2023-509935-23-00已完成2 期
A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 8
- 主要终点
- Part A: Incidence of Adverse Events (AEs), Baseline up to 75 weeks
研究概览
简要总结
Part A: To evaluate the safety and tolerability of multiple ascending doses of SRP-5051, administered intravenously (IV) every 4 weeks, and determine the maximum tolerated dose (MTD) Part B: To evaluate dystrophin protein level in skeletal muscle tissue following SRP-5051 treatment, administered IV every 4 weeks at the doses selected based on data from Part A
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Part B Screening; Safety Lead-in Period; Treatment and Observation Period; Study Safety FU Period
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 64 years（0-17 Years, 18-64 Years）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •Inclusion Criteria for participants previously treated with Vesleteplirsen: - Has received prior Vesleteplirsen treatment in Part A of this study or in Study 5051-
- •Inclusion Criteria for treatment-naïve participants enrolling into Part B: - Has a genetic diagnosis of Duchenne muscular dystrophy (DMD) and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment. - Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight), or has not received corticosteroids for at least 12 weeks prior to study drug administration. - Has stable pulmonary function (forced vital capacity [FVC] ≥40% of predicted and no requirement for nocturnal ventilation).
排除标准
- •Exclusion Criteria for participants previously treated with Vesleteplirsen and new participants enrolling into Part B: - Presence of other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or any other condition that, in the Investigator's opinion, could interfere with participation in the trial.
- •Exclusion Criteria for treatment-naive participants enrolling into Part B: - History of hypomagnesemia within 12 weeks prior to Screening. - Initiation or change of dosing (except for modifications to accommodate changes in weight or changes in standard of care) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, β-blockers, or potassium. - Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations. - Has a left ventricular ejection fraction (LVEF) <40.0% based on an echocardiogram (ECHO) performed within 12 weeks prior to Screening or at the Screening Visit. - Treatment with any exon 51-skipping therapy within 4 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time. - Other inclusion/exclusion criteria apply.
结局指标
主要结局
Part A: Incidence of Adverse Events (AEs), Baseline up to 75 weeks
Part A: Incidence of Adverse Events (AEs), Baseline up to 75 weeks
Part B: Change From Baseline in Dystrophin Protein Level at Week 28
Part B: Change From Baseline in Dystrophin Protein Level at Week 28
次要结局
- Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen, Pre-dose and at multiple time points (up to 32 hours) after end of infusion
- Part A: PK: Urine Concentration of Vesleteplirsen, Pre-dose and at multiple time periods (up to 48 hours) after end of infusion
- Part B: Change From Baseline in Exon-Skipping Levels at Week 28
- Part B: Incidence of Adverse Events (AEs), Baseline up to Week 304
- Part B: PK: Plasma Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusion
- Part B: PK: Urine Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusion
- Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay at Week 28
研究者
Patient Recruitment
Scientific
Sarepta Therapeutics Inc.
研究点 (8)
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