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Treatment of Children With Autistic Spectrum Disorder With Autologous Umbilical Cord Blood, a Pilot Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04243382
Recruitment Status : Not yet recruiting
First Posted : January 28, 2020
Last Update Posted : January 28, 2020
Sponsor:
Information provided by (Responsible Party):
Dr. Omer Bar-Yosef, Sheba Medical Center

Brief Summary:

This study is a phase II, prospective, double blind, placebo-controlled study of the efficacy of autologous umbilical cord blood infusion.

The study population will consist of 60 children ages 18 months to 12 years with ASD. The population will be randomly assigned to 2 groups, the study group be treated by cord blood in the beginning of the study and the control group by placebo product.

The study will consist of 4 stages Stage 1: initial assessment by physiotherapist and occupational therapist / treatment by cord blood or placebo / blood work before and after treatment Stage 2: at stage 1 + 6 months assessment by physiotherapist and occupational therapist / cross-over treatment by cord blood or placebo / blood work before and after treatment Stage 4: at stage 1 + 12 months assessment by physiotherapist and occupational therapist The primary outcome is improvement of social communication skills six months after treatment at stage 1


Condition or disease Intervention/treatment Phase
Autistic Spectrum Disorder Biological: Autologous umbilical cord blood Biological: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: The randomization will be done by external source and the assignment will disclosed to cord bank bank only. They will produce either a cord blood or placebo unit which will be completely covered. Each unit will have its own index number that will be documented by the research coordinator. Neither the researcher or the family will know the nature of the unit.
Primary Purpose: Treatment
Official Title: Treatment of Children With Autistic Spectrum Disorder With Autologous Umbilical Cord Blood, a Pilot Study
Estimated Study Start Date : January 2020
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: group 1
Autologous umbilical cord blood transfusion Single dose of an Autologous umbilical cord blood transfusion
Biological: Autologous umbilical cord blood
Single infusion of autologous umbilical cord blood cells

Experimental: group 2
The placebo product will consist of the standard ingredients of the acellular content of the UCB unit. It will consist of 20 ml Dextran (Plander 40.000 - 50g/500ml, solution for infusion) and 20 ml of human Albumin 5% (solution for infusion). The volume of placebo product will be 40 ml,
Biological: Placebo
Placebo




Primary Outcome Measures :
  1. Improvement of social communication skills [ Time Frame: 6 months ]
    Vineland Adaptive Behavior Scales—Second Edition (VINELAND-II)

  2. Improvement of social communication skills [ Time Frame: 6 months ]
    Pediatric Evaluation of Disability Inventory-Computer Adaptive Test-ASD


Secondary Outcome Measures :
  1. Improvement of social communication skills [ Time Frame: 6 months ]
    Theory of Mind Inventory - 2

  2. Functional assessment [ Time Frame: 6 months ]
    Adaptive Behavior Assessment System - Second edition (ABAS-2)



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Months to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age ≥ 1.5 years to ≤ 12 years (11 years, 364 days) at the time of visit 1
  • Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5 criteria
  • Fragile X testing performed and negative
  • Available and qualified umbilical cord blood unit with a minimum banked total nucleated cell dose of ≥ 2 x 10e7 cells/kg
  • Stable on current psychiatric medication regimen (dose and dosing schedule) for at least 2 months prior to infusion of study product
  • Normal absolute lymphocyte count (≥1500/uL)
  • Able to travel to Sheba Medical Center University three times (baseline, 6 and 12 months post-baseline), and parent/guardian is able to participate in interim surveys and interviews monthly
  • Parental consent

Exclusion Criteria

  • General:

    • Review of medical records indicates ASD diagnosis not likely
    • Known diagnosis of any of the following coexisting psychiatric conditions: depression, bipolar disorder, schizophrenia, obsessive compulsive disorder
    • Screening data suggests that participant would not be able to comply with the requirements of the study procedures, including study outcome measures, as assessed by the study team
    • Family is unwilling or unable to commit to participation in all study-related assessments, including follow up for approximately 12 months
  • Genetic:

    • Records indicate that child has a known genetic syndrome such as (but not limited to) Fragile X syndrome, neurofibromatosis, Rett syndrome, tuberous sclerosis, PTEN mutation, cystic fibrosis, muscular dystrophy
    • Known pathogenic copy number variation (CNV) associated with ASD (e.g., 16p11.2, 15q13.2, 2q13.3)
  • Infectious:

    • Known active CNS infection
    • Evidence of uncontrolled infection based on records or clinical assessment
    • HIV positivity
  • 4 Medical:

    • Known metabolic disorder
    • Known mitochondrial dysfunction
    • History of unstable epilepsy or uncontrolled seizure disorder, Lennox Gastaut syndrome, Dravet syndrome, or other similar epileptic encephalopathy
    • Concurrent genetic or acquired disease or comorbidity(ies) that could require a future stem cell transplant
    • Significant sensory (e.g., blindness, deafness, uncorrected hearing impairment) or motor (e.g., cerebral palsy) impairment
    • Evidence of clinically relevant physical dysmorphology indicative of a genetic syndrome as assessed by the PIs or other investigators, including a medical geneticist and psychiatrists trained in identifying dysmporphic features associated with neurodevelopmental conditions.
  • Current/Prior Therapy:

    • History of prior cell therapy
    • Current or prior use of IVIG or other anti-inflammatory medications with the exception of NSAIDs
    • No systemic steroid therapy that has lasted >2 weeks, and no systemic steroids within 3 months prior to enrollment. Topical and inhaled steroids are permitted.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04243382


Contacts
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Contact: Omer Bar-Yosef, MD.PHD 972-35302895 Omer.BarYosef@sheba.health.gov.il

Sponsors and Collaborators
Sheba Medical Center
Investigators
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Principal Investigator: Omer Bar-Yosef, MD.PHD Sheba Medical Center
Publications:
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Responsible Party: Dr. Omer Bar-Yosef, M.D.-Ph.D. Peadiatric Neurology and Child Development. The Edmond and Lily Safra Children's Hospital, Sheba Medical Center
ClinicalTrials.gov Identifier: NCT04243382    
Other Study ID Numbers: SHEBA-18-5105-OBY-CTIL
First Posted: January 28, 2020    Key Record Dates
Last Update Posted: January 28, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Yes data set of the baseline of the children, the quantity of cord blood transfusion they received and the clinical followup information.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Time Frame: within 2 years from the end of data collection

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Dr. Omer Bar-Yosef, Sheba Medical Center:
Autistic Spectrum Disorder, autologous umblical cord blood
Additional relevant MeSH terms:
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Disease
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Pathologic Processes
Neurodevelopmental Disorders
Mental Disorders