UCB Stem Cells for Autism Spectrum Disorders

NCT ID: NCT04710810

Last Updated: 2021-08-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-11-04

Study Completion Date

2021-07-31

Brief Summary

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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".

Detailed Description

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Conditions

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Autism Spectrum Disorder

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Experimental

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.

Group Type EXPERIMENTAL

umbilical cord blood mononuclear stem cells

Intervention Type OTHER

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.

Control

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

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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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.

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

3 Years

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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St. Petersburg Bekhterev Research Psychoneurological Institute

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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St.-Petersburg Bekhterev Reserach Psychoneurological Institute

Saint Petersburg, Sankt-Peterburg, Russia

Site Status

Countries

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Russia

Other Identifiers

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SCA1512

Identifier Type: -

Identifier Source: org_study_id

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