EUCTR2016-001654-18-ES进行中（未招募）1 期
A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 160
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •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 (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 3 months 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.
- •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.
- •Males 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 [BMS-986089; 50 days] plus 90 days (duration of sperm turnover) for a total of 140 days (5 months) post-treatment completion.
- •For the rest of Inclusion Criteria, please refer to study protocol Section 6.1 (page 50).
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 160
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •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 corticosteroid 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 3 months 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 3 months 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, or any other investigational drug (excluding deflazacort and exon skipping therapies) currently or within 3 months prior to the start of study drug administration.
- •Participants with a FVC of < 50% (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 3 months prior to the start of study drug administration.
- •Current or prior treatment within 3 months prior to the start of study drug administration with androgens or human growth hormone.
- •Prior treatment with BMS-986089 or any other anti-myostatin agent.
- •History of lower limb fracture within 3 months prior to the start of study drug administration.
- •History of upper limb fracture within 2 months prior to the start of study drug administration.
- •Any injury which 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 1 month 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.
- •Uncontrolled clinical signs or symptoms of congestive heart failure (American College of Cardiology/American Heart Associated Stage C or Stage D).
- •For participants participating in cMRI substudy: implanted ferromagnetic metal (implanted metal that is not ferromagnetic, such as surgical steel or titanium implants may be allowed if the implants will not compromise the quality of the cMRI).
- •History of hypersensitivity to components of the study drug (histidine, trehalose, diethylenetriaminepentaacetic acid, polysorbate 80).
- •Unwilling or unable to administer study drug at home.
- •For the rest of Exclusion Criteria, please refer to study protocol Section 6.2 (page 51).
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