Can a mitochondrial drug restore sight in friedreich ataxia?

NCT ID NCT05168774

Completed Symptom relief Sponsor: Children's Hospital of Philadelphia Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This pilot study tested the drug elamipretide in 20 people with Friedreich ataxia to see if it could safely improve vision loss. Participants received either a low or high dose, and researchers measured changes in eyesight. The study is complete, but results are not yet available.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: elamipretide
What this could lead to: If it works, this could point toward a treatment for vision loss in Friedreich ataxia.
What could go wrong: This is a very small, early-phase pilot study with only 20 participants. The drug may not improve vision, and results may not apply to other symptoms of Friedreich ataxia.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Friedreich ataxia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

FRIEDREICH ATAXIA

Contacts and locations

Locations

Children's Hospital of Philadelphia - Neurology

Philadelphia, Pennsylvania, 19104, United States

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