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

NCT04243382 | Unknown Phase 2

• 1 other identifier: SHEBA-18-5105-OBY-CTIL
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

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

Conditions

Autistic Spectrum Disorder

Keywords

Autistic Spectrum Disorder, autologous umblical cord blood

Outcome Measures

Primary Outcomes (2)

• Improvement of social communication skills

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

  6 months

• Improvement of social communication skills

  Pediatric Evaluation of Disability Inventory-Computer Adaptive Test-ASD

  6 months

Secondary Outcomes (2)

• Improvement of social communication skills

  6 months

• Functional assessment

  6 months

Study Arms (2)

group 1

EXPERIMENTAL

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

Biological: Autologous umbilical cord blood

group 2

EXPERIMENTAL

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

Autologous umbilical cord blood BIOLOGICAL

Single infusion of autologous umbilical cord blood cells

group 1

Placebo BIOLOGICAL

Placebo

group 2

Eligibility Criteria

• Age: 18 Months - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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
• Medical:
• Known metabolic disorder
• Known mitochondrial dysfunction

Sponsors & Collaborators

• Sheba Medical Center: lead

Study Sites (1)

Chaim Seba Medical Center

Ramat Gan, Israel

RECRUITING

Related Publications (3)

• 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.

  PMID: 24074734 BACKGROUND

• 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.

  PMID: 27031587 BACKGROUND

• 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.

  PMID: 28870209 BACKGROUND

MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Omer Bar-Yosef, MD.PHD

  Sheba Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Omer Bar-Yosef, MD.PHD

CONTACT

972-35302895; Omer.BarYosef@sheba.health.gov.il

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: M.D.-Ph.D. Peadiatric Neurology and Child Development. The Edmond and Lily Safra Children's Hospital

Study Record Dates

• First Submitted: January 15, 2020
• First Posted: January 28, 2020
• Study Start: February 27, 2020
• Primary Completion: December 1, 2024
• Study Completion: December 1, 2024
• Last Updated: January 20, 2021

Record last verified: 2021-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: within 2 years from the end of data collection

Locations

IL (1)