Trial Outcomes & Findings for Dose Escalation Study of AST-OPC1 in Spinal Cord Injury (NCT NCT02302157)
NCT ID: NCT02302157
Last Updated: 2021-07-14
Results Overview
Numbers of adverse events within 1 year (365 days) that are related to AST-OPC1 injection
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
25 participants
Primary outcome timeframe
One Year
Results posted on
2021-07-14
Participant Flow
Participant milestones
| Measure |
AST-OPC1, Cohort 1
n=3 subjects Dosage of 2 million cells AIS score A
|
AST-OPC1, Cohort 2
n=6 subjects Dosage 10 million cells AIS score A
|
AST-OPC1, Cohort 3
n=6 subjects Dosage of 20 million cells AIS score A
|
AST-OPC1, Cohort 4
n=6 subjects Dosage of 10 million AIS score B
|
AST-OPC1, Cohort 5
n=4 subjects Dosage of 20 million cells AIS score B
|
|---|---|---|---|---|---|
|
Overall Study
STARTED
|
3
|
6
|
6
|
6
|
4
|
|
Overall Study
COMPLETED
|
3
|
6
|
6
|
6
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Dose Escalation Study of AST-OPC1 in Spinal Cord Injury
Baseline characteristics by cohort
| Measure |
AST-OPC1, Cohort 1
n=3 Participants
n=3 Dosage of 2 million cells AIS score A
|
AST-OPC1, Cohort 2
n=6 Participants
n=6 Dosage of 10 million cells AIS score A
|
AST-OPC1, Cohort 3
n=6 Participants
n=6 Dosage of 20 million cells AIS score A
|
AST-OPC1, Cohort 4
n=6 Participants
n=6 Dosage of 10 million cells AIS score B
|
AST-OPC1, Cohort 5
n=4 Participants
n=4 Dosage of 20 million cells AIS score B
|
Total
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
4 Participants
n=21 Participants
|
25 Participants
n=8 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Age, Continuous
|
24.3 years
STANDARD_DEVIATION 6.1 • n=5 Participants
|
24.5 years
STANDARD_DEVIATION 7.4 • n=7 Participants
|
44.7 years
STANDARD_DEVIATION 14.7 • n=5 Participants
|
28.5 years
STANDARD_DEVIATION 14.1 • n=4 Participants
|
34.3 years
STANDARD_DEVIATION 20.7 • n=21 Participants
|
30.8 years
STANDARD_DEVIATION 14.8 • n=8 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
4 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
21 Participants
n=8 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
1 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
1 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
1 Participants
n=8 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
22 Participants
n=8 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
6 participants
n=7 Participants
|
6 participants
n=5 Participants
|
6 participants
n=4 Participants
|
4 participants
n=21 Participants
|
25 participants
n=8 Participants
|
|
The Abbreviated Injury Scale (AIS)
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
4 Participants
n=21 Participants
|
25 Participants
n=8 Participants
|
PRIMARY outcome
Timeframe: One YearPopulation: All 25 subject experienced at least one (1) AEs. The population description described on comment \[1\] above.
