NCT06191354进行中（未招募）不适用
A Multicenter, Open, Dose-escalation Clinical Study Evaluating the Safety, Initial Efficacy, and Immunogenicity of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1
Kun Sun4 个研究点 分布在 1 个国家目标入组 12 人开始时间: 2023年6月25日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 12
- 试验地点
- 4
- 主要终点
- Incidence of AEs and SAEs
研究概览
简要总结
This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).
详细描述
This is a multicenter, open, dose-escalation clinical study to evaluate the safety, initial efficacy, and immunogenicity of SKG0201 injection in patients with spinal muscular atrophy type 1 (SMA 1).
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
- — 至 180 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Type 1 SMA, defined by bi-allelic mutations in the SMN1 gene.
- •Age 180 days or younger at day of infusion.
- •Clinical history and signs are consistent with type I SMA, that is hypotonia on clinical examination, with delay in motor skills, poor head control, rounded shoulder posture, and joint hypermobility.
- •The legal guardian of the subject understands the purpose of the study, the possible risks and rights of the study, agrees that the subject can participate in the study, complete all research steps, tests and visits, and sign the ICF voluntarily.
- •During the study period, according to the change of the subject's condition, the subject's legal guardian is willing to perform standard treatment requirements as suggested by the researcher.
排除标准
- •Pulse oximetry < 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support.
- •Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards (WHO 2006).
- •Active viral infection with significant signs or symptoms and require systematic hospitalization.
- •In the presence of other severe infections or diseases.
- •Known allergy to prednisolone, other glucocorticoids, or their excipients.
- •Clinically significant abnormal laboratory values prior to administration.
- •Previously used other SMA drugs (such as Spinraza, Evrysdi, Zolgensma, etc.) or participated in clinical studies of other SMA drugs.
- •Had received previous or anticipated major surgical procedures during the study assessment period.
结局指标
主要结局
Incidence of AEs and SAEs
时间窗: 18 months of age
AEs: adverse events; SAEs: serious Adverse events
Incidence and characteristics of DLT
时间窗: 4 weeks
DLT: dose-limiting toxicity
次要结局
- CHOP-INTEND score changes from baseline(24 weeks)
- Proportion of subjects who achieve developmental milestones with improvement in exercise intensity and function assessed according to BSID-III(24 weeks)
- Survival rate(14 months of age)
研究者
Kun Sun
Professor of Department of Pediatric Cardiology
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
研究点 (4)
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