NCT03652259终止1 期
A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
研究概览
简要总结
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males or females of any ethnic group
- •β-SG deoxyribonucleic acid (DNA) gene mutations at both alleles
- •Weakness demonstrated based on history of difficulty in running, jumping and climbing stairs
- •A 100 meter walk/run (MWR) test result: ≥40 % of predicted for age-, height-, gender-, and weight-matched healthy controls at the screening visit
排除标准
- •Active viral infection based on clinical observations
- •Cardiac magnetic resonance imaging (MRI) determined left ventricular ejection fraction (LVEF) <40%
- •Serological evidence of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection
- •Diagnosis of (or ongoing treatment for) an autoimmune disease
- •Abnormal laboratory values considered clinically significant
- •Concomitant illness or requirement for chronic drug treatment that, in the opinion of the Principal Investigator, creates unnecessary risks for gene transfer.
- •Other inclusion/exclusion criteria apply.
结局指标
主要结局
Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
时间窗: Baseline up to 7 years
次要结局
- Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers(Baseline, Day 60)
- Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence(Baseline, Day 60)
- Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot(Baseline, Day 60)
研究者
研究点 (2)
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