NCT05906251终止1 期
An Open-Label, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP 6004 Administered by Systemic Infusion in Ambulatory Subjects With Limb Girdle Muscular Dystrophy Type 2B/R2 (LGMD2B/R2, Dysferlin Related)
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 1
- 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 primary purpose of this study is to evaluate the safety of SRP-6004 administered by intravenous (IV) infusion in ambulatory participants with LGMD2B/R2 (DYSF related).
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
- 18 Years 至 50 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic DYSF Deoxyribonucleic acid (DNA) gene mutations as documented prior to screening visits.
- •Participants must be ambulatory per protocol specified criteria.
- •Ability to cooperate with motor assessment testing.
- •Has accessible and intact lower and upper extremity musculature for biopsy.
- •Have adeno-associated virus rhesus serotype 74 (rAAVrh74) antibody titers < 1:400 (that is, not elevated) as determined by enzyme-linked immunosorbent assay (ELISA).
排除标准
- •Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.
- •Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- •Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.
- •Note: Other inclusion or exclusion criteria could apply.
研究组 & 干预措施
SRP-6004
Experimental
Participants will receive single IV infusion of SRP-6004 on Day 1.
干预措施: SRP-6004 (Genetic)
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 Month 60
次要结局
- Change from Baseline in Percent of Normal DYSF Protein Expression as Measured by Western Blot(Baseline, Day 90 and Month 15)
- Change from Baseline in Percent of Normal DYSF Protein Expression as Assessed by IF Percent DYSF Positive Fibers (PPF: DYSF)(Baseline, Day 90 and Month 15)
- Change from Baseline in Percent of Normal DYSF Protein Expression as Measured by Immunofluorescence (IF) Fiber Intensity(Baseline, Day 90 and Month 15)
研究者
研究点 (1)
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