A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension

What Was Tested

This registered phase 3 trial tested SS-31 (Elamipretide).

This will be followed by an open-label treatment extension.

It had three arms (groups): Part 1: Elamipretide (experimental), Part 1: Placebo (placebo comparator) and Part 2: Elamipretide open label (experimental).

Placebo SC daily.

The registry classes the interventions as combination product.

Who Took Part

The registry lists 218 actual participants took part.

It was open to people of any sex aged from 16 years up to 80 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 prior to participation in any trial-related procedures. Agrees to adhere to the trial requirements for the length of the trial, including the use of the elamipretide delivery system. Subject is ≥ 16 and ≤ 80 years of age. Diagnosed with PMM in the opinion of the investigator and confirmed by an Adjudication Committee.

The full record lists 5 inclusion and 24 exclusion criteria; among the exclusions: female who are pregnant, planning to become pregnant, or breastfeeding/lactating; ECG evidence of acute ischemia, atrial fibrillation, or active conduction system abnormalities with the exception of any of the following:; first degree atrioventricular bock (av-block).

How the Study Was Run

Design: randomized, parallel-group and quadruple-blind.

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

The SS-31 (Elamipretide) group and the comparison group ran side by side rather than switching.

Neither the participants nor the staff measuring them knew who was on SS-31 (Elamipretide), so expectations could not easily colour the readings.

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

It started in 2017-10-09 with completion listed for 2020-02-10.

It ran at 27 sites in United States (13), Italy (6), Canada (2), Germany (2), United Kingdom (2) and Denmark (1).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Six-minute Walk Test (6MWT) (time frame: Baseline to 24 weeks). Total Fatigue Score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) (time frame: Baseline to 24 weeks).

It also tracked 4 secondary outcomes, for example Fatigue During Activities Score on the Primary Mitochondrial Disease Symptom Assessment (PMMSA), Neuro-QoL Fatigue Activities of Daily Living, Change From Baseline in the Most Bothersome Symptom Score on the Primary Mitochondrial Myopathy Symptoms Assessment and Neuro-QoL Fatigue Short Form Score.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of SS-31 (Elamipretide) in 218 people.

It ended early (terminated); results are posted on the registry, and this page does not restate them.

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