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Autologous Cord Blood Stem Cells for Autism

This study is currently recruiting participants.
Verified August 2012 by Sutter Health
Sponsor:
Information provided by (Responsible Party):
Michael Chez, MD, Sutter Health
ClinicalTrials.gov Identifier:
NCT01638819
First received: June 26, 2012
Last updated: August 20, 2012
Last verified: August 2012
History of Changes
  Purpose

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
Endpoint Classification: Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:

MedlinePlus related topics: Autism
U.S. FDA Resources

Further study details as provided by Sutter Health:

Primary Outcome Measures:
  • Change in language [ Time Frame: Baseline and 6 months ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    Change in the Vineland Adaptive Behavior Scales between baseline and six months after infusion of AUCB containing stem cells

  • Improved Behavior [ Time Frame: Baseline and 6 months ] [ Designated as safety issue: No ]
    Change in Pervasive Developmental Disorders Behavior Index (PDDBI) between baseline and six months after infusion of AUCB containing stem cells

  • Change in Serum Values [ Time Frame: Baseline and 6 months ] [ Designated as safety issue: No ]

    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



Estimated Enrollment: 30
Study Start Date: August 2012
Estimated Study Completion Date: August 2013
Estimated Primary Completion Date: August 2013 (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
Saline
 Biological: Placebo
Saline

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.

  Eligibility

Ages Eligible for Study:   2 Years to 7 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 2 to 7 years of age
  • Diagnosis of Autistic Disorder as diagnosed by the 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, CD34+ count of less than 0.3% 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)
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01638819

Contacts
Contact: Heather Harris, MS 1-888-536-9826

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

Additional Information:
Sutter Institute for Medical Research  This link exits the ClinicalTrials.gov site

No publications provided by Sutter Health

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Michael Chez, MD, Principal Investigator, Sutter Health
ClinicalTrials.gov Identifier: NCT01638819     History of Changes
Other Study ID Numbers: CB2011Chez
Study First Received: June 26, 2012
Last Updated: August 20, 2012
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Autistic Disorder
Child Development Disorders, Pervasive
Mental Disorders Diagnosed in Childhood
Mental Disorders

ClinicalTrials.gov processed this record on May 16, 2013