NCT06747273终止1 期
A Phase 1b, Multicenter, Single Dose Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Subjects in the United States
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESI) and Treatment-emergent Serious Adverse Events (SAEs)
研究概览
简要总结
The primary objective of this study is to evaluate the safety of SRP-9004.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ambulatory participants, defined as able to walk without assistive aid, 10MWR <30 seconds, and NSAD total score ≥25; non-ambulatory participant, defined as 10MWR ≥30 seconds or unable to perform, and PUL 2.0 entry scale score ≥
- •Ambulatory participants must be 4 to 20 years of age and the non-ambulatory participant must be ≥4 years of age.
- •All participants must be ≤70 kilograms
- •Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic α-SG deoxyribonucleic acid (DNA) gene mutations as documented prior to screening.
- •Able to cooperate with muscle testing.
- •Participants must have adeno-associated virus (AAV) serotype Rh74 (rh74) antibody titers <1:400 (that is, not elevated) as determined by an enzyme-linked immunosorbent assay (ELISA).
排除标准
- •Left ventricular ejection fraction <40% or clinical signs and/or symptoms of cardiomyopathy
- •FVC ≤40% of predicted value and/or requirement for nocturnal ventilation
- •Any other clinically significant illness, including neuromuscular (other than limb girdle muscular dystrophy type 2D/R3 [LGMD2D/R3]), that in the opinion of the Investigator might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safety, or clinical interpretability.
- •Other inclusion/exclusion criteria apply.
研究组 & 干预措施
SRP-9004
Experimental
Participants will receive a single dose of SRP-9004 on Day 1.
干预措施: SRP-9004 (Drug)
结局指标
主要结局
Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESI) and Treatment-emergent Serious Adverse Events (SAEs)
时间窗: Up to 60 months
次要结局
- Change from Baseline Through Day 60 in Quantity of Alpha-sarcoglycan (α-SG) Protein Expression(Baseline, Day 60)
- Change from Baseline Through Month 24 in Quantity of α-SG Protein Expression(Baseline, Month 24)
- Change from Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score(Baseline, Month 60)
- Change from Baseline Through Month 60 in Performance of the Upper Limb (PUL) Version 2.0 Total Score(Baseline, Month 60)
- Change from Baseline Through Month 60 in Time to Rise From Floor(Baseline, Month 60)
- Change from Baseline Through Month 60 in Time to Ascend 4 Steps(Baseline, Month 60)
- Change from Baseline Through Month 60 in Time to Complete 10-meter Walk/Run (10MWR)(Baseline, Month 60)
- Change from Baseline Through Month 60 in Time to Complete 100-meter Walk/Run (100MWR)(Baseline, Month 60)
- Change from Baseline through Month 60 in Creatine Kinase (CK) Level(Baseline, Month 60)
- Time to Change of Disease Milestones (Loss of Ambulation)(Baseline, Month 60)
- Change from Baseline Through Month 60 in Skeletal Muscle Magnetic Resonance Imagining (MRI) Findings(Baseline, Month 60)
- Change from Baseline Through Month 60 in Wearable Device Stride Velocity 95% (SV95C) (Ambulatory Participants Only)(Baseline, Month 60)
- Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Vital Capacity (FVC)(Baseline, Month 60)
- Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by FVC Percentage (%) Predicted(Baseline, Month 60)
- Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Expiratory Volume in 1 Second (FEV1)(Baseline, Month 60)
- Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by FEV1 % Predicted(Baseline, Month 60)
- Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Peak Expiratory Flow Rate (PEFR)(Baseline, Month 60)
- Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Maximal Inspiratory Pressure (MIP)(Baseline, Month 60)
- Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Maximal Expiratory Pressure (MEP)(Baseline, Month 60)
研究者
研究点 (2)
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