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

NCT ID: NCT04099381

Last Updated: 2023-01-18

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

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Total Enrollment

150 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-08-10

Study Completion Date

2024-05-26

Brief Summary

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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.

Detailed Description

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Autism Spectrum Disorders (ASD) are heterogeneous neuro-developed disorders. Autism is the most common disorder of the diseases characterized by dysfunctions in response to social interaction and communication, as well as the presence of repetitive and stereotyped behaviors. Recent reports of a sharp increase in the number of children with autism. The exact etiology of autism remains unclear. Compliance, the definition of effective treatments for autism is particularly difficult.

Although it has been understood, it can be a question of immune dysregulation. Examination of the inflammatory cytokines, dysfunction of the immune system and the immune system. 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. Our study suggest that infusion of cord blood mononuclear cells will affect Autism.

This protocol was developed on the basis of the results of the previously approved protocol of the center NCT03786744 (Transfusion of allogeneic cord blood samples in patients with autism spectrum disorders), which showed high efficiency in the rehabilitation of patients. The present protocol is intended for revealing the dependence of the clinical effect on the degree of tissue compatibility of umbilical cord blood samples and the recipient.

Conditions

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Autism Spectrum Disorder Autism

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

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.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Group 1 Low HLA compatibility

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.

Group Type EXPERIMENTAL

ASD CB-MNC infusion low level HLA compatibility.

Intervention Type 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 2 High HLA compatibility

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.

Group Type EXPERIMENTAL

ASD CB-MNC infusion high level HLA compatibility.

Intervention Type 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 3 Control

Patients with standard therapy as a control group.

Group Type OTHER

Standard therapy.

Intervention Type OTHER

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

Interventions

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ASD CB-MNC infusion low level HLA compatibility.

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.

Intervention Type BIOLOGICAL

ASD CB-MNC infusion high level HLA compatibility.

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.

Intervention Type BIOLOGICAL

Standard therapy.

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

Intervention Type OTHER

Eligibility Criteria

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Exclusion Criteria

* 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;
* Epileptic seizures with or without medication in the last 6 months before inclusion in the protocol.
Minimum Eligible Age

3 Years

Maximum Eligible Age

14 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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INBIO, LLC

UNKNOWN

Sponsor Role collaborator

State-Financed Health Facility "Samara Regional Medical Center Dinasty"

OTHER

Sponsor Role lead

Responsible Party

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Volchkov Stanislav

Deputy director, Quality assurance director

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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STANISLAV VOLCHKOV, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Medical Centre Dinasty

Locations

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Medical Centre Dinasty

Samara, , Russia

Site Status RECRUITING

Countries

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Russia

Central Contacts

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STANISLAV VOLCHKOV, MD, PhD

Role: CONTACT

+79608159408

Olga Tyumina, M.D, PhD

Role: CONTACT

Facility Contacts

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Stanislav Volchkov, MD, PhD

Role: primary

+79277811532

Olga Tyumina, MD, PhD

Role: backup

+78469564455

Other Identifiers

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ASD-HLA2019

Identifier Type: -

Identifier Source: org_study_id

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