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

Phase 1

Recruiting

• Conditions: Duchenne Muscular Dystrophy; MedDRA version: 20.0Level: PTClassification code: 10013801Term: Duchenne muscular dystrophy Class: 100000004850; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: CTIS2023-506737-30-00
• Lead Sponsor: Biomarin Pharmaceutical Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-08-04
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Male
• Target Recruitment: 18

Inclusion Criteria

1. Is male and age 4 through 10 years at Screening., 2. Clinical diagnosis of Duchenne muscular dystrophy in the opinion of the investigator resulting from a documented dystrophin mutation in the DMD gene amenable to exon 51 skipping as reviewed by a central genetic counselor., 3. Ambulatory at Screening, defined as able to walk independently without assistive devices and complete the timed 10 meter walk/run test in 8 seconds or less., 4. Not currently daytime ventilator dependent and not expected to need daytime mechanical or noninvasive ventilation within the next year in the opinion of the investigator., 5. Currently receiving treatment with oral corticosteroids, on a stable dose for at least 12 weeks prior to Baseline, and must remain on a consistent dose/dose regimen throughout the study except for modifications to accommodate changes in weight., 6. Normal urinalysis at Screening (trace protein allowed).

Exclusion Criteria

1. For children 7 years of age or older, forced expiratory volume (FEV1) < 60% of predicted., 2. Current or history of liver or renal disease., 3. Left ventricular ejection fraction (LVEF) < 55% based on an echocardiogram (ECHO) performed within 3 months prior to the Baseline (Day 1) visit., 4. Mean QT interval corrected with Fridericia’s method (QTcF) = 450 msec on the Screening electrocardiogram (ECG) conducted in triplicate., 5. Platelet count of < 150 x 10^9/L at Screening., 6. Renal function laboratory parameters outside of prespecified values as defined per protocol., 7. Treatment with any exon skipping therapy within 12 weeks prior to Baseline (Day 1) or with any gene therapy for the treatment of DMD at any time.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified