Recruiting
Phase 1
Phase 2

BMN 351

Sponsor:

BioMarin Pharmaceutical

Code:

NCT06280209

Conditions

Duchenne Muscular Dystrophy

Eligibility Criteria

Sex: Male

Age: 4 - 10

Healthy Volunteers: Not accepted

Interventions

BMN 351

Study Details

Brief 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.

Conditions

Duchenne Muscular Dystrophy

Study ID

NCT06280209

Start date

Jan 3, 2024

Status verified date

Apr, 2026

Completion date

Sep 30, 2026

Anticipated

Primary completion date

Sep 30, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 4 - 10

Healthy Volunteers: Not accepted

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 therapy

Study Design

Enrollment

18 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1A

Cohort 1A will consist of both a single ascending dose (SAD) part and a multiple ascending dose (MAD). BMN 351 will be administered once every 2 weeks during the SAD portion of the study for up to 8 weeks and once weekly during the MAD portion for up to 89 weeks.

experimental: Cohort 1B

BMN 351 low dose will be administered once weekly for up to 97 weeks

experimental: Cohort 2

BMN 351 medium dose will be administered once weekly for up to 73 weeks

experimental: Cohort 3

BMN 351 high dose will be administered once weekly for up to 48 weeks

Interventions

BMN 351

Anti-sense Oligonucleotide BMN 351 will be administered intravenously.

Primary outcome measure

  • To evaluate and safety and tolerability of single and multiple doses of BMN 351 (incidence, severity, and dose-relationship of adverse effects and changes in laboratory parameters). [ Time Frame: Up to 97 weeks. ]

Central Contacts and Locations

Locations

Children's Hospital LHSC

Recruiting

London, Ontario, Canada, N6A 5W9

Contacts

Carolyn Twible

519-685-8500carolyn.twible@lhsc.on.ca

Principal Investigator:

Craig Campbell, MD

More Information

Sponsor

BioMarin Pharmaceutical

Last update posted

Apr 8, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by BioMarin Pharmaceutical on 2026-04-08.