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 sites166 target enrollmentJuly 12, 2017

Overview

No summary available.

Registry

who.int

Start Date

July 12, 2017

End Date

April 28, 2020

Last Updated

5 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

Male

Sponsor

•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) 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
•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

•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 investigational drug (excluding deflazacort
•in CS dose\-finding trials) currently or within 12 weeks prior to the start