Neurodisability and Rehabilitation, Murdoch Children's Research Institute, Parkville, Victoria, Australia
Neurodevelopment and Disability, The Royal Children's Hospital Melbourne, Parkville, Victoria, Australia.
BMJ Open. 2020 Mar 8;10(3):e034974. doi: 10.1136/bmjopen-2019-034974.
Cerebral palsy (CP) is the most common physical disability of childhood but has no cure. Stem cells have the potential to improve brain injury and are proposed as a therapy for CP. However, many questions remain unanswered about the most appropriate cell type, timing of infusions, dose required and associated risks. Therefore, human safety and efficacy trials are necessary to progress knowledge in the field.
This is a single group study with sample size n=12 to investigate safety of single-dose intravenous 12/12 human leucocyte antigen-matched sibling cord blood cell infusion to children with CP aged 1-16 years without immune suppression. The study is similar to a 3+3 design, where the first two groups of participants have severe CP, and the final six participants include children with all motor severities. Children will be monitored for adverse events and the duration that donor cells are detected. Assessments at baseline, 3 and 12 months will investigate safety and preliminary evidence of change in gross motor, fine motor, cognitive and quality of life outcomes.
Full approval was obtained from The Royal Children's Hospital Human Research Ethics Committee, and a clinical trial notification was accepted by Australia's Therapeutic Goods Administration. Participant guardian informed consent will be obtained before any study procedures. The main results of this study will be submitted for publication in a peer-reviewed journal.
ACTRN12616000403437, NCT03087110.
脑瘫（CP）是儿童最常见的身体残疾，但目前尚无治愈方法。干细胞具有改善脑损伤的潜力，被提议作为脑瘫的一种治疗方法。然而，关于最合适的细胞类型、输注时间、所需剂量和相关风险等问题仍存在许多未解答的疑问。因此，需要进行人体安全性和疗效试验，以推动该领域的知识发展。
这是一项单组研究，样本量为 n=12，旨在研究 1-16 岁无免疫抑制的脑瘫儿童单次静脉输注 12/12 人类白细胞抗原匹配同胞脐带血细胞的安全性。该研究类似于 3+3 设计，前两组参与者患有严重脑瘫，最后六名参与者包括所有运动严重程度的儿童。将监测参与者的不良事件和供体细胞的检测持续时间。在基线、3 个月和 12 个月进行评估，以调查安全性和粗大运动、精细运动、认知和生活质量结果的初步变化证据。
已获得皇家儿童医院人类研究伦理委员会的全面批准，并获得澳大利亚治疗商品管理局的临床试验通知。在进行任何研究程序之前，将获得参与者监护人的知情同意。该研究的主要结果将提交给同行评议的期刊发表。
ACTRN12616000403437，NCT03087110。
