Clinical trial NCT01638819: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autism
Lead sponsor: Sutter Health
Sponsor class: OTHER
First posted: 2012-07-12
Enrollment: 30
Sex: ALL
Minimum age (years): 2
Maximum age (years): 7
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in Language (Total Standard Score, Range 40 - 160)
timeFrame: Baseline and 6 months
Secondary outcomes:
measure: Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)
timeFrame: Baseline and 6 months
measure: Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest)
timeFrame: Baseline and 6 months
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: Sutter Pediatric Neurology
state: California
Study officials:
affiliation: Sutter Health
name: Michael Chez, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Mauron A. [Autism, stem cells, and magical powder]. Rev Med Suisse. 2012 Sep 19;8(354):1795. No abstract available. French.