UCB Stem Cells for Autism Spectrum Disorders

NCT04710810 | Completed Phase 1

• 1 other identifier: SCA1512
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief 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

Outcome Measures

Primary Outcomes (3)

• Incidence of Treatment Emergent Adverse Events

  Assessed by Adverse Event Grading Scale. It categorized adverse events according to severity. Mild Adverse Event - event results in mild or transient discomfort, not requiring intervention or treatment; does not limit or interfere with daily activities (e.g., insomnia, mild headache). Moderate Adverse Event - event is sufficiently discomforting so as to limit or interfere with daily activities; may require interventional treatment (e.g., fever requiring antipyretic medication). Severe and undesirable Adverse Event - event results in significant symptoms that prevent normal daily activities; may require hospitalization or invasive intervention (e.g., anemia resulting in blood transfusion).

  24 hours after infusion

• Changes in severity of autistic symptoms from baseline at 3d and 6th month (measured by Checklist for Autism Spectrum Disorders (CASD)

  The CASD rates 30 symptoms of autism, covering six domains: problems with social interaction, perseveration, somatosensory disturbance, atypical communication and development, mood, problems with attention and safety. Symptoms are scored as present or absent based on information drawn from various sources, including teacher, caregiver, parent, observation of the child, and available records. The scores from each subscale are combined in order to calculate a Total Score, which ranges from 0 to 30 points. A lower score indicates less severe symptoms of ASD and a higher score correlates with more severe symptoms of ASD.

  at baseline, 3d and 6th month

• Changes in severity of autistic symptoms from baseline at 3d and 6th month (measured by Autism Treatment Evaluation Checklist (ATEC)

  The purpose of the ATEC is to measure a change in a patient due to various interventions. It consists of four subtests: Speech/Language Communication (14 items); Sociability (20 items); Sensory/ Cognitive Awareness (18 items); Health/Physical/Behavior (25 items). The scores from each subscale are combined in order to calculate a Total Score, which ranges from 0 to 179 points. A lower score indicates less severe symptoms of ASD and a higher score correlates with more severe symptoms of ASD.

  at baseline, 3d and 6th month

Secondary Outcomes (4)

• "Digit Span" subscale of Wechsler Intelligence Scale for Children (WISC)

  at baseline, 6th month

• "Picture completion" subscale of Wechsler Intelligence Scale for Children (WISC)

  at baseline, 6th month

• "Block design" subscale of Wechsler Intelligence Scale for Children (WISC)

  at baseline, 6th month

• "Coding" subscale of Wechsler Intelligence Scale for Children (WISC)

  at baseline, 6th month

Study Arms (2)

Experimental

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.

Other: umbilical cord blood mononuclear stem cells

Control

NO INTERVENTION

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

Interventions

umbilical cord blood mononuclear stem cells 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.

Experimental

Eligibility Criteria

• Age: 3 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• diagnosed ASD by ICD-10 criteria

You may not qualify if:

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

Sponsors & Collaborators

• St. Petersburg Bekhterev Research Psychoneurological Institute: lead

Study Sites (1)

St.-Petersburg Bekhterev Reserach Psychoneurological Institute

Saint Petersburg, Sankt-Peterburg, 192019, Russia

Location

MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 28, 2020
• First Posted: January 15, 2021
• Study Start: November 4, 2019
• Primary Completion: January 31, 2021
• Study Completion: July 31, 2021
• Last Updated: August 4, 2021

Record last verified: 2021-08

Locations

RU (1)