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History of Changes for Study: NCT01638819
Autologous Cord Blood Stem Cells for Autism
Latest version (submitted August 14, 2018) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 July 9, 2012 None (earliest Version on record)
2 July 16, 2012 Recruitment Status, Study Status and Contacts/Locations
3 August 20, 2012 Recruitment Status, Study Status and Contacts/Locations
4 July 30, 2013 Study Status, Outcome Measures and Eligibility
5 November 20, 2014 Recruitment Status, Study Status and Contacts/Locations
6 May 18, 2015 Study Status
7 November 23, 2015 Study Status
8 March 1, 2016 Study Status and Study Design
9 November 4, 2016 Study Status
10 May 10, 2017 Recruitment Status, Study Status
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Results Submission Events
11 August 14, 2018 Study Status, Outcome Measures, Results and Study Design
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Study NCT01638819
Submitted Date:  July 9, 2012 (v1)

Open or close this module Study Identification
Unique Protocol ID: CB2011Chez
Brief Title: Autologous Cord Blood Stem Cells for Autism
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
Secondary IDs:
Open or close this module Study Status
Record Verification: July 2012
Overall Status: Recruiting
Study Start: July 2012
Primary Completion: July 2013 [Anticipated]
Study Completion: July 2013 [Anticipated]
First Submitted: June 26, 2012
First Submitted that
Met QC Criteria:
July 9, 2012
First Posted: July 12, 2012 [Estimate]
Last Update Submitted that
Met QC Criteria:
July 9, 2012
Last Update Posted: July 12, 2012 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: Sutter Health
Responsible Party: Principal Investigator
Investigator: Michael Chez, MD
Official Title: Principal Investigator
Affiliation: Sutter Health
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring: Yes
Open or close this module Study Description
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.

Open or close this module Conditions
Conditions: Autism
Keywords:
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 2
Interventional Study Model: Crossover Assignment
Number of Arms: 2
Masking: Triple (Participant, Care Provider, Investigator)
Allocation: Randomized
Enrollment: 30 [Anticipated]
Open or close this module Arms and Interventions
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
Open or close this module Outcome Measures
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. 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
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

Open or close this module Eligibility
Minimum Age: 2 Years
Maximum Age: 7 Years
Sex: All
Gender Based:
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)
Open or close this module Contacts/Locations
Central Contact Person: Tammy Donnel, MS
Telephone: (916) 453-5994
Email: DonnelT@sutterhealth.org
Central Contact Backup: Carol Parise, PhD
Telephone: (916) 733-4413
Email: PariseC@sutterhealth.org
Study Officials: Michael Chez, MD
Principal Investigator
Sutter Health
Locations: United States, California
Sutter Pediatric Neurology
[Recruiting]
Sacramento, California, United States, 95816
Contact:Principal Investigator: Michael Chez, MD
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Links: Description: Sutter Institute for Medical Research
Available IPD/Information:

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