Trial Outcomes & Findings for A Study of Modified Stem Cells in Traumatic Brain Injury (TBI) (NCT NCT02416492)
NCT ID: NCT02416492
Last Updated: 2021-12-27
Results Overview
The FMMS motor component consists of the 33-item upper extremity subscale (UE-FMMS) and the 17-item lower extremity subscale (LE-FMMS). Items were scored on a 3-point ordinal scale: 0= cannot perform; 1= partial motion; 2= full motion Individual items were then summed to determine scores for the 2 subscale scores, as well as a motor total score (total of all item scores including the 2 subscales UE-FMMS and LE-FMMS). As a result, the UE-FMMS subscale score ranged from 0 to 66 and the LE-FMMS subscale score ranged from 0 to 34. The FMMS motor total score ranged from 0 (hemiplegia) to a maximum of 100 points (normal motor performance).
COMPLETED
PHASE2
63 participants
24 weeks
2021-12-27
Participant Flow
Participant milestones
| Measure |
Treatment Group: SB623 2.5 Million Cells
2.5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: SB623 5 Million Cells
5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: SB623 10 Million Cells
10 million SB623 cells
SB623 surgically implanted
|
Treatment Group: Sham Surgery
Control Sham Surgery
Sham Control: Sham Surgery
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
15
|
15
|
16
|
15
|
|
Overall Study
COMPLETED
|
15
|
15
|
16
|
15
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of Modified Stem Cells in Traumatic Brain Injury (TBI)
Baseline characteristics by cohort
| Measure |
Sham Surgery
n=15 Participants
Control Sham Surgery
Sham Control: Sham Surgery
|
Total
n=61 Participants
Total of all reporting groups
|
Treatment Group: 2.5 Million Cells
n=15 Participants
2.5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 5 Million Cells
n=15 Participants
5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 10 Million Cells
n=16 Participants
10 million SB623 cells
SB623 surgically implanted
|
|---|---|---|---|---|---|
|
Age, Continuous
|
35.48 years
STANDARD_DEVIATION 12.96 • n=4 Participants
|
34.40 years
STANDARD_DEVIATION 11.77 • n=21 Participants
|
36.66 years
STANDARD_DEVIATION 13.57 • n=5 Participants
|
31.22 years
STANDARD_DEVIATION 9.15 • n=7 Participants
|
34.23 years
STANDARD_DEVIATION 11.46 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=4 Participants
|
18 Participants
n=21 Participants
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=4 Participants
|
43 Participants
n=21 Participants
|
11 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
15 Participants
n=4 Participants
|
59 Participants
n=21 Participants
|
15 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=4 Participants
|
18 Participants
n=21 Participants
|
4 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=4 Participants
|
42 Participants
n=21 Participants
|
11 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 weeksPopulation: Modified Intent-to-Treat (mITT) Population: Included all randomized subjects who completed the surgery treatment procedure (61 subjects)
The FMMS motor component consists of the 33-item upper extremity subscale (UE-FMMS) and the 17-item lower extremity subscale (LE-FMMS). Items were scored on a 3-point ordinal scale: 0= cannot perform; 1= partial motion; 2= full motion Individual items were then summed to determine scores for the 2 subscale scores, as well as a motor total score (total of all item scores including the 2 subscales UE-FMMS and LE-FMMS). As a result, the UE-FMMS subscale score ranged from 0 to 66 and the LE-FMMS subscale score ranged from 0 to 34. The FMMS motor total score ranged from 0 (hemiplegia) to a maximum of 100 points (normal motor performance).
