Spinal Cord Injury, Spinal Cord Injury, Acute, Spinal Cord Injury Cervical, Spinal Cord Injury, Chronic, Spinal Cord Injury Thoracic
Conditions
Keywords
Spinal Cord Injury, DOSED, Subacute spinal cord injury, Oligodendrocyte Progenitor Cell, Chronic spinal cord injury, Stem Cell Transplant, Cervical spinal cord injury, Thoracic spinal cord injury, Paralysis, Quadriplegia, Tetraplegia
Brief summary
The DOSED clinical study evaluates the safety and utility of a novel delivery device to deliver LCTOPC1, a cell therapy, to the spinal cord of patients with a spinal cord injury (SCI). LCTOPC1 is designed to replace or support cells that are absent or dysfunctional due to traumatic injury, with a goal to help improve the quality of life and restore or augment functional activity in persons suffering from a traumatic cervical or thoracic injuries.
Detailed description
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.
Interventions
BIOLOGICALOPC1
One injection of 10 million LCTOPC1 cells will be delivered to the target injection site of the damaged spinal tissue.
Sponsors
Lineage Cell Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. Sensorimotor complete, traumatic SCI (ASIA Impairment Scale A) or sensory incomplete, traumatic SCI (ASIA Impairment Scale B) 2. 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 3. 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 4. Individuals must have at least one upper extremity ISNCSCI key muscle with at least 1/5 strength 5. 18 through 65 years of age, inclusive, at time of consent 6. Single injury with sufficient visualization of the spinal cord injury epicenter and lesion margins to enable post-injection safety monitoring 7. Informed consent for this protocol must be provided and documented (i.e., signed ICF) 8. Able to participate in an elective surgical procedure to inject LCTOPC1 21 days or later following SCI 9. 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
1. SCI due to penetrating trauma 2. Traumatic anatomical transection, laceration, or inadequate decompression of the spinal cord based on prior surgery or MRI 3. 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 4. Subjects with a cavity structure that would preclude successful transplantation, as identified on MRI, which may include septations or irregularities in tissue structure 5. Persons with syringomyelia, defined as those with progressively enlarging cysts on T2-weighted images associated with neurological decline 6. Inability to communicate effectively with neurological examiner such that the validity of patient data could be compromised 7. Organ damage or systemic disease that would create an unacceptable risk for surgery or immunosuppression 8. Need for mechanical support of ventilation (ventilator, continuous positive airway pressure \[CPAP\], bi-level positive airway pressure \[BiPAP\]), excluding supplemental oxygen, at baseline 9. 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 10. Pregnant or nursing women 11. Subjects with an implanted spinal cord stimulator (SCS), whether temporary or permanent.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Frequency and Severity of Adverse Events Related to the Delivery Device or Injection Procedure Through 30 Days | One month after LCTOPC1 injection | Frequency and severity of the MI PSD System or injection procedure related adverse events (AEs) through 30 days (1 month). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Frequency and Severity of Adverse Events Related to LCTOPC1 and/or the Concomitant Immunosuppression Through 90 Days | Three months after LCTOPC1 injection | Frequency and severity of AEs through 90 days (3 months) following injection of LCTOPC1 and/or the concomitant immunosuppression administered. |
| Incidence of MRI Findings Indicative of Deterioration or Safety-Related Changes at 90 Days | Three months after LCTOPC1 injection | Incidence of MRI findings indicative of deterioration or safety-related changes at 90 days post-administration of LCTOPC1. Central radiologists will assess MRI data for the presence and severity of: (1) Intramedullary hemorrhage, CSF leak, epidural abscess, or infection; (2) Expanding cyst or mass at the injection site or elsewhere in the CNS; (3) Inflammatory lesions at the injection site or elsewhere in the CNS; (4) CSF flow obstruction. Severity will be categorized as \[mild, moderate, severe\] based on predefined radiological criteria. |
Countries
United States
Contacts
CONTACTReference Study ID Number: LCTOPC1-SCI-03 https://lineagecell.com/products-pipeline/opc1/
Outcome results
None listed