Autologous Cord Blood Stem Cells for Autism

Sutter Health

Status and phase

Completed

Phase 2

Conditions

Autism

Treatments

Biological: Autologous Cord Blood Stem Cells

Biological: Placebo

Study type

Interventional

Funder types

Other

Identifiers

NCT01638819

CB2011Chez

Details and patient eligibility

About

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).

Full 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.

Enrollment

30 patients

Sex

All

Ages

2 to 7 years old

Volunteers

No Healthy Volunteers

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