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A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants with Duchenne Muscular Dystrophy

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Trial Status Recruiting

Trial Identifier

NCT06280209

Condition

Duchenne Muscular Dystrophy

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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 Criteria

Birth Sex

Male

Age

4 Years to 10 Years

Healthy Volunteers

No

Drug/Treatment:

BMN 351

Phase:

Phase 1/Phase 2

Study Type:

Interventional

Number of Participants:

18

Study Started:

2024-01-03

Study Updated:

2024-12-05

Trial Locations

  • Fondazione Serena Onlus - 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

    Sevilla, , Spain

  • Yeditepe University Kosuyolu Hospital

    Istanbul, , Turkey

  • Great Ormond Street Hospital NHS Foundation Trust

    London, , United Kingdom

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 therap

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