Clinical trial NCT04243382: Treatment of Children With Autistic Spectrum Disorder With Autologous Umbilical Cord Blood, a Pilot Study
Official title: Treatment of Children With Autistic Spectrum Disorder With Autologous Umbilical Cord Blood, a Pilot Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Autistic Spectrum Disorder
Keywords: Autistic Spectrum Disorder, autologous umblical cord blood
Lead sponsor: Sheba Medical Center
Sponsor class: OTHER_GOV
First posted: 2020-01-28
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


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.

Primary outcomes:
measure: Improvement of social communication skills
timeFrame: 6 months
measure: Improvement of social communication skills
timeFrame: 6 months
Secondary outcomes:
measure: Improvement of social communication skills
timeFrame: 6 months
measure: Functional assessment
timeFrame: 6 months
Other outcomes:
Locations:
city: Ramat Gan
country: Israel
facility: Chaim Seba Medical Center
Study officials:
affiliation: Sheba Medical Center
name: Omer Bar-Yosef, MD.PHD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.
citation: 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.