UCB Stem Cells for Autism Spectrum Disorders

St. Petersburg Bekhterev Research Psychoneurological Institute

Status and phase: Completed; Phase 1

Conditions: Autism Spectrum Disorder

Treatments: Other: umbilical cord blood mononuclear stem cells

Study type: Interventional

Funder types: Other

Identifiers

NCT04710810

SCA1512

Details and patient eligibility

About

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".

Enrollment: 30 patients

Sex: All

Ages: 3 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• diagnosed ASD by ICD-10 criteria

Exclusion criteria

• acute somatic diseases
• indication for surgical (neurosurgical) therapy
• acute or chronic infectious diseases
• history of acute allergic reactions

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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30 participants in 2 patient groups

Experimental

Experimental group

Description:

Patients receive group and rhesus compatible UCB cells through intravenous bolus injections (4 injections at 2-week intervals) after pre-medication with Clemastine (0.025 mg/kg body weight, intravenously). One dose is 250±10 x 10⁶ viable cells per sample.

Treatment:

Other: umbilical cord blood mononuclear stem cells

Control

No Intervention group

Description:

Patients receives standard therapy (applied behavioral analysis, speech therapy).