Outcome measures
| Measure |
Treatment Group: 2.5 Million Cells
n=15 Participants
2.5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 5 Million Cells
n=15 Participants
5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 10 Million Cells
n=16 Participants
10 million SB623 cells
SB623 surgically implanted
|
Sham Surgery
n=15 Participants
Control Sham Surgery
Sham Control: Sham Surgery
|
|---|---|---|---|---|
|
Change From Baseline in Fugl-Meyer Motor Scale (FMMS) Score at Week 24 Among All Patients
|
6.0 Change in score on a scale from baseline
Standard Deviation 10.1
|
11.0 Change in score on a scale from baseline
Standard Deviation 8.4
|
8.1 Change in score on a scale from baseline
Standard Deviation 12.8
|
2.3 Change in score on a scale from baseline
Standard Deviation 4.7
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: The Modified Intent-to-Treat (mITT) population included all randomized subjects who completed the surgery treatment procedure. Only subjects in the mITT population who have the value available for Change from Baseline in DRS at 24 weeks are included in this analysis.
DRS is an observer rated, 30-point ordinal scale that evaluates eight areas of functioning in four categories: 1. Consciousness (eye opening, verbal response, motor response) 2. Cognitive ability (feeding, toileting, grooming) 3. Dependence on others 4. Employability Each area of functioning was rated on a scale of 0 to either 3 or 5. The maximum score is 29 (extreme vegetative state) and the minimum score is 0 (person without disability).
Outcome measures
| Measure |
Treatment Group: 2.5 Million Cells
n=15 Participants
2.5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 5 Million Cells
n=14 Participants
5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 10 Million Cells
n=16 Participants
10 million SB623 cells
SB623 surgically implanted
|
Sham Surgery
n=15 Participants
Control Sham Surgery
Sham Control: Sham Surgery
|
|---|---|---|---|---|
|
Change From Baseline in Disability Rating Scale Score at Week 24 Among All Patients
|
-0.1 Change in score on a scale from baseline
Standard Deviation 1.2
|
-1.4 Change in score on a scale from baseline
Standard Deviation 2.6
|
-0.6 Change in score on a scale from baseline
Standard Deviation 2.2
|
0.6 Change in score on a scale from baseline
Standard Deviation 1.6
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: The Modified Intent-to-Treat (mITT) population included all randomized subjects who completed the surgery treatment procedure. Only subjects in the mITT population who have the value available for Change from Baseline in ARAT at 24 weeks are included in this analysis.
The ARAT total score is the sum of the scores from 19 tests spread across four subscales: grasp, grip, pinch, and gross movement. Each test is scored on an ordinal 4-point scale with 0= non movement, 1 = the movement task is partially performed, 2 = the movement task is completed but takes abnormally long, and 3 = the movement is performed normally. Summation of a 0-3 score in each item yields a total score between 0 and 57.
Outcome measures
| Measure |
Treatment Group: 2.5 Million Cells
n=14 Participants
2.5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 5 Million Cells
n=13 Participants
5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 10 Million Cells
n=14 Participants
10 million SB623 cells
SB623 surgically implanted
|
Sham Surgery
n=14 Participants
Control Sham Surgery
Sham Control: Sham Surgery
|
|---|---|---|---|---|
|
Change From Baseline in ARAT Total Score at Week 24 Among Upper Extremity Deficit Patients
|
3.0 Change in score on a scale from baseline
Standard Deviation 6.7
|
4.2 Change in score on a scale from baseline
Standard Deviation 5.2
|
-0.3 Change in score on a scale from baseline
Standard Deviation 10.3
|
-0.4 Change in score on a scale from baseline
Standard Deviation 11.5
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: The Modified Intent-to-Treat (mITT) population included all randomized subjects who completed the surgery treatment procedure. Only subjects in the mITT population who have the value available for Change from Baseline in Gait Velocity at 24 weeks are included in this analysis.
Gait Velocity was measured on a standard 10 meter walk.
