Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD

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Trial ID: NCT02515669,25513,26554

Age - 5 Years-10 Years Condition - Pediatric Neurology, Pediatric Medical Genetics Gender - Male Accepting Healthy Volunteers - No

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

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Intervention(s):

drug: RO7239361

drug: Placebo

Recruiting

I'm Interested

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305

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