Following completion of the ALS Early Feasibility Study of the MyoRegulator® device for treatment of ALS (NCT06165172), the CALM study will further assess the feasibility of the MyoRegulator® device to treat ALS in an expanded number of individuals with ALS. CALM will gather additional preliminary evidence of clinical safety and potential effectiveness in this patient population with a longer follow-up period and additional secondary endpoints in a single-arm study prior to commencing a larger sham-controlled pivotal trial.
18-80 years of age inclusive
Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS as defined by revised El Escorial criteria
Less than or equal to 3 years since ALS symptom onset
Slow Vital Capacity ≥ 50% of predicted capacity at the time of Screening as determined using a portable spirometer
Study participants who are on permanent assisted ventilation (PAV) defined as >22h of noninvasive or invasive ventilation a day for > 7 consecutive days.
Study participants who have been diagnosed with ALS having only clinical bulbar involvement
Implanted intrathecal pump
Prior botulinum toxin injection(s) at any site within 12 weeks of study enrollment
Single group
Multi-site direct current stimulation (DCS)
DeviceThe MyoRegulator® treatment is a non-invasive intervention that suppresses motor neuron hyperexcitability and activates protein degradation pathways through the use of multi-site direct current stimulation (multi-site DCS).
Feasibility of treating ALS patients using multi-site DCS
The feasibility of treating ALS patients using multi-site DCS will be evaluated by determining the number of patients who can complete all of the study treatments at the target stimulation levels:
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Safety will be evaluated by recording the frequency and duration of any adverse events reported by study participants or observed by physical examination during or following treatment and throughout the study duration, with particular attention to any device-related serious adverse events or unanticipated adverse events.
ALS Functional Rating Scale (ALSFRS-R)
The Revised (ALSFRS-R) is a clinician administered outcome measure that provides an evaluation of function in ALS patients. The ALSFRS-R measures 12 aspects of physical function, ranging from one's ability to swallow and use utensils to climbing stairs and breathing. Each function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0.
Rasch Overall ALS Disability Scale (ROADS)
ROADS is a patient-reported outcome measure used to evaluate functional abilities for activities of daily living in patients with ALS. ROADS is scored by assigning each of its 28 items a value of 0, 1, or 2: 0: Unable to perform the task 1. Able to perform the task with difficulty or with assistance 2. Able to perform the task without difficulty ROADS then provides a normed total score that ranges from 0 to 146. The normed score is linearly weighted, meaning that a 1-point change represents a consistent and quantifiable measurement of disability. A 2-point change reflects twice the amount of disability.
ALS Assessment Questionnaire (ALSAQ-40)
The ALSAQ-40 is a patient-reported, 40-item questionnaire that measures five areas of health status (domains) in patients with Amyotrophic Lateral Sclerosis (ALS) or other motor neuron diseases: 1) physical mobility; 2) activities of daily living and independence; 3) eating and drinking; 4) communication; 5), and emotional functioning. Each item on the ALSAQ-40 is scored using a 5-point Likert scale from 0 to 4. The sum of the item scores for each domain is divided by the maximum possible score for that domain, and then multiplied by 100. A lower score indicates a higher health-related quality of life (HRQoL).
Accurate Test of Limb Isometric Strength (ATLIS)
The ATLIS machine will be used to measure any changes in isometric muscle strength in the study patients over the course of the study.
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PathMaker Neurosystems Inc.
Lead sponsor
Beth Israel Deaconess Medical Center
Collaborator
United States Department of Defense
Collaborator
