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

NCT07542314 | Not Yet Recruiting Phase 4 FDA Drug

• 1 other identifier: SRP-9001-402
• Study Type: interventional
• Target: 20
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne Muscular Dystrophy; DMD; ELEVIDYS; Delandistrogene moxeparvovec-rokl; SRP-9001

Outcome Measures

Primary Outcomes (1)

• Cohort 1: Number of Participants with ALI

  12 weeks

Secondary Outcomes (9)

• Cohort 1: Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESIs) and Serious Adverse Events (SAEs)

  Day 1 up to Week 24

• Cohort 1: Number of Participants with Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Interstitial Lung Disease/Non-infectious Pneumonitis

  Day 1 up to Week 24

• Cohort 1: Number of Participants with Hepatic Adverse Events, Hepatic Biomarkers, and Laboratory Assessments Indicative of Either Acute Hepatocellular Injury or Acute Liver Dysfunction

  Day 1 up to Week 24

• Cohort 1: Number of Participants with Severe ALI

  Day 1 up to Week 24

• Cohort 1: Number of Participants with ALI

  Day 1 up to Week 24

Study Arms (2)

Cohort 1: ELEVIDYS

OTHER

Participants will receive ELEVIDYS in a commercial setting on Day 1. Participants will also receive sirolimus, glucocorticoids and antibiotics orally.

Drug: ELEVIDYS; Drug: Sirolimus; Drug: Glucocorticoids; Drug: Antibiotics

Cohort 2: ELEVIDYS

NO INTERVENTION

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.

Interventions

ELEVIDYS DRUG

Administered via an intravenous infusion.

Also known as: Delandistrogene moxeparvovec-rokl, SRP-9001

Cohort 1: ELEVIDYS

Sirolimus DRUG

Administered orally.

Cohort 1: ELEVIDYS

Glucocorticoids DRUG

Administered orally.

Cohort 1: ELEVIDYS

Antibiotics DRUG

Administered orally.

Cohort 1: ELEVIDYS

Eligibility Criteria

• Age: 4 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• Cohort 1 only: Contraindicated to receive ELEVIDYS per the United States Package Insert (USPI).
• Cohort 1 only: Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.
• 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: 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: 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.

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Sirolimus; Glucocorticoids; Anti-Bacterial Agents

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals; Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Physiological Effects of Drugs; Pharmacologic Actions; Chemical Actions and Uses; Anti-Infective Agents; Therapeutic Uses

Central Study Contacts

Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4,

CONTACT

1-888-SAREPTA (1-888-727-3782); SareptAlly@sarepta.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 2, 2026
• First Posted: April 21, 2026
• Study Start: April 30, 2026
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): March 31, 2027
• Last Updated: April 21, 2026

Record last verified: 2026-04