An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 15
- 试验地点
- 8
- 主要终点
- Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 (vesleteplirsen) in participants with Duchenne muscular dystrophy (DMD) who participated in studies of SRP-5051.
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
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Has completed a study of SRP-5051 and continues to meet the Eligibility Criteria of Study 5051-102.
排除标准
- •Initiation or change of dosing (except for modifications to accommodate changes in weight or changes in standard of care) since completing a study administering SRP-5051 and while participating in this study for any of the following: angiotensin converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents (ARBs), beta-blockers, potassium and steroids*.
- •Requires antiarrhythmic and/or diuretic therapy for heart failure.
- •Use of any herbal medication/supplement containing aristolochic acid.
- •Treatment with any experimental therapy since entering original study or any experimental gene therapy for the treatment of DMD at any time.
- •Participation in an interventional clinical trial since completing original study.
- •Other inclusion/exclusion criteria apply.
- •* The dose of steroids must remain constant except for modifications to accommodate changes in weight.
研究组 & 干预措施
SRP-5051
Participants will receive SRP-5051 via intravenous (IV) infusion. Dosage and frequency will be determined from the safety profile of other ongoing SRP-5051 studies.
干预措施: SRP-5051 (Drug)
结局指标
主要结局
Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)
时间窗: Up to approximately 135 weeks
A TEAE was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module.
次要结局
- Plasma Concentration of SRP-5051(Day 1, Day 84, every 84 days after Day 84 (up to a maximum of approximately 135 weeks) (pre-dose, immediately prior to end of infusion, up to 4-6 hours post-dose))