Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2020-04-28
• Target enrollment: 0
• Participant gender: Male

Summary

This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hoffmann-La Roche

Criteria

Inclusion Criteria:

• Diagnosed with DMD by confirmed medical history and genetic testing

• Able to walk without assistance

• Minimum North Star Ambulatory Assessment score of 15 at screening

• Able to walk up 4 stairs in 8 seconds or less

• Weigh at least 15 kg (33 lbs)

• Taking corticosteroids for DMD

Exclusion Criteria:

• 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

• Concomitant or previous participation at any time in a gene therapy study

Other protocol defined Inclusion/Exclusion Criteria could apply.