Dose Escalation Study of AST-OPC1 in Spinal Cord Injury

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ClinicalTrials.gov Identifier: NCT02302157

Recruitment Status : Completed

First Posted : November 26, 2014

Results First Posted : July 13, 2021

Last Update Posted : July 14, 2021

View this study on Beta.ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

Lineage Cell Therapeutics, Inc.

Study Type	Interventional
Study Design	Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Conditions	Cervical Spinal Cord Injury Spine Injury Spinal Cord Trauma
Intervention	Biological: AST-OPC1
Enrollment	25

Participant Flow

Recruitment Details
Pre-assignment Details


Arm/Group Title	AST-OPC1, Cohort 1	AST-OPC1, Cohort 2	AST-OPC1, Cohort 3	AST-OPC1, Cohort 4	AST-OPC1, Cohort 5
Arm/Group Description	n=3 subjects Dosage of 2 million ce...	n=6 subjects Dosage 10 million cell...	n=6 subjects Dosage of 20 million c...	n=6 subjects Dosage of 10 million A...	n=4 subjects Dosage of 20 million c...
Arm/Group Description	n=3 subjects Dosage of 2 million cells AIS score A	n=6 subjects Dosage 10 million cells AIS score A	n=6 subjects Dosage of 20 million cells AIS score A	n=6 subjects Dosage of 10 million AIS score B	n=4 subjects Dosage of 20 million cells AIS score B
Period Title: Overall Study
Started	3	6	6	6	4
Completed	3	6	6	6	4
Not Completed	0	0	0	0	0

Baseline Characteristics

Arm/Group Title		AST-OPC1, Cohort 1	AST-OPC1, Cohort 2	AST-OPC1, Cohort 3	AST-OPC1, Cohort 4	AST-OPC1, Cohort 5	Total
Arm/Group Description		n=3 Dosage of 2 million cells AIS s...	n=6 Dosage of 10 million cells AIS ...	n=6 Dosage of 20 million cells AIS ...	n=6 Dosage of 10 million cells AIS ...	n=4 Dosage of 20 million cells AIS ...	Total of all reporting groups
Arm/Group Description		n=3 Dosage of 2 million cells AIS score A	n=6 Dosage of 10 million cells AIS score A	n=6 Dosage of 20 million cells AIS score A	n=6 Dosage of 10 million cells AIS score B	n=4 Dosage of 20 million cells AIS score B	Total of all reporting groups
Overall Number of Baseline Participants		3	6	6	6	4	25
Baseline Analysis Population Description		...
Baseline Analysis Population Description		The baseline characteristics and demographic are described separately by each cohorts per Age, gender, race, height, weight, BMI
Age, Categorical Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	3 participants	6 participants	6 participants	6 participants	4 participants	25 participants
	<=18 years	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	Between 18 and 65 years	3 100.0%	6 100.0%	6 100.0%	6 100.0%	4 100.0%	25 100.0%
	>=65 years	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%
Age, Continuous Mean (Standard Deviation) Unit of measure: Years
	Number Analyzed	3 participants	6 participants	6 participants	6 participants	4 participants	25 participants
		24.3 (6.1)	24.5 (7.4)	44.7 (14.7)	28.5 (14.1)	34.3 (20.7)	30.8 (14.8)
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	3 participants	6 participants	6 participants	6 participants	4 participants	25 participants
	Female	0 0.0%	1 16.7%	1 16.7%	0 0.0%	2 50.0%	4 16.0%
	Male	3 100.0%	5 83.3%	5 83.3%	6 100.0%	2 50.0%	21 84.0%
Race (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	3 participants	6 participants	6 participants	6 participants	4 participants	25 participants
	American Indian or Alaska Native	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	Asian	0 0.0%	0 0.0%	0 0.0%	0 0.0%	1 25.0%	1 4.0%
	Native Hawaiian or Other Pacific Islander	0 0.0%	0 0.0%	0 0.0%	0 0.0%	1 25.0%	1 4.0%
	Black or African American	0 0.0%	1 16.7%	0 0.0%	0 0.0%	0 0.0%	1 4.0%
	White	3 100.0%	5 83.3%	6 100.0%	6 100.0%	2 50.0%	22 88.0%
	More than one race	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	Unknown or Not Reported	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%
Region of Enrollment Measure Type: Number Unit of measure: Participants
United States	Number Analyzed	3 participants	6 participants	6 participants	6 participants	4 participants	25 participants
United States		3	6	6	6	4	25
The Abbreviated Injury Scale (AIS) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	3 participants	6 participants	6 participants	6 participants	4 participants	25 participants
		3 100.0%	6 100.0%	6 100.0%	6 100.0%	4 100.0%	25 100.0%

