Clinical trial NCT05307536: Autologous BMMNCs Combined With Educational Intervention for ASD
Official title: Autologous Bone Marrow Mononuclear Cell Transplantation Combined With Educational Intervention for Autism Spectrum Disorders (ASD): A Phase 2 Randomised Controlled Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autism Spectrum Disorder
Lead sponsor: Vinmec Research Institute of Stem Cell and Gene Technology
Sponsor class: OTHER
First posted: 2022-04-01
Enrollment: 54
Sex: ALL
Minimum age (years): 3
Maximum age (years): 7
Standard ages: CHILD
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 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)
Primary purpose: TREATMENT

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.

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

Eligibility criteria: 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

Primary outcomes:
measure: Change of severity of ASD after intervention through the Diagnostic and Statistical Manual of Mental Disorders
timeFrame: 2 months, 6 months, 12 months after the first BMMNCs infusion
measure: Change of severity of ASD after intervention through the Childhood Autism Rating Scale
timeFrame: 2 months, 6 months, 12 months after the first BMMNCs infusion
measure: Changes in social interaction; adaptive behaviour; daily living skills through Vineland Adaptive Behaviour Scale
timeFrame: 2 months, 6 months, 12 months after the first BMMNCs infusion
measure: Changes in social interaction; adaptive behaviour; daily living skills through Clinical Global Impression Improvement
timeFrame: 2 months, 6 months, 12 months after the first BMMNCs infusion
Secondary outcomes:
measure: Changes in health-related quality of life using Pediatric Quality of Life Inventory (PedsQL)
timeFrame: 2 months, 6 months, 12 months after the first BMMNCs infusion
Other outcomes:
Locations:
city: Hanoi
country: Vietnam
facility: Vinmec Research Institute of Stem Cell and Gene Technology
Study officials:
affiliation: Vinmec Research Institute of Stem Cell and Gene Technology
name: Liemg Nguyen, PhD
role: PRINCIPAL_INVESTIGATOR
References: