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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

Phase 2
2 Years
7 Years
Open (Enrolling)

Thank you

Trial Information

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

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.

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

- 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)

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Outcome Measure:

Change in language

Outcome Description:

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.

Outcome Time Frame:

Baseline and 6 months

Safety Issue:


Principal Investigator

Michael Chez, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Sutter Health


United States: Food and Drug Administration

Study ID:




Start Date:

August 2012

Completion Date:

August 2013

Related Keywords:

  • Autism
  • Autistic Disorder



Sutter Pediatric Neurology Sacramento, California  95816