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

Официальное название

Delivery of Oligodendrocyte Progenitor Cells for Spinal Cord Injury: Evaluation of a Novel Device (DOSED)

Обзор

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.

Подробное описание

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.

Вмешательства

• Биопрепарат OPC1
  One injection of 10 million LCTOPC1 cells will be delivered to the target injection site of the damaged spinal tissue.

Первичные конечные точки

• Frequency and Severity of Adverse Events Related to the Delivery Device or Injection Procedure Through 30 Days [Срок оценки: One month after LCTOPC1 injection]

Вторичные конечные точки (2)

• Frequency and Severity of Adverse Events Related to LCTOPC1 and/or the Concomitant Immunosuppression Through 90 Days [Срок оценки: Three months after LCTOPC1 injection]
• Incidence of MRI Findings Indicative of Deterioration or Safety-Related Changes at 90 Days [Срок оценки: Three months after LCTOPC1 injection]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 2 центра

• Rancho Research Institue — Downey
• University of California, San Diego — La Jolla

Идентификаторы

NCT: NCT06841770 · LCTOPC1-SCI-03

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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