Phase 2 Study of Rasagiline for Treatment of Amyotrophic Lateral Sclerosis

Study Purpose:

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).

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis (ALS)

Study Type:


Type of Intervention:


Intervention Name:

Rasagiline, Placebo



Phase 2

Study Chair(s)/Principal Investigator(s):

Richard Barohn, MD, University of Kansas Medical Center ID:


Neals Affiliated?


Coordinating Center Contact Information

University of Kansas

Kansas City, Kansas, 66160 United States

Full Study Summary:

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 Sponsor:

Richard Barohn, MD

Participant Duration:

12 months

Estimated Enrollment:


Estimated Study Start Date:

11 / 21 / 2013

Estimated Study Completion Date:

07 / 27 / 2016

Posting Last Modified Date:

01 / 27 / 2020

Date Study Added to

02 / 08 / 2013

Minimum Age:

21 Years

Maximum Age:

80 Years

Can participants use Riluzole?


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, citalopram > 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 systolic blood pressure (SBP) > 160 mmHg and/or diastolic (DBP) > 95 mmHg.

18. Use of BiPAP at screening.

Phoenix Neurological Associates

Phoenix