Phase 1/2 Dose Escalation Study to assess BMN 351 in patients with DMD
Research type
Research Study
Full title
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
IRAS ID
1008332
Contact name
Parvin Perrino
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Research summary
Patients with Duchenne Muscular Dystrophy (DMD) have a mutation in the gene that makes a protein called dystrophin. With this mutation, the muscles of patients with DMD are unable to make dystrophin and the muscles become weak. BioMarin is developing a drug called BMN 351 that could enable muscles to produce a shorter and potentially functional form of dystrophin. The study hypothesis is that selected dose(s) of BMN 351 is safe and tolerable and participants receiving BMN 351 will demonstrate increase in dystrophin in muscles when compared to their own previous level. This study is the first time BMN 351 has been studied in people (Phase I, First in Human). Given the progressive nature of DMD, this is an open-label study, and all participants will receive BMN 351. This study will investigate BMN 351 at 6 escalating doses: the dose level or levels participant initially receives will depend on when they enroll in the study. An independent expert safety panel will review the study data at each dose level to recommend whether it is safe to move to a higher dose or not. The main objective is to assess the safety and tolerability of BMN 351 at different dose levels in participants with DMD. The secondary objective is to evaluate the plasma and urine pharmacokinetics (how study drug is absorbed, broken down and eliminated from the body) and muscle distribution of study drug BMN 351. Participants are eligible if they are male and age 4 through 10 years at Screening, have confirmed DMD genetic mutation, can walk independently and complete walk/run test in a required timeframe. The study is planned to open at one site within the United Kingdom, Great Ormond Street Hospital for Children. Up to 18 participants will be enrolled in the study globally.
REC name
London - Central Research Ethics Committee
REC reference
23/LO/0717
Date of REC Opinion
7 Nov 2023
REC opinion
Further Information Favourable Opinion