
Key limitationsExtremely small rare-disease sample; open-label extension is vulnerable to bias and should be separated from blinded results.
Registry / identifierPMID 33077895