Phase 1 Study Evaluating the Safety and Preliminary Efficacy of a Novel Cell-Based and Small Molecule Combination Therapy for White Matter Injury in Patients With Periventricular Leukomalacia
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 发起方
- 入组人数
- 10
- 主要终点
- Change in White Matter Integrity
研究概览
简要总结
The goal of this study is to explore a new treatment that may help repair brain damage in individuals with periventricular leukomalacia (PVL), a condition that affects white matter in the brain. Researchers are testing whether a combination of a novel cell therapy and specific molecular agents can support brain repair.
The main questions the study aims to answer are:
Can the treatment help regrow white matter and improve myelin repair? Does the treatment reduce scarring in the brain? Is the treatment safe and well-tolerated?
The study uses several components, including:
A specific type of neural progenitor cell to form the basis of the therapy. A small molecule compound to support cell function and survival. An agent designed to promote the repair of the myelin sheath. An enzyme intended to break down scar tissue in the brain. Researchers will study how these components work together to protect and repair the brain by influencing key pathways involved in damage and recovery.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of periventricular leukomalacia (PVL) confirmed by MRI
- •Clinically stable at time of intervention
- •Parental or legal guardian consent if participant is a minor
排除标准
- •Severe congenital brain malformations unrelated to PVL
- •Active CNS infection or systemic inflammatory disease
- •History of severe intraventricular hemorrhage (Grade III/IV)
- •Known allergy or history of a significant hypersensitivity reaction to the investigational product or any of its components.
- •Participation in another interventional study within the past 30 days
结局指标
主要结局
Change in White Matter Integrity
时间窗: At baseline and 12 weeks post-intervention
Measured using fractional anisotropy (FA) values derived from diffusion tensor imaging (DTI) MRI to assess structural white matter characteristics in brain regions affected by periventricular leukomalacia (PVL).
次要结局
- Change in Functional Motor Score(Baseline, 6 weeks, and 12 weeks post-intervention)
- Change in Glial Scar Density(12 weeks post-intervention)