Outcome measures
| Measure |
Treatment Group: 2.5 Million Cells
n=14 Participants
2.5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 5 Million Cells
n=13 Participants
5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 10 Million Cells
n=14 Participants
10 million SB623 cells
SB623 surgically implanted
|
Sham Surgery
n=14 Participants
Control Sham Surgery
Sham Control: Sham Surgery
|
|---|---|---|---|---|
|
Change From Baseline in Gait Velocity (10 Meter Walk Time in Seconds) at Week 24 Among Lower Extremity Deficit Patients
|
-3.1 10 meter walk change of time in seconds
Standard Deviation 87.0
|
-3.9 10 meter walk change of time in seconds
Standard Deviation 6.7
|
-2.2 10 meter walk change of time in seconds
Standard Deviation 113.6
|
-2.4 10 meter walk change of time in seconds
Standard Deviation 6.7
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: The Modified Intent-to-Treat (mITT) population included all randomized subjects who completed the surgery treatment procedure. Only subjects in the mITT population who have the value available for Change from Baseline in NeuroQOL T-scores at 24 weeks are included in this analysis.
Two NeuroQoL short form assessments were used (upper extremity function and lower extremity function); each has 8 items with 5 possible scores (e.g. 1= not at all, 2=a little bit, 3= somewhat, 4=quite a bit, 5=very much) or frequency ("never"to "always"); Raw scores are converted to T-scores based on a consistent metric (i.e., the T distribution) and data from the US general population. The theoretical range in scale for Upper extremity T-score and Lower extremity T-score are 12.8 to 53.8 and 16.5 to 58.6 respectively. When interpreting these T-scores, higher scores correspond to higher levels of functioning whereas lower scores correspond to lower levels of functioning.
Outcome measures
| Measure |
Treatment Group: 2.5 Million Cells
n=14 Participants
2.5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 5 Million Cells
n=13 Participants
5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 10 Million Cells
n=15 Participants
10 million SB623 cells
SB623 surgically implanted
|
Sham Surgery
n=13 Participants
Control Sham Surgery
Sham Control: Sham Surgery
|
|---|---|---|---|---|
|
Change From Baseline in NeuroQOL T-scores at Week 24 of NeuroQOL Domains
Change in T-score from baseline (upper extremity function)
|
5.66 Change in score on a scale from baseline
Standard Deviation 8.06
|
5.27 Change in score on a scale from baseline
Standard Deviation 8.90
|
-0.77 Change in score on a scale from baseline
Standard Deviation 6.42
|
2.48 Change in score on a scale from baseline
Standard Deviation 9.89
|
|
Change From Baseline in NeuroQOL T-scores at Week 24 of NeuroQOL Domains
Change in T-score from baseline (lower extremity function)
|
3.06 Change in score on a scale from baseline
Standard Deviation 6.58
|
1.95 Change in score on a scale from baseline
Standard Deviation 6.82
|
3.38 Change in score on a scale from baseline
Standard Deviation 7.10
|
1.75 Change in score on a scale from baseline
Standard Deviation 7.05
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: Modified Intent-to-Treat (mITT) Population: Included all randomized subjects who completed the surgery treatment procedure
