©2025 Stanford Medicine
Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
Not Recruiting
Trial ID: NCT02515669,25513,26554
Purpose
The purpose of this study is to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.
Official Title
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
Eligibility
Inclusion Criteria:
* 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
Exclusion Criteria:
* 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
Intervention(s):
drug: RO7239361
drug: Placebo
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305