Duchenne Muscular Dystrophy
Conditions
Interventions
Each participant will be administered weekly, SC doses of RO7239361 or placebo
for 48 weeks in a randomized, double blind design.
1/3 - RO7239361 dose 1 (7,5 mg of 15 mg based on weight) SC weekly
ambulatory boys 6 tot < 12 years
anti-myostatin agent
corticosteroids
Duchenne Muscular Dystrophy
Sponsors
Hoffmann-La Roche
Eligibility
Age
2 Years to 17 Years
Inclusion criteria
Inclusion criteria: * Males, * 6 to 0.2 mg/kg prednisone or > 0.24 mg/kg deflazacort) or dosing regimen for at least 12 weeks prior to the start of study drug, with the expectation that dosage and dosing regimen will not change significantly for the duration of the study. * North Star Ambulatory Assessment (NSAA) score >
Exclusion criteria
Exclusion criteria: * Participants with cognitive impairment or behavioral issues that, in the judgement of the investigator, will compromise their ability to comply with study procedures. * Participants on intermittent CS regimens with off periods of 20 days or longer (eg.: 10 days on, 20 days off). * Any change (initiation, change in drug class, dose modification unrelated to change in body weight, interruption or re-initiation) in prophylaxis/treatment for congestive heart failure (CHF) within 12 weeks prior to start of study treatment. * Any change (initiation, change in drug class, dose modification unrelated to change in body weight, interruption or re-initiation) in prophylaxis/treatment for bone density within 12 weeks prior to start of study treatment. * Treatment with exon skipping therapies within 6 months prior to the start of study drug administration. * Treatment with ataluren currently or within 12 weeks prior to the start of study drug administration. * Concurrent or previous participation at any time in a gene therapy study. * Participants with a FVC of
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The change from baseline in the North Star Ambulatory Assessment (NSAA) total score at Week 48 in RO723936-treated participants compared to placebo-treated participants. | — |
Secondary
| Measure | Time frame |
|---|---|
| * Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in the following: - 4 stair climb velocity (4SCV) - Stand from supine velocity - 10 M walk/run velocity - PODCI transfers and basic mobility subscale - Proximal lower extremity flexor (knee extension and knee flexion) strength, measured using manual myometry - 6-Minute Walk Distance (6MWD) - Clinical Global Impression of Change (CGI-C) - Stride velocity recorded with ActiMyo * Tabulations of the numbers of unique participants with new or worsening laboratory abnormalities, SAEs and AEs leading to discontinuation, in RO7239361 arms compared to the placebo arm. | — |
Countries
Argentina, Australia, Belgium, Canada, France, Germany, Italy, Japan, Netherlands, Spain, Sweden, United Kingdom, United States of America
Outcome results
None listed