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

• Status: Terminated
• Trial end date: 2020-04-15
• Target enrollment: 0
• Participant gender: Male

Summary

The purpose of this study is to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hoffmann-La Roche

Criteria

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