Outcome Measures

1.Primary Outcome


Title	Number of Adverse Events Within 1 Year (365 Days) That Are Related to AST-OPC1 Injection
Description	Numbers of adverse events within 1 year (365 days) that are related to...
Description	Numbers of adverse events within 1 year (365 days) that are related to AST-OPC1 injection
Time Frame	One Year

Outcome Measure Data

Analysis Population Description

All 25 subject experienced at least one (1) AEs. The population description described on comment [1] above.


Arm/Group Title	AST-OPC1, Cohort 1	AST-OPC1, Cohort 2	AST-OPC1, Cohort 3	AST-OPC1, Cohort 4	AST-OPC1, Cohort 5
Arm/Group Description:	n=3 Dosage of 2 million cells AIS s...	n=6 Dosage of 10 million cells AIS ...	n=6 Dosage of 20 million cells AIS ...	n=6 Dosage of 10 million cells AIS ...	n=4 Dosage of 20 million cells AIS ...
Arm/Group Description:	n=3 Dosage of 2 million cells AIS score A	n=6 Dosage of 10 million cells AIS score A	n=6 Dosage of 20 million cells AIS score A	n=6 Dosage of 10 million cells AIS score B	n=4 Dosage of 20 million cells AIS score B
Overall Number of Participants Analyzed	3	6	6	6	4
Measure Type: Number Unit of Measure: Adverse Events
	92	122	133	118	69

2.Secondary Outcome


Title	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
Description	The scale is based on the ISNCSCI (International Standards for Neurolo...
Description	The 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)
Time Frame	One Year

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	AST-OPC1, Cohort 1	AST-OPC1, Cohort 2	AST-OPC1, Cohort 3	AST-OPC1, Cohort 4	AST-OPC1, Cohort 5
Arm/Group Description:	n=3 Dosage of 2 million AIS A	n=6 Dosage of 10 million AIS A	n=6 Dosage of 20 million AIS A	n=6 Dosage of 10 million AIS B	n=4 Dosage of 20 million AIS B
Arm/Group Description:	n=3 Dosage of 2 million AIS A	n=6 Dosage of 10 million AIS A	n=6 Dosage of 20 million AIS A	n=6 Dosage of 10 million AIS B	n=4 Dosage of 20 million AIS B
Overall Number of Participants Analyzed	3	6	6	6	4
Mean (Full Range) Unit of Measure: Score on a scale
UEMS (Upper Extremities Motor Scale) - Baseline	19 (0 to 50)	21 (0 to 50)	17 (0 to 50)	22 (0 to 50)	18 (0 to 50)
UEMS (Upper Extremities Motor Scale) - Day 365	24 (0 to 50)	34 (0 to 50)	26 (0 to 50)	28 (0 to 50)	24 (0 to 50)
LEMS (Lower Extremities Motor Scale) - Baseline	0 (0 to 50)	0 (0 to 50)	0 (0 to 50)	0 (0 to 50)	0 (0 to 50)
LEMS (Lower Extremities Motor Scale) - Day 365	0 (0 to 50)	0 (0 to 50)	0 (0 to 50)	1 (0 to 50)	1 (0 to 50)
LT (Light Touch) - Baseline	21 (0 to 112)	27 (0 to 112)	22 (0 to 112)	35 (0 to 112)	53 (0 to 112)
LT (Light Touch) - Day 365	21 (0 to 112)	32 (0 to 112)	31 (0 to 112)	46 (0 to 112)	65 (0 to 112)
PP (Pin Prick) - Baseline	22 (0 to 112)	23 (0 to 112)	19 (0 to 112)	25 (0 to 112)	22 (0 to 112)
PP (Pin Prick) - Day 365	20 (0 to 112)	25 (0 to 112)	21 (0 to 112)	28 (0 to 112)	34 (0 to 112)

