An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 24
- 试验地点
- 9
- 主要终点
- Number of Participants With Treatment Emergent Adverse Events
研究概览
简要总结
The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
详细描述
This is an open-label, multi-center study to explore the safety and tolerability of eteplirsen injection in participants with advanced stage DMD with confirmed genetic mutations amenable to treatment by exon 51 skipping.
Participants will be evaluated for inclusion during a Screening/Baseline period of up to 4 weeks. Eligible participants will receive once weekly intravenous (IV) infusions of 30 mg/kg eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 7 Years 至 21 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male 7 - 21 years of age
- •Diagnosis of DMD with a mutation that is amenable to exon 51 skipping, confirmed by a genetic report
- •Stable dose of oral corticosteroids for at least 24 weeks or has not received corticosteroids for at least 24 weeks
- •Non-ambulatory, or incapable of walking ≥300 meters on the 6-Minute Walk Test (6MWT).
- •Score of ≤4 on the Brooke Score for Arms and Shoulders.
- •Stable cardiac and pulmonary function
- •Use of contraceptives for sexually active males throughout the study
- •Willing to provide consent and comply with the study
排除标准
- •Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids).
- •Previous treatment with SMT C1100/BMN 195 at any time.
- •Previous treatment with drisapersen (PRO051) within the last 6 months.
- •Participation in any other DMD interventional clinical study within 12 weeks
- •Major change in physiotherapy regimen within the past 3 months
- •Major surgery within 3 months
- •Presence of other clinically significant illness
- •Use of an aminoglycoside antibiotic within 12 weeks or the need for this antibiotic or statin during study
- •Forced vital capacity % predicted [FVC % predicted] <40%, or requiring daytime ventilation.
- •Require antiarrhythmic and/or antidiuretic therapy for heart failure.
- •Have a left ventricular ejection fraction (LVEF) of <40%.
- •Prior or ongoing medical condition that could adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results.
研究组 & 干预措施
Eteplirsen 30 mg/kg
Participants will receive eteplirsen 30 mg/kg/week intravenous (IV) infusions, weekly, for up to 96 weeks.
干预措施: Eteplirsen (Drug)
结局指标
主要结局
Number of Participants With Treatment Emergent Adverse Events
时间窗: From first dose of drug up to 100 weeks
An adverse event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. A serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; Required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug (up to 100 weeks) that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious adverse events.
次要结局
- Number of Participants With Potentially Clinically Significant Laboratory Abnormalities(Baseline up to 100 weeks)
- Number of Participants With at Least One Potentially Clinically Significant Abnormalities in Physical Examinations(Baseline up to 100 weeks)
- Number of Participants With Abnormalities in Echocardiograms (ECHO)(Baseline up to 100 weeks)
- Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs(Baseline up to 100 weeks)
- Number of Participants With Abnormalities in Electrocardiograms (ECGs)(Baseline up to 100 weeks)