A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism

What Was Tested

The paper reports a study of SS-31 (Elamipretide), published as a randomized controlled trial.

The researchers performed a randomized, double-blind, placebo-controlled crossover trial followed by an open-label extension in BTHS to test the effect of elamipretide, a mitochondrial tetrapeptide that interacts with cardiolipin.

Who Took Part

According to the abstract, 1, subjects were involved.

How the Study Was Run

In design terms this was a randomized, double-blind, open-label, placebo-controlled and crossover study.

Random assignment meant chance, not the researchers, decided who got SS-31 (Elamipretide) and who got the comparison, which limits bias.

Blinding on both sides kept the subjects and researchers unaware of who received SS-31 (Elamipretide).

Everyone knew when SS-31 (Elamipretide) was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

The comparison arm used a placebo, so changes seen with SS-31 (Elamipretide) could be set against what happens without it.

The timeline in the abstract includes 12 weeks.

What Was Measured

The subject headings attached to it are Barth Syndrome, Cardiolipins, Mitochondria and Oligopeptides.

Author keywords: 6-minute walk test and elamipretide.

What This Means in Plain Language

The paper is a randomized, double-blind and open-label study of SS-31 (Elamipretide) in 1, subjects over 12 weeks.

With fewer than 40 people, it is a small study, so it is better read as an early signal for further research than as a settled answer.

Its findings take up roughly 4 sentences of the abstract and are left out here on purpose; the source paper is the place to read them.

The publication is from 2021 (Genetics in medicine : official journal of the American College of Medical Genetics), a journal based in United States.

More context: the SS-31 (Elamipretide) compound page, all human trials.