A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome

What Was Tested

This registered phase 2, phase 3 trial tested 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.

Who Took Part

The registry lists 12 actual participants took part.

It was open to men only aged from 12 years with no upper age limit.

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

Examples of who could join: Genetically confirmed Barth Syndrome. Male aged 12 and above. At the screening visit, eGFR must meet the following:. Ambulatory and impaired during the baseline 6MWT. On stable medication for 30 days prior to the baseline visit.

The full record lists 7 inclusion and 11 exclusion criteria; among the exclusions: undergone any inpatient hospitalizations within 30 days of the baseline visit; is undergoing an apparent pubertal growth spurt; has uncontrolled hypertension.

How the Study Was Run

Design: randomized, crossover 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.

In the crossover set-up each of the participants took both SS-31 (Elamipretide) and the comparison in turn, acting as their own control.

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 2017-07-05 with completion listed for 2018-10-05.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit. Part 1: Total Fatigue Score Based on the BTHS-SA by Visit.

It also tracked 12 secondary outcomes, for example Part 2: Distance Walked During the 6MWT, Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit, Part 1: Muscle Strength as Measured by HHD by Visit, Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit, Part 1: 5XSST by Visit and Part 2: Change From Baseline: 5X Sit to Stand by Visit.

What This Means in Plain Language

Put simply, this is a phase 2, phase 3, randomized trial of SS-31 (Elamipretide) in 12 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.