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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
Sponsor:
Information provided by (Responsible Party):
Michael Chez, MD, Sutter Health

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Autism
Interventions Biological: Autologous Cord Blood Stem Cells
Biological: Placebo
Enrollment 30
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Autologous Cord Blood Stem Cells First/Placebo Second Placebo First/Autologous Cord Blood Stem Cells Second
Hide Arm/Group Description

Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product

Placebo: Saline

Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product

Placebo: Saline

Period Title: Overall Study
Started 15 15
First Intervention (8 Weeks) 15 15
Second Intervention (24 Weeks) 14 15
Completed 14 15
Not Completed 1 0
Reason Not Completed
Lost to Follow-up             1             0
Arm/Group Title Autologous Cord Blood Stem Cells First/Placebo Second Placebo First/Autologous Cord Blood Stem Cells Second Total
Hide Arm/Group Description

Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product

Placebo: Saline

Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product

Placebo: Saline

Total of all reporting groups
Overall Number of Baseline Participants 14 15 29
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 14 participants 15 participants 29 participants
4.50
(2.42 to 6.80)
4.57
(3.00 to 6.30)
4.53
(2.42 to 6.80)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants 15 participants 29 participants
Female
3
  21.4%
1
   6.7%
4
  13.8%
Male
11
  78.6%
14
  93.3%
25
  86.2%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants 15 participants 29 participants
Hispanic or Latino
3
  21.4%
2
  13.3%
5
  17.2%
Not Hispanic or Latino
11
  78.6%
13
  86.7%
24
  82.8%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants 15 participants 29 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
2
  14.3%
2
  13.3%
4
  13.8%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
12
  85.7%
12
  80.0%
24
  82.8%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
1
   6.7%
1
   3.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 14 participants 15 participants 29 participants
14 15 29
Abnormal EEG at baseline  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants 15 participants 29 participants
5
  35.7%
4
  26.7%
9
  31.0%
Autism Diagnostic Observation Schedule (ADOS)   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 14 participants 15 participants 29 participants
7.93  (1.39) 6.60  (3.14) 7.24  (2.50)
[1]
Measure Description: The Autism Diagnostic Observation Schedule (ADOS) is a semi-structured assessment of communication, social interaction, and play (or imaginative use of materials) for individuals suspected of having autism or other pervasive developmental disorders. The total score ranges from 0 to 28 with 0 representing no abnormality present. A child is suspected to have Autism if the total ADOS score is greater than 7. The mean of the total ADOS scores is listed below.
Compartment infused   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Large compartment (80%) Number Analyzed 14 participants 15 participants 29 participants
9
  64.3%
12
  80.0%
21
  72.4%
Small compartment (20%) Number Analyzed 14 participants 15 participants 29 participants
5
  35.7%
3
  20.0%
8
  27.6%
[1]
Measure Description: Each subject's cord blood unit consists of two compartments. One contains 80% total volume and the other contains 20% total volume.
Weight  
Mean (Standard Deviation)
Unit of measure:  Kilograms (kg)
Number Analyzed 14 participants 15 participants 29 participants
19.79  (4.44) 18.67  (3.02) 19.21  (3.75)
TNC dose infused x 10^6/kg   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Number of cells infused x 10^6/kg
Number Analyzed 12 participants 10 participants 22 participants
14.49  (6.12) 18.17  (8.30) 16.16  (7.26)
[1]
Measure Description: The Total Nucleated Cell count or TNC is the total cell count after cord blood processing.
[2]
Measure Analysis Population Description: TNC data was not available for 2 cord blood first subjects and 5 placebo first subjects.
Viable total nucleated cell (vTNC) count infused   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Number of alive cells x 10^6/kg
Number Analyzed 12 participants 10 participants 22 participants
314.88  (236.52) 359.34  (163.22) 335.09  (203.06)
[1]
Measure Description: The viable Total Nucleated Cell count or vTNC is the count of alive cells.
[2]
Measure Analysis Population Description: TNC dose data was not available for 2 cord blood first subjects and 5 placebo first subjects.
Percent viability post-thaw, (flow cytometry 7-AAD)   [1] 
Mean (Standard Deviation)
Unit of measure:  Percent viable cells
Number Analyzed 14 participants 15 participants 29 participants
51.36  (11.36) 55.75  (11.38) 53.73  (11.37)
[1]
Measure Description: This measure determines the percent of stem cells that are alive.
Percent viable CD34 (cluster of differentiation) – post-thaw   [1] 
Mean (Standard Deviation)
Unit of measure:  Percent viable cells
Number Analyzed 14 participants 15 participants 29 participants
49.54  (36.66) 45.06  (37.46) 47.30  (36.41)
[1]
Measure Description: CD34, a protein found on the surface of stem cells, is the best known marker for blood-forming stem cells. It is used to predict the quality of white cell count yield.
1.Primary Outcome
Title Change in Language (Total Standard Score, Range 40 - 160)
Hide Description

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.

