Dose Escalation Study of AST-OPC1 in Spinal Cord Injury

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety of cross sequential escalating doses of AST-OPC1 administered among 5 cohorts at a single time-point between 21 and 42 days post injury, inclusively, to subjects with subacute cervical spinal cord injuries (SCI).

Official Title
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A Phase 1/2a Dose Escalation Study of AST-OPC1 in Subjects With Subacute Cervical Spinal Cord Injury

Conditions
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Cervical Spinal Cord Injury, Spine Injury, Spinal Cord Trauma

Intervention / Treatment
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* Biological: AST-OPC1


Participation Criteria
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Eligibility Criteria
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Major Inclusion Criteria: Sensorimotor complete, traumatic SCI (ASIA Impairment Scale A) for cohorts 1,2,3 Sensorimotor incomplete, traumatic SCI (ASIA Impairment Scale B) for cohorts 4,5 Last fully preserved single neurological level (SNL) from C-4 to C-7 From 18 through 69 years of age at time of injury Single spinal cord lesion on a post-stabilization magnetic resonance imaging (MRI) scan, with sufficient visualization of the spinal cord injury epicenter and lesion margins to enable post-injection safety monitoring Informed consent for this protocol and the companion long term follow-up protocol must be provided and documented (i.e., signed informed consent forms) no later than 37 days following injury Able to participate in an elective surgical procedure to inject AST-OPC1 21-42 days following SCI Major Exclusion Criteria: SCI due to penetrating trauma Traumatic anatomical transection or laceration of the spinal cord based on prior surgery or MRI Any concomitant injury that interferes with the performance, interpretation or validity of neurological examinations Inability to communicate effectively with neurological examiner such that the validity of patient data could be compromised Significant organ damage or systemic disease that would create an unacceptable risk for surgery or immunosuppression History of any malignancy (except non-melanoma skin cancers) Pregnant or nursing women Body mass index (BMI) > 35 or weight > 300 lbs. Active participation in another experimental procedure/intervention

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 69 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: AST-OPC1<br>Open label, dose escalation, cross-sequential cohort of subjects who receive an injection or two injections of AST-OPC1 at a single time-point | Biological: AST-OPC1<br>* One injection of 2 million or 10 million AST-OPC1 cells, or 2 injections of 10 million AST-OPC1 cells for a total of 20 million cells; cohort dependent<br>|

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 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) | One Year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cervical spinal cord injury, AST-OPC1, SCiStar, OPC1, Neural cell, Trauma spinal cord, stemcell transplant, Cervical spine injury, Paralysis, Quadriplegia, Tetraplegia, Spine injury, oligodendrocyte progenitor cell, Subacute spinal cord injury