EUCTR2021-005478-24-BE进行中（未招募）1 期
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Dyne Therapeutics, Inc0 个研究点目标入组 112 人开始时间: 2022年9月29日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 112
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- Male
入选标准
- •Age 4 to 16 years inclusive, at the time of informed consent/assent.
- •Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping.
- •Upper extremity muscle group that is amenable to muscle biopsy.
- •Brooke Upper Extremity Scale score of 1 or 2.
- •Ambulatory or non-ambulatory. A non-ambulatory participant must have been non-ambulatory for <2 years before enrolment.
- •Receiving a stable dosage of glucocorticoids for at least 12 weeks prior to the start of study drug administration.
- •Left ventricular ejection fraction of =50% by echocardiogram or =55% by cardiac magnetic resonance imaging (MRI).
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 112
- •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
- •Uncontrolled clinical symptoms and signs of congestive heart failure (CHF).
- •Any change in prophylaxis/treatment for CHF within 3 months prior to the start of study treatment.
- •History of major surgical procedure within 12 weeks prior to the start of study drug administration or an expectation of a major surgical procedure during the study.
- •Requirement of daytime ventilator assistance.
- •Percent predicted FVC <40 % (applies only for participants who are age =7 years).
- •Receipt of eteplirsen, or alternative exon-skipping/dystrophin-modifying therapy, within 12 weeks of randomization.
- •Receipt of non-exon skipping investigational drug within 4 months before the start of study drug administration.
- •Receipt of gene therapy at any time.
研究者
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