Clinical trials: Mitochondrial Respiratory Chain Deficiencies, active

Leigh Syndrome

Sirolimus for Leigh Syndrome

• Enrolling by invitation
• Age: 6 months to 55 years
• Phase 2

The purpose of this study is to evaluate the safety and efficacy of the drug Sirolimus in participants with Leigh syndrome.

Trial name

NCT06843811

Testing

Sirolimus

Where

United States

Volunteers

15 people

Sponsor

Matthew Demczko