Can a mitochondrial drug restore sight in friedreich ataxia?
NCT ID NCT05168774
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.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Children's Hospital of Philadelphia - Neurology
Philadelphia, Pennsylvania, 19104, United States