Intrathecal Allogeneic Umbilical Cord Tissue-Derived Mesenchymal Stromal Cells in Children With Cerebral Palsy: A Pilot Study of Feasibility, Short-Term Safety, and Exploratory Outcomes
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 7
- 试验地点
- 1
- 主要终点
- Number of Participants Who Successfully Completed Intrathecal Administration of the Intended UCT-MSC Dose Without Technical Failure and Completed the 6-Month Follow-up
研究概览
简要总结
This is a single-center, open-label, single-arm Phase I pilot study designed to evaluate the feasibility and short-term safety of a single intrathecal administration of allogeneic umbilical cord tissue-derived mesenchymal stromal cells (UCT-MSCs) in children with cerebral palsy (CP).
详细描述
Seven children aged 2-5 years with Gross Motor Function Classification System (GMFCS) levels II-V will receive a single intrathecal dose of allogeneic UCT-MSCs in addition to standard multidisciplinary care. The primary outcomes are procedural feasibility and short-term safety, including the occurrence of adverse events during the procedure, hospitalization, and throughout a 6-month follow-up period. Exploratory outcomes include changes in functional classification and spasticity. This pilot study is intended to provide preliminary safety and feasibility data to support the design of future controlled clinical trials and is not powered to evaluate treatment efficacy.
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
- 2 Years 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children aged 2-5 years. Diagnosis of cerebral palsy, independently confirmed by two experienced pediatric neurologists.
- •Gross Motor Function Classification System (GMFCS) level II-V. Parent(s) or legal guardian(s) able and willing to provide written informed consent.
排除标准
- •History of congenital infection, including toxoplasmosis, rubella, cytomegalovirus, herpes simplex virus, or other clinically relevant congenital infections.
- •Normal findings on brain neuroimaging. Known malignancy. Confirmed genetic or congenital disorder unrelated to cerebral palsy. Advanced systemic disease, including renal insufficiency, hepatic dysfunction, or severe anemia.
- •Previous treatment with stem cell or stromal cell therapy. Participation in another interventional clinical study.
研究组 & 干预措施
Intrathecal UCT-MSC
Participants receive a single intrathecal administration of allogeneic umbilical cord-derived mesenchymal stromal cells 2 × 10⁶ viable UCT-MSCs/kg body weight administered by lumbal puncture.
干预措施: allogeneic umbilical cord tissue-derived mesenchymal stromal cells (UCT-MSCs) (Biological)
结局指标
主要结局
Number of Participants Who Successfully Completed Intrathecal Administration of the Intended UCT-MSC Dose Without Technical Failure and Completed the 6-Month Follow-up
时间窗: From the intrathecal administration through 6 months after intervention.
Feasibility was defined as successful delivery of the intended dose of allogeneic umbilical cord-derived mesenchymal stromal cells (UCT-MSCs), completion of the intrathecal administration without technical failure, completion of the planned post-procedural observation, participant retention through the 6-month follow-up, and absence of protocol deviations preventing completion of the intervention or follow-up.
Number of Participants With Treatment-Emergent Adverse Events Following Intrathecal Administration of Allogeneic Umbilical Cord-Derived Mesenchymal Stromal Cells
时间窗: From the intrathecal administration through 6 months after intervention.
Safety was assessed by recording immediate and delayed treatment-emergent adverse events occurring during the procedure, during hospitalization, and throughout the 6-month follow-up period. Events of interest included fever, vomiting, headache, irritability, back pain, local procedural complications, infection, allergic or infusion-related reactions, seizure, altered consciousness, neurological deterioration, prolonged hospitalization, and serious adverse events. For each adverse event, the time of onset, maximum severity, duration, management, clinical outcome, seriousness, and investigator-assessed relationship to the intervention were documented.
次要结局
- Gross Motor Function Classification System (GMFCS) Level(Baseline, Month 1, Month 3, and Month 6)
- Manual Ability Classification System (MACS) Level(Baseline, Month 1, Month 3, and Month 6)
- Communication Function Classification System (CFCS) Level(Baseline, Month 1, Month 3, and Month 6)
- Eating and Drinking Ability Classification System (EDACS) Level(Baseline, Month 1, Month 3, and Month 6)
- Modified Ashworth Scale (MAS) Score(Baseline, Month 1, Month 3, and Month 6)
研究者
Elisabeth Siti Herini
Professor
Gadjah Mada University