Clinical trial NCT04099381: Safety and Efficacy of the Transfusion of UCB in Patients With an ASD Depending on the Degree of HLA Compatibility.
Official title: Protocol for Evaluating the Safety and Efficacy of Transfusion of Umbilical Cord Blood in Patients With a Diagnosis of Autism Spectrum Disorder Depending on the Degree of Tissue Compatibility of the Donor and the Recipient
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Autism Spectrum Disorder
Autism
Keywords: Autism, ASD, Cord blood, HLA
Lead sponsor: State-Financed Health Facility "Samara Regional Medical Center Dinasty"
Sponsor class: OTHER
Collaborators: name: INBIO, LLC
First posted: 2019-09-23
Enrollment: 150
Sex: ALL
Minimum age (years): 3
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 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 purpose: TREATMENT

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.

Detailed 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.

Eligibility 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.

Primary outcomes:
measure: Number of participants with non-serious and serious adverse events.
timeFrame: baseline, change from baseline at 12 month
measure: The Childhood Autism Rating Scale (CARS2).
timeFrame: baseline, change from baseline at 6,12 month
measure: Autism Treatment Evaluation Checklist (ATEC).
timeFrame: baseline, change from baseline every month (total 12 evaluations)
Secondary outcomes:
measure: Evaluation of the survival of donor cells in the host body without immune responses
timeFrame: baseline, change from baseline at 6 month
measure: The correlation of the concentration of T-regulatory cells (CD4/CD25/FoxP3) in the sample of umbilical cord blood and the treatment effectiveness degree
timeFrame: At 1, at 6 months (At first and second infusion)
measure: The correlation of the concentration of T-regulatory cells (CD4/CD25/FoxP3) in the patient depending on the severity of the disease.
timeFrame: At 1, at 6 months (At first and second infusion)
measure: The correlation of the concentration of CD34+ cells in the cord blood sample and the treatment effectiveness degree
timeFrame: At 1, at 6 months (At first and second infusion)
Other outcomes:
Locations:
city: Samara
country: Russian Federation
facility: Medical Centre Dinasty
Study officials:
affiliation: Medical Centre Dinasty
name: STANISLAV VOLCHKOV, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: