This site became the new on June 19th. Learn more.
Show more Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more... Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more... Menu
Give us feedback

Autologous Cord Blood Stem Cells for Autism

This study has been completed.
Information provided by (Responsible Party):
Michael Chez, MD, Sutter Health Identifier:
First received: June 26, 2012
Last updated: May 10, 2017
Last verified: May 2017

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 Intervention Phase
Autism Biological: Autologous Cord Blood Stem Cells Biological: Placebo Phase 2

Study Type: Interventional
Study Design: 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

Further study details as provided by Michael Chez, MD, Sutter Health:

Primary Outcome Measures:
  • 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:
  • 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

  • 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)

  • 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

Enrollment: 29
Study Start Date: August 2012
Study Completion Date: December 2016
Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.


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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01638819

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

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
Study First Received: June 26, 2012
Last Updated: May 10, 2017

Additional relevant MeSH terms:
Autistic Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders processed this record on September 21, 2017