Rasagiline in Subjects With Amyotrophic Lateral Sclerosis (ALS)
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.
Amyotrophic Lateral Sclerosis (ALS)
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
|Official Title:||Phase 2 Study of Rasagiline for Treatment of Amyotrophic Lateral Sclerosis IND# 104,360|
- Change in the ALS Functional Rating Scale-Revised (ALSFRS-R) [ Time Frame: Change from Baseline in ALSFRS-R at 12 months ] [ Designated as safety issue: No ]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).
- Change in vital capacity (VC) [ Time Frame: Change from Baseline in VC at 12 months ] [ Designated as safety issue: No ]Determine if decline in vital capacity is slower in patients taking 2 mg rasagiline than controls.
- Change in quality of life [ Time Frame: Change from Baseline in Quality of Life at 12 months ] [ Designated as safety issue: No ]determine if quality of life improves in patients taking 2 mg rasagiline
- Number of participants with adverse events [ Time Frame: up to 12 months ] [ Designated as safety issue: Yes ]Determine if patients 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.
- Difference in survival status between study groups [ Time Frame: Change from Baseline in survival status at 12 months ] [ Designated as safety issue: No ]Determine if there is a difference in survival between patients on rasagiline than patients not on rasagiline
- Bcl2Bax expression ratio in RNA samples [ Time Frame: up to 12 months ] [ Designated as safety issue: No ]Test to determine if rasagiline targets the Bcl2/Bax expression ration in RNA.
- Biomarker Assays of Mitochondrial Function [ Time Frame: Change from Baseline in Biomarker Assays at 12 months ] [ Designated as safety issue: No ]Determine if rasagiline targets mitochondrial membrane potentials. Effect determined by comparing mean slopes of the study groups.
- Effect of study drug on apoptosis markers [ Time Frame: Change from Baseline in Apoptosis Markers at 12 months ] [ Designated as safety issue: No ]Effect of rasagiline on the apoptosis markers in patients with ALS
- Effect of study drug on oxidative stress [ Time Frame: Change from Baseline in Oxidative Stress at 12 months ] [ Designated as safety issue: No ]Determine if oxygen radical antioxidant capacity is targeted by rasagiline in patients with ALS.
|Study Start Date:||September 2012|
|Estimated Study Completion Date:||August 2016|
|Estimated Primary Completion Date:||August 2016 (Final data collection date for primary outcome measure)|
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Rasagiline 2mg once a day for 12 months.
Other Name: Azilect
Placebo Comparator: Placebo
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01786603
|United States, Arizona|
|Phoenix Neurological Associates|
|Phoeniz, Arizona, United States, 85018|
|United States, California|
|University of California - Irvine|
|Irvine, California, United States, 92868|
|California Pacific Medical Center|
|San Francisco, California, United States, 94115|
|United States, Kansas|
|University of Kansas Medical Center|
|Kansas City, Kansas, United States, 66160|
|United States, Missouri|
|St. Louis University|
|St. Louis, Missouri, United States, 63104|
|United States, Nebraska|
|University of Nebraska|
|Omaha, Nebraska, United States, 68198|
|United States, New York|
|New York, New York, United States, 10032|
|United States, Oregon|
|Oregon Health and Science University|
|Portland, Oregon, United States, 97239|
|United States, Pennsylvania|
|University of Pennsylvania|
|Philadelphia, Pennsylvania, United States, 19107|
|United States, Texas|
|UT Southwestern Medical Center|
|Dallas, Texas, United States, 75390|
|Principal Investigator:||Richard Barohn, MD||University of Kansas Medical Center|