The proportions of SB623 treated subjects (pooling all SB623 doses) scoring either 7 (much better) or 6 (a little better, meaningful) on the Global Rating of Perceived Change (from Baseline) - Subject at Week 24 and on the Global Rating of Perceived Change (from Baseline) - Clinician at Week 24 was compared to the corresponding proportions of sham surgery control subjects using logistic regression models with adjustment for the baseline Fugl-Meyer Motor Scale score and the GOS-E score at screening as continuous covariates. The following 7-point Likert scale was used * Score 7 = Much better * Score 6 = A little better, meaningful * Score 5 = A little better, not meaningful * Score 4 = About the same * Score 3 = A little worse, not meaningful * Score 2 = A little worse, meaningful * Score 1 = Much worse
Outcome measures
| Measure |
Treatment Group: 2.5 Million Cells
n=15 Participants
2.5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 5 Million Cells
n=15 Participants
5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: 10 Million Cells
n=16 Participants
10 million SB623 cells
SB623 surgically implanted
|
Sham Surgery
n=15 Participants
Control Sham Surgery
Sham Control: Sham Surgery
|
|---|---|---|---|---|
|
Global Rating of Perceived Change: The Percentage of Subjects Scoring Either 6 or 7 on the Global Rating of Perceived Change by Both Subject and Physician
Clinician at Week 24 · Scoring either 6 or 7 on the Global Rating (Yes)
|
5 Participants
|
6 Participants
|
4 Participants
|
2 Participants
|
|
Global Rating of Perceived Change: The Percentage of Subjects Scoring Either 6 or 7 on the Global Rating of Perceived Change by Both Subject and Physician
Clinician at Week 24 · Scoring either 6 or 7 on the Global Rating (No)
|
10 Participants
|
9 Participants
|
12 Participants
|
13 Participants
|
|
Global Rating of Perceived Change: The Percentage of Subjects Scoring Either 6 or 7 on the Global Rating of Perceived Change by Both Subject and Physician
Subject at Week 24 · Scoring either 6 or 7 on the Global Rating (Yes)
|
8 Participants
|
12 Participants
|
6 Participants
|
4 Participants
|
|
Global Rating of Perceived Change: The Percentage of Subjects Scoring Either 6 or 7 on the Global Rating of Perceived Change by Both Subject and Physician
Subject at Week 24 · Scoring either 6 or 7 on the Global Rating (No)
|
7 Participants
|
3 Participants
|
10 Participants
|
11 Participants
|
Adverse Events
Treatment Group: SB623 2.5 Million Cells
Treatment Group: SB623 5 Million Cells
Treatment Group: SB623 10 Million Cells
Sham Surgery
Serious adverse events
| Measure |
Treatment Group: SB623 2.5 Million Cells
n=15 participants at risk
2.5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: SB623 5 Million Cells
n=15 participants at risk
5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: SB623 10 Million Cells
n=16 participants at risk
10 million SB623 cells
SB623 surgically implanted
|
Sham Surgery
n=15 participants at risk
Control Sham Surgery
Sham Control: Sham Surgery
|
|---|---|---|---|---|
|
Nervous system disorders
Transient ischaemic attack
|
0.00%
0/15 • 1 year
|
6.7%
1/15 • 1 year
|
0.00%
0/16 • 1 year
|
0.00%
0/15 • 1 year
|
|
Infections and infestations
Wound infection
|
0.00%
0/15 • 1 year
|
0.00%
0/15 • 1 year
|
0.00%
0/16 • 1 year
|
6.7%
1/15 • 1 year
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.00%
0/15 • 1 year
|
0.00%
0/15 • 1 year
|
0.00%
0/16 • 1 year
|
6.7%
1/15 • 1 year
|
|
Nervous system disorders
Balance disorder
|
0.00%
0/15 • 1 year
|
0.00%
0/15 • 1 year
|
6.2%
