Safety and Efficacy of the Transfusion of UCB in Patients With an ASD Depending on the Degree of HLA Compatibility. (ASD-HLA2019)

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

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Autism Spectrum Disorder; Autism

Treatments: Biological: ASD CB-MNC infusion high level HLA compatibility.; Other: Standard therapy.; Biological: ASD CB-MNC infusion low level HLA compatibility.

Study type: Interventional

Funder types: Other

Identifiers

NCT04099381

ASD-HLA2019

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 150 estimated patients

Sex: All

Ages: 3 to 14 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Patient selection criteria (indications for this type of treatment):

• Patient's age from 4 to 14 years;
• Diagnosis: autistic spectrum disorder;
• The severity of the disease on the ATEC scale of at least 16 points;
• The presence of a compatible allogeneic sample suitable for infusion;
• Parental consent (official guardians).

Patient exclusion criteria (contraindications for this type of treatment):

• 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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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150 participants in 3 patient groups

Group 1 Low HLA compatibility

Experimental group

Description:

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.

Treatment:

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

Group 2 High HLA compatibility

Experimental group

Description:

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.

Treatment:

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

Group 3 Control

Other group

Description:

Patients with standard therapy as a control group.

Treatment:

Other: Standard therapy.