Trial Outcomes & Findings for Autologous Cord Blood Stem Cells for Autism (NCT NCT01638819)
NCT ID: NCT01638819
Last Updated: 2018-08-20
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
Baseline and 6 months
Results posted on
2018-08-20
Participant Flow
Participant milestones
| Measure |
Autologous Cord Blood Stem Cells First/Placebo Second
Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product
Placebo: Saline
|
Placebo First/Autologous Cord Blood Stem Cells Second
Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product
Placebo: Saline
|
|---|---|---|
|
Overall Study
STARTED
|
15
|
15
|
|
Overall Study
First Intervention (8 Weeks)
|
15
|
15
|
|
Overall Study
Second Intervention (24 Weeks)
|
14
|
15
|
|
Overall Study
COMPLETED
|
14
|
15
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Autologous Cord Blood Stem Cells First/Placebo Second
Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product
Placebo: Saline
|
Placebo First/Autologous Cord Blood Stem Cells Second
Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product
Placebo: Saline
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
Baseline Characteristics
TNC data was not available for 2 cord blood first subjects and 5 placebo first subjects.
Baseline characteristics by cohort
| Measure |
Autologous Cord Blood Stem Cells First/Placebo Second
n=14 Participants
Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product
Placebo: Saline
|
Placebo First/Autologous Cord Blood Stem Cells Second
n=15 Participants
Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product
Placebo: Saline
|
Total
n=29 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
4.50 years
n=14 Participants
|
4.57 years
n=15 Participants
|
4.53 years
n=29 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=14 Participants
|
1 Participants
n=15 Participants
|
4 Participants
n=29 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=14 Participants
|
14 Participants
n=15 Participants
|
25 Participants
n=29 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=14 Participants
|
2 Participants
n=15 Participants
|
5 Participants
n=29 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=14 Participants
|
13 Participants
n=15 Participants
|
24 Participants
n=29 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=14 Participants
|
0 Participants
n=15 Participants
|
0 Participants
n=29 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=14 Participants
|
0 Participants
n=15 Participants
|
0 Participants
n=29 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=14 Participants
|
2 Participants
n=15 Participants
|
4 Participants
n=29 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=14 Participants
|
0 Participants
n=15 Participants
|
0 Participants
n=29 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=14 Participants
|
0 Participants
n=15 Participants
|
0 Participants
n=29 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=14 Participants
|
12 Participants
n=15 Participants
|
24 Participants
n=29 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=14 Participants
|
0 Participants
n=15 Participants
|
0 Participants
n=29 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=14 Participants
|
1 Participants
n=15 Participants
|
1 Participants
n=29 Participants
|
|
Region of Enrollment
United States
|
14 participants
n=14 Participants
|
15 participants
n=15 Participants
|
29 participants
n=29 Participants
|
|
Abnormal EEG at baseline
|
5 Participants
n=14 Participants
|
4 Participants
n=15 Participants
|
9 Participants
n=29 Participants
|
|
Autism Diagnostic Observation Schedule (ADOS)
|
7.93 units on a scale
STANDARD_DEVIATION 1.39 • n=14 Participants
|
6.60 units on a scale
STANDARD_DEVIATION 3.14 • n=15 Participants
|
7.24 units on a scale
STANDARD_DEVIATION 2.50 • n=29 Participants
|
|
Compartment infused
Large compartment (80%)
|
9 Participants
n=14 Participants
|
12 Participants
n=15 Participants
|
21 Participants
n=29 Participants
|
|
Compartment infused
Small compartment (20%)
|
5 Participants
n=14 Participants
|
3 Participants
n=15 Participants
|
8 Participants
n=29 Participants
|
|
Weight
|
19.79 kilograms (kg)
STANDARD_DEVIATION 4.44 • n=14 Participants
|
18.67 kilograms (kg)
STANDARD_DEVIATION 3.02 • n=15 Participants
|
19.21 kilograms (kg)
STANDARD_DEVIATION 3.75 • n=29 Participants
|
|
TNC dose infused x 10^6/kg
|
14.49 Number of cells infused x 10^6/kg
STANDARD_DEVIATION 6.12 • n=12 Participants • TNC data was not available for 2 cord blood first subjects and 5 placebo first subjects.
|
18.17 Number of cells infused x 10^6/kg
STANDARD_DEVIATION 8.30 • n=10 Participants • TNC data was not available for 2 cord blood first subjects and 5 placebo first subjects.
|
16.16 Number of cells infused x 10^6/kg
STANDARD_DEVIATION 7.26 • n=22 Participants • TNC data was not available for 2 cord blood first subjects and 5 placebo first subjects.
|
|
Viable total nucleated cell (vTNC) count infused
|
314.88 number of alive cells x 10^6/kg
STANDARD_DEVIATION 236.52 • n=12 Participants • TNC dose data was not available for 2 cord blood first subjects and 5 placebo first subjects.
|
359.34 number of alive cells x 10^6/kg
STANDARD_DEVIATION 163.22 • n=10 Participants • TNC dose data was not available for 2 cord blood first subjects and 5 placebo first subjects.
|
335.09 number of alive cells x 10^6/kg
STANDARD_DEVIATION 203.06 • n=22 Participants • 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)
|
51.36 Percent viable cells
STANDARD_DEVIATION 11.36 • n=14 Participants
|
55.75 Percent viable cells
STANDARD_DEVIATION 11.38 • n=15 Participants
|
53.73 Percent viable cells
STANDARD_DEVIATION 11.37 • n=29 Participants
|
|
Percent viable CD34 (cluster of differentiation) - post-thaw
|
49.54 Percent viable cells
STANDARD_DEVIATION 36.66 • n=14 Participants
|
45.06 Percent viable cells
STANDARD_DEVIATION 37.46 • n=15 Participants
|
47.30 Percent viable cells
STANDARD_DEVIATION 36.41 • n=29 Participants
|
PRIMARY outcome
Timeframe: Baseline and 6 monthsPopulation: 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).
