Autologous Cord Blood Stem Cells for Autism
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).
|Autism||Biological: Autologous Cord Blood Stem Cells Biological: Placebo||Phase 2|
|Study Design:||Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
|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|
- Change in language [ Time Frame: Baseline and 6 months ]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.
- Improved Behavior/Learning [ Time Frame: Baseline and 6 months ]Change in the Vineland Adaptive Behavior Scales between baseline and six months after infusion of AUCB containing stem cells
- Change in Symptoms of Autism [ Time Frame: Baseline and 6 months ]
Change at baseline and 6 months in symptoms of Autism as measured by:
- Autism Diagnostic Observation Schedule (ADOS)
- Clinical Global Impression (CGI)
- Change in Serum Values [ Time Frame: Baseline and 6 months ]
Change in the following between baseline and six months after infusion of AUCB containing stem cells as measured by:
• Serum (TNF) alpha, Interleukin 1-alpha (IL-1α), interleukin 13( IL-13), Interleukin -1β, Interleukins 6, 10, 13
|Study Start Date:||August 2012|
|Study Completion Date:||December 2016|
|Primary Completion Date:||December 2015 (Final data collection date for primary outcome measure)|
|Experimental: Autologous Cord Blood Stem Cells||
Biological: Autologous Cord Blood Stem Cells
One infusion of 60 ml syringe of study product
Placebo Comparator: Placebo
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01638819
|United States, California|
|Sutter Pediatric Neurology|
|Sacramento, California, United States, 95816|
|Principal Investigator:||Michael Chez, MD||Sutter Health|