An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Sarepta Therapeutics, Inc0 个研究点目标入组 12 人开始时间: 2017年7月17日最近更新: 2021年4月19日

研究概览

暂无简介。

•1. Be a male between 6 months to 48 months of age, inclusive.
•2. Have an established clinical diagnosis of DMD with a deletion mutation amenable to exon 51 skipping (e.g., deletions of exons 45-50, 47-50, 48-50, 49-50, 50, 52, 52-63).
•3. Have a parent(s) or legal guardian(s) who is able to understand and comply with the study requirements and is willing to provide written informed consent for the patient to participate in the study.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 12
•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

•1. Has received any pharmacologic treatment that might have an effect on muscle strength or function within 12 weeks prior to dosing at Week 1 (e.g., growth hormone, anabolic steroids).
•2. Has received previous or current treatment with any experimental treatment. Prior drisapersen therapy is permitted if a patient has not received drisapersen for 6 months prior to the Week 1 dose.
•3. Has a clinically significant illness other than DMD, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, behavioural disease, or malignancy likely to impair the patient’s ability to participate in this study.
•4. Has a clinically significant laboratory abnormality that is either not expected or is of a greater severity than what is expected in DMD patients.
•5. Has any other condition that, in the Investigator’s opinion, could interfere with the patient’s participation in the study.

发起方

Sarepta Therapeutics, Inc