NCT06152237进行中（未招募）1 期
A Multicenter, Open Label, Randomized, Dose-Escalation and Dose-Expansion Study of the Safety, Tolerability, and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females With Rett Syndrome
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
- 5
- 主要终点
- Primary Safety
研究概览
简要总结
The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome.
The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is up to 6 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 8 Years（Child）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Participant has a confirmed diagnosis of classical/typical Rett Syndrome with a documented mutation of the MECP2 gene that results in loss of function.
- •Participant is between ≥5 to ≤8 years of age at the time of consent.
- •Participant must be up to date with all relevant local vaccination requirements, with last vaccination dose received at least 42 days prior to the start of the immunosuppression regimen.
- •Participant's parent/caregiver must be willing to allow participant to receive blood or blood products for the treatment of an AE if medically needed.
排除标准
- •Participant has another neurodevelopmental disorder independent of the MECP2 gene loss of function mutation, or any other genetic syndrome with a progressive course.
- •Participant has a history of brain injury that causes neurological problems.
- •Participant had grossly abnormal psychomotor development in the first 6 months of life.
- •Participant has a diagnosis of atypical Rett syndrome.
- •Participant has an MECP2 mutation that does not cause Rett syndrome.
- •Participant requires non-invasive and invasive ventilatory support.
- •Participant has contraindications for IT administration of TSHA-102 or lumbar puncture procedure, other medical conditions, or contraindications to any medications required for IT administration.
- •Participant has acute or chronic hepatitis B or C infections.
研究组 & 干预措施
Cohort 2
Experimental
Dose Level 2
干预措施: TSHA-102 (Genetic)
Cohort 1
Experimental
Dose Level 1
干预措施: TSHA-102 (Genetic)
结局指标
主要结局
Primary Safety
时间窗: Baseline through week 52
The incidence of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
次要结局
- Exploratory Efficacy(Baseline through week 52)
研究者
研究点 (5)
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