Phase 4 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Patients With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting (ENHANCE)

Sarepta Therapeutics, Inc.

Summary

The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.

Eligibility

Age range: 4+ years
Sex: Male
Healthy volunteers: No

Detailed criteria

Key Inclusion Criteria: 1. Cohort 1 only: Is male at birth, ambulatory, and ≥ 4 years of age at the time of dosing. 2. Cohort 1 only: Is eligible for commercial ELEVIDYS. 3. Cohort 2 only: Is male at birth and has previously received ELEVIDYS in a commercial setting after pre-treatment with sirolimus and corticosteroids. 4. Cohort 1 only: Participants who are sexually active must agree to use, for the entire duration of the study, a condom and the female sexual partner must also use a medically acceptable form of birth control (eg, oral contraceptive). 5. Has (a) parent(s) or legal guardian(s…

Interventions

Drug
ELEVIDYS
Administered via an intravenous infusion.
Drug
Sirolimus
Administered orally.
Drug
Glucocorticoids
Administered orally.
Drug
Antibiotics
Administered orally.

Location

Baylor College of Medicine
Houston, Texas

Phase 4 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Patients With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting (ENHANCE)

Summary

Eligibility

Interventions

Location

Compensation

Run this study?

Details

Conditions