A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

NCT06128564 | Active Not Recruiting Phase 2 DMC FDA Drug

• 3 other identifiers: BN438812022-000691-192023-509901-57-00
• Study Type: interventional
• Target: 13
• Locations: 6 countries
• Sites: 7

Brief Summary

This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD; Duchenne; Dystrophin; Gene-Delivery; Pediatric

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)

  Baseline up to Week 260

Secondary Outcomes (1)

• Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot

  Baseline, Week 12

Study Arms (1)

Delandistrogene Moxeparvovec

EXPERIMENTAL

Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.

Genetic: delandistrogene moxeparvovec

Interventions

delandistrogene moxeparvovec GENETIC

Single IV infusion of delandistrogene moxeparvovec

Also known as: SRP-9001, ELEVIDYS, RO7494222, delandistrogene moxeparvovec-rokl

Delandistrogene Moxeparvovec

Eligibility Criteria

• Age: 2 Years - 3 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Cohort A: \>=3 years of age to \<4 years of age
• Cohort B: \>=2 years of age to \<3 years of age
• Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
• Able to cooperate with age-appropriate motor assessment testing
• A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive)

You may not qualify if:

• Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression, within protocol-specified time limits
• Recombinant Adeno-Associated Virus Serotype rh74 (rAArh74) antibody titers are elevated, as per protocol-specified criteria
• Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline
• Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer
• Medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the participant's ability to comply with the protocol required testing or procedures, or compromise the participant's well-being or safety, or clinical interpretability

Sponsors & Collaborators

• Hoffmann-La Roche: lead
• Sarepta Therapeutics, Inc.: collaborator

Study Sites (7)

Chr de La Citadelle

Liège, 3500, Belgium

Location

Hôpital Necker-Enfants Malades

Paris, 75015, France

Location

Universitätsklinikum Essen

Essen, 45147, Germany

Location

PU A. Gemelli, Università Cattolica del Sacro Cuore

Rome, Lazio, 00168, Italy

Location

Hospital Sant Joan De Deu

Esplugues de Llobregas, Barcelona, 08950, Spain

Location

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

Location

John Radcliffe Hospital

Oxford, OX3 9DU, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

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

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 8, 2023
• First Posted: November 13, 2023
• Study Start: November 29, 2023
• Primary Completion (Estimated): February 28, 2030
• Study Completion (Estimated): January 30, 2034
• Last Updated: February 10, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share

Locations

BE (1); FR (1); GB (2); IT (1); ES (1); DE (1)