Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)

What Was Tested

In this phase 3 trial, researchers set out to test SS-31 (Elamipretide).

It had two arms (groups): Elamipretide (experimental) and Placebo (placebo comparator).

The registry classes the interventions as drug.

Who Took Part

The registry lists 102 actual participants took part.

It was open to people of any sex aged from 18 years up to 70 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Willing and able to provide a signed informed consent form (ICF) prior to participation in any trial-related procedures. Agrees and is able to adhere to the trial requirements for the length of the trial, including administration of assigned treatment. Is ≥18 years and ≤ 70 years of age at the time of screening. Diagnosed with nPMD with a predominant clinical manifestation of myopathy, which must include progressive external ophthalmoplegia (PEO) and exercise intolerance and/or skeletal muscle weakness, with genetic confirmation of either:. Nuclear DNA mutation of the mitochondrial replisome (replisome-related mutations), which include the following genes:.

The full record lists 25 inclusion and 15 exclusion criteria; among the exclusions: female subjects who are pregnant, planning to become pregnant, or breastfeeding/lactating; walks \< 150 meters or \> 450 meters during the 6mwt (screening visit only); has had any prior interventional cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months prior to screening.

How the Study Was Run

It was run as a randomized, parallel-group and quadruple-blind study.

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

Groups ran in parallel: each of the participants stayed in one arm throughout.

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

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2022-04-29 with completion listed for 2024-09-30.

It ran at 32 sites in United States (9), Italy (6), Spain (4), Germany (3), United Kingdom (3) and Australia (2).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Six-minute walk test (6MWT) (time frame: Baseline, Weeks 12, 24, 36, 48, 52 (End of Trial Visit)).

It also tracked 3 secondary outcomes, for example 5 times sit-to-stand test (5XSST), Triple Timed up-and-go test (3TUG) and Patient Global Impression of Severity (PGI-S) Scale.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of SS-31 (Elamipretide) in 102 people.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

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