RECRUITINGPhase 1 / Phase 2INTERVENTIONAL
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
About This Trial
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.
Who May Be Eligible (Plain English)
Who May Qualify:
- 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
Who Should NOT Join This Trial:
- 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
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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
Treatments Being Tested
DRUG
BMN 351
Anti-sense Oligonucleotide BMN 351 will be administered intravenously.
Locations (8)
Children's Hospital LHSC
London, Ontario, Canada
Fondazione Serena ETS - Centro Clinico NeMO Milano
Milan, Italy
UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, Italy
Leids Universitair Medisch Centrum
Leiden, Netherlands
Hospital Sant Joan de Deu
Barcelona, Spain
Hospital Viamed Santa Angela De la Cruz
Seville, Spain
Yeditepe University Kosuyolu Hospital
Istanbul, Turkey (Türkiye)
Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom