Safety and Efficacy of the Transfusion of UCB in Patients With an ASD Depending on the Degree of HLA Compatibility.

NCT04099381 | Unknown Phase 1

• 1 other identifier: ASD-HLA2019
• Study Type: interventional
• Target: 150
• 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 dysregulation. 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 with regarding to HLA compatibility.

Conditions

Autism Spectrum Disorder; Autism

Keywords

Autism, ASD, Cord blood, HLA

Outcome Measures

Primary Outcomes (3)

• Number of participants with non-serious and serious adverse events.

  Safety assessment. Adverse events rate will be assessed in all patients.

  baseline, change from baseline at 12 month

• The Childhood Autism Rating Scale (CARS2).

  Rating for behavior, characteristics, and abilities against the expected developmental growth. Will be assessed fifteen items, including: relationship to people; imitation; emotional response; body; object use; adaptation to change; visual response; listening response; taste-smell-touch response and use; fear and nervousness; verbal communication; non-verbal communication; activity level; level and consistency of intellectual response; general impressions. Total score will be assessed. Scores range form 15 to 60 with 30 being the cutoff rate for a diagnosis of mild autism. Scores 30-37 indicate mild to moderate autism, while scores between 38 and 60 are characterized as severe autism.

  baseline, change from baseline at 6,12 month

• Autism Treatment Evaluation Checklist (ATEC).

  Will be assessed: I. Speech/Language Communication (14 items); II. Sociability (20 items); III. Sensory/ Cognitive Awareness (18 items); and IV. Health/Physical/Behavior (25 items).

  baseline, change from baseline every month (total 12 evaluations)

Secondary Outcomes (4)

• Evaluation of the survival of donor cells in the host body without immune responses

  baseline, change from baseline at 6 month

• The correlation of the concentration of T-regulatory cells (CD4/CD25/FoxP3) in the sample of umbilical cord blood and the treatment effectiveness degree

  At 1, at 6 months (At first and second infusion)

• The correlation of the concentration of T-regulatory cells (CD4/CD25/FoxP3) in the patient depending on the severity of the disease.

  At 1, at 6 months (At first and second infusion)

• The correlation of the concentration of CD34+ cells in the cord blood sample and the treatment effectiveness degree

  At 1, at 6 months (At first and second infusion)

Study Arms (3)

Group 1 Low HLA compatibility

EXPERIMENTAL

ASD CB-MNC infusion from different donors and standard therapy. CBU with 3 or less HLA compatibility degree in A, B, DRB1 loci will be used.

Biological: ASD CB-MNC infusion low level HLA compatibility.

Group 2 High HLA compatibility

EXPERIMENTAL

ASD CB-MNC infusion from different donors and standard therapy. CBU with 3 or more HLA compatibility degree in A, B, DRB1 loci will be used.

Biological: ASD CB-MNC infusion high level HLA compatibility.

Group 3 Control

OTHER

Patients with standard therapy as a control group.

Other: Standard therapy.

Interventions

ASD CB-MNC infusion low level HLA compatibility. BIOLOGICAL

CB-MNC infusion from different donors. One dose consists of 2-15х10\^7 cells per 1 kg of patient weight for each infusion. The protocol includes 2 infusions with an interval of 6-x months. HLA compatibility of the cord blood is 3 or fewer by A, B, DRB1 loci.

Group 1 Low HLA compatibility

ASD CB-MNC infusion high level HLA compatibility. BIOLOGICAL

CB-MNC infusion from different donors. One dose consists of 2-15х10\^7 cells per 1 kg of patient weight for each infusion. The protocol includes 2 infusions with an interval of 6-x months. HLA compatibility of the cord blood is 4 or more by A, B, DRB1 loci.

Group 2 High HLA compatibility

Standard therapy. OTHER

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

Group 3 Control

Eligibility Criteria

• Age: 3 Years - 14 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may not qualify if:

• The patient's age under 4 years, after 14 years;
• The presence of the following diseases in the history: heart failure at the stage of decompensation, stroke in the history of less than 1 year ago, anemia and other blood diseases;
• Decompensation for chronic and endocrinological diseases;
• Acute viral and bacterial infections during the acute clinical phase of the disease;
• HIV infection, hepatitis B and C;
• Cancer, chemotherapy, and history of cancer;
• Tuberculosis;
• Severe form of intellectual disability as a concomitant disease (diagnosis can be ignored, according to the decision of the Medical Committee of the Center);
• Fragile X chromosome syndrome;

Sponsors & Collaborators

• State-Financed Health Facility "Samara Regional Medical Center Dinasty": lead
• INBIO, LLC: collaborator

Study Sites (1)

Medical Centre Dinasty

Samara, 443095, Russia

RECRUITING

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

Study Officials

• STANISLAV VOLCHKOV, MD, PhD

  Medical Centre Dinasty

  PRINCIPAL INVESTIGATOR

Central Study Contacts

STANISLAV VOLCHKOV, MD, PhD

CONTACT

+79608159408; bioen07@gmail.com

Olga Tyumina, M.D, PhD

CONTACT

centr123@bk.ru

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. In total, the study will involve 150 patients in three groups. The first and second groups will receive transfusions of umbilical cord blood hematopoietic cells, with varying degrees of selection for the genes of the HLA system. The third group will be the control group. In the control group, 50 patients with similar pathology will be examined, comparable in age, sex and degree of disorder of the type of "para-copies" against the background of standard therapy.

Study Record Dates

• First Submitted: September 17, 2019
• First Posted: September 23, 2019
• Study Start: August 10, 2019
• Primary Completion: December 26, 2023
• Study Completion: May 26, 2024
• Last Updated: January 18, 2023

Record last verified: 2023-01

Locations

RU (1)