NCT03907072终止2 期
A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 6
- 试验地点
- 22
- 主要终点
- Change From Baseline in Dystrophin Level (% Normal Dystrophin)
研究概览
简要总结
This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension period to evaluate the safety and efficacy of WVE-210201 (suvodirsen) in ambulatory male pediatric patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping intervention (DYSTANCE 51)
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 5 Years 至 12 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of DMD based on clinical phenotype with increased serum creatine kinase
- •Documented mutation in the Dystrophin gene associated with DMD that is amenable to exon 51 skipping
- •Ambulatory male, able to walk independently for at least 10 meters in 10 seconds or less at the time of Screening visit (performed as part of the NSAA)
- •Stable pulmonary and cardiac function, as measured by:
- •Reproducible percent predicted forced vital capacity (FVC) ≥50%
- •Left ventricular ejection fraction (LVEF) >55% in patients <10 years of age and >45% in patients ≥10 years of age, as measured (and documented) by echocardiogram
- •Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy occurred ≥6 months prior to Screening, and no changes in dosing ≤3 months prior to Screening visit
排除标准
- •Cardiac insufficiency:
- •Severe cardiomyopathy that, in the opinion of the Investigator, prohibits participation in this study; however, cardiomyopathy that is managed by angiotensin-converting-enzyme (ACE) inhibitors or beta blockers is acceptable provided the patient meets the LVEF inclusion criterion
- •Any other evidence of clinically significant structural or functional heart abnormality
- •A cardiac troponin I value > 0.2 ng/mL
- •Need for daytime mechanical or non-invasive ventilation OR anticipated need for daytime mechanical or non-invasive ventilation within the next year, in the opinion of the Investigator. Nighttime non-invasive ventilation is permitted
- •Received prior treatment with drisapersen or with an investigational peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
- •Received prior treatment with gene therapy for DMD
- •Received treatment with ataluren or eteplirsen within the 14 weeks prior to the planned Baseline biopsy collection
- •Received any investigational drug within 3 months or 5 half-lives, whichever is longer, prior to the planned Baseline biopsy collection
研究组 & 干预措施
WVE-210201 (3 mg/kg)
Experimental
Weekly IV administrations of WVE-210210 at 3 mg/kg
干预措施: WVE-210201 (suvodirsen) (Drug)
WVE-210201 (4.5 mg/kg)
Experimental
Weekly IV administrations of WVE-210210 at 4.5 mg/kg
干预措施: WVE-210201 (suvodirsen) (Drug)
Placebo
Placebo Comparator
Weekly IV administrations of phosphate buffered saline solution visually identical in appearance to WVE-21021
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline in Dystrophin Level (% Normal Dystrophin)
时间窗: Day 1 to Week 12, Week 22, or Week 46
US/other regions (as applicable)
Change From Baseline in North Star Ambulatory Assessment (NSAA)
时间窗: Day 1 through Week 48
European Union (EU)/other regions (as applicable)
次要结局
- Change From Baseline in 4-stair Climb(Day 1 through Week 48)
- Change From Baseline in Forced Vital Capacity(Day 1 through Week 48)
- Change From Baseline in North Star Ambulatory Assessment (NSAA)(Day 1 through Week 48)
- Change From Baseline in the 10-meter Walk/Run Test(Day 1 through Week 48)
- Change From Baseline in Dystrophin Level (% Normal Dystrophin)(Day 1 to Week 12, Week 22, or Week 46)
- Change From Baseline in Upper Limb Proximal Strength(Day 1 through Week 48)
- Change From Baseline in NSAA(Day 1 through Week 96)
- Change From Baseline in the 95th Percentile of Stride Velocity(Day 1 through Week 48)
研究者
研究点 (22)
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