FRDA Investigator Initiated Study (IIS) With Elamipretide

What Was Tested

The trial (phase 1, phase 2) is built around SS-31 (Elamipretide).

It had two arms (groups): one (experimental) and one (experimental).

The registry classes the interventions as drug.

More on the Plan

The interim analysis will be based on data from a 36-week visit.

For subjects worse than 20/800 at study start, they will be followed using low vision alternatives only.

Who Took Part

20 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: Genetically confirmed FRDA (point mutations allowed). Age \>16 years. Disease onset before 18 years of age.

The full record lists 8 inclusion and 11 exclusion criteria; among the exclusions: any unstable illness that in the investigator's opinion precludes participation in the study; use of any investigational product within 30 days prior to screening; A history of substance abuse.

How the Study Was Run

It was run as a randomized, parallel-group and open-label study.

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.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Being open-label, the SS-31 (Elamipretide) study did not mask who received what.

It started in 2022-03-03 with completion listed for 2024-06-25.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Change in High Contrast Visual Acuity (time frame: Baseline to 52 weeks).

It also tracked 7 secondary outcomes, for example Change in Low Contrast Visual Acuity, Change in Low Luminescence Visual Activity, Change in Retinal Nerve Fiber Layer by Optical Coherence Tomography (OCT), Change in Visual Quality of Life by Visual Functioning Questionnaire (VFQ), Change in Cardiac Strain and Change in Cardiac Fibrosis.

What This Means in Plain Language

Put simply, this is a phase 1, phase 2, randomized trial of SS-31 (Elamipretide) in 20 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.