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8月24日最近更新: 2024年7月15日

适应症

试验速览

暂无简介。

•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

•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.

发起方

Dyne Therapeutics, Inc