NL-OMON48814已完成3 期
A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy. - RO7239361
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 17（—）
入选标准
- •* 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
排除标准
- •* 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.
研究者
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