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Allogenic Cord Blood Transfusion in Patients With Autism

Primary Purpose

Autism Spectrum Disorder, Autism

Status
Completed
Phase
Phase 1
Locations
Russian Federation
Study Type
Interventional
Intervention
ASD CB-MNC injection.
Standard therapy.
Sponsored by
State-Financed Health Facility "Samara Regional Medical Center Dinasty"
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Autism Spectrum Disorder focused on measuring Autism, ASD, Cord blood

Eligibility Criteria

3 Years - 15 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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

Exclusion Criteria:

  • 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.

Sites / Locations

  • Medical Centre Dinasty

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Other

Arm Label

ASD CB-MNC injection.

Standard therapy.

Arm Description

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

Patients with standard therapy as control group.

Outcomes

Primary Outcome Measures

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.
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.

Secondary Outcome Measures

Cytokine Analysis.
IL1b, IL6, TNF-alpha, IL8, y-IFN measure in patient peripheral blood
Immune Status as Determined by Percentage of Peripheral Blood Cells
Peripheral blood flow cytometry cell count for CD3; CD4; CD8; CD19; CD16-56 (%)
Immune Status as Determined by Absolute Cell Count of Peripheral Blood Cells
Peripheral blood flow cytometry cell count for CD3; CD4; CD8; CD19; CD16-56 (absolute v.)

Full Information

First Posted
December 21, 2018
Last Updated
October 28, 2020
Sponsor
State-Financed Health Facility "Samara Regional Medical Center Dinasty"
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1. Study Identification

Unique Protocol Identification Number
NCT03786744
Brief Title
Allogenic Cord Blood Transfusion in Patients With Autism
Official Title
Efficiency Evaluation of Allogenic Umbilical Cord Blood (UCB) Transfusion in Patients With Autism
Study Type
Interventional

2. Study Status

Record Verification Date
October 2020
Overall Recruitment Status
Completed
Study Start Date
October 12, 2018 (Actual)
Primary Completion Date
October 12, 2019 (Actual)
Study Completion Date
December 12, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
State-Financed Health Facility "Samara Regional Medical Center Dinasty"

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.
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 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. The Our study suggest that infusion of cord blood mononuclear cells will affect Autism.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autism Spectrum Disorder, Autism
Keywords
Autism, ASD, Cord blood

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Model Description
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).
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ASD CB-MNC injection.
Arm Type
Experimental
Arm Description
ASD CB-MNC injection from different donors and standard therapy.
Arm Title
Standard therapy.
Arm Type
Other
Arm Description
Patients with standard therapy as control group.
Intervention Type
Biological
Intervention Name(s)
ASD CB-MNC injection.
Intervention Description
CB-MNC injection from different donors. One dose consist 20-50 mil MNC/kg.The protocol include 3 injection at monthly intervals.
Intervention Type
Other
Intervention Name(s)
Standard therapy.
Intervention Description
The standard therapy can include drugs, special psychology training etc.
Primary Outcome Measure Information:
Title
A Number of Participants With Non-serious and Serious Adverse Events 24 Hrs Post-transfusion
Description
Safety assessment. The adverse events rate will be assessed in all patients.
Time Frame
24hrs post transfusion
Title
Autism Treatment Evaluation Checklist (ATEC).
Description
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.
Time Frame
score at baseline, 1, 2, 6, 12 months
Secondary Outcome Measure Information:
Title
Cytokine Analysis.
Description
IL1b, IL6, TNF-alpha, IL8, y-IFN measure in patient peripheral blood
Time Frame
score at baseline, 1, 2, 6 months
Title
Immune Status as Determined by Percentage of Peripheral Blood Cells
Description
Peripheral blood flow cytometry cell count for CD3; CD4; CD8; CD19; CD16-56 (%)
Time Frame
score at baseline, 6 month
Title
Immune Status as Determined by Absolute Cell Count of Peripheral Blood Cells
Description
Peripheral blood flow cytometry cell count for CD3; CD4; CD8; CD19; CD16-56 (absolute v.)
Time Frame
score at baseline, 6 month

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
15 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Confirmed diagnosis: autistic type disorder (ASD). Systemic speech underdevelopment The presence of attention deficit hyperactivity disorder as a comorbid state Cognitive impairment Exclusion Criteria: 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
STANISLAV VOLCHKOV, MD, PhD
Organizational Affiliation
Medical Centre Dinasty
Official's Role
Principal Investigator
Facility Information:
Facility Name
Medical Centre Dinasty
City
Samara
ZIP/Postal Code
443095
Country
Russian Federation

12. IPD Sharing Statement

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Allogenic Cord Blood Transfusion in Patients With Autism

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