Randomized controlled single blind clinical trial for evaluating safety and efficacy of autologous bone marrow mononuclear cells intrathecal transplantation in children with Autism
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 50
- 试验地点
- 1
研究概览
简要总结
The proposed project titled Randomized Controlled Single-Blind Clinical Trial for Evaluating Safety and Efficacy of Autologous Bone Marrow Mononuclear Cells (BMMNCs) Intrathecal Transplantation in Children with Autism will be conducted at NeuroGen Brain and Spine Institute, Navi Mumbai. Approved by the Institutional Ethics Committee, this study aims to scientifically assess the safety and therapeutic potential of autologous BMMNCs delivered intrathecally in children diagnosed with Autism Spectrum Disorder (ASD).
Previous preclinical and clinical studies suggests that this intervention may improve autism-related symptoms by addressing core neuropathological mechanisms such as neuroinflammation, immune dysregulation, and cerebral hypoperfusion.
In this randomized controlled single-blind trial, 50 children (ages 3–15) diagnosed per DSM-V criteria will be recruited and divided into two groups:
1. Group A (Intervention): Intrathecal autologous BMMNC transplantation plus standard medical and rehabilitation therapy.
2. Group B (Control): Standard medical and rehabilitation therapy alone.
Independent blinded assessors will evaluate participants using validated outcome measures such as ISAA, CARS, GARS, at baseline and at 1, 3 and 6 months. Comparative Brain PET-CT scans done before intervention and 6 months after intervention will be used to monitor the efficacy of the intervention. Adverse events will be closely monitored by an independent Data Safety Monitoring Board.
This study seeks to establish robust scientific evidence for the safety and efficacy of autologous bone marrow-derived mononuclear cells as a novel adjunctive treatment for autism, potentially improving core behavioral, social, and functional outcomes in affected children.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 盲法
- Outcome Assessor Blinded
入排标准
- 年龄范围
- 3.00 Year(s) 至 15.00 Year(s)（—）
- 性别
- All
入选标准
- •Patients who are diagnosed as Autism on the basis of DSM V criteria
- •Patients whose parents are willing to give an informed written consent
- •Patients whose parents agree to adhere to the protocol and are willing to come for mandatory follow up assessments at regular intervals.
排除标准
- •1.Patients with other co-morbid neurodevelopmental disorders like mental retardation or cerebral palsy.
- •2.Patients with present malignancies or history of malignancies 3.Patients with positive HIV, Hepatitis – B, Hepatitis – C or any other acute infections 4.Patients with severe anemia, bleeding tendency; with blood hemoglobin levels below 8 g/dl.
- •5.Patients with any bone marrow disorders.
- •6.Severe renal or liver dysfunction.
- •7.Uncontrolled seizures.
研究者
Dr Alok Sharma
NeuroGen Brain and Spine Institute