Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)

What Was Tested

The trial (phase 2) is built around SS-31 (Elamipretide).

This is a Phase 2 Open-Label extension study to evaluate the long term safety and tolerability of daily elamipretide injections in patients with genetically confirmed Primary Mitochondrial Disease who previously participated in the SPIMM-202 Clinical Trial.

It had 1 arm (groups): elamipretide (experimental).

The registry classes the interventions as drug.

More on the Plan

This open-label, non-comparative, extension trial will enroll subjects with genetically confirmed PMD who have completed the End-of-Study Visit in the SPIMM-202 trial.

Who Took Part

Enrollment: 28 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 completed the End-of-Study Visit in SPIMM-202.

The full record lists 2 inclusion and 5 exclusion criteria; among the exclusions: subject experienced an adverse reaction attributed to study drug resulting in permanent discontinuation of study drug in the spimm-202 trial; female subjects who are pregnant, planning to become pregnant, or lactating; subject has undergone an in-patient hospitalization within the 1 month prior to the spimm-203 baseline visit.

How the Study Was Run

It was run as a single-group and open-label study.

Everyone knew when SS-31 (Elamipretide) was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2016-12 with completion listed for 2020-03-09.

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

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Distance Walked on Six Minute Walk Test (time frame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks).

It also tracked 11 secondary outcomes, for example EuroQol 5 Dimensions 5 Levels (EQ-5D-5L), Euroquol Visual Analog Scale, NeuroQOL Fatigue Questionnaire, Patient Global Assessment : Categorical, Patient Global Assessment: Continuous Variable and Work Limitations Questionnaire.

What This Means in Plain Language

Put simply, this is a phase 2 trial of SS-31 (Elamipretide) in 28 people.

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

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.