Autologous Cord Blood Stem Cells for Autism

NCT01638819 | Completed Phase 2 Results DMC

• 1 other identifier: CB2011Chez
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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).

Conditions

Autism

Outcome Measures

Primary Outcomes (1)

• Change in Language (Total Standard Score, Range 40 - 160)

  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.

  Baseline and 6 months

Secondary Outcomes (2)

• Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)

  Baseline and 6 months

• Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest)

  Baseline and 6 months

Study Arms (2)

Autologous Cord Blood Stem Cells

EXPERIMENTAL

Biological: Autologous Cord Blood Stem Cells

Placebo

PLACEBO COMPARATOR

Saline

Biological: Placebo

Interventions

Autologous Cord Blood Stem Cells BIOLOGICAL

One infusion of 60 ml syringe of study product

Autologous Cord Blood Stem Cells

Placebo BIOLOGICAL

Saline

Placebo

Eligibility Criteria

• Age: 2 Years - 7 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Sutter Health: lead

Study Sites (1)

Sutter Pediatric Neurology

Sacramento, California, 95816, United States

Location

Related Publications (1)

• Mauron A. [Autism, stem cells, and magical powder]. Rev Med Suisse. 2012 Sep 19;8(354):1795. No abstract available. French.

  PMID: 23097920 DERIVED

Related Links

• Sutter Institute for Medical Research

MeSH Terms

Conditions

Autistic Disorder

Condition Hierarchy (Ancestors)

Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Michael Chez, MD
• Organization: Sutter Health

ChezM@sutterhealth.org

(916) 773-8711

Study Officials

• Michael Chez, MD

  Sutter Health

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: June 26, 2012
• First Posted: July 12, 2012
• Study Start: August 1, 2012
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2016
• Last Updated: August 20, 2018
• Results First Posted: August 20, 2018

Record last verified: 2018-08

Locations

US (1)