A Phase 1/2a Open-Label Study to Investigate the Safety of the Transplantation (by Injection) of Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) Into Subjects With Transverse Myelitis (TM)
This study is a non-randomized, open-label, partially blinded, sequential cohort, dose-escalation study designed to obtain preliminary data on the safety, tolerability, and early activity of Q-Cells® transplantation in subjects with Transverse Myelitis. For each of the dose levels, transplantation of Q-Cells® unilaterally into spinal cord demyelinated lesions will be evaluated. Subjects will be blinded to side of treatment.
• Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to collect and use protected health information (PHI) in accordance with national and local subject privacy regulations.
• Live within reasonable travel distance to center or have reliable mechanism to travel to the center.
• Have a caregiver willing/able to assist in the transportation and care required by study participation.
• Subject is 18 - 70 years of age (inclusive) on day of Screening Visit.
• Subject is diagnosed with idiopathic TM within the past 120 months in accord with the Transverse Myelitis Consortium Working Group (2002).
• Subject has an MRI with a single focus of T2 hyperintensity with its most rostral extent at or below C8 myotome/dermatome level.
• Subject has negative NMO IgG (anti-AQP4) test at two separate time points, separated by at least 6 months.
• Subject has brain MRI not consistent with multiple sclerosis or other autoimmune or demyelinating disease.
• Subject is more than 12 months from TM onset.
⁃ Subject has ASIA A, B or C categorization, and is unable to ambulate (Cohort A) or unable to ambulate 100 feet without significant bilateral support. (Cohort B).
⁃ Subject's neurological deficits related to TM have been stable for at least 3 months.
⁃ Subject is medically able to undergo the study procedures and physically able to adhere to the visit schedule at the time of study entry.
⁃ For women of child bearing capacity, negative pregnancy test during the Screening Period and at the Pre-Operative Visit.
⁃ Males and females will agree to practice effective birth control during study participation and up to one year after.
⁃ Current, up to date, COVID vaccination.
⁃ Current, up to date, varicella zoster vaccination.
⁃ Current, seasonally up to date, influenza vaccination.