Could a common drug help kids with rare leigh syndrome?
NCT ID NCT06843811
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests the drug sirolimus (rapamycin) in 15 people aged 6 months to 55 years with genetically confirmed Leigh syndrome, a rare and serious mitochondrial disease. The goal is to see if the drug is safe and can help with symptoms like muscle weakness and developmental delays. Participants take sirolimus twice daily, and researchers monitor side effects, infections, and changes in muscle function over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- sirolimus (also called rapamycin)
- What this could lead to
- If it works, this could point toward a treatment that slows or stabilizes Leigh syndrome symptoms.
- What could go wrong
- This is a very small, early-phase trial with only 15 participants. It may not show clear benefit, and sirolimus can cause side effects like infections and abnormal lab values.
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.
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
-
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19146, United States
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