Autologous Cord Blood Stem Cells for Autism

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01638819
Recruitment Status : Completed
First Posted : July 12, 2012
Last Update Posted : May 12, 2017
Information provided by (Responsible Party):
Michael Chez, MD, Sutter Health

Brief Summary:

Evaluate the efficacy of one infusion of stem cells from autologous umbilical cord blood in patients with autism over six months after infusion as measured by changes in expressive and receptive language.

Also demonstrate improved behavior, learning, and changes in Serum tumor necrosis factor alpha (TNF-α), tumor necrosis factor beta (TNF-β), interleukin 1-alpha (IL-1α), interleukin 1-beta (IL-1β), interleukin 6 (IL-6), interleukin 10 (IL-10), and interleukin 13 (IL-13).

Condition or disease Intervention/treatment Phase
Autism Biological: Autologous Cord Blood Stem Cells Biological: Placebo Phase 2

Detailed Description:

This is a single-center, randomized, placebo-controlled, crossover outpatient study with 15 subjects receiving one infusion of autologous umbilical cord blood (AUCB) containing a minimum of 10 million total nucleated cells per kilogram (TNC/kg) and 15 subjects receiving an infusion of placebo (saline). After the 24-week follow-up testing is conducted, the groups will crossover so that patients who initially received AUCB will receive placebo and patients who received placebo at baseline will receive the cord blood. Both groups will be tested again 24-weeks after infusion. The neuropsychologist, PI, staff from Cord Blood Registry (CBR), and parents will be blinded as to the infusion sequence.

The duration of participation for each study subject is approximately 55 weeks. This includes one screening visit over a period of approximately 6 weeks, one visit for baseline testing, one day for infusion of TNC (minimum 10 million/kg) or saline placebo followed by 24 weeks of follow-up. A second baseline visit is conducted at week-24 with the second infusion of TNC or saline placebo occurring 5-7 days after. Twenty-four additional weeks of follow-up occur after the second infusion.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 29 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Blinded, Placebo-controlled, Crossover Study to Assess the Efficacy of Stem Cells From Autologous Umbilical Cord Blood to Improve Language and Behavior in Children With Autism
Study Start Date : August 2012
Actual Primary Completion Date : December 2015
Actual Study Completion Date : December 2016

Arm Intervention/treatment
Experimental: Autologous Cord Blood Stem Cells Biological: Autologous Cord Blood Stem Cells
One infusion of 60 ml syringe of study product

Placebo Comparator: Placebo
Biological: Placebo

Primary Outcome Measures :
  1. Change in language [ Time Frame: Baseline and 6 months ]
    Change in language as measured by the Receptive One-Word Vocabulary Test (ROWVT) and Expressive One-Word Vocabulary Test (EOWVT) at baseline and six months following infusion of stem cells from AUCB or infusion of placebo.

Secondary Outcome Measures :
  1. Improved Behavior/Learning [ Time Frame: Baseline and 6 months ]
    Change in the Vineland Adaptive Behavior Scales between baseline and six months after infusion of AUCB containing stem cells

  2. Change in Symptoms of Autism [ Time Frame: Baseline and 6 months ]

    Change at baseline and 6 months in symptoms of Autism as measured by:

    • Autism Diagnostic Observation Schedule (ADOS)
    • Clinical Global Impression (CGI)

  3. Change in Serum Values [ Time Frame: Baseline and 6 months ]

    Change in the following between baseline and six months after infusion of AUCB containing stem cells as measured by:

    • Serum (TNF) alpha, Interleukin 1-alpha (IL-1α), interleukin 13( IL-13), Interleukin -1β, Interleukins 6, 10, 13

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 7 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age 2 to 7 years of age
  • Diagnosis of Autistic Disorder as diagnosed by the Diagnostic and Statistical Manual, 4th Edition, Text Revision (DSM-IV-TR) developmental delays, and ADOS
  • A sufficient quantity of autologous cord blood stored at Cord Blood Registry that was stored and processed using the Thermogenesis AutoXpress Platform
  • Stable on any current medications for at least 2 months prior to infusion of cord blood
  • Medical records indicating that patient does not have genetic conditions such as cerebral palsy, cystic fibrosis, muscular dystrophy, crohns disease, rheumatoid disease, fragile X, Retts Syndrome, Angelman Syndrome, tuberous sclerosis, epilepsy, or known genetic defects that overlap autism spectrum.
  • Results of an EEG within 12-months of baseline
  • English speaking

Exclusion Criteria:

  • CNS infection
  • Extreme prematurity (< 34 weeks gestation)
  • Severe Cognitive Disability IQ below 45 with autism
  • Clinical seizure activity within 6 months of baseline
  • Lennox Gastaut syndrome or infantile spasms
  • Dravet syndrome
  • HIV, renal or hepatic impairment
  • Prior hematological or malignant disease
  • Fever of 101 F within 2 weeks prior to infusion
  • Serious CNS infection or trauma
  • Unwilling to commit to follow-up
  • Mental illness including schizophrenia
  • Pervasive Developmental Disorder—Not Otherwise Specified
  • Asperger's Disorder
  • Cord blood unit is less than 85% viable, has a TNC of less than 10 million/kg, or sterility testing results are positive
  • Garlic allergy
  • Previous adverse reaction to Dimethyl Sulfoxide (DMSO)
  • Maternal medical records indicate communicable diseases including HIV, Hepatitis B or C, syphilis, cytomegalovirus (CMV)
  • Currently taking anti-inflammatory medications
  • History of asthma who may potentially require treatment with steroids
  • Inflammatory Disease
  • Renal/hepatic disease: serum Creatinine > 1.5 mg/dl and total Bilirubin > 1.5 mg/dl
  • Allergic to diphenhydramine (Benadryl)
  • Treatment with chelation therapy, hyperbaric oxygen therapy, pig worm therapy, or other alternative therapies the investigator deems clinically relevant

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01638819

United States, California
Sutter Pediatric Neurology
Sacramento, California, United States, 95816
Sponsors and Collaborators
Sutter Health
Principal Investigator: Michael Chez, MD Sutter Health

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Michael Chez, MD, Principal Investigator, Sutter Health Identifier: NCT01638819     History of Changes
Other Study ID Numbers: CB2011Chez
First Posted: July 12, 2012    Key Record Dates
Last Update Posted: May 12, 2017
Last Verified: May 2017

Additional relevant MeSH terms:
Autistic Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders