NCT06280209Active, not recruitingPhase 1/2
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants 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
Source: ClinicalTrials.gov · Last updated 25 Jun 2026
Brief summary (registry, in English)
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 18 (actual)
- First submitted
- 26 Jan 2024
- Start date
- 3 Jan 2024
- Primary completion date
- 14 Apr 2027
- Lead sponsor
- BioMarin Pharmaceutical
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT06280209
- EU CT number
- 2023-506737-30-00
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 18
- From 3 Jan 2024 to the primary completion date, 14 Apr 2027
- 7 sites in 5 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Treatment with an exon skipping therapy within 12 weeks prior to the first visit.
- Any history of treatment with gene therapy
Other illness
- The participant will have some initial clinical labs and studies to assess baseline level of heart and lung function.
Who the study is looking for
- 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
Age in the registry: 4 years to 10 years · Sex: Male · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Sant Joan De Deu Barcelona Hospital Coordinator of this site? Confirm its status | Esplugues De Llobregat | Spain | Completed |
| Hospital Viamed Santa Ángela de la Cruz Coordinator of this site? Confirm its status | Sevilla | Spain | Completed |
| Great Ormond Street Hospital NHS Foundation Trust Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Centro Clinico Nemo Coordinator of this site? Confirm its status | Milan | Italy | Completed |
| Fondazione Policlinico Universitario Agostino Gemelli IRCCS Coordinator of this site? Confirm its status | Rome | Italy | Completed |
| Leids Universitair Medisch Centrum (LUMC) Coordinator of this site? Confirm its status | Leiden | Netherlands | Completed |
| Yeditepe University Kosuyolu Hospital Coordinator of this site? Confirm its status | Istanbul | Turkey (Türkiye) | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.