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Sirolimus for Leigh Syndrome

NCT06843811 · Status: ENROLLING_BY_INVITATION · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2026-03-31

No results posted yet for this study

Summary

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

Conditions

  • Leigh Syndrome

Interventions

DRUG

Sirolimus

Sirolimus will be given at a starting dose of 0.8 to 1.3 mg/m2 twice daily, depending on subject age and weight.

Sponsors & Collaborators

  • Matthew Demczko

    lead OTHER

Principal Investigators

  • Matthew Demczko, MD · Children's Hospital of Philadelphia

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-02-27
Primary Completion
2027-12-31
Completion
2027-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06843811 on ClinicalTrials.gov