Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting

Part of paid clinical trials in Houston, Texas.

Sponsor: Sarepta Therapeutics, Inc.
Study ID: NCT07542314
Phase: PHASE4
Status: Not Yet Recruiting

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Conditions

Duchenne Muscular Dystrophy

Eligibility Criteria

Sex: MALE
Age: 4 Years - N/A
Healthy Volunteers: Not accepted

Interventions

ELEVIDYS — DRUG
Administered via an intravenous infusion.
Sirolimus — DRUG
Administered orally.
Glucocorticoids — DRUG
Administered orally.
Antibiotics — DRUG
Administered orally.

Study Details

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.

Key Dates

First listed: Apr 21, 2026
Start date: Aug 31, 2026
Status verified: Aug 2026
Primary completion: Aug 31, 2027
Completion: Aug 31, 2027

Study Design

Enrollment: 20 participants (estimated)
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Arms

Other: Cohort 1: ELEVIDYS
Participants will receive ELEVIDYS in a commercial setting on Day 1. Participants will also receive sirolimus, glucocorticoids and antibiotics orally.
No Intervention: Cohort 2: ELEVIDYS
Participants who have previously received ELEVIDYS in a commercial setting after prophylactic treatment with sirolimus and corticosteroids will participate in one study visit during which a muscle biopsy will be performed. No study treatment will be administered in this cohort.