Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER

What Was Tested

This randomized controlled trial paper looks at SS-31 (Elamipretide).

Who Took Part

By PubMed's age tags, the people studied were teenagers, young adults, adults and middle-aged adults, both men and women.

OLE primary endpoints were safety and tolerability; secondary endpoints included change from baseline in the 6-minute walk test (6MWT) and BarTH Syndrome Symptom Assessment (BTHS-SA) Total Fatigue score.

Muscle strength, physician- and patient-assessed outcomes, echocardiographic parameters, and biomarkers, including cardiolipin (CL) and monolysocardiolipin (MLCL), were assessed.

How the Study Was Run

The researchers set it up as a randomized, double-blind, open-label and placebo-controlled study.

Because people in this SS-31 (Elamipretide) study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

As a double-blind SS-31 (Elamipretide) study, the assignments were hidden from both participants and investigators until the end.

Being open-label, the SS-31 (Elamipretide) study did not mask who received what.

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

TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE.

What Was Measured

The subject headings attached to it are Barth Syndrome, Oligopeptides, Muscle Strength, Fatigue and Cardiolipins.

Author keywords: Cardiomyopathy, Elamipretide and Mitochondrial disease.

What This Means in Plain Language

The paper is a randomized, double-blind and open-label study of SS-31 (Elamipretide).

Results are reported in around 7 sentences of the abstract; we leave them to the paper itself rather than summarise them.

The publication is from 2024 (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.