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.
Outcome measures
| Measure |
Autologous Cord Blood Stem Cells
n=14 Participants
Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product
|
Placebo
n=15 Participants
Placebo: Saline
|
|---|---|---|
|
Change in Language (Total Standard Score, Range 40 - 160)
ROWPVT-4 baseline
|
79.62 units on a scale
Standard Deviation 25.85
|
79.66 units on a scale
Standard Deviation 21.21
|
|
Change in Language (Total Standard Score, Range 40 - 160)
ROWPVT-4 6 months
|
80.45 units on a scale
Standard Deviation 22.07
|
80.59 units on a scale
Standard Deviation 26.33
|
|
Change in Language (Total Standard Score, Range 40 - 160)
EOWPVT-4 baseline
|
74.55 units on a scale
Standard Deviation 23.23
|
73.10 units on a scale
Standard Deviation 20.88
|
|
Change in Language (Total Standard Score, Range 40 - 160)
EOWPVT-4 6 months
|
74.03 units on a scale
Standard Deviation 22.00
|
75.38 units on a scale
Standard Deviation 23.22
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: 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).
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).
Outcome measures
| Measure |
Autologous Cord Blood Stem Cells
n=14 Participants
Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product
|
Placebo
n=15 Participants
Placebo: Saline
|
|---|---|---|
|
Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)
ABC - baseline
|
66.00 units on a scale
Standard Deviation 12.20
|
74.27 units on a scale
Standard Deviation 16.00
|
|
Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)
ABC - 6 month
|
67.57 units on a scale
Standard Deviation 16.52
|
75.00 units on a scale
Standard Deviation 11.60
|
|
Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)
Communication - baseline
|
66.00 units on a scale
Standard Deviation 15.56
|
79.60 units on a scale
Standard Deviation 17.18
|
|
Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)
Communication - 6 month
|
68.00 units on a scale
Standard Deviation 18.82
|
83.33 units on a scale
Standard Deviation 15.46
|
|
Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)
Motor - baseline
|
73.86 units on a scale
Standard Deviation 13.17
|
77.67 units on a scale
Standard Deviation 13.57
|
|
Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)
Motor - 6 month
|
69.71 units on a scale
Standard Deviation 15.10
|
76.67 units on a scale
Standard Deviation 7.61
|
|
Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)
Daily - baseline
|
68.14 units on a scale
Standard Deviation 15.55
|
77.13 units on a scale
Standard Deviation 16.61
|
|
Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)
Daily - 6 month
|
70.36 units on a scale
Standard Deviation 17.73
|
77.33 units on a scale
Standard Deviation 15.30
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: 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).
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.
Outcome measures
| Measure |
Autologous Cord Blood Stem Cells
n=14 Participants
Autologous Cord Blood Stem Cells: One infusion of 60 ml syringe of study product
|
Placebo
n=15 Participants
Placebo: Saline
|
|---|---|---|
|
Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest)
Fluid Reasoning - baseline
|
5.86 units on a scale
Standard Deviation 5.46
|
5.97 units on a scale
Standard Deviation 5.28
|
|
Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest)
Fluid Reasoning - 6 months
|
6.79 units on a scale
Standard Deviation 5.75
|
6.41 units on a scale
Standard Deviation 5.69
|
|
Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest)
Knowledge - baseline
|
3.97 units on a scale
Standard Deviation 3.46
|
4.45 units on a scale
Standard Deviation 3.63
|
|
Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest)
Knowledge - 6 months
|
4.62 units on a scale
Standard Deviation 3.75
|
4.41 units on a scale
Standard Deviation 3.70
|
Adverse Events
All Participants
Serious events: 0 serious events
Other events: 28 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
All Participants
n=29 participants at risk
Adverse events were measured for all participants throughout the study and not by arm since both arms received stem cell cord blood infusion.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Seasonal cold
|
41.4%
12/29 • Number of events 18 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.9%
2/29 • Number of events 2 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Renal and urinary disorders
Dark urine
|
31.0%
9/29 • Number of events 9 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
General disorders
Flu
|
10.3%
3/29 • Number of events 3 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Gastrointestinal disorders
Diarrhea
|
20.7%
6/29 • Number of events 9 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
General disorders
Fever
|
44.8%
13/29 • Number of events 15 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Infections and infestations
Strep infection
|
10.3%
3/29 • Number of events 4 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Infections and infestations
Upper respiratory infection
|
10.3%
3/29 • Number of events 3 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Gastrointestinal disorders
Vomiting
|
37.9%
11/29 • Number of events 17 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Gastrointestinal disorders
Loose stool
|
3.4%
1/29 • Number of events 1 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
General disorders
Mononucleosis
|
3.4%
1/29 • Number of events 1 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Infections and infestations
Viral infection
|
3.4%
1/29 • Number of events 1 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Infections and infestations
Scarlet fever
|
3.4%
1/29 • Number of events 1 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Infections and infestations
Sinus infection
|
3.4%
1/29 • Number of events 1 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
6.9%
2/29 • Number of events 2 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Ear and labyrinth disorders
Ear infection
|
10.3%
3/29 • Number of events 3 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
|
Skin and subcutaneous tissue disorders
Rash
|
13.8%
4/29 • Number of events 4 • Adverse event data were collected over the entire study time frame (12 months).
Adverse events were measured for all participants throughout the study and not classified by arm since both arms received stem cell cord blood infusion.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place