LTFU Study of Subjects With Cervical SCI Who Received AST-OPC1

Study Overview
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Brief Summary
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This is a LTFU study for cervical SCI subjects that were administered AST-OPC1 cells in the main study AST-OPC1-01.

Detailed Description
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Study AST-OPC1-02 is a Phase 1/2a open-label, multi-center long term follow-up (LTFU) study for twenty-five (25) subjects with cervical SCI that were administered AST-OPC1 cells in the main dose-escalation study AST-OPC1-01. The purpose of this study is to monitor long-term safety in subjects for 15 years post AST-OPC1 administration.

Official Title
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A Long-term Follow-up Study of Subjects With Cervical Spinal Cord Injuries Who Received AST-OPC1 in Protocol AST-OPC1-01

Conditions
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Spinal Cord Injuries

Intervention / Treatment
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* Diagnostic Test: Magnetic Resonance Imaging (MRI)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects who received AST-OPC1 under Study AST-OPC1-01 Reconfirmation of consent for long-term follow-up Exclusion Criteria: •Subjects who, for geographic or compliance reasons, are inappropriate candidates for participation in a long-term follow-up study in the opinion of the Investigator

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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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Subjects treated with AST-OPC1 in the initial dosing study AST-OPC1-01<br>Subjects treated with AST-OPC1 in the initial dosing study AST-OPC1-01 will be followed for 15-year long-term safety monitoring | Diagnostic Test: Magnetic Resonance Imaging (MRI)<br>* Observational study; annual MRI for first 5 years only to monitor changes in the injection site<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence and severity of adverse events (AEs) | Information will be collected on all adverse events through Year 5 and on late-occurring adverse events (Years 6-15) related to any changes in neurological condition, a fever of unknown etiology, and the development of neoplasias and/or neurological disorders. | Up to 15 years after AST-OPC1 injection | 
| Changes from baseline greater than 1.5x or 2x in chemistry and hematology panel results over 5 years after injection of AST-OPC1 | Summary statistics will be tabulated for chemistry and hematology laboratory parameter results and change from baseline to Year 5, as applicable, by cohort and overall. A table counting abnormal flags based on the respective lab's reference ranges by time point will be generated for each cohort and overall. At each visit, for each parameter, the number of subjects with low, normal, high, and clinically significant assessments will be tabulated. | Up to 5 years after AST-OPC1 injection | 
| Changes at the injection site as monitored by MRI | MRI scans will monitor the brain and cervical spine for any possible long-term changes that could be related to AST-OPC1 | Up to 5 years after AST-OPC1 injection | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in neurologic function as assessed by the ISNCSCI | Subject baseline results will be compared against changes in motor and sensory examination results | Up to 5 years after AST-OPC1 injection |