Autologous BMMNCs Combined With Educational Intervention for ASD

NCT05307536 | Completed Phase 2 DMC

• 1 other identifier: ISC.19.50
• Study Type: interventional
• Target: 54
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (4)

• 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").

  2 months, 6 months, 12 months after the first BMMNCs infusion

• Change of severity of ASD after intervention through the Childhood Autism Rating Scale

  The Childhood Autism Rating Scale (CARS) consists of 14 domains assessing behaviors associated with ASD, with a 15th domain rating general impressions of ASD. Each domain is scored on a scale ranging from one to four; higher scores are associated with a higher level of impairment. Total scores can range from a low of 15 to a high of 60; scores below 30 indicate that the individual is in the non-autistic range, scores between 30 and 36.5 indicate mild to moderate autism, and scores from 37 to 60 indicate severe autism.

  2 months, 6 months, 12 months after the first BMMNCs infusion

• Changes in social interaction; adaptive behaviour; daily living skills through Vineland Adaptive Behaviour Scale

  The Vineland Adaptive Behaviour Scale second version (VABS-2) is a semi-structured interview measuring adaptive behavior with standard scores (M = 100, SD = 15). We define proficiency as a standard score of 85 or above (i.e., 1 SD below the mean). This metric for "not impaired" is used in other optimal outcome studies and the learning disability literature. We define growth as an improvement of at least 1 SD in standard scores (15 points) from T1 to T2. Improvement of 1 SD in clinical trials is considered a large effect size.

  2 months, 6 months, 12 months after the first BMMNCs infusion

• Changes in social interaction; adaptive behaviour; daily living skills through Clinical Global Impression Improvement

  The Clinical Global Impression Improvement (CGI) is a rating scale that measures symptom severity and treatment response. The severity is categorized into seven levels: 1) not present (no ASD); 2) barely evident ASD symptoms; 3) mild ASD symptoms; 4) moderate ASD symptoms; 5) moderately severe ASD symptoms; 6) severe ASD symptoms or 7) very severe ASD symptoms. The response of each patient is also divided into seven levels: level 1) very much improved; level 2) much improved; level 3) minimally improved; level 4) no change; level 5) minimally worse; level 6) much worse; level 7) very much worse.

  2 months, 6 months, 12 months after the first BMMNCs infusion

Secondary Outcomes (1)

• Changes in health-related quality of life using Pediatric Quality of Life Inventory (PedsQL)

  2 months, 6 months, 12 months after the first BMMNCs infusion

Study Arms (2)

Autologous BMMNCs infusion combined with educational intervention

EXPERIMENTAL

Bone marrows are harvested from the patient's iliac depending on the patient's body weight as follows: 8 ml/kg for patients under 10 kg; \[80 ml + (body weight in kg - 10) × 7 ml\] for patients above 10 kg. Mononuclear cells from collected bone marrow are infused intrathecally through a space between number vertebrae 4th and 5th. Two transplantations will be conducted at an interval of 6 months. The educational intervention will be developed based on the Early Start Denver Model for 6 months after the first infusion. Evaluation: The efficacy outcomes will be measured using the CARS, Vineland Adaptive Behavior Scales - second Edition (VABS), Autism Behavior Checklists (ABC), and Clinical Global Impression (CGI). In addition, Health-related quality of life (HRQoL) in patients will be evaluated at baseline, two months, six months, and 12 months with those in the control group (educational intervention only) using the Pediatric Quality of Life Inventory (PedsQL)

Combination Product: Autologous BMMNCs transplantation and educational intervention

Educational intervention (controlled group)

ACTIVE COMPARATOR

The educational intervention will be developed based on the Early Start Denver Model for six months after enrollment. Evaluation: The efficacy outcomes will be measured using the CARS, Vineland Adaptive Behavior Scales - second Edition (VABS), Autism Behavior Checklists (ABC), and Clinical Global Impression (CGI). In addition, Health-related quality of life (HRQoL) in patients will be evaluated at baseline, two months, six months, and 12 months with those in the control group (educational intervention only) using the Pediatric Quality of Life Inventory (PedsQL)

Combination Product: Educational intervention

Interventions

Autologous BMMNCs transplantation and educational intervention COMBINATION_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

Autologous BMMNCs infusion combined with educational intervention

Educational intervention COMBINATION_PRODUCT

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

Educational intervention (controlled group)

Eligibility Criteria

• Age: 3 Years - 7 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Vinmec Research Institute of Stem Cell and Gene Technology: lead

Study Sites (1)

Vinmec Research Institute of Stem Cell and Gene Technology

Hanoi, 10000, Vietnam

Location

Related Publications (2)

• Nguyen LT, Nguyen PM, Nguyen HP, Bui HT, Dao LTM, Van Pham M, Hoang CK, Nguyen PT, Nguyen TTP, Nguyen ATP, Hoang VT, Bui HTP, Vuong NK, Van Ngo D. Outcomes of autologous bone marrow mononuclear cell administration combined with educational intervention in the treatment of autism spectrum disorder: a randomized, open-label, controlled phase II clinical trial. Stem Cell Res Ther. 2025 May 30;16(1):268. doi: 10.1186/s13287-025-04404-4.

  PMID: 40442857 DERIVED

• Than UTT, Nguyen LT, Nguyen PH, Nguyen XH, Trinh DP, Hoang DH, Nguyen PAT, Dang VD. Inflammatory mediators drive neuroinflammation in autism spectrum disorder and cerebral palsy. Sci Rep. 2023 Dec 18;13(1):22587. doi: 10.1038/s41598-023-49902-8.

  PMID: 38114596 DERIVED

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Early Intervention, Educational

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Child Health Services; Community Health Services; Health Services; Health Care Facilities Workforce and Services; Preventive Health Services

Study Officials

• Liemg Nguyen, PhD

  Vinmec Research Institute of Stem Cell and Gene Technology

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: A total of 54 patients with autism spectrum disorder (3-7 years old) will be recruited and divided into two groups: the controlled group (only intervention education) (n = 27) and the treatment group (Bone marrow mononuclear cell infusions combined educational intervention (n =27)

Study Record Dates

• First Submitted: February 12, 2022
• First Posted: April 1, 2022
• Study Start: December 26, 2021
• Primary Completion: June 30, 2023
• Study Completion: December 30, 2023
• Last Updated: January 22, 2026

Record last verified: 2026-01

Locations

VN (1)