A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular DystrophyMuscular Disorder10028396
- Registration Number
- NL-OMON48814
- Lead Sponsor
- Hoffmann-La Roche
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 10
* 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
* 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.
* Prior treatment with RO7239361 or any other anti-myostatin agent.
* History of lower limb fracture within 12 weeks prior to the start of study drug administration.
* History of upper limb fracture within 8 weeks prior to the start of study drug administration.
* Any injury that may impact functional testing. Previous injuries must be fully healed prior to consenting.
* Expectation of major surgical procedure, such as scoliosis surgery, during the double blind phase of this study.
* Requirement of daytime ventilator assistance
* Initiation of nighttime ventilation less than 4 weeks prior to the start of study drug administration
* Expectation that daytime or nighttime ventilation may be initiated during the double blind phase of this study.
* Clinical signs or symptoms of uncontrolled congestive heart failure (CHF) (American College of Cardiology/American Heart Associated Stage C or Stage D).
* Unwilling or unable to administer study drug at home.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p> The change from baseline in the North Star Ambulatory Assessment (NSAA) total<br /><br>score at Week 48 in RO723936-treated participants<br /><br>compared to placebo-treated participants. </p><br>
- Secondary Outcome Measures
Name Time Method <p>* Change from baseline at Week 48 in RO7239361 treated participants compared<br /><br>to placebo treated participants in the following:<br /><br><br /><br>- 4 stair climb velocity (4SCV)<br /><br>- Stand from supine velocity<br /><br>- 10 M walk/run velocity<br /><br>- PODCI transfers and basic mobility subscale<br /><br>- Proximal lower extremity flexor (knee extension and knee flexion)<br /><br>strength, measured using manual myometry<br /><br>- 6-Minute Walk Distance (6MWD)<br /><br>- Clinical Global Impression of Change (CGI-C)<br /><br>- Stride velocity recorded with ActiMyo<br /><br><br /><br>* Tabulations of the numbers of unique participants with new or worsening<br /><br>laboratory abnormalities, SAEs and AEs leading to discontinuation,<br /><br>in RO7239361 arms compared to the placebo arm.</p><br>