Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
Trial ID or NCT#
The purpose of this study is to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.
A Multi-Site, Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Subcutaneous Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of RO7239361 (BMS-986089) in Ambulatory Boys With Duchenne Muscular Dystrophy
- - Diagnosed with DMD - Able to walk without assistance - Able to walk up 4 stairs in 8 seconds or less - Weigh at least 15 kg - Taking corticosteroids for DMD
- - Ejection fraction < 55% on echocardiogram, based on central read - Any behavior or mental issue that will affect the ability to complete the required study procedures - Previously or currently taking medications like androgens or human growth hormone - Use of a ventilator during the day - Unable to have blood samples collected or receive an injection under the skin - Treatment with exon skipping therapies 6 months prior to study start - Treatment with ataluren or any investigational drug currently or within 5 half-lives prior to study start