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

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

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Conditions

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

Start date
Jul 31, 2026
Status verified
May 2026
Primary completion
Mar 31, 2027
Completion
Mar 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.

Primary Outcome Measure

Cohort 1: Number of Participants with ALI [ Time Frame: 12 weeks ]

Central Contacts

  • Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4,
    1-888-SAREPTA (1-888-727-3782)
    [email protected]

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