A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy

F. Hoffmann-La Roche Ltd0 sites159 target enrollmentStarted: June 27, 2017Last updated: June 8, 2020

Overview

No summary available.

•Key Inclusion Criteria:
•Males, 6 \>\= to \< 12 years of age at time of randomization.
•Diagnosis of DMD, confirmed by medical history (eg., onset of clinical signs or symptoms before 5 years of age together with an elevated serum creatine kinase level observed before or after initial diagnosis) and by genotyping.
•Participants \>\= 15 kg.
•Ambulatory without assistance.
•Participants must be receiving corticosteroids (CS, prednisone, prednisolone, or deflazacort) for at least 6 months prior to the start of study drug, with no significant change in dosage (\> 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 \>\= 15 points at screening
•4SC\<\= 8 seconds at screening.
•Participants must agree to avoid major changes in their physical or respiratory therapy regimen during the double blind phase, to the extent possible.
•Subjects who are sexually active with WOCBP must agree to follow instructions for method(s) of contraception for the duration of treatment with study treatment(s) plus 5 half\-lives of the study treatment \[RO7239361; 50 days] plus 90 days (duration of sperm turnover) for a total of 140 days (5 months) post\-treatment completion.

•Key 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.
•Treatment with any other investigtional drug (excluding deflzacort in CS dose\-finding trials) 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 \< 50% of predicted value (in participants able to produce a valid FVC, as judged by the clinical evaluator or respiratory therapist).

Sponsor