Adverse Events

Time Frame	1 year
Adverse Event Reporting Description	[Not Specified]

Arm/Group Title	AST-OPC1, Cohort 1		AST-OPC1, Cohort 2		AST-OPC1, Cohort 3		AST-OPC1, Cohort 4		AST-OPC1, Cohort 5
Arm/Group Description	n=3 Dosage of 2 million cells AIS s...		n=6 Dosage of 10 million cells AIS ...		n=6 Dosage of 20 million cells AIS ...		n=6 Dosage of 10 million cells AIS ...		n=4 Dosage of 20 million cells AIS ...
Arm/Group Description	n=3 Dosage of 2 million cells AIS score A		n=6 Dosage of 10 million cells AIS score A		n=6 Dosage of 20 million cells AIS score A		n=6 Dosage of 10 million cells AIS score B		n=4 Dosage of 20 million cells AIS score B
All-Cause Mortality
	AST-OPC1, Cohort 1		AST-OPC1, Cohort 2		AST-OPC1, Cohort 3		AST-OPC1, Cohort 4		AST-OPC1, Cohort 5
	Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)
Total	0/3 (0.00%)		0/6 (0.00%)		0/6 (0.00%)		0/6 (0.00%)		0/4 (0.00%)
Serious Adverse Events Serious Adverse Events
	AST-OPC1, Cohort 1		AST-OPC1, Cohort 2		AST-OPC1, Cohort 3		AST-OPC1, Cohort 4		AST-OPC1, Cohort 5
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	2/3 (66.67%)		2/6 (33.33%)		4/6 (66.67%)		2/6 (33.33%)		1/4 (25.00%)
Blood and lymphatic system disorders
Hypokalemia ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	1/6 (16.67%)	1	0/6 (0.00%)	0	0/4 (0.00%)	0
Cardiac disorders
Atrial Fibrillation ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	0/6 (0.00%)	0	0/6 (0.00%)	0	1/4 (25.00%)	1
Gastrointestinal disorders
Bilateral Decubitus Ulcers ^† 1	0/3 (0.00%)	0	1/6 (16.67%)	1	0/6 (0.00%)	0	0/6 (0.00%)	0	0/4 (0.00%)	0
Gastrointestinal Bleeding ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	1/6 (16.67%)	1	0/6 (0.00%)	0	0/4 (0.00%)	0
General disorders
Altered Mental Status ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	2/6 (33.33%)	2	0/6 (0.00%)	0	0/4 (0.00%)	0
Infections and infestations
Aspiration Pneumonia ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	1/6 (16.67%)	1	0/6 (0.00%)	0	0/4 (0.00%)	0
Bilateral Pyelonephritis due to UTI ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	1/6 (16.67%)	1	0/6 (0.00%)	0	0/4 (0.00%)	0
Buttock Abscess ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	1/6 (16.67%)	1	0/6 (0.00%)	0	0/4 (0.00%)	0
Escherichia Coli Urinary Sepsis ^† 1	0/3 (0.00%)	0	1/6 (16.67%)	1	0/6 (0.00%)	0	0/6 (0.00%)	0	0/4 (0.00%)	0
Infected Epidural Fluid ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	0/6 (0.00%)	0	1/6 (16.67%)	1	0/4 (0.00%)	0
Sepsis ^† 1	1/3 (33.33%)	1	0/6 (0.00%)	0	0/6 (0.00%)	0	0/6 (0.00%)	0	0/4 (0.00%)	0
Urosepsis ^† 1	0/3 (0.00%)	0	1/6 (16.67%)	1	0/6 (0.00%)	0	0/6 (0.00%)	0	0/4 (0.00%)	0
Urinary Tract Infection (UTI) ^† 1	2/3 (66.67%)	2	1/6 (16.67%)	1	2/6 (33.33%)	2	2/6 (33.33%)	2	0/4 (0.00%)	0
Wound Infection ^† 1	0/3 (0.00%)	0	1/6 (16.67%)	1	0/6 (0.00%)	0	0/6 (0.00%)	0	0/4 (0.00%)	0
Injury, poisoning and procedural complications
Cerebrospinal Fluid Leak ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	0/6 (0.00%)	0	1/6 (16.67%)	1	0/4 (0.00%)	0
Musculoskeletal and connective tissue disorders
Bilateral Hip Effusions ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	1/6 (16.67%)	1	0/6 (0.00%)	0	0/4 (0.00%)	0
Left Femoral Shaft Fracture ^† 1	1/3 (33.33%)	1	0/6 (0.00%)	0	0/6 (0.00%)	0	0/6 (0.00%)	0	0/4 (0.00%)	0
Renal and urinary disorders
Acute Renal Failure ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	0/6 (0.00%)	0	1/6 (16.67%)	1	0/4 (0.00%)	0
Non-Traumatic Kidney Injury ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	0/6 (0.00%)	0	0/6 (0.00%)	0	1/4 (25.00%)	1
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	1/6 (16.67%)	1	0/6 (0.00%)	0	0/4 (0.00%)	0
Vascular disorders
Pulmonary Embolism ^† 1	0/3 (0.00%)	0	0/6 (0.00%)	0	2/6 (33.33%)	2	0/6 (0.00%)	0	0/4 (0.00%)	0
1 Term from vocabulary, MedDRA (18.0) † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	AST-OPC1, Cohort 1		AST-OPC1, Cohort 2		AST-OPC1, Cohort 3		AST-OPC1, Cohort 4		AST-OPC1, Cohort 5
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/3 (0.00%)		0/6 (0.00%)		0/6 (0.00%)		0/6 (0.00%)		0/4 (0.00%)

