This trial is active, not recruiting.

Condition amyotrophic lateral sclerosis (als)
Treatments rasagiline, placebo
Phase phase 2
Sponsor Richard Barohn, MD
Start date September 2012
End date August 2016
Trial size 80 participants
Trial identifier NCT01786603, 12312, RO1FD003739


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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model crossover assignment
Masking double blind (subject, caregiver, investigator)
Primary purpose treatment
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
rasagiline Azilect
Rasagiline 2mg once a day for 12 months.
(Placebo Comparator)
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.

Primary Outcomes

Change in the ALS Functional Rating Scale-Revised (ALSFRS-R)
time frame: Change from Baseline in ALSFRS-R at 12 months

Secondary Outcomes

Change in vital capacity (VC)
time frame: Change from Baseline in VC at 12 months
Change in quality of life
time frame: Change from Baseline in Quality of Life at 12 months
Number of participants with adverse events
time frame: up to 12 months
Difference in survival status between study groups
time frame: Change from Baseline in survival status at 12 months
Bcl2Bax expression ratio in RNA samples
time frame: up to 12 months
Biomarker Assays of Mitochondrial Function
time frame: Change from Baseline in Biomarker Assays at 12 months
Effect of study drug on apoptosis markers
time frame: Change from Baseline in Apoptosis Markers at 12 months
Effect of study drug on oxidative stress
time frame: Change from Baseline in Oxidative Stress at 12 months

Eligibility Criteria

Male or female participants from 21 years up to 80 years old.

Inclusion Criteria: 1. A clinical diagnosis of laboratory-supported probable, probable, or definite ALS, according to a modified El Escorial criteria, by the study investigator (Appendix IV). 2. 21 to 80 years of age inclusive. 3. VC greater or equal to 75% of predicted at screening and baseline. 4. Onset of weakness within 2 years prior to enrollment. 5. If patients are taking riluzole for ALS, they must be on a stable dose for at least thirty days prior to the baseline visit. 6. Women of childbearing age must be non-lactating and surgically sterile or using an effective method of birth control and have a negative pregnancy test. 7. Willing and able to give signed informed consent that has been approved by the Institutional Review Board (IRB). Exclusion criteria 1. Requirement for tracheotomy ventilation or non-invasive ventilation for > 23 hours per day. 2. Patients on sympathomimetic agents. This includes pseudoephedrine, phenylephrine, phenylpropanolamine, and ephedrine. 3. Patients on analgesics with serotoninergic properties such as meperidine, tramadol, methadone and propoxyphene, flexeril. 4. Patients on fluoxetine or fluvoxamine. 5. Patients taking amitriptyline > 50 mg/d, trazodone and sertraline > 100 mg/d, citalogram > 20 mg/d or paroxetine > 30 mg/d. 6. Diagnosis of other neurodegenerative diseases (Parkinson disease, Alzheimer disease, etc). 7. Clinically significant history of unstable medical illness (unstable angina, advanced cancer, etc) over the last 30 days. 8. Has a diaphragm pacing device or plan on obtaining a diaphragm pacing device during the course of the study. 9. History of renal disease. 10. History of liver disease. 11. Current pregnancy or lactation. 12. Limited mental capacity such that the patient cannot provide written informed consent or comply with evaluation procedures. 13. History of recent alcohol or drug abuse or noncompliance with treatment or other experimental protocols. 14. Vital Capacity (VC) < 75% of predicted. 15. Receipt of any investigational drug within the past 30 days. 16. Women with the potential to become pregnant who are not practicing effective birth control. 17. Poorly controlled hypertensive subjects or resting blood pressure SBP > 160 mmHg and/or DBP > 95 mmHg. 18. Use of BiPAP at screening.

Additional Information

Official title Phase 2 Study of Rasagiline for Treatment of Amyotrophic Lateral Sclerosis IND# 104,360
Principal investigator Richard Barohn, MD
Description 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.
Trial information was received from ClinicalTrials.gov and was last updated in May 2016.
Information provided to ClinicalTrials.gov by University of Kansas Medical Center.