Autologous BMMNCs Combined With Educational Intervention for ASD

Vinmec Research Institute of Stem Cell and Gene Technology54 enrolled

Overview

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.

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Interventions

Autologous BMMNCs transplantation and educational interventionCOMBINATION_PRODUCT

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

Educational interventionCOMBINATION_PRODUCT

Educational intervention: 6 months based on the Early Start Denver Model

Eligibility

Sex: ALLMin age: 3 YearsMax age: 7 YearsHealthy volunteers:

Medical Language ↔ Plain English

Inclusion Criteria: * Patient is between 3 to 7 years old * Male/Female patient diagnosed with Autism disorder according to the diagnostic criteria for Autism Spectrum Disorder in Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) * Patient has a medium level to the most severe level of autism (CARS score \>=34 to 50) * Patient's parent or caregiver must have an educational level of high school or above * Patient's family has given consent to participate in the study Exclusion Criteria: * Patient is above seven years of age * Autistic patient having epilepsy * Patient with coagulation disorders * Hydrocephalus with ventricular drain * Allergy to anesthetic agents * Sever health conditions such as cancer, failure of heart, lung, liver, or kidney * Active infections

Outcomes

Primary Outcomes

Change of severity of ASD after intervention through the Diagnostic and Statistical Manual of Mental Disorders

The Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5). DSM-5 describes three levels of increasing severity of ASD.DSM-5 describes three levels of increasing severity of ASD, from (1) ("requiring support") to (2) ("requiring substantial support"), and finally to (3) ("requiring very substantial support").