Sirolimus for Leigh Syndrome

Conditions

• Leigh Syndrome

Eligibility Criteria

Sex

ALL

Age

6 Months - 55 Years

Healthy Volunteers

Not accepted

Interventions

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

Study Details

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

Key Dates

Start date

Feb 27, 2025

Status verified

Mar 2026

Primary completion

Dec 31, 2027

Completion

Dec 31, 2027

Study Design

Enrollment

15 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Phase 2A
  Participants will receive Sirolimus for at least 24 weeks at a starting dose of 0.8 to 1.3 mg/m2 two (2) times daily.
• Experimental: Long-Term Extension
  Eligible participants may continue Sirolimus treatment for up to two (2) years.

Primary Outcome Measure

Rate of Adverse Events [ Time Frame: Up to 2.5 years ]

Locations (1)

Find similar trials in Philadelphia, PA

Related Studies

• Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
  Recruiting · Sanford Health · Sioux Falls, South Dakota
• The International Registry for Leigh Syndrome
  Recruiting · The University of Texas Health Science Center, Houston · Houston, Texas