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ENROLLING BY INVITATION
NCT07573631
PHASE2

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

Sponsor: BioMarin Pharmaceutical

View on ClinicalTrials.gov

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.

Official title: An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Weekly Intravenous Infusions of BMN 351 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Key Details

Gender

MALE

Age Range

4 Years - Any

Study Type

INTERVENTIONAL

Enrollment

18

Start Date

2026-04-20

Completion Date

2031-12

Last Updated

2026-07-24

Healthy Volunteers

No

Interventions

DRUG

BMN 351

Anti-sense Oligonucleotide BMN 351 will be administered intravenously

Locations (6)

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