NCT05876780进行中（未招募）1 期
A Multicenter, Open-label, Single-dose, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 on Subjects With Limb Girdle Muscular Dystrophy, Type 2E/R4 (β-Sarcoglycan Deficiency)
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Change from Baseline in β-SG Protein Expression Quantity Assessed by Immunofluorescence (IF) Fiber Intensity at Day 60
研究概览
简要总结
The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.
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 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Cohort 1 only: Ambulatory per protocol specified criteria. - Cohort 2 only: Non-ambulatory per protocol specified criteria and 4 to 50 years of age.
- •Possesses 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-sarcoglycan gene Deoxyribonucleic acid (DNA) (SGCB) gene mutations based on documented clinical findings.
- •Ability to cooperate with muscle testing.
排除标准
- •Presence of any other clinically significant illness or medical condition, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, neuromuscular (other than LGMD2E/R4), or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for gene transfer or a medical condition or extenuating circumstance 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.
- •Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.
- •Any contraindication to use of corticosteroid.
- •Note: Other inclusion or exclusion criteria could apply.
研究组 & 干预措施
SRP-9003
Experimental
Participants will receive single IV infusion of SRP-9003 on Day 1.
干预措施: SRP-9003 (Genetic)
结局指标
主要结局
Change from Baseline in β-SG Protein Expression Quantity Assessed by Immunofluorescence (IF) Fiber Intensity at Day 60
时间窗: Baseline, Day 60
Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
时间窗: Baseline up to Month 60
Change from Baseline in β-SG Protein Expression Quantity Assessed by IF Percent Protein Fibers (PPF) at Day 60
时间窗: Baseline, Day 60
Change from Baseline in β-SG Protein Expression Quantity Assessed by Western Blot at Day 60
时间窗: Baseline, Day 60
次要结局
- Change from Baseline in Ability Captured Through Interactive Video Evaluation (ACTIVE) - Seated Workspace Volume Task at Month 60(Baseline, Month 60)
- Change from Baseline in North Star Assessment for Dysferlinopathy (NSAD) at Month 60(Baseline, Month 60)
- Change From Baseline in Time to Rise From the Floor, Time to Complete 100 and 10 meter Walk/Run, and the Timed Stair Ascend 4 Steps Test(Baseline, Month 60)
- Change from Baseline in Performance of Upper Limb Version 2.0 (PUL 2.0) Score at Month 60(Baseline, Month 60)
- Cohort 1 (Ambulatory): Change from Baseline in Stride Velocity (95%) and Stair-climbing Velocity (95%) as Assessed by a Wearable Device at Month 60(Baseline, Month 60)
- Cohort 1 (Ambulatory): Change from Baseline in Number of Stairs Climbed per Hour as Assessed by a Wearable Device at Month 60(Baseline, Month 60)
- Cohort 1 (Ambulatory): Change from Baseline in Stride Length (95%) as Assessed by a Wearable Device at Month 60(Baseline, Month 60)
- Change from Baseline in β-SG Protein Expression Quantity Assessed by IF Fiber Intensity at Month 24(Baseline, Month 24)
- Change from Baseline in β-SG Protein Expression Quantity Assessed by IF Percent Protein Fibers (PPF) at Month 24(Baseline, Month 24)
- Change from Baseline in β-SG Protein Expression Quantity Assessed by Western Blot at Month 24(Baseline, Month 24)
- Cohort 1 (Ambulatory): Change from Baseline Distance Walked per Hour as Assessed by a Wearable Device at Month 60(Baseline, Month 60)
- Change from Baseline in Upper Extremity Activity at Month 60(Baseline, Month 60)
- Change from Baseline in Angular Wrist Velocity at Month 60(Baseline, Month 60)
- Change from Baseline in Vector Genome Copies Using Droplet Digital Polymerase Chain Reaction (ddPCR) in the Target Muscle Tissue(Baseline, Day 60 and Month 24)
研究者
研究点 (2)
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