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Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial

Plain-language guide to PMID 37268435 (2023): double-blind randomized controlled trial study of SS-31 (Elamipretide).

Key Facts

PMID
37268435
Journal
Neurology
Year
2023
Study type
Randomized Controlled Trial
Authors
Karaa A et al. (32 authors)
DOI
10.1212/WNL.0000000000207402

What This Study Did, in Plain Language

What Was Tested

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

Primary mitochondrial myopathies (PMMs) encompass a group of genetic disorders that impair mitochondrial oxidative phosphorylation, adversely affecting physical function, exercise capacity, and quality of life (QoL).

Why It Was Studied

Trial registered with clinicaltrials.gov, Clinical Trials Identifier: NCT03323749; submitted on October 12, 2017; first patient enrolled October 9, 2017.

Gov/ct2/show/NCT03323749?term = elamipretide&draw = 2&rank = 9.

Who Took Part

The age groups tagged for this paper are middle-aged adults, both men and women.

Secondary endpoints included most bothersome symptom score on the PMMSA, NeuroQoL Fatigue Short-Form scores, and the patient global impression and clinician global impression of PMM symptoms.

How the Study Was Run

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

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

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

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

What Was Measured

The subject headings attached to it are Quality of Life, Merozoite Surface Protein 1, Mitochondrial Myopathies, Fatigue and Oligopeptides.

What This Means in Plain Language

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

The abstract goes on to report its results in about 10 sentences; this page does not restate them, so read the paper for the findings and their limits.

The publication is from 2023 (Neurology), a journal based in United States.

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

Our Notes on the Compound

SS-31 (Elamipretide): A mitochondria-targeted peptide, described as a "bodyguard" for the inner mitochondrial membrane.

Related Pages

Source: PubMed record. This explanation is written by PeptideWhiz from the public record. For research and education only. Not medical advice; no dosing information is given.