Autologous Cord Blood Stem Cells for Autism
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|ClinicalTrials.gov Identifier: NCT01638819|
Recruitment Status : Completed
First Posted : July 12, 2012
Results First Posted : August 20, 2018
Last Update Posted : August 20, 2018
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).
|Condition or disease||Intervention/treatment||Phase|
|Autism||Biological: Autologous Cord Blood Stem Cells Biological: Placebo||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||30 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|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 Start Date :||August 2012|
|Actual Primary Completion Date :||December 2015|
|Actual Study Completion Date :||December 2016|
|Experimental: Autologous Cord Blood Stem Cells||
Biological: Autologous Cord Blood Stem Cells
One infusion of 60 ml syringe of study product
Placebo Comparator: Placebo
- Change in Language (Total Standard Score, Range 40 - 160) [ Time Frame: Baseline and 6 months ]
Change in language as measured by the Receptive One-Word Vocabulary Test (ROWVT-4) and Expressive One-Word Vocabulary Test (EOWVT-4) at baseline and six months following infusion of stem cells from AUCB or infusion of placebo.
The ROWPVT-4 and EOWPVT-4 were administered by a neuropsychologist and are norm-referenced assessments. The ROWPVT-4 tests an individual's ability to match a spoken word with an image of an object, action, or concept. The EOWPVT-4 tests an individual's ability to name, with one word, objects, actions, and concepts when presented with color illustrations. The tests target the ability to understand the meaning of words spoken and name what is depicted on a test plate without context.
Scores of 85-115 are considered to be within the average range of functioning.
- Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest) [ Time Frame: Baseline and 6 months ]
Change in the Vineland Adaptive Behavior and Socialization Scales (2nd edition) between baseline and six months after infusion of AUCB containing stem cells.
Vineland Adaptive Behavior and Socialization Scales consist of the following subparts: Daily Living Skills, Socialization, and Adaptive Behavior Composite (ABC). These are questionnaires completed by a parent or caregiver. Scores above 80 are classified using approximately the same ranges as IQ tests. Scores below 80 are categorized as borderline adaptive functioning (70-80); mildly deficient adaptive functioning (51-69); moderately deficient adaptive behavior (36-50); severely deficient adaptive behavior; (20-35); and markedly or profoundly deficient adaptive behavior (<20).
- Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest) [ Time Frame: Baseline and 6 months ]The Stanford Binet, version 5, was used to assess the brain function. It can assess the level of intelligence across several age spans and ability levels. The Stanford-Binet looks at intelligence in five areas. In this study 2 areas were looked at: Knowledge and Fluid Reasoning that were age and condition appropriate. Each sub-test has a mean of 10 and a standard deviation of 3. The standard deviation indicates how far above or below the norm the subject's score is. Scores of 7 to 13 are considered to be within the average range of functioning.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01638819
|United States, California|
|Sutter Pediatric Neurology|
|Sacramento, California, United States, 95816|
|Principal Investigator:||Michael Chez, MD||Sutter Health|