Education and Training
Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
The purpose of this study is to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.
Stanford is currently accepting patients for this trial.
Intervention(s):
- drug: RO7239361
- drug: Placebo
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
Ages Eligible for Study
5 Years - 10 Years
Genders Eligible for Study
Male
Now accepting new patients
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Recruiting