ELEVIDYS With Added Sirolimus in Duchenne Muscular Dystrophy, Looking at Liver Injury
Tests ELEVIDYS plus sirolimus in people with Duchenne muscular dystrophy, watching for liver injury.
Plain-language title generated from the registry record
Registry title: Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
Key facts
- Where
- Houston, United StatesSee the site
- Phase
- Phase 4
Where in United States
- Baylor College of MedicineHouston, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Sarepta Therapeutics, Inc.
- Trial code
- NCT07542314
- Record updated
- 6 October 2026
- Original description
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.Read full original descriptionShow less
Phase 4 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Patients With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting (ENHANCE)
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.
Inclusion: Cohort 1 only: Is male at birth, ambulatory, and ≥ 4 years of age at the time of dosing; Cohort 1 only: Is eligible for commercial ELEVIDYS; Cohort 2 only: Is male at birth and has previously received ELEVIDYS in a commercial setting after pre-treatment with sirolimus and corticosteroids; 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); Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with the study visit schedule and all other protocol requirements, or is ≥ 18 years of age and personally able to understand and comply with the protocol requirements; Either has a parent or legal guardian who is willing to provide informed consent, or is ≥ 18 years of age and able to provide informed consent independently.
Exclusion: Has a medical condition or confounding circumstances (eg, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:; The participant's ability to comply with the protocol-required procedures, and/or; The participant's well-being or safety, and/or; The clinical interpretability of the data collected from the participant; Cohort 1 only: Has a symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1; Cohort 1 only: Has received a live virus vaccine within 4 weeks or inactive vaccine within 2 weeks of the Day 1 visit or expects to receive a vaccination during the first 3 months after Day 1; Cohort 1 only: Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection; Cohort 1 only: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients; Cohort 1 only: Contraindicated to receive ELEVIDYS per the United States Package Insert (USPI); Cohort 1 only: Any wounds or recent injuries that, in the opinion of the Investigator, would be at risk of dehiscence or impaired healing in the setting of sirolimus administration; Other inclusion/exclusion criteria may apply, per protocol.