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 with causes of weakness, sensory loss and/or autonomic dysfunction other than TM have not been practically excluded.
Subject with significant cognitive impairment, clinical dementia, or major psychiatric illness including psychosis, bipolar disease, major depression, as determined by the DSM-V that will interfere with participation in the trial.
Subject with a diagnosis of a neurodegenerative disease (e.g., ALS, Parkinson's disease, Alzheimer's disease).
Subject suffering with medical conditions that impair nerve or muscle function (e.g., notable peripheral neuropathy, metabolic muscle disease) or any disease or condition that would impair the subject's neuromuscular function or impair the adequate assessment of the subject's function (e.g., severe osteoarthritis).
Sequential
Q-Cells
Biological/VaccineCellular therapeutic comprised of human cells of the glial lineage
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability].
Safety will be measured by the number of therapy related adverse events.
Scores on American Spinal Injury Association (ASIA) scale testing including motor and sensory evaluations
The patient's grade is based on how much sensation he or she can feel at multiple points on the body, as well as tests of motor function. Sensory grading is 0 (sensation absent), 1 (impaired sensitivity), or 2 (normal sensation) for each of light touch and pin prick sensation at each of 28 locations bilaterally: a maximum of 224 for total normal sensation. Motor function is graded from 0 (total paralysis) to 5 (normal active movement) for each of ten motor functions (e.g. knee extension; 10 total motions bilaterally) for a maximum of 100.
Quantitative muscle strength values from a hand held dynamometer (HHD)
Assess changes in neurological function as measured by muscle strength
Pain Scores on the Visual Analog Scale (VAS)
VAS is a linear score where subjects rank their current pain on a scale of 0 to 10 where 0 is no pain and 10 is the worst pain imaginable.
Score on Quality of Life (QOL) Questionnaire
The National Institutes of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS) QOL questionnaire will be used to document patient reported outcomes in the following areas: Lower extremity function, Urinary/Bladder functions, Bowel functions, and Sexual functions.
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