Autologous BMMNCs Combined With Educational Intervention for ASD

Vinmec Research Institute of Stem Cell and Gene Technology

Status and phase: Completed; Phase 2

Conditions: Autism Spectrum Disorder

Treatments: Combination Product: Educational intervention; Combination Product: Autologous BMMNCs transplantation and educational intervention

Study type: Interventional

Funder types: Other

Identifiers

NCT05307536

ISC.19.50

Details and patient eligibility

About

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.

Full 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

Enrollment: 54 patients

Sex: All

Ages: 3 to 7 years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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54 participants in 2 patient groups

Autologous BMMNCs infusion combined with educational intervention

Experimental group

Description:

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)

Treatment:

Combination Product: Autologous BMMNCs transplantation and educational intervention

Educational intervention (controlled group)

Active Comparator group

Description:

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)

Treatment:

Combination Product: Educational intervention