Testing BMN 351 for safety in children with Duchenne Muscular Dystrophy
A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants With Duchenne Muscular Dystrophy
This study is testing a new treatment called BMN 351 to see if it is safe for children aged 4 to 10 with Duchenne Muscular Dystrophy who have a specific genetic mutation.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 18 (estimated) |
| Ages | 4 Years to 10 Years |
| Sex | Male |
| Sponsor | BioMarin Pharmaceutical Industry-sponsored |
| Locations | 8 sites (London, Ontario and 7 other locations) |
| Trial ID | NCT06280209 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety and tolerability of BMN 351 in children aged 4 to 10 with Duchenne Muscular Dystrophy (DMD) who have a specific genetic mutation. It is a Phase 1/2, open-label, multi-center trial that consists of two parts, with participants assigned to different cohorts receiving escalating doses of the treatment. The first part involves increasing doses every two weeks, while the second part transitions to weekly dosing. The study aims to enroll approximately 18 participants to assess the pharmacokinetics and pharmacodynamics of BMN 351.
Who should consider this trial
Good fit: Ideal candidates are boys aged 4 to 10 diagnosed with Duchenne Muscular Dystrophy and a specific genetic mutation suitable for exon 51 skipping.
Not a fit: Patients who have received exon skipping therapy or gene therapy within the last 12 weeks may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve the management of Duchenne Muscular Dystrophy in affected children.
How similar studies have performed: Other studies have shown promise with similar exon skipping approaches, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age 4 to 10 * Diagnosis of Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping * Able to walk * Not requiring assistance from a ventilator to breathe * Currently on consistent doses of steroid treatment for the last 12 weeks Exclusion Criteria: * The participant will have some initial clinical labs and studies to assess baseline level of heart and lung function. * Treatment with an exon skipping therapy within 12 weeks prior to the first visit. * Any history of treatment with gene therapy
Where this trial is running
London, Ontario and 7 other locations
- Children's Hospital LHSC — London, Ontario, Canada (Recruiting)
- Fondazione Serena ETS - Centro Clinico NeMO Milano — Milan, Italy (Recruiting)
- UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore — Rome, Italy (Recruiting)
- Leids Universitair Medisch Centrum — Leiden, Netherlands (Recruiting)
- Hospital Sant Joan de Deu — Barcelona, Spain (Recruiting)
- Hospital Viamed Santa Angela De la Cruz — Seville, Spain (Recruiting)
- Yeditepe University Kosuyolu Hospital — Istanbul, Turkey (Türkiye) (Recruiting)
- Great Ormond Street Hospital NHS Foundation Trust — London, United Kingdom (Recruiting)
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.