A Study to Evaluate the Safety of a Delivery Device for Administering LCTOPC1 in Participants With Spinal Cord Injury

LCTOPC1-SCI-03 DOSED study is a Phase 1b, open-label, multi-center, device safety study designed to assess the safety of a novel delivery device for administering a one-time injection of LCTOPC1. The study will enroll 3-5 participants with subacute (21 to 42 days post-injury) spinal cord injuries and 3-5 participants with chronic (1 to 5 years post-injury) spinal cord injuries. Eligible participants must have either sensorimotor complete (AIS-A) or motor complete / sensory incomplete spinal cord injuries (AIS-B), located in the cervical (C4-C7) or thoracic (T1-T10) regions. The first four patients will be enrolled sequentially subsequent to meeting safety requirements. Participants will be monitored for long-term safety for up to 10 years following the administration of LCTOPC1.

Inclusion criteria

• Sensorimotor complete, traumatic SCI (ASIA Impairment Scale A) or sensory incomplete, traumatic SCI (ASIA Impairment Scale B)
• For subjects with subacute injury, International Standards for Neurological Classification of SCI (ISNCSCI) Neurological Level of Injury (NLI) from C-4 to T-10 occurring 21 to 42 days prior to LCTOPC1 injection
• For subjects with chronic injury, ISNCSCI NLI from C-4 to T-10 and more than 30 days without clinical improvement from last assessment, occurring 1 to 5 years prior to LCTOPC1 injection
• Individuals must have at least one upper extremity ISNCSCI key muscle with at least 1/5 strength
• 18 through 65 years of age, inclusive, at time of consent
• Single injury with sufficient visualization of the spinal cord injury epicenter and lesion margins to enable post-injection safety monitoring
• Informed consent for this protocol must be provided and documented (i.e., signed ICF)
• Able to participate in an elective surgical procedure to inject LCTOPC1 21 days or later following SCI
• Female subjects of child-bearing potential must agree to the use of contraception for 1 year following LCTOPC1 injection; male subjects must agree to use contraception to prevent pregnancy in any female partners of child-bearing potential for 1 year following LCTOPC1 injection

Exclusion criteria

• SCI due to penetrating trauma
• Traumatic anatomical transection, laceration, or inadequate decompression of the spinal cord based on prior surgery or MRI
• Any concomitant injury that interferes with the performance, interpretation, or validity of neurological examinations, such as multiple spinal cord lesions, brachial/lumbar plexus injury, cauda equina injury or traumatic brain injury
• Subjects with a cavity structure that would preclude successful transplantation, as identified on MRI, which may include septations or irregularities in tissue structure
• Persons with syringomyelia, defined as those with progressively enlarging cysts on T2-weighted images associated with neurological decline
• Inability to communicate effectively with neurological examiner such that the validity of patient data could be compromised
• Organ damage or systemic disease that would create an unacceptable risk for surgery or immunosuppression
• Need for mechanical support of ventilation (ventilator, continuous positive airway pressure [CPAP], bi-level positive airway pressure [BiPAP]), excluding supplemental oxygen, at baseline
• History of any malignancy (except non-melanoma skin cancers). For cancers in remission for more than five years, enrollment is allowed with concurred documented approval of principal investigator, oncologist, and Sponsor's medical monitor prior to enrollment
• Pregnant or nursing women
• Subjects with an implanted spinal cord stimulator (SCS), whether temporary or permanent.