A Randomized Controlled Trial Evaluating the Efficacy and Safety of Autologous Bone Marrow-Derived Mononuclear Cell Transplantation Combined With Rehabilitation in Children With Sequelae of Hypoxic-Ischemic Encephalopathy or Intracranial Hemorrhage
概览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- National Children's Hospital, Vietnam
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- Change in Gross Motor Function Measure (GMFM-88) total score from baseline to 12 months
概览
简要总结
This study evaluates the safety and preliminary efficacy of autologous bone marrow mononuclear cell transplantation combined with rehabilitation in children with neurological sequelae caused by hypoxic-ischemic brain injury or intracranial hemorrhage. Participants in the intervention group will receive autologous bone marrow aspiration, mononuclear cell transplantation via intrathecal route, and rehabilitation, while the control group will receive rehabilitation alone. Outcomes will be compared between groups over follow-up time points.
详细描述
Neurological sequelae following hypoxic-ischemic brain injury and intracranial hemorrhage in children remain a major clinical challenge with limited effective treatment options. Rehabilitation is the current standard of care but often results in incomplete recovery. Autologous bone marrow mononuclear cell transplantation has emerged as a promising therapeutic approach due to its potential neuroregenerative and neuroprotective effects. This phase II study aims to evaluate the safety and preliminary efficacy of combining autologous bone marrow mononuclear cell transplantation with rehabilitation compared to rehabilitation alone. The study will also contribute to the development of a standardized technical protocol for this intervention.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children diagnosed with cerebral palsy
- •Age from 12 months to 72 months
- •Cerebral palsy caused by hypoxic-ischemic brain injury or intracranial hemorrhage
- •Gross Motor Function Classification System (GMFCS) level III to V
- •Completed required laboratory tests according to the study protocol
- •Written informed consent obtained from the child's parent or legal guardian
排除标准
- •Children with acute infection at the time of screening or enrollment
- •Children with severe coagulopathy or contraindications to bone marrow aspiration
- •Children with severe systemic diseases (e.g., severe cardiac, hepatic, or renal failure)
- •Children with contraindications to anesthesia or intrathecal transplantation procedures
- •Children with a history of malignancy
- •Children who are unable to participate in or complete the rehabilitation program
- •Parents or legal guardians who do not provide informed consent
研究组 & 干预措施
BMMNC Transplantation Plus Rehabilitation
Participants receive autologous bone marrow mononuclear cell transplantation via intrathecal administration combined with rehabilitation.
干预措施: Autologous Bone Marrow Mononuclear Cell Transplantation (Procedure)
Rehabilitation Alone
Participants receive rehabilitation without cell transplantation.
结局指标
主要结局
Change in Gross Motor Function Measure (GMFM-88) total score from baseline to 12 months
时间窗: Baseline and 12 months
Gross motor function will be assessed using the Gross Motor Function Measure (GMFM-88), a validated and standardized clinical scale for evaluating motor function in children with neurological impairment. The primary outcome is the change in GMFM-88 total score from baseline to 12 months. The change in score will be calculated and compared between the intervention group (autologous bone marrow mononuclear cell transplantation plus rehabilitation) and the control group (rehabilitation alone).
次要结局
- Change in GMFM score at 3, 6, 9 months(Baseline and 3, 6, 9 months)
- Change in Gross Motor Function Classification System (GMFCS) level from baseline to 12 months(Baseline and 12 months)
- Change in Manual Ability Classification System (MACS or Mini-MACS) level from baseline to 12 months(Baseline and 12 months)
- Incidence of transplantation-related adverse events(From intervention through 12 months follow-up)
研究者
Dat Tran
Principal Investigator, MD, PhD
National Children's Hospital, Vietnam