ClinicalTrialsFinder
RecruitingPhase 1Phase 2NCT06280209

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

Sponsor

BioMarin Pharmaceutical

Enrollment

18 participants

Start Date

Jan 3, 2024

Study Type

INTERVENTIONAL

Conditions

Duchenne Muscular Dystrophy

Summary

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.

Eligibility

Sex: MALEMin Age: 4 YearsMax Age: 10 Years

Inclusion Criteria5

  • 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 Criteria3

  • 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

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGBMN 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

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT06280209

Related Trials

Vasodilator and Exercise Study for DMD (VASO-REx)

NCT062907131 location

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

NCT0728718921 locations

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

NCT071606342 locations

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

NCT0613863915 locations

The Duchenne Registry

NCT020697561 location