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Clinical Trials in the UK / NCT07573631
Enrolling by invitation Phase 2

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

NCT07573631 · tracked via the Priya Life Science UK tracker
Sponsor
BioMarin Pharmaceutical
Phase
Phase 2
Started
2026-04-20
Last updated
2026-07-24

Condition(s) studied

Duchenne Muscular Dystrophy (DMD)

Investigational drug(s) / intervention(s)

BMN 351

BMN 351: Anti-sense Oligonucleotide BMN 351 will be administered intravenously

Study summary

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.

Eligibility

Sex
MALE
Min age
4 Years
Max age
Healthy volunteers
No
Inclusion Criteria: * 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 Exclusion Criteria: * 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

Primary outcome measure(s)

Trial sites (6)

FacilityCityRegionStatus
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 Viamed Santa Angela De la Cruz Seville Spain
Yeditepe University Kosuyolu Hospital Istanbul Turkey
Great Ormond Street Hospital NHS Foundation Trust London United Kingdom

More BioMarin Pharmaceutical trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07573631 on ClinicalTrials.gov ↗ ← All trials in the UK