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 efficacy of Q-Cells® transplantation in subjects with ALS. Following an initial cohort receiving cell transplants unilaterally in the lumbar spinal cord, subsequent cohorts will receive escalating doses transplanted unilaterally in cervical spinal cord. Subjects and outcome measure assessors will be blinded to side of treatment. The study will be conducted at sites with extensive clinical experience with the care of patients with ALS.
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 efficacy of Q-Cells® transplantation in subjects with ALS. Following an initial cohort receiving cell transplants unilaterally in the lumbar spinal cord, subsequent cohorts will receive escalating doses transplanted unilaterally in cervical spinal cord. Subjects and outcome measure assessors will be blinded to side of treatment. The study will be conducted at sites with extensive clinical experience with the care of patients with ALS. The study is planned to enroll up to 30 subjects over 24 months. Each subject will receive a single time point administration of Q-Cells®: with 5 or 10 (dependent upon dose level) transplantation foci targeted to the anterior horn in either the lumbar or cervical spinal cord. The study consists of Screening, Pre-operative/Treatment, and Post-treatment study periods. The study data will be assessed for safety and efficacy after the last subject has completed the 9-month study visit. Following the 9-month study period, subjects who consent will continue to be followed for safety and efficacy long-term in a separate protocol.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
30
cellular therapeutic comprised of human cells of the glial lineage
Safety of human glial restricted progenitor cell transplantation in patients with Amyotrophic Lateral Sclerosis. (adverse events)
Safety will be measured by the number of therapy related adverse events.
Time frame: 9-month
Score on the amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Time frame: 9-month
Quantitative muscle strength values from a hand held dynamometer (HHD) will be tested bilaterally
Time frame: 9-month
Hand grip (bilateral) values from a grip dynamometer
Time frame: 9-month
Forced vital capacity (FVC)
Time frame: 9-month
Maximum sniff nasal inspiratory pressure (SNIP)
Time frame: 9-month
Electrical Impedance Myography (EIM) values on bilateral upper and lower limbs
Time frame: 9-month
Pain scores on the Visual Analog Scale (VAS)
Time frame: 9-month
Score on the ALS Specific Quality of Life Questionnaire - Revised
Time frame: 9-month
Score on the Ashworth Spasticity Scale
Time frame: 9-month
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