Numbers of adverse events within 1 year (365 days) that are related to AST-OPC1 injection
Outcome measures
| Measure |
AST-OPC1, Cohort 1
n=3 Participants
n=3 Dosage of 2 million cells AIS score A
|
AST-OPC1, Cohort 2
n=6 Participants
n=6 Dosage of 10 million cells AIS score A
|
AST-OPC1, Cohort 3
n=6 Participants
n=6 Dosage of 20 million cells AIS score A
|
AST-OPC1, Cohort 4
n=6 Participants
n=6 Dosage of 10 million cells AIS score B
|
AST-OPC1, Cohort 5
n=4 Participants
n=4 Dosage of 20 million cells AIS score B
|
|---|---|---|---|---|---|
|
Number of Adverse Events Within 1 Year (365 Days) That Are Related to AST-OPC1 Injection
|
92 Adverse Events
|
122 Adverse Events
|
133 Adverse Events
|
118 Adverse Events
|
69 Adverse Events
|
SECONDARY outcome
Timeframe: One YearThe scale is based on the ISNCSCI (International Standards for Neurological Classification of Spinal Cord Injury) that measures the severity of Spinal cord injury. The data was collected per time point during the first year (30,60,90,180,270,365). The 365-days time point was pre-specified as Secondary Outcome Measure. High score means better outcome. Units are "Scores on a scale". The score is divided to motor scale (0-100) and sensor scale (0-224). Motor scale is divided to subscale of upper extremity (0-50) and lower extremity (0-50). Upper and lower extremity are divided to subscale of Right (0-25) + Left (0-25). Each upper \& lower extremity is divided to subscale of 5 motor nerve function (0-5). Sensor scale is divided to subscale of light touch (0-112) and pin prick (0-112). light touch and pin prick are divided to subscales of Right (0-56) + Left (0-56). Each right and left light touch and pin prick are divided to subscales of 28 sensor nerve function (0-2)
Outcome measures
| Measure |
AST-OPC1, Cohort 1
n=3 Participants
n=3 Dosage of 2 million cells AIS score A
|
AST-OPC1, Cohort 2
n=6 Participants
n=6 Dosage of 10 million cells AIS score A
|
AST-OPC1, Cohort 3
n=6 Participants
n=6 Dosage of 20 million cells AIS score A
|
AST-OPC1, Cohort 4
n=6 Participants
n=6 Dosage of 10 million cells AIS score B
|
AST-OPC1, Cohort 5
n=4 Participants
n=4 Dosage of 20 million cells AIS score B
|
|---|---|---|---|---|---|
|
Neurological Function as Measured by Upper Extremity Motor Scores and Motor Level on International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) Examinations as Secondary End Point - 365 Days After Injection of AST-OPC1
UEMS (Upper Extremities Motor Scale) - Baseline
|
19 Score on a scale
Interval 0.0 to 50.0
|
21 Score on a scale
Interval 0.0 to 50.0
|
17 Score on a scale
Interval 0.0 to 50.0
|
22 Score on a scale
Interval 0.0 to 50.0
|
18 Score on a scale
Interval 0.0 to 50.0
|
|
Neurological Function as Measured by Upper Extremity Motor Scores and Motor Level on International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) Examinations as Secondary End Point - 365 Days After Injection of AST-OPC1
UEMS (Upper Extremities Motor Scale) - Day 365
|
24 Score on a scale
Interval 0.0 to 50.0
|
34 Score on a scale
Interval 0.0 to 50.0
|
26 Score on a scale
Interval 0.0 to 50.0
|
28 Score on a scale
Interval 0.0 to 50.0
|
24 Score on a scale
Interval 0.0 to 50.0
|
|
Neurological Function as Measured by Upper Extremity Motor Scores and Motor Level on International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) Examinations as Secondary End Point - 365 Days After Injection of AST-OPC1
LEMS (Lower Extremities Motor Scale) - Baseline
|
0 Score on a scale
Interval 0.0 to 50.0
|
0 Score on a scale
Interval 0.0 to 50.0
|
0 Score on a scale
Interval 0.0 to 50.0
|
0 Score on a scale
Interval 0.0 to 50.0
|
0 Score on a scale
Interval 0.0 to 50.0
|
|
Neurological Function as Measured by Upper Extremity Motor Scores and Motor Level on International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) Examinations as Secondary End Point - 365 Days After Injection of AST-OPC1
LEMS (Lower Extremities Motor Scale) - Day 365
|
0 Score on a scale
Interval 0.0 to 50.0
|
0 Score on a scale
Interval 0.0 to 50.0
|
0 Score on a scale
Interval 0.0 to 50.0
|
1 Score on a scale
Interval 0.0 to 50.0
|
1 Score on a scale
Interval 0.0 to 50.0
|
|
Neurological Function as Measured by Upper Extremity Motor Scores and Motor Level on International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) Examinations as Secondary End Point - 365 Days After Injection of AST-OPC1
LT (Light Touch) - Baseline
|
21 Score on a scale
Interval 0.0 to 112.0
|
27 Score on a scale
Interval 0.0 to 112.0
|
22 Score on a scale
Interval 0.0 to 112.0
|
35 Score on a scale
Interval 0.0 to 112.0
|
53 Score on a scale
Interval 0.0 to 112.0
|
|
Neurological Function as Measured by Upper Extremity Motor Scores and Motor Level on International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) Examinations as Secondary End Point - 365 Days After Injection of AST-OPC1
