The purpose of this study is to assess the long-term safety and tolerability of tirasemtiv in patients with ALS who had completed the double-blind placebo-controlled study of tirasemtiv in ALS (CY 4031).
Enrolled participants will begin dosing of tirasemtiv 125 mg twice daily (250 mg/day) for a period of 4 weeks and will titrate to their tolerated dose, the maximum dose being 250 mg twice daily (500 mg/day). This study will also compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 with those who completed treatment with placebo in CY 4031 during continued treatment of both groups with tirasemtiv during CY 4033, compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 during that study with their clinical course during continued treatment with tirasemtiv during CY 4033, and compare the clinical course of patients who completed treatment with placebo in CY 4031 during that study with their clinical course during treatment with tirasemtiv during CY 4033.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
280
Oral
Number of Participants With Adverse Events
The number of participants with adverse events was used as the measure for the long-term safety and tolerability of tirasemtiv.
Time frame: From the first dose of tirasemtiv through 28 days after the last dose
Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 24 in CY 4033
Slow vital capacity (SVC) was measured using a spirometer (in units of liters). Following 3 to 5 breaths at rest, patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs). Values obtained were converted to percent predicted values (ie, the test result as a percent of predicted values for patients of similar demographic and baseline characteristics \[eg, height, age, sex\]).
Time frame: baseline and 24 weeks
Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 48 in CY 4033
Slow vital capacity was measured using a spirometer (in units of liters). Following 3 to 5 breaths at rest, patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs). Values obtained were converted to percent predicted values (ie, the test result as a percent of predicted values for patients of similar demographic and baseline characteristics \[eg, height, age, sex\]).
Time frame: baseline and 48 weeks
Change From CY 4031 Baseline in ALS Functional Rating Scale - Revised (ALSFRS-R) Total Score at Week 24
The ALSFRS-R is used to measure the progression and severity of disability in patients with ALS. The ALSFRS-R consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in the following 4 domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The total score ranges from 0 to 48, with higher scores reflecting more normal function and lower scores reflecting more impaired function. Comparing postbaseline to baseline assessments, a negative value indicates a worsening in function.
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St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
Phoenix, Arizona, United States
Cedars-Sinai Medical Center
Los Angeles, California, United States
University of California, Irvine
Orange, California, United States
UC Davis Medical Center
Sacramento, California, United States
Forbes Norris MDA/ALS Research Center
San Francisco, California, United States
Stanford Hospital and Clinics
Stanford, California, United States
University of Colorado Hospital Anschutz Outpatient Pavilion
Aurora, Colorado, United States
Hospital for Special Care
New Britain, Connecticut, United States
George Washington University Medical Center
Washington D.C., District of Columbia, United States
Mayo Clinic
Jacksonville, Florida, United States
...and 57 more locations
Time frame: baseline and 24 weeks
Change From CY 4031 Baseline in ALSFRS-R Total Score at Week 48
The ALSFRS-R is used to measure the progression and severity of disability in patients with ALS. The ALSFRS-R consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in the following 4 domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The total score ranges from 0 to 48, with higher scores reflecting more normal function and lower scores reflecting more impaired function. Comparing postbaseline to baseline assessments, a negative value indicates a worsening in function.
Time frame: baseline and 48 weeks