Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study

What Was Tested

In this phase 2 trial, researchers set out to test SS-31 (Elamipretide).

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

The registry classes the interventions as drug.

More on the Plan

Each sequence group went through 5 distinct periods: Screening, Treatment Period 1, Washout, Treatment Period 2, and Follow-Up.

Who Took Part

Enrollment: 30 actual participants took part.

It was open to people of any sex aged from 16 years with no upper age limit.

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

Examples of who could join: Subject must reside in North America for the duration of the study. Subject has not received study drug in the SPIMM-201 study within 3 weeks prior to Screening. Subject has been on stable (unchanged and constant) medications (including over-the counter treatments, vitamins, or supplements) for at least 1 month prior to the Baseline Visit.

The full record lists 5 inclusion and 10 exclusion criteria; among the exclusions: female subjects who are pregnant, planning to become pregnant, or lactating; subject has a history of active alcoholism or drug addiction during the year before the screening visit.

How the Study Was Run

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

The participants were allocated by chance, so the SS-31 (Elamipretide) group and the comparison group should start out alike.

Each person switched between SS-31 (Elamipretide) and the comparison at some point, usually with a gap in between.

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 2016-08-22 with completion listed for 2017-03-10.

It ran at 4 sites in United States (4).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Distance Walked on the 6-minute Walk Test (6MWT) by Visit (time frame: End of Week 4 and End of Week 12).

It also tracked 9 secondary outcomes, for example Wrist Accelerometer Counts by Day, Average Hip Accelerator Counts by Day, Neuro-QoL Fatigue Short Form Score: Total T-Scores (Question 1-8), Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) Total Fatigue Score by Week, Triple Timed Up and Go (3TUG) Test by Visit and Patient Global Assessment [PGA] Score by Visit, Continuous.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of SS-31 (Elamipretide) in 30 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

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