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Status unknownNCT04243382Updated Jan 20, 2021

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

A Phase 2 interventional study of Autologous umbilical cord blood and Placebo in Autistic Spectrum Disorder, sponsored by Sheba Medical Center. Status unknown at 1 site in Israel. Open to participants aged 18 Months to 12 Years. Per ClinicalTrials.gov, last updated 2021-01-20.

Sponsored by Sheba Medical Center · Phase 2, Interventional, and Treatment

The sponsor has not verified this record recently (last verified Jan 2021), so the status shown — last known as Recruiting — may be out of date.
Phase
Phase 2
Study type
Interventional
Enrollment
60
Allocation
Randomized
Ages
18 Months to 12 Years
Sex
All
01

Study 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

02

Conditions studied

  • Autistic Spectrum Disorder

Keywords

  • Autistic Spectrum Disorder, autologous umblical cord blood
03

In context

Autistic Disorder

1,344 studies on the registry are indexed under Autistic Disorder; 334 are open to participants now.

This study's planned enrollment of 60 is above the median of 45 across 1,044 interventional studies indexed under Autistic Disorder.

Browse Autistic Disorder studies →

Lead sponsor

Sheba Medical Center is the lead sponsor of 660 studies on the registry; 63 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Months to 12 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
60 participants (estimated)

Study arms

  • Experimental
    group 1

    Autologous umbilical cord blood transfusion Single dose of an Autologous umbilical cord blood transfusion

    Biological: Autologous umbilical cord blood

  • 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

Interventions

  • BiologicalAutologous umbilical cord blood

    Single infusion of autologous umbilical cord blood cells

  • BiologicalPlacebo

    Placebo

06

What researchers measure

Primary outcomes

  1. Improvement of social communication skills

    Vineland Adaptive Behavior Scales-Second Edition (VINELAND-II)

    Time frame: 6 months

  2. Improvement of social communication skills

    Pediatric Evaluation of Disability Inventory-Computer Adaptive Test-ASD

    Time frame: 6 months

Secondary outcomes

  1. Improvement of social communication skills

    Theory of Mind Inventory - 2

    Time frame: 6 months

  2. Functional assessment

    Adaptive Behavior Assessment System - Second edition (ABAS-2)

    Time frame: 6 months

07

Study locations

1 of 1 sites recruiting
  • Chaim Seba Medical Center
    Ramat Gan, Israel
    • Omer Bar-Yosef · Contact
    Recruiting
08

References and documents

Publications

  • Lai MC, Lombardo MV, Baron-Cohen S. Autism. Lancet. 2014 Mar 8;383(9920):896-910. doi: 10.1016/S0140-6736(13)61539-1. Epub 2013 Sep 26. PubMed 24074734 ↗
  • Christensen DL, Baio J, Van Naarden Braun K, Bilder D, Charles J, Constantino JN, Daniels J, Durkin MS, Fitzgerald RT, Kurzius-Spencer M, Lee LC, Pettygrove S, Robinson C, Schulz E, Wells C, Wingate MS, Zahorodny W, Yeargin-Allsopp M; Centers for Disease Control and Prevention (CDC). Prevalence and Characteristics of Autism Spectrum Disorder Among Children Aged 8 Years--Autism and Developmental Disabilities Monitoring Network, 11 Sites, United States, 2012. MMWR Surveill Summ. 2016 Apr 1;65(3):1-23. doi: 10.15585/mmwr.ss6503a1. Erratum In: MMWR Morb Mortal Wkly Rep. 2016 Apr 22;65(15):404. doi: 10.15585/mmwr.mm6515a7. MMWR Morb Mortal Wkly Rep. 2018 Nov 16;67(45):1279. doi: 10.15585/mmwr.mm6745a7. PubMed 27031587 ↗
  • Prata J, Santos SG, Almeida MI, Coelho R, Barbosa MA. Bridging Autism Spectrum Disorders and Schizophrenia through inflammation and biomarkers - pre-clinical and clinical investigations. J Neuroinflammation. 2017 Sep 4;14(1):179. doi: 10.1186/s12974-017-0938-y. PubMed 28870209 ↗

Individual participant data

Plan to share: Yes — Yes data set of the baseline of the children, the quantity of cord blood transfusion they received and the clinical followup information.

Supporting information: Study protocol, Sap, Icf, Csr

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jan 20, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT04243382
Lead sponsor
Sheba Medical Center
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) — Principal investigator
First posted
Jan 28, 2020
Start date
Feb 27, 2020
Primary completion
Dec 2024 (estimated)
Completion
Dec 2024 (estimated)
Last update
Jan 20, 2021

Study contacts

Omer Bar-Yosef, MD.PHD
Contact
Omer.BarYosef@sheba.health.gov.il
972-35302895
Omer Bar-Yosef, MD.PHD
principal investigator · Sheba Medical Center

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in Jan 2021. You cannot join it, but the record below documents what was studied.

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