An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy

Enrolling by invitation · Phase 2

Conditions studied: Duchenne Muscular Dystrophy (DMD)

In brief

This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the 351-201 study.

Key facts

Study ID

NCT07573631

Run by

BioMarin Pharmaceutical

People needed

18

Starts

2026-04-20

Expected to finish

2031-12-01

Last updated by the study team

2026-07-24

Who can join

Age: 4 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Participants must have completed 351-201 without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study
• Currently receiving treatment with oral corticosteroids, on a stable dose regimen during 351-201, and must remain on a consistent dose regimen throughout 351-202 or 351-203 except for modifications to accommodate changes in weight
• Transition to the equivalent dose of vamorolone is permitted in 351-202 where approved in participating countries.
• Willing and able to adhere to the study visit schedule and other protocol requirements
• Willing to use contraception (sexually mature males) throughout the study and for 90 days after the final dose, if sexually active
• Contraceptive use by males should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
• Willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure
• Willing and able to provide written, signed informed consent as parent or guardian after the nature of the study has been explained and prior to performance of any research-related procedure

You may not qualify if…

• Have known coagulation disorder
• Are taking any prohibited medications
• any approved exon skipping therapy within 12 weeks prior to baseline or with any gene therapy for the treatment of DMD at any time
• anti-coagulants, anti-thrombotics, or anti-platelet agents
• immunosuppressants

Where it is running

• Fondazione Serena ETS - Centro Clinico NeMO Milano — Milan, Italy, Italy
• UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore — Rome, Italy, Italy
• Leids Universitair Medisch Centrum — Leiden, Netherlands, Netherlands
• Hospital Viamed Santa Angela De la Cruz — Seville, Spain, Spain
• Yeditepe University Kosuyolu Hospital — Istanbul, Turkey, Turkey (Türkiye)
• Great Ormond Street Hospital NHS Foundation Trust — London, United Kingdom, United Kingdom

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.