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

NCT ID: NCT04243382

Last Updated: 2021-01-20

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 60 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-02-27
• Study Completion Date: 2024-12-31

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

group 1

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

Group Type: EXPERIMENTAL

Autologous umbilical cord blood

Intervention Type: BIOLOGICAL

Single infusion of autologous umbilical cord blood cells

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,

Group Type: EXPERIMENTAL

Placebo

Intervention Type: BIOLOGICAL

Placebo

Interventions

Autologous umbilical cord blood

Single infusion of autologous umbilical cord blood cells

Intervention Type: BIOLOGICAL

Placebo

Placebo

Intervention Type: BIOLOGICAL

Eligibility 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.

• Minimum Eligible Age: 18 Months
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sheba Medical Center

OTHER_GOV

Sponsor Role: lead

Responsible Party

Dr. Omer Bar-Yosef

M.D.-Ph.D. Peadiatric Neurology and Child Development. The Edmond and Lily Safra Children's Hospital

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Omer Bar-Yosef, MD.PHD

Role: PRINCIPAL_INVESTIGATOR

Sheba Medical Center

Locations

Chaim Seba Medical Center

Ramat Gan, , Israel

Site Status: RECRUITING

Countries

Israel

Central Contacts

Omer Bar-Yosef, MD.PHD

Role: CONTACT

Omer.BarYosef@sheba.health.gov.il

972-35302895

Facility Contacts

Omer Bar-Yosef

Role: primary

References

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.

Reference Type: BACKGROUND

PMID: 24074734 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 27031587 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 28870209 (View on PubMed)

Other Identifiers

SHEBA-18-5105-OBY-CTIL

Identifier Type: -

Identifier Source: org_study_id