Human evidence 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.

Quick read

Human evidence record

What this study tells us

Participants were assigned to comparison groups by chance, usually with placebo and/or blinding to reduce bias. This is a strong design for testing cause and effect, but the population, duration and endpoints still limit what can be concluded.

Who was studied?

Barth syndrome

How many?

n=12

What happened?

Blinded primary endpoints were not significantly different; longer open-label extension produced signals used in the broader rare-disease evidence package.

What stops us overclaiming?

Extremely small rare-disease sample; open-label extension is vulnerable to bias and should be separated from blinded results.

Need help with terms such as randomized, placebo, endpoint or sample size? Open the glossary →

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

Open Elamipretide page →Open primary source →