Gene delivery for treating Duchenne Muscular Dystrophy in young children
A Two-Part, Open-Label Systemic Gene Delivery Study to Evaluate the Safety and Expression of RO7494222 (SRP-9001) in Subjects Under the Age of Four With Duchenne Muscular Dystrophy
This study is testing a new gene therapy in young children with Duchenne Muscular Dystrophy to see if it can help their muscles work better.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 21 (estimated) |
| Ages | N/A to 3 Years |
| Sex | Male |
| Sponsor | Hoffmann-La Roche Industry-sponsored |
| Locations | 7 sites (Liege and 6 other locations) |
| Trial ID | NCT06128564 on ClinicalTrials.gov |
What this trial studies
This open-label, single-arm study aims to evaluate the safety and expression of delandistrogene moxeparvovec in young participants diagnosed with Duchenne Muscular Dystrophy (DMD). The study will include children from birth to under four years of age, who will be monitored for approximately 264 weeks. Participants must have a confirmed diagnosis of DMD and meet specific genetic criteria. The study seeks to assess the effectiveness of this gene therapy in increasing dystrophin expression, which is crucial for muscle function.
Who should consider this trial
Good fit: Ideal candidates are children under four years old with a confirmed diagnosis of DMD and specific genetic mutations.
Not a fit: Patients who have previously received gene therapy or are on corticosteroids for DMD may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve muscle function and quality of life for children with DMD.
How similar studies have performed: Other studies using gene therapy approaches for DMD have shown promising results, indicating potential for success in this novel application.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Cohort A: \>=3 years of age to \<4 years of age * Cohort B: \>=2 years of age to \<3 years of age * Cohort C: \>6 months to \<2 years of age * Cohort D: \<=6 months 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) Exclusion Criteria: * 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 Other inclusion or exclusion criteria could apply
Where this trial is running
Liege and 6 other locations
- Chr de La Citadelle — Liege, Belgium (Recruiting)
- Hôpital Necker-Enfants Malades — Paris, France (Recruiting)
- Universitätsklinikum Essen — Essen, Germany (Recruiting)
- PU A. Gemelli, Università Cattolica del Sacro Cuore — Roma, Lazio, Italy (Recruiting)
- Hospital Sant Joan De Deu — Esplugues De Llobregas, Barcelona, Spain (Recruiting)
- Great Ormond Street Hospital for Children — London, United Kingdom (Recruiting)
- John Radcliffe Hospital — Oxford, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Reference Study ID Number: BN43881 https://forpatients.roche.com/
- Email: global-roche-genentech-trials@gene.com
- Phone: 888-662-6728 (U.S. and Canada)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.