| CTRI Number |
CTRI/2026/01/100104 [Registered on: 01/01/2026] Trial Registered Prospectively |
| Last Modified On: |
27/12/2025 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Biological |
| Study Design |
Randomized, Parallel Group, Active Controlled Trial |
|
Public Title of Study
|
Studying the therapeutic effect of Intrathecal administration of autologous Bone marrow mononuclear cells in children with autism |
|
Scientific Title of Study
|
Randomized controlled single blind clinical trial for evaluating safety and efficacy of autologous bone marrow mononuclear cells intrathecal transplantation in children with Autism |
| Trial Acronym |
NIL |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| NIL |
NIL |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
Dr Alok Sharma |
| Designation |
Director |
| Affiliation |
NeuroGen Brain and Spine Institute |
| Address |
Department of Medical Services, Second floor, Room 201, Plot 19, Sector 40, Seawoods West, Navi Mumbai
Thane
MAHARASHTRA
400706
India |
| Phone |
9820046663 |
| Fax |
|
| Email |
alok276@gmail.com |
|
Details of Contact Person
Scientific Query
|
| Name |
Dr Hemangi Sane |
| Designation |
Head Research and Development |
| Affiliation |
NeuroGen Brain and Spine Institute |
| Address |
Department of Research and Development, 1st Floor, Room no 105, Plot 19, Sector 40, Seawoods West, Navi Mumbai
Thane
MAHARASHTRA
400706
India |
| Phone |
9819741333 |
| Fax |
|
| Email |
doc.hemangi@gmail.com |
|
Details of Contact Person
Public Query
|
| Name |
Dr Nandini Gokulchandran |
| Designation |
Head Medical Services |
| Affiliation |
NeuroGen Brain and Spine Institute |
| Address |
Department of Medical Services, Room no 106, 1st floor, Plot 19, Sector 40, Seawoods West, Navi Mumbai
Thane
MAHARASHTRA
400706
India |
| Phone |
9987703080 |
| Fax |
|
| Email |
drnandini76@gmail.com |
|
|
Source of Monetary or Material Support
|
| NeuroGen Brain and Spine Institute, Plot no 19, Sector 40, Seawoods, Navi Mumbai, India. 400706 |
|
|
Primary Sponsor
|
| Name |
NeuroGen Brain and Spine Institute |
| Address |
Plot 19, Sector 40, Seawoods (W), Navi Mumbai-400706 |
| Type of Sponsor |
Private hospital/clinic |
|
|
Details of Secondary Sponsor
|
|
|
Countries of Recruitment
|
India |
|
Sites of Study
|
| No of Sites = 1 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Alok Sharma |
NeuroGen Brain and Spine Institute |
Department of Medicine, Plot 19, Sector 40, Seawoods (W), Navi Mumbai-400706
Thane
MAHARASHTRA |
9820046663
alok276@gmail.com |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 1 |
| Name of Committee |
Approval Status |
| NeuroGen Brain and Spine Institute |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: F840||Autistic disorder, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Intervention |
Intrathecal autologous BMMNC transplantation plus standard medical and rehabilitation therapy. |
This randomized, controlled, single blind trial includes 50 children (ages 3–15) diagnosed with autism based on DSM-V criteria. Independent blinded assessors will evaluate each child using standard tools (ISAA, CARS, GARS) at baseline and at 1, 3, and 6 months. Brain PET-CT scans will be done before the intervention and again at 6 months to assess changes. An independent Data Safety Monitoring Board will oversee and monitor any adverse events. The trial aims to generate scientific evidence on the safety and effectiveness of autologous bone marrow derived mononuclear cells as an additional treatment option for autism. |
| Comparator Agent |
Standard Medical and rehabilitation therapy |
All participants in the control group will be provided with standard medical and rehabilitation for autism |
|
|
Inclusion Criteria
|
| Age From |
3.00 Year(s) |
| Age To |
15.00 Year(s) |
| Gender |
Both |
| Details |
1. Patients who are diagnosed as Autism on the basis of DSM V criteria
2. Patients whose parents are willing to give an informed written consent
3. Patients whose parents agree to adhere to the protocol and are willing to come for mandatory follow up assessments at regular intervals.
|
|
| ExclusionCriteria |
| Details |
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
|
|
|
Method of Generating Random Sequence
|
Computer generated randomization |
|
Method of Concealment
|
Centralized |
|
Blinding/Masking
|
Outcome Assessor Blinded |
|
Primary Outcome
|
| Outcome |
TimePoints |
| Safety of the intervention |
1 month
3 months
6 months |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
| Efficacy of intervention |
1 month
3 months
6 months |
|
|
Target Sample Size
|
Total Sample Size="50"
Sample Size from India="50"
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" |
|
Phase of Trial
|
N/A |
|
Date of First Enrollment (India)
|
10/01/2026 |
| Date of Study Completion (India) |
Applicable only for Completed/Terminated trials |
| Date of First Enrollment (Global) |
Date Missing |
| Date of Study Completion (Global) |
Applicable only for Completed/Terminated trials |
|
Estimated Duration of Trial
|
Years="0"
Months="6"
Days="0" |
|
Recruitment Status of Trial (Global)
|
Open to Recruitment |
| Recruitment Status of Trial (India) |
Not Yet Recruiting |
|
Publication Details
|
N/A |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
|
Brief Summary
|
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. |