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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Drug: ELEVIDYS; Drug: Sirolimus; Drug: Glucocorticoids; Drug: Antibiotics

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4,; Phone Number: 1-888-SAREPTA (1-888-727-3782); Email: SareptAlly@sarepta.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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) 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.
6. 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.

Key Exclusion Criteria:

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

3. 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:

   1. The participant's ability to comply with the protocol-required procedures, and/or
   2. The participant's well-being or safety, and/or
   3. The clinical interpretability of the data collected from the participant
4. Cohort 1 only: Has a symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
5. 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.
6. 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.
7. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Cohort 1: ELEVIDYS; Participants will receive ELEVIDYS in a commercial setting on Day 1. Participants will also receive sirolimus, glucocorticoids and antibiotics orally.	Drug: ELEVIDYS; Administered via an intravenous infusion.; Other Names: SRP-9001; Delandistrogene moxeparvovec-rokl; Drug: Sirolimus; Administered orally.; Drug: Glucocorticoids; Administered orally.; Drug: Antibiotics; Administered 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Cohort 1: Number of Participants with ALI	12 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
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
Cohort 1: Duration of ALI	Day 1 up to Week 24
Cohort 1: Amount of Steroid Use	Day 1 up to Week 24
Cohort 1: Duration of Steroid Use	Day 1 up to Week 24
Cohort 1: Quantity of ELEVIDYS Dystrophin Protein Expression as Measured by Western Blot	Week 12

Collaborators and Investigators

• Sponsor: Sarepta Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Estimated): July 31, 2026
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): March 31, 2027

Study Registration Dates

• First Submitted: April 2, 2026
• First Submitted That Met QC Criteria: April 14, 2026
• First Posted (Actual): April 21, 2026

Study Record Updates

• Last Update Posted (Actual): May 26, 2026
• Last Update Submitted That Met QC Criteria: May 22, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Duchenne Muscular Dystrophy; DMD; ELEVIDYS; Delandistrogene moxeparvovec-rokl; SRP-9001
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophy, Duchenne; Anti-Infective Agents; Physiological Effects of Drugs; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Organic Chemicals; Pharmacologic Actions; Chemical Actions and Uses; Therapeutic Uses; Macrolides; Lactones; Adrenal Cortex Hormones; Sirolimus; Anti-Bacterial Agents; Glucocorticoids
• Other Study ID Numbers: SRP-9001-402

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No