Limitations and Caveats

Open label study

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

Layout table for Results Point of Contact information

Name/Title:	Avi Ben Shabat, MD
Organization:	Lineage
Phone:	+972-509105205
EMail:	avi@cellcure.co.il

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

Fessler RG, Ehsanian R, Liu CY, Steinberg GK, Jones L, Lebkowski JS, Wirth ED, McKenna SL. A phase 1/2a dose-escalation study of oligodendrocyte progenitor cells in individuals with subacute cervical spinal cord injury. J Neurosurg Spine. 2022 Jul 8;37(6):812-820. doi: 10.3171/2022.5.SPINE22167. Print 2022 Dec 1.

McKenna SL, Ehsanian R, Liu CY, Steinberg GK, Jones L, Lebkowski JS, Wirth E, Fessler RG. Ten-year safety of pluripotent stem cell transplantation in acute thoracic spinal cord injury. J Neurosurg Spine. 2022 Apr 1:1-10. doi: 10.3171/2021.12.SPINE21622. Online ahead of print.

Layout table for additonal information

Responsible Party:	Lineage Cell Therapeutics, Inc.
ClinicalTrials.gov Identifier:	NCT02302157
Other Study ID Numbers:	AST-OPC1-01
First Submitted:	November 24, 2014
First Posted:	November 26, 2014
Results First Submitted:	December 15, 2020
Results First Posted:	July 13, 2021
Last Update Posted:	July 14, 2021

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