ALS is a disorder that weakens motor strength and lung function. Rapid loss of motor neurons in the brain and spinal cord of ALS patients causes the symptoms of increasing weakness and loss of muscle function. Motor neurons are responsible for sending signals to muscles in our bodies to trigger movement. While there are drugs to help relieve symptoms of ALS, there is no cure for ALS. Rasagiline is a drug with possible neuroprotective characteristics. Neuroprotective means that the nervous system may be protected against weakening. It is known that rasagiline has possible neuroprotective characteristics, but the effectiveness of rasagiline for patients with ALS has not been tested. Rasagiline is approved for the treatment of Parkinson's disease. Rasagiline for treatment of ALS is not approved by the U.S. Food and Drug Administration (FDA) and is investigational. Investigational drugs are studied to find out if they are safe and effective in the treatment of diseases or conditions. By doing this study, researchers hope to learn if rasagiline is safe and slows disease progression in patients with ALS. Funding Source - FDA OOPD (FDA Orphan Products Division).
The study is a phase II, double-blind, placebo-controlled, multicenter study of rasagiline 2mg/day. Subjects will be assigned to either active agent or placebo (3:1) for twelve months. Subjects will undergo outpatient evaluations at screening, baseline, and months 1, 2, 4, 6, 8, 10 and 12 and telephone assessments at months 3, 5, 7 and 9. There will be a close-out phone call 30 days post month 12.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
80
Rasagiline 2mg once a day for 12 months.
Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
Phoenix Neurological Associates
Phoenix, Arizona, United States
University of California - Irvine
Irvine, California, United States
California Pacific Medical Center
San Francisco, California, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
St. Louis University
St Louis, Missouri, United States
University of Nebraska
Omaha, Nebraska, United States
Columbia University
New York, New York, United States
Oregon Health and Science University
Portland, Oregon, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
UT Southwestern Medical Center
Dallas, Texas, United States
ALS Functional Rating Scale-Revised (ALSFRS-R)
Difference in ALS Functional Rating Scale - Revised (ALSFRS-R) score. The ALSFRS-R is an ordinal rating scale that assesses 12 functional activities. Each activity is scored between 0-4, with a total score ranging from 48 (normal function) to 0 (no function).
Time frame: ALS Functional Rating Scale-Revised (ALSFRS-R) Difference from Baseline to Month 12
Change in Vital Capacity (VC)
Determine if decline in vital capacity is slower in participants taking 2 mg rasagiline than controls.
Time frame: Vital Capacity Change from Baseline to Month 12
Change in Quality of Life
Participants completed the single-item ALSQOL (ALS Quality of Life) which asks participants to rank their global quality of life, considering all parts of their lives - physical, emotional, social, spiritual and financial - in the last 7 days and rate on a scale of 0 (very bad) to 10 (excellent).
Time frame: Quality of Life Change from Baseline to Month 12
Number of Participants With Adverse Events
Determine if participants on rasagiline 2 mg had a different safety profile than patients not on rasagiline. Adverse event information to be collected from date of enrollment until end of study participation.
Time frame: Adverse Events from Baseline to Month 12
Difference in Survival Status Between Study Groups
Determine if there is a difference in survival between participants on rasagiline than patients not on rasagiline
Time frame: Survival status at Month 12
Effect of Study Drug on Apoptosis Markers
Effect of rasagiline on the apoptosis markers (Annexin V stain) in participants with ALS. Assessed at baseline, month 6, and month 12; change from baseline to month 12 reported. Extra time point was not a pre-specified Primary or Secondary Outcome Measure.
Time frame: Apoptosis Marker change from Baseline to Month 12
Effect of Study Drug on Oxidative Stress
Determine if oxygen radical antioxidant capacity is targeted by rasagiline in participants with ALS. Assessed at baseline, month 6, and month 12; change from baseline to month 12 reported. Extra time point was not a pre-specified Primary or Secondary Outcome Measure.
Time frame: Oxidative Stress change from Baseline to Month 12
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