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.
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
Change in language
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.
Baseline and 6 months
Michael Chez, MD
United States: Food and Drug Administration
|Sutter Pediatric Neurology||Sacramento, California 95816|