NCT04710810

UCB Stem Cells for Autism Spectrum Disorders

Transplantation of Human Allogenic Cord Blood Mononuclear Stem Cells in Autism: Safety and Efficacy of the Method

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: St. Petersburg Bekhterev Research Psychoneurological Institute · Academic / Other
• Sex: All
• Age: 3 Years – 11 Years
• Healthy volunteers: Not accepted

Summary

Purpose. The goals of the study are to assess the safety of the intravenous infusion of umbilical cord blood (UCB) cells in patients with autism and to confirm changes in social/ communicative skills and cognitive functioning after four infusions of ABO/Rh-matched UCB stem cells. Material and methods. The sample comprises 30 patients (27 males, 3 females) aged between 3 and 11 years with ASD under the care of the National Medical Research Center for Psychiatry and Neurology (Saint-Petersburg, Russia). Participants are randomly assigned to either the control group (14 males, 1 female) or the experimental group (13 males, 2 females). The experimental group receives intravenous injections of UCB cells four times with a two-week gap between injections. The control group receives standard therapy. The dynamic of cognitive functions and social/communicative skills assess with Checklist for autism spectrum disorders (CASD), Autism treatment evaluation checklist (ATEC), subscales of Wechsler Intelligence Scale for Children (WISC) - "Digit Span", "Picture completion", "Block design", "Coding".

Conditions

• Autism Spectrum Disorder

Interventions

Type	Name	Description
OTHER	umbilical cord blood mononuclear stem cells	UCB samples (70-130 ml of initial volume) were aseptically drained in blood-collection bags with 35 mL of citrate phosphate dextrose (CPDA-1) anticoagulant. Red blood cell-depleted/plasma-reduced nucleated cells were isolated by sedimentation in dextran-40, resuspended in autologous plasma with 10% dimethylsulfoxide and 1% dextran-40, and aliquoted in 3.8 ml vials with target doses of 3.0±0.2 x 10⁶ cells per vial. Vials were cooled to -90⁰C, quarantined in the vapor phase of liquid nitrogen. Tested for HIV-1/2, hepatitis B and C, HTLV-1/2, HSV-1 and -2, CMV, syphilis, and sterility. Characterized by AB0/Rh, CD34/CD45-positive cell content. Before injection sample thaws in the water bath at +37⁰C and washes from an excess amount of DMSO in 40 mL of physiological saline containing 2.5% human serum albumin and 5% dextran-40. After centrifugation and supernatant withdrawal, the UCB resuspends in 40 ml of the same solution at a target dose of 250±10 x 10⁶ viable cells per sample.

Timeline

• Start date: 2019-11-04
• Primary completion: 2021-01-31
• Completion: 2021-07-31
• First posted: 2021-01-15
• Last updated: 2021-08-04

Locations

1 site across 1 country: Russia