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. Idiopathic Transverse Myelitis is a monophasic disorder characterized predominantly by demyelination. Patients are left with disability from damage to ascending and descending white matter tracts. Q-Cells® are comprised of glial progenitor cells.It is postulated that the Q-Cells® glial progeny (healthy astrocytes and oligodendrocytes) will integrate into the spinal cord lesion site and remyelinate demyelinated axons as well as provide trophic support for damaged axons. Therefore, Q-Cells® have the potential to repair damage that has occurred and could be clinically useful for patients with disability caused by TM. The study is planned to enroll up to 9 subjects. Each subject will be followed for 9 months after transplantation of Q-Cells®. Each subject will receive a single time point administration of Q-Cells®: with transplantation foci targeted to posterior columns in the spinal cord (all transplantation foci below C7) on one side. Study participation consists of Screening, Pre-operative/Treatment, and Post-treatment study periods that will generally last from 9 to 12 months in total. The study data will be assessed for safety and activity until the last subject has completed the 9-month study visit. Following completion of the 9-month follow-up period, subjects who consent will continue to be followed for safety and activity in a separate long-term follow-up protocol.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
9
cellular therapeutic comprised of human cells of the glial lineage
UT Southwestern
Dallas, Texas, United States
RECRUITINGIncidence of Treatment-Emergent Adverse Events [Safety and Tolerability].
Safety will be measured by the number of therapy related adverse events.
Time frame: 9-months
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.
Time frame: 9-months
Quantitative muscle strength values from a hand held dynamometer (HHD)
Time frame: 9-months
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.
Time frame: 9-months
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.
Time frame: 9-months
Score on Ashworth Spasticity Scale
The Modified Ashworth Scale (MAS) will be used to measure spasticity. During the administration of the MAS the examiner passively moves the joint being tested and rates the perceived level of resistance in the muscle groups opposing the movement. The scale is single-item measures ranging from 0 to 4, where 0 indicates no increase in muscle tone and 4 indicates that the affected part is rigid in flexion or extension.
Time frame: 9-months
Latency and conduction velocities from tibial somatosensory evoked potentials (SSEPs)
Somatosensory evoked potentials (SEPs or SSEPs) are a useful, noninvasive means of assessing the somatosensory pathway functioning. Electrodes are placed on the lower extremity, the back, neck and head of a patient. A nonpainful electrical stimulus is applied to the lower extremity and the signal transduction is captured by the electrodes along the back, neck and scalp. The speed of signal transduction and amplitude of the signal is measured. Standardized values are recorded and can be monitored over time. Changes in the latency (conduction velocity) will be tracked from baseline to the 9 month outcome. Transverse Myeltitis decreases the conduction speed (an increase in latency); shorter latency (increased conduction velocity) would be an indication of recovery of function.
Time frame: 9-months
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