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Autistic Spectrum DisorderPhase 2NCT04243382

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

A Phase 2 study testing Autologous umbilical cord blood in people with Autistic Spectrum Disorder, run by Sheba Medical Center at 1 site in Israel.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 months to 12 years
Conditions the study is for
Autistic Spectrum Disorder
What is being tested?
Autologous umbilical cord blood
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
60 participants
Where
1 siteIsrael
Study dates
27 Feb 2020to Dec 2024
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 months to 12 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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

Who cannot join

  • 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:
Show all 23Show fewer
  • 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.

Trial locations and contacts

1 site in 1 country.

Omer Bar-Yosef, MD.PHD contact

972-35302895

Omer.BarYosef@sheba.health.gov.il

Israel1 site, 1 recruiting

  • Chaim Seba Medical Center

    Ramat Gan

    Omer Bar-Yosef

    Recruiting

Other trials in Israel

About this study

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

Being tested
Autologous umbilical cord blood, Placebo
Keywords
Autistic Spectrum Disorder, autologous umblical cord blood
Medical subject headings
Autism Spectrum Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Autologous umbilical cord bloodBiological

    Single infusion of autologous umbilical cord blood cells

  • PlaceboBiological

    Placebo

Study groups in NCT04243382
Study groupRoleWhat they receive
group 1ExperimentalAutologous umbilical cord blood transfusion Single dose of an Autologous umbilical cord blood transfusion
Receives: Biological: Autologous umbilical cord blood
group 2ExperimentalThe 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,
Receives: Biological: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Improvement of social communication skills

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

    Measured: 6 months

  2. Improvement of social communication skills

    Pediatric Evaluation of Disability Inventory-Computer Adaptive Test-ASD

    Measured: 6 months

Secondary outcomes (2)

  1. Improvement of social communication skills

    Theory of Mind Inventory - 2

    Measured: 6 months

  2. Functional assessment

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

    Measured: 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Jan 2020
  2. Study start27 Feb 2020
  3. Primary completionDec 2024 (estimated)
  4. Study completionDec 2024 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
60 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04243382
Sponsor's protocol ID
SHEBA-18-5105-OBY-CTIL
Status last verified
Jan 2021
First submitted
15 Jan 2020
Last updated
20 Jan 2021
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Yes data set of the baseline of the children, the quantity of cord blood transfusion they received and the clinical followup information.

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.

    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

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Autologous umbilical cord blood in people with Autistic Spectrum Disorder, run by Sheba Medical Center at 1 site in Israel.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 20 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Omer Bar-Yosef, MD.PHD (972-35302895, Omer.BarYosef@sheba.health.gov.il), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Improvement of social communication skills (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04243382, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2021.

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