Time Frame Baseline and 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Please note that endpoints for only the first 24 weeks were recorded due to the fact that the placebo second group may not be a true placebo group (stem cell infusion does not necessarily wash out like a drug).
Arm/Group Title Autologous Cord Blood Stem Cells Placebo
Hide Arm/Group Description:
Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product
Placebo: Saline
Overall Number of Participants Analyzed 14 15
Mean (Standard Deviation)
Unit of Measure: units on a scale
ROWPVT-4 baseline 79.62  (25.85) 79.66  (21.21)
ROWPVT-4 6 months 80.45  (22.07) 80.59  (26.33)
EOWPVT-4 baseline 74.55  (23.23) 73.10  (20.88)
EOWPVT-4 6 months 74.03  (22.00) 75.38  (23.22)
2.Secondary Outcome
Title Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)
Hide Description

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).

Time Frame Baseline and 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Please note that endpoints for only the first 24 weeks were recorded due to the fact that the placebo second group may not be a true placebo group (stem cell infusion does not necessarily wash out like a drug).
Arm/Group Title Autologous Cord Blood Stem Cells Placebo
Hide Arm/Group Description:
Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product
Placebo: Saline
Overall Number of Participants Analyzed 14 15
Mean (Standard Deviation)
Unit of Measure: units on a scale
ABC - baseline 66.00  (12.20) 74.27  (16.00)
ABC - 6 month 67.57  (16.52) 75.00  (11.60)
Communication - baseline 66.00  (15.56) 79.60  (17.18)
Communication - 6 month 68.00  (18.82) 83.33  (15.46)
Motor - baseline 73.86  (13.17) 77.67  (13.57)
Motor - 6 month 69.71  (15.10) 76.67  (7.61)
Daily - baseline 68.14  (15.55) 77.13  (16.61)
Daily - 6 month 70.36  (17.73) 77.33  (15.30)
3.Secondary Outcome
Title Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest)
Hide Description 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.
Time Frame Baseline and 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Please note that endpoints for only the first 24 weeks were recorded due to the fact that the placebo second group may not be a true placebo group (stem cell infusion does not necessarily wash out like a drug).
Arm/Group Title Autologous Cord Blood Stem Cells Placebo
Hide Arm/Group Description:
Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product
Placebo: Saline
Overall Number of Participants Analyzed 14 15
Mean (Standard Deviation)
Unit of Measure: units on a scale
Fluid Reasoning - baseline 5.86  (5.46) 5.97  (5.28)
Fluid Reasoning - 6 months 6.79  (5.75) 6.41  (5.69)
Knowledge - baseline 3.97  (3.46) 4.45  (3.63)
Knowledge - 6 months 4.62  (3.75) 4.41  (3.70)
Time Frame Adverse event data were collected over the entire study time frame (12 months).
Adverse Event Reporting Description Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
 
Arm/Group Title All Participants
Hide Arm/Group Description Adverse events were measured for all participants throughout the study and not by arm since both arms received stem cell cord blood infusion.
All-Cause Mortality
All Participants
Affected / at Risk (%)
Total   0/29 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
All Participants
Affected / at Risk (%) # Events
Total   0/29 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
All Participants
Affected / at Risk (%) # Events
Total   28/29 (96.55%)    
Ear and labyrinth disorders   
Ear infection   3/29 (10.34%)  3
Gastrointestinal disorders   
Diarrhea   6/29 (20.69%)  9
Vomiting   11/29 (37.93%)  17
Loose stool   1/29 (3.45%)  1
General disorders   
Flu   3/29 (10.34%)  3
Fever   13/29 (44.83%)  15
Mononucleosis   1/29 (3.45%)  1
Infections and infestations   
Strep infection   3/29 (10.34%)  4
Upper respiratory infection   3/29 (10.34%)  3
Viral infection   1/29 (3.45%)  1
Scarlet fever   1/29 (3.45%)  1
Sinus infection   1/29 (3.45%)  1
Renal and urinary disorders   
Dark urine   9/29 (31.03%)  9
Respiratory, thoracic and mediastinal disorders   
Seasonal cold   12/29 (41.38%)  18
Cough   2/29 (6.90%)  2
Pneumonia   2/29 (6.90%)  2
Skin and subcutaneous tissue disorders   
Rash   4/29 (13.79%)  4
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Michael Chez, MD
Organization: Sutter Health
Phone: (916) 773-8711
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Michael Chez, MD, Sutter Health
ClinicalTrials.gov Identifier: NCT01638819     History of Changes
Other Study ID Numbers: CB2011Chez
First Submitted: June 26, 2012
First Posted: July 12, 2012
Results First Submitted: May 1, 2018
Results First Posted: August 20, 2018
Last Update Posted: August 20, 2018