LT (Light Touch) - Day 365
|
21 Score on a scale
Interval 0.0 to 112.0
|
32 Score on a scale
Interval 0.0 to 112.0
|
31 Score on a scale
Interval 0.0 to 112.0
|
46 Score on a scale
Interval 0.0 to 112.0
|
65 Score on a scale
Interval 0.0 to 112.0
|
|
Neurological Function as Measured by Upper Extremity Motor Scores and Motor Level on International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) Examinations as Secondary End Point - 365 Days After Injection of AST-OPC1
PP (Pin Prick) - Baseline
|
22 Score on a scale
Interval 0.0 to 112.0
|
23 Score on a scale
Interval 0.0 to 112.0
|
19 Score on a scale
Interval 0.0 to 112.0
|
25 Score on a scale
Interval 0.0 to 112.0
|
22 Score on a scale
Interval 0.0 to 112.0
|
|
Neurological Function as Measured by Upper Extremity Motor Scores and Motor Level on International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) Examinations as Secondary End Point - 365 Days After Injection of AST-OPC1
PP (Pin Prick) - Day 365
|
20 Score on a scale
Interval 0.0 to 112.0
|
25 Score on a scale
Interval 0.0 to 112.0
|
21 Score on a scale
Interval 0.0 to 112.0
|
28 Score on a scale
Interval 0.0 to 112.0
|
34 Score on a scale
Interval 0.0 to 112.0
|
Adverse Events
AST-OPC1, Cohort 1
Serious events: 2 serious events
Other events: 0 other events
Deaths: 0 deaths
AST-OPC1, Cohort 2
Serious events: 2 serious events
Other events: 0 other events
Deaths: 0 deaths
AST-OPC1, Cohort 3
Serious events: 4 serious events
Other events: 0 other events
Deaths: 0 deaths
AST-OPC1, Cohort 4
Serious events: 2 serious events
Other events: 0 other events
Deaths: 0 deaths
AST-OPC1, Cohort 5
Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
AST-OPC1, Cohort 1
n=3 participants at risk
n=3 Dosage of 2 million cells AIS score A
|
AST-OPC1, Cohort 2
n=6 participants at risk
n=6 Dosage of 10 million cells AIS score A
|
AST-OPC1, Cohort 3
n=6 participants at risk
n=6 Dosage of 20 million cells AIS score A
|
AST-OPC1, Cohort 4
n=6 participants at risk
n=6 Dosage of 10 million cells AIS score B
|
AST-OPC1, Cohort 5
n=4 participants at risk
n=4 Dosage of 20 million cells AIS score B
|
|---|---|---|---|---|---|
|
Infections and infestations
Escherichia Coli Urinary Sepsis
|
0.00%
0/3 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Gastrointestinal disorders
Gastrointestinal Bleeding
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Blood and lymphatic system disorders
Hypokalemia
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Infections and infestations
Infected Epidural Fluid
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/4 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Left Femoral Shaft Fracture
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Renal and urinary disorders
Non-Traumatic Kidney Injury
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Vascular disorders
Pulmonary Embolism
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
33.3%
2/6 • Number of events 2 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Infections and infestations
Sepsis
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Infections and infestations
Urosepsis
|
0.00%
0/3 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Infections and infestations
Urinary Tract Infection (UTI)
|
66.7%
2/3 • Number of events 2 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
33.3%
2/6 • Number of events 2 • 1 year
|
33.3%
2/6 • Number of events 2 • 1 year
|
0.00%
0/4 • 1 year
|
|
Infections and infestations
Wound Infection
|
0.00%
0/3 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Renal and urinary disorders
Acute Renal Failure
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/4 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
General disorders
Altered Mental Status
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
33.3%
2/6 • Number of events 2 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Infections and infestations
Aspiration Pneumonia
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Cardiac disorders
Atrial Fibrillation
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Bilateral Decubitus Ulcers
|
0.00%
0/3 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Bilateral Hip Effusions
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Infections and infestations
Bilateral Pyelonephritis due to UTI
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Infections and infestations
Buttock Abscess
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/4 • 1 year
|
|
Injury, poisoning and procedural complications
Cerebrospinal Fluid Leak
|
0.00%
0/3 • 1 year
|
0.00%
0/6 • 1 year
|
0.00%
0/6 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
0.00%
0/4 • 1 year
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place