Allogenic Cord Blood Transfusion in Patients With Autism

Efficiency Evaluation of Allogenic Umbilical Cord Blood (UCB) Transfusion in Patients With Autism

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.

Study Overview

• Status: Completed
• Conditions: Autism Spectrum Disorder; Autism
• Intervention / Treatment: Other: Standard therapy.; Biological: ASD CB-MNC injection.

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.

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Russian Federation
  • Samara, Russian Federation, 443095
    • Medical Centre Dinasty

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 15 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NON_RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: ASD CB-MNC injection.; ASD CB-MNC injection from different donors and standard therapy.	Other: Standard therapy.; The standard therapy can include drugs, special psychology training etc.; Biological: ASD CB-MNC injection.; CB-MNC injection from different donors. One dose consist 20-50 mil MNC/kg.The protocol include 3 injection at monthly intervals.
OTHER: Standard therapy.; Patients with standard therapy as control group.	Other: Standard therapy.; The standard therapy can include drugs, special psychology training etc.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	24hrs post transfusion
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.	score at baseline, 1, 2, 6, 12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cytokine Analysis.	IL1b, IL6, TNF-alpha, IL8, y-IFN measure in patient peripheral blood	score at baseline, 1, 2, 6 months
Immune Status as Determined by Percentage of Peripheral Blood Cells	Peripheral blood flow cytometry cell count for CD3; CD4; CD8; CD19; CD16-56 (%)	score at baseline, 6 month
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.)	score at baseline, 6 month

Collaborators and Investigators

• Sponsor: State-Financed Health Facility "Samara Regional Medical Center Dinasty"

Study record dates

Study Major Dates

• Study Start (ACTUAL): October 12, 2018
• Primary Completion (ACTUAL): October 12, 2019
• Study Completion (ACTUAL): December 12, 2019

Study Registration Dates

• First Submitted: December 21, 2018
• First Submitted That Met QC Criteria: December 24, 2018
• First Posted (ACTUAL): December 26, 2018

Study Record Updates

• Last Update Posted (ACTUAL): November 19, 2020
• Last Update Submitted That Met QC Criteria: October 28, 2020
• Last Verified: October 1, 2020

More Information

Terms related to this study

• Keywords: Autism, ASD, Cord blood
• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Child Development Disorders, Pervasive; Autistic Disorder; Autism Spectrum Disorder
• Other Study ID Numbers: 12112018

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No