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Autologous Cord Blood Stem Cells for Autism

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ClinicalTrials.gov Identifier: NCT01638819
Recruitment Status : Completed
First Posted : July 12, 2012
Results First Posted : August 20, 2018
Last Update Posted : August 20, 2018
Sponsor:
Information provided by (Responsible Party):
Michael Chez, MD, Sutter Health

Tracking Information
First Submitted Date  ICMJE June 26, 2012
First Posted Date  ICMJE July 12, 2012
Results First Submitted Date  ICMJE May 1, 2018
Results First Posted Date  ICMJE August 20, 2018
Last Update Posted Date August 20, 2018
Study Start Date  ICMJE August 2012
Actual Primary Completion Date December 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 14, 2018)
Change in Language (Total Standard Score, Range 40 - 160) [ Time Frame: Baseline and 6 months ]
Change in language as measured by the Receptive One-Word Vocabulary Test (ROWVT-4) and Expressive One-Word Vocabulary Test (EOWVT-4) at baseline and six months following infusion of stem cells from AUCB or infusion of placebo. The ROWPVT-4 and EOWPVT-4 were administered by a neuropsychologist and are norm-referenced assessments. The ROWPVT-4 tests an individual's ability to match a spoken word with an image of an object, action, or concept. The EOWPVT-4 tests an individual's ability to name, with one word, objects, actions, and concepts when presented with color illustrations. The tests target the ability to understand the meaning of words spoken and name what is depicted on a test plate without context. Scores of 85-115 are considered to be within the average range of functioning.
Original Primary Outcome Measures  ICMJE
 (submitted: July 9, 2012)
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.
Change History Complete list of historical versions of study NCT01638819 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 14, 2018)
  • Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest) [ Time Frame: Baseline and 6 months ]
    Change in the Vineland Adaptive Behavior and Socialization Scales (2nd edition) between baseline and six months after infusion of AUCB containing stem cells. Vineland Adaptive Behavior and Socialization Scales consist of the following subparts: Daily Living Skills, Socialization, and Adaptive Behavior Composite (ABC). These are questionnaires completed by a parent or caregiver. Scores above 80 are classified using approximately the same ranges as IQ tests. Scores below 80 are categorized as borderline adaptive functioning (70-80); mildly deficient adaptive functioning (51-69); moderately deficient adaptive behavior (36-50); severely deficient adaptive behavior; (20-35); and markedly or profoundly deficient adaptive behavior (<20).
  • Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest) [ Time Frame: Baseline and 6 months ]
    The Stanford Binet, version 5, was used to assess the brain function. It can assess the level of intelligence across several age spans and ability levels. The Stanford-Binet looks at intelligence in five areas. In this study 2 areas were looked at: Knowledge and Fluid Reasoning that were age and condition appropriate. Each sub-test has a mean of 10 and a standard deviation of 3. The standard deviation indicates how far above or below the norm the subject's score is. Scores of 7 to 13 are considered to be within the average range of functioning.
Original Secondary Outcome Measures  ICMJE
 (submitted: July 9, 2012)
  • 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
  • Improved Behavior [ Time Frame: Baseline and 6 months ]
    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 ]
    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
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Autologous Cord Blood Stem Cells for Autism
Official Title  ICMJE 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
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).

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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Autism
Intervention  ICMJE
  • Biological: Autologous Cord Blood Stem Cells
    One infusion of 60 ml syringe of study product
  • Biological: Placebo
    Saline
Study Arms  ICMJE
  • Experimental: Autologous Cord Blood Stem Cells
    Intervention: Biological: Autologous Cord Blood Stem Cells
  • Placebo Comparator: Placebo
    Saline
    Intervention: Biological: Placebo
Publications * Mauron A. [Autism, stem cells, and magical powder]. Rev Med Suisse. 2012 Sep 19;8(354):1795. French.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 9, 2012)
30
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE December 2016
Actual Primary Completion Date December 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 7 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01638819
Other Study ID Numbers  ICMJE CB2011Chez
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Michael Chez, MD, Sutter Health
Study Sponsor  ICMJE Sutter Health
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Michael Chez, MD Sutter Health
PRS Account Sutter Health
Verification Date August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP