Allogenic Cord Blood Transfusion in Patients With Autism

NCT03786744 | Completed Phase 1 Results

• 1 other identifier: 12112018
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Autism is one of those disorders in Autism spectrum disorders (ASD), which characterized by social interaction abnormalities, impaired verbal and non-verbal communication, and repetitive, obsessive behavior, while the therapeutic effect of current treatments remains limited progress. The possible reason for ASD is neural hypoperfusion and immune deregulation. The Human Umbilical Cord Blood Mononuclear Cells (hUCB-MNCs) have been shown to have the ability to modulate the immune response and enhance angiogenesis, suggesting the novel and promising therapeutic strategy. In this study, the safety and efficacy of hUCB-MNCs infusion will be evaluated in patients with Autism.

Conditions

Autism Spectrum Disorder; Autism

Keywords

Autism, ASD, Cord blood

Outcome Measures

Primary Outcomes (2)

• A Number of Participants With Non-serious and Serious Adverse Events 24 Hrs Post-transfusion

  Safety assessment. The adverse events rate will be assessed in all patients.

  24hrs post transfusion

• Autism Treatment Evaluation Checklist (ATEC).

  The ATEC is designed to measure changes in the severity of ASD in response to treatment. A total score and 4 subscale scores are reported. Questions in the first 3 subscales are scored using a 0-3 scale (not true/partially true/absolutely true). The 4 subscale, Health/Physical/Behavior, is scored using a 0-4 point scale (Not a problem/Minor problem/Moderate problem/Serious problem). The first subscale, Speech/Language/Communication, contains 14 items where the score ranges from 0-28 points. The Sociability subscale contains 20 items and participants can score from 0-40. The third subscale, Sensory/Cognitive awareness, has 18 items and scores range from 0-36. Finally, the Health/Physical/Behavior subscale contains 25 items and scores range from 0-75. The scores from each subscale are combined in order to calculate a Total Score, which ranges from 0 to179 points. A lower score indicates less severe symptoms of ASD and a higher score correlates with more severe symptoms of ASD.

  score at baseline, 1, 2, 6, 12 months

Secondary Outcomes (3)

• Cytokine Analysis.

  score at baseline, 1, 2, 6 months

• Immune Status as Determined by Percentage of Peripheral Blood Cells

  score at baseline, 6 month

• Immune Status as Determined by Absolute Cell Count of Peripheral Blood Cells

  score at baseline, 6 month

Study Arms (2)

ASD CB-MNC injection.

EXPERIMENTAL

ASD CB-MNC injection from different donors and standard therapy.

Biological: ASD CB-MNC injection.; Other: Standard therapy.

Standard therapy.

OTHER

Patients with standard therapy as control group.

Other: Standard therapy.

Interventions

ASD CB-MNC injection. BIOLOGICAL

CB-MNC injection from different donors. One dose consist 20-50 mil MNC/kg.The protocol include 3 injection at monthly intervals.

ASD CB-MNC injection.

Standard therapy. OTHER

The standard therapy can include drugs, special psychology training etc.

ASD CB-MNC injection.; Standard therapy.

Eligibility Criteria

• Age: 3 Years - 15 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Confirmed diagnosis: autistic type disorder (ASD).
• Systemic speech underdevelopment
• The presence of attention deficit hyperactivity disorder as a comorbid state
• Cognitive impairment

You may not qualify if:

• organic pathology of the brain according to CT, MRI
• the presence of the following diseases in history: heart failure in the stage of decompensation, stroke in history less than 1 year ago, blood diseases;
• decompensation of chronic and endocrinological diseases;
• acute respiratory viral and bacterial infections, period less than 1 month after the acute phase.
• HIV infection, hepatitis B and C.
• oncological diseases, chemotherapy in the anamnesis;
• tuberculosis.
• Severe form of intellectual disability.
• Cerebral palsy.
• epilepsy.

Sponsors & Collaborators

• State-Financed Health Facility "Samara Regional Medical Center Dinasty": lead

Study Sites (1)

Medical Centre Dinasty

Samara, 443095, Russia

Location

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Results Point of Contact

• Title: Volchkov Stanislav
• Organization: Samara Medical Center Dinasty

quality@cordbank.ru

+78469564455

Study Officials

• STANISLAV VOLCHKOV, MD, PhD

  Medical Centre Dinasty

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Deputy director, Quality assurance director

Model Details: This is prospective, non-randomized (open label) with control group study. The total 20 patient will be enrolled in this study. In 10 patient with ASD will be infused with umbilical corb blood. Other 10 patient with ASD will be treated with standard therapy (control group).

Study Record Dates

• First Submitted: December 21, 2018
• First Posted: December 26, 2018
• Study Start: October 12, 2018
• Primary Completion: October 12, 2019
• Study Completion: December 12, 2019
• Last Updated: November 19, 2020
• Results First Posted: November 19, 2020

Record last verified: 2020-10

Locations

RU (1)