Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Numbers of participants with adverse events (AEs), serious adverse events (SAEs)
研究概览
简要总结
The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA.
The specific objectives are:
- To determine clinical effectiveness of Vesemnogene lantuparvovec therapy for SMA as evaluated by developmental gross motor milestone and survival.
- To describe the safety profile of Vesemnogene therapy for SMA as evaluated by adverse events reporting and laboratory tests, and monitoring of Adverse events of special interest.
详细描述
This is an observational study designed to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec therapy for SMA. Potential patients with genetic diagnosis of SMA will be evaluated for eligibility to undergo available gene therapies. Following the administration of Vesemnogene therapy, patient will be monitored for toxicity and response to treatment. No subjects will be withdrawn from the study, and subjects could freely drop out from the study anytime, simply by not showing up.
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
- 6 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Willing and able to give written informed consent for participation in the study.
- •Genetic confirmation of SMA (biallelic deletion or mutation of SMN1).
- •SMA clinical phenotype and condition, that in the opinion of the treating physician, treatment with Vesemnogene will likely be beneficial.
- •Absence of contraindications for spinal tap procedure or administration of intrathecal therapy.
- •Total AAV antibody titres < 1:20 as determined by ELISA assay.
- •Normal liver function (AST/ALT < 3XULN, Bilirubin <3.0 mg/dL).
- •Unable to access or failure to respond to currently available curative treatments for SMA.
排除标准
- 未提供
研究组 & 干预措施
The selected dose inchildren ≥ 6 months of age
Administration the selected dose of Vesemnogene Lantuparvovec in children > 6 months of age
干预措施: vesemnogene lantuparvovec (Biological)
结局指标
主要结局
Numbers of participants with adverse events (AEs), serious adverse events (SAEs)
时间窗: Baseline up to 5 years old
Participants are monitored for safety from baseline up to the end of the follow-up period.
次要结局
- Change from baseline in developmental gross motor milestones achieved according to WHO criteria(Baseline up to 5 years old)
- Event-free Survival until the 5th year or the last follow-up(Baseline up to 5 vears old)