The purpose of the study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.
Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive, degenerative disease of motor neurons in the brain and spinal cord that leads to muscle atrophy and spasticity in limb and bulbar muscles resulting in weakness and loss of ambulation, oropharyngeal dysfunction, weight loss, and ultimately respiratory failure. The purpose of this study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
616
Oral tablet 150 mg given twice daily (BID)
Number of Subjects Who Reported an Adverse Event
The number of subjects who reported an adverse event during the study
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Subjects Who Experienced a Serious Adverse Event
The number of subjects enrolled who reported a serious adverse event during the study
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event
The number of subjects enrolled who discontinued the study treatment due to an adverse event during the study
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Participants With Potentially Clinically Significant Vital Sign Results
Number of Participants with Potentially Clinically Significant Vital Sign Abnormalities. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Participants With Potentially Clinically Significant Hematology Results
Number of Participants with Potentially Clinically Significant Hematology Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Participants With Potentially Clinically Significant Blood Chemistry Results
Number of Participants with Potentially Clinically Significant Blood Chemistry Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Barrow Neurological Institute - St. Joseph's Hospital
Phoenix, Arizona, United States
University of California at San Francisco - Fresno
Fresno, California, United States
University of California, Irvine
Orange, California, United States
University of California, Davis
Sacramento, California, United States
California Pacific Medical Center
San Francisco, California, United States
Hospital for Special Care
New Britain, Connecticut, United States
Mayo Clinic - Jacksonville
Jacksonville, Florida, United States
University of Miami Miller School of Medicine
Miami, Florida, United States
University of South Florida Medical Center
Tampa, Florida, United States
Emory University
Atlanta, Georgia, United States
...and 70 more locations
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Number of Participants With Potentially Clinically Significant ECG Results
Number of Participants with Potentially Clinically Significant ECG Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
Time frame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study
The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score between 0 to 48, with higher scores representing better function. Slope is calculated using a linear mixed effects model with x-axis time in months and y-axis ALSFRS-R score. Units for slope are change per month in units on the ALSFRS-R scale.
Time frame: Up to maximum 226 days: approximately 32.2 weeks
Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study
SNIP is a test of inspiratory force (sternocleidomastoid and diaphragm) measured via a nasal cannula and is used to assess respiratory muscle weakness and to monitor changes in respiratory muscle strength over time. During the SNIP maneuver, the patient is asked to perform a strong, sharp, maximal sniff, whereby nasal pressure is measured via nasal cannula. The maximum recorded value after several attempts, with rest in between attempts, was use in the analysis.
Time frame: Up to maximum 226 days: approximately 32.2 weeks
Death up to 6 Months
Kaplan-Meier estimate of percentage of subjects who died up to 6 months
Time frame: 6 Months
Percentage of Participants With Death or Death Equivalent up to 6 Months
Kaplan-Meier estimate of percentage of subjects who died or had a death equivalent event (tracheostomy or permanent assisted ventilation \[PAV\], defined as use of noninvasive ventilation \[NIV\] for ≥22 hours per day for ≥10 days) up to 6 months
Time frame: 6 months