Clinical trial

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

Name

351-201

Description

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.

Trial arms

Trial start

2024-01-03

Estimated PCD

2025-12-01

Trial end

2025-12-01

Status

Recruiting

Phase

Early phase I

Treatment

BMN 351

Anti-sense Oligonucleotide BMN 351 will be administered intravenously.

Arms:

Cohort 1A, Cohort 1B, Cohort 2, Cohort 3

Size

Primary endpoint

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).

Up to 73 weeks.

Eligibility criteria

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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 18, 'type': 'ESTIMATED'}}

Updated at

2024-04-16

1 organization

1 product

1 indication

Organization

BioMarin Pharmaceutical

Product

BMN 351

Indication

Duchenne muscular dystrophy