NCT05307536

Autologous BMMNCs Combined With Educational Intervention for ASD

Autologous Bone Marrow Mononuclear Cell Transplantation Combined With Educational Intervention for Autism Spectrum Disorders (ASD): A Phase 2 Randomised Controlled Trial

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 54 (actual)
• Sponsor: Vinmec Research Institute of Stem Cell and Gene Technology · Academic / Other
• Sex: All
• Age: 3 Years – 7 Years
• Healthy volunteers: Accepted

Summary

The purpose of this study is to evaluate the safety and effectiveness of autologous bone marrow mononuclear cell infusion combined with educational intervention for children with autism spectrum disorder (ASD). Inclusion criteria: Male/Female patients diagnosed with Autism disorder based on DSM-V scale, the patient is between 3 to 7 years old, a patient has a medium level to server level of autism (CARS score\>=30 to \<50), VARS-2 \>=50, patient's parent or caregiver must have the educational level of high school or above, patient's family has given consent to participate in the study. Exclusion criteria for treatment group: patient above seven years of age and smaller than 3-year old, autistic patients having epilepsy, hydrocephalus with ventricular drain, allergy to anesthetic agents, severe health conditions such as cancer, failure of heart, lung, liver, or kidney, active infections, children with birth defects of the spine.

Detailed description

A total of 54 patients with autism spectrum disorder (aged 3-7) will be recruited and divided into two groups: the control group (only intervention education) (n = 27) and the treatment group (bone marrow mononuclear cell transplantation combined intervention education (n =27). The bone marrow mononuclear cell will be applied to 27 patients in the treatment group infuse via the intrathecal administration. The primary outcome is measured by the number of AEs/SAEs during BMMNCs infusion. The secondary outcome was measured by clinical examinations performed at baseline and then at 02, 06, and 12 months after the first infusion. Multiple tools were used to diagnose and determine the severity of ASD, including DSM-5, CARS, the Vineland Adaptive Behavior Scales Second Edition (VABS-II), and the Clinical Global Impression (CGI). DSM-5, which was published in May 2013, provides new diagnostic criteria for ASD

Conditions

• Autism Spectrum Disorder

Interventions

Type	Name	Description
COMBINATION_PRODUCT	Autologous BMMNCs transplantation and educational intervention	Biological/ Vaccine: Autologous bone marrow-derived mononuclear cell infusion (BM-MNCs) will be administrated at baseline, and the second infusion will be performed six months after the first administration. For each transplantation, the bone marrow will be harvested through an anterior iliac crest puncture under general anesthesia in the operating theatre. The volume collected depends on the patient's body weight based on our experience from a previous study as follows: 8 ml/kg for patients under 10 kg; \[80 ml + (body weight in kg - 10) x 7 ml\] for patients above 10 kg but no more than 250 ml in total. Educational intervention: 6 months based on the Early Start Denver Model
COMBINATION_PRODUCT	Educational intervention	Educational intervention: 6 months based on the Early Start Denver Model

Timeline

• Start date: 2021-12-26
• Primary completion: 2023-06-30
• Completion: 2023-12-30
• First posted: 2022-04-01
• Last updated: 2026-01-22

Locations

1 site across 1 country: Vietnam