NCT03508947已完成1 期
A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 36
- 试验地点
- 13
- 主要终点
- Safety: Severity of AEs
研究概览
简要总结
This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with DMD amenable to exon 51 skipping intervention.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 5 Years 至 18 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Duchenne muscular dystrophy (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 or non-ambulatory male patients aged ≥5 - ≤18 years
- •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 within one year prior to enrollment into the study.
排除标准
- •Severe cardiomyopathy; cardiomyopathy that is managed by angiotensin-converting enzyme (ACE) inhibitors or beta blockers is acceptable provided the patient meets the LVEF inclusion criteria.
- •Need for mechanical or non-invasive ventilation OR anticipated need for mechanical or non-invasive ventilation within the next year, in the opinion of the Investigator.
- •Changes in nutritional or herbal supplements or concomitant medications within 1 month prior to Screening visit or plans to modify dose or regimen during the study.
- •Currently on anticoagulants or antithrombotics.
- •Received treatment with eteplirsen or ataluren within the past 14 weeks.
- •Received prior treatment with drisapersen.
- •Received any investigational drug within the past 3 months or 5 half-lives, whichever is longer.
研究组 & 干预措施
WVE-210201 (Dose B) or placebo
Experimental
干预措施: Placebo (Drug)
WVE-210201 (Dose C) or placebo
Experimental
干预措施: WVE-210201 (Drug)
WVE-210201 (Dose C) or placebo
Experimental
干预措施: Placebo (Drug)
WVE-210201 (Dose D) or placebo
Experimental
干预措施: WVE-210201 (Drug)
WVE-210201 (Dose D) or placebo
Experimental
干预措施: Placebo (Drug)
WVE-210201 (Dose E) or placebo
Experimental
干预措施: WVE-210201 (Drug)
WVE-210201 (Dose E) or placebo
Experimental
干预措施: Placebo (Drug)
WVE-210201 (Dose A) or placebo
Experimental
干预措施: WVE-210201 (Drug)
WVE-210201 (Dose A) or placebo
Experimental
干预措施: Placebo (Drug)
WVE-210201 (Dose B) or placebo
Experimental
干预措施: WVE-210201 (Drug)
结局指标
主要结局
Safety: Severity of AEs
时间窗: Day 1 to Day 85 (end of study)
Safety: Number of patients with serious AEs (SAEs)
时间窗: Day 1 to Day 85 (end of study)
Safety: Number of patients with adverse events (AEs)
时间窗: Day 1 to Day 85 (end of study)
Safety and Tolerability: Number of patients who withdraw due to AEs
时间窗: Day 1 to Day 85 (end of study)
次要结局
- PK: Area under the plasma concentration-time curve (AUC 0-t)(Day 1, Day 2, and Day 8)
- Pharmacokinetics (PK): Maximum observed concentration (Cmax)(Day 1, Day 2, and Day 8)
- PK: Time of occurrence of Cmax (tmax)(Day 1, Day 2, and Day 8)
研究者
研究点 (13)
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