Autologous Cord Blood Stem Cells for Autism

• 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: Sutter Health
• 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

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Autologous Cord Blood Stem Cells First/Placebo Second	Placebo First/Autologous Cord Blood Stem Cells Second
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

Baseline Characteristics

Arm/Group Title		Autologous Cord Blood Stem Cells First/Placebo Second	Placebo First/Autologous Cord Blood Stem Cells Second	Total
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
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.

Outcome Measures

1. Primary Outcome

Title	Change in Language (Total Standard Score, Range 40 - 160)
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

Outcome Measure Data

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
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)
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

Outcome Measure Data

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
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)
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

Outcome Measure Data

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
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)

Adverse Events

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
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%)
Serious Adverse Events
	All Participants
	Affected / at Risk (%)	# Events
Total	0/29 (0.00%)
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

Limitations and Caveats

[Not Specified]

More Information

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
• EMail: ChezM@sutterhealth.org

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Mauron A. [Autism, stem cells, and magical powder]. Rev Med Suisse. 2012 Sep 19;8(354):1795. No abstract available. French.

• Responsible Party: Michael Chez, MD, Sutter Health
• ClinicalTrials.gov Identifier: NCT01638819
• 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