NL-OMON48814
Completed
Phase 3
A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy. - RO7239361
Overview
- Phase
- Phase 3
- Intervention
- Not specified
- Conditions
- Duchenne Muscular Dystrophy
- Sponsor
- Hoffmann-La Roche
- Enrollment
- 10
- Status
- Completed
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
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.
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 \< 50% of predicted value (in participants able to produce a valid FVC, as judged by the clinical evaluator or respiratory therapist)
- •\* Cutaneous AEs sustained during participation in a prior clinical trial that resolved less than 12 weeks prior to the start of study drug administration.
- •\* Current or prior treatment within 12 weeks prior to the start of study drug administration with androgens or human growth hormone.
Outcomes
Primary Outcomes
Not specified
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