Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy

What Was Tested

This registered phase 1, phase 2 trial tested SS-31 (Elamipretide).

It had four arms (groups): one (experimental), one (experimental), one (experimental) and Placebo (placebo comparator).

The registry classes the interventions as drug.

More on the Plan

Each cohort went through 3 distinct periods: Screening, Treatment, and Follow-up.

The Screening Period started with informed consent and may have lasted up to 40 days.

During this period, screening procedures to determine subject eligibility for the study occurred, including confirmation of disease, which incorporated a committee review of the investigator-submitted diagnosis and genetic results.

The Treatment Period began on Day 1 (Visit 2) and lasted for 5 days (until Day 5 \[Visit 6\]).

Who Took Part

The registry lists 36 actual participants took part.

It was open to people of any sex aged from 16 years up to 65 years.

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

Examples of who could join: Diagnosis of mitochondrial disease believed to impair the mitochondrial respiratory chain. Eligibility requires prior genetic confirmation of mitochondrial disease. Diagnosis of mitochondrial myopathy judged by the Investigators to be due to existing mitochondrial disease. Must be able to complete a Screening Visit 6MWT. Body mass index (BMI) score \>15.0 and \<35.0 kg/m2 at Screening Visit.

The full record lists 6 inclusion and 13 exclusion criteria; among the exclusions: had any exclusionary newcastle mitochondrial disease adult scale (nmdas) scores at screening visit; hospitalized (admitted as in-patient) within 1 month prior to the baseline visit; A history of type 1 diabetes mellitus (T1DM).

How the Study Was Run

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

Because people in this SS-31 (Elamipretide) study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

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

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

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

It started in 2015-02 with completion listed for 2016-04.

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

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) (time frame: Assessed at Baseline, Day 5 (end-of-treatment visit)).

It also tracked 27 secondary outcomes, for example Change in Ventilatory Efficiency (VE/VCO2 Slope), Change in Aerobic Efficiency (ΔO2 Consumption/Δ Work Ratio), Change in Oxygen Utilization (ΔVO2/ΔlogVE Ratio), Change in Oxygen Uptake Kinetics (Mean Response Time as Measured by Seconds), Change in Pre-exercise Lactate Levels (mg/dL) and Change in Post-exercise Lactate Levels (mg/dL).

What This Means in Plain Language

Put simply, this is a phase 1, phase 2, randomized trial of SS-31 (Elamipretide) in 36 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.