1/16 • 1 year
|
0.00%
0/15 • 1 year
|
|
Nervous system disorders
Seizure
|
0.00%
0/15 • 1 year
|
0.00%
0/15 • 1 year
|
6.2%
1/16 • 1 year
|
6.7%
1/15 • 1 year
|
|
Psychiatric disorders
Delirium
|
6.7%
1/15 • 1 year
|
0.00%
0/15 • 1 year
|
6.2%
1/16 • 1 year
|
0.00%
0/15 • 1 year
|
Other adverse events
| Measure |
Treatment Group: SB623 2.5 Million Cells
n=15 participants at risk
2.5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: SB623 5 Million Cells
n=15 participants at risk
5 million SB623 cells
SB623 surgically implanted
|
Treatment Group: SB623 10 Million Cells
n=16 participants at risk
10 million SB623 cells
SB623 surgically implanted
|
Sham Surgery
n=15 participants at risk
Control Sham Surgery
Sham Control: Sham Surgery
|
|---|---|---|---|---|
|
Nervous system disorders
Headache
|
33.3%
5/15 • 1 year
|
40.0%
6/15 • 1 year
|
50.0%
8/16 • 1 year
|
26.7%
4/15 • 1 year
|
|
Injury, poisoning and procedural complications
Wound complication
|
20.0%
3/15 • 1 year
|
33.3%
5/15 • 1 year
|
25.0%
4/16 • 1 year
|
20.0%
3/15 • 1 year
|
|
Gastrointestinal disorders
Nausea
|
20.0%
3/15 • 1 year
|
20.0%
3/15 • 1 year
|
12.5%
2/16 • 1 year
|
6.7%
1/15 • 1 year
|
|
Gastrointestinal disorders
Vomiting
|
13.3%
2/15 • 1 year
|
13.3%
2/15 • 1 year
|
25.0%
4/16 • 1 year
|
6.7%
1/15 • 1 year
|
|
General disorders
Pyrexia
|
26.7%
4/15 • 1 year
|
13.3%
2/15 • 1 year
|
0.00%
0/16 • 1 year
|
0.00%
0/15 • 1 year
|
|
General disorders
Asthenia
|
13.3%
2/15 • 1 year
|
13.3%
2/15 • 1 year
|
6.2%
1/16 • 1 year
|
6.7%
1/15 • 1 year
|
|
Nervous system disorders
Dizziness
|
20.0%
3/15 • 1 year
|
6.7%
1/15 • 1 year
|
6.2%
1/16 • 1 year
|
6.7%
1/15 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.7%
1/15 • 1 year
|
13.3%
2/15 • 1 year
|
6.2%
1/16 • 1 year
|
13.3%
2/15 • 1 year
|
|
Injury, poisoning and procedural complications
Incision site pain
|
20.0%
3/15 • 1 year
|
0.00%
0/15 • 1 year
|
6.2%
1/16 • 1 year
|
6.7%
1/15 • 1 year
|
|
Injury, poisoning and procedural complications
Procedural headache
|
13.3%
2/15 • 1 year
|
0.00%
0/15 • 1 year
|
12.5%
2/16 • 1 year
|
6.7%
1/15 • 1 year
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/15 • 1 year
|
20.0%
3/15 • 1 year
|
6.2%
1/16 • 1 year
|
0.00%
0/15 • 1 year
|
|
Psychiatric disorders
Agitation
|
13.3%
2/15 • 1 year
|
0.00%
0/15 • 1 year
|
6.2%
1/16 • 1 year
|
0.00%
0/15 • 1 year
|
|
Investigations
Alanine aminotransferase increased
|
6.7%
1/15 • 1 year
|
6.7%
1/15 • 1 year
|
6.2%
1/16 • 1 year
|
0.00%
0/15 • 1 year
|
|
Investigations
Aspartate aminotransferase increased
|
6.7%
1/15 • 1 year
|
6.7%
1/15 • 1 year
|
6.2%
1/16 • 1 year
|
0.00%
0/15 • 1 year
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/15 • 1 year
|
6.7%
1/15 • 1 year
|
12.5%
2/16 • 1 year
|
0.00%
0/15 • 1 year
|
|
Nervous system disorders
Head discomfort
|
6.7%
1/15 • 1 year
|
6.7%
1/15 • 1 year
|
6.2%
1/16 • 1 year
|
0.00%
0/15 • 1 year
|
|
Infections and infestations
Influenza
|
6.7%
1/15 • 1 year
|
0.00%
0/15 • 1 year
|
12.5%
2/16 • 1 year
|
13.3%
2/15 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
13.3%
2/15 • 1 year
|
6.7%
1/15 • 1 year
|
0.00%
0/16 • 1 year
|
6.7%
1/15 • 1 year
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
6.7%
1/15 • 1 year
|
0.00%
0/15 • 1 year
|
12.5%
2/16 • 1 year
|
13.3%
2/15 • 1 year
|
Additional Information
Bijan Nejadnik M.D.; Chief Medical Officer
SanBio, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place