Clinical trial NCT06841770: A Study to Evaluate the Safety of a Delivery Device for Administering LCTOPC1 in Participants With Spinal Cord Injury
Official title: Delivery of Oligodendrocyte Progenitor Cells for Spinal Cord Injury: Evaluation of a Novel Device (DOSED)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Spinal Cord Injury Cervical
Spinal Cord Injury Thoracic
Spinal Cord Injury, Acute
Spinal Cord Injury
Spinal Cord Injury, Chronic
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
Lead sponsor: Lineage Cell Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2025-02-24
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Frequency and Severity of Adverse Events Related to the Delivery Device or Injection Procedure Through 30 Days
timeFrame: One month after LCTOPC1 injection
Secondary outcomes:
measure: Frequency and Severity of Adverse Events Related to LCTOPC1 and/or the Concomitant Immunosuppression Through 90 Days
timeFrame: Three months after LCTOPC1 injection
measure: Incidence of MRI Findings Indicative of Deterioration or Safety-Related Changes at 90 Days
timeFrame: Three months after LCTOPC1 injection
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: University of California, San Diego
state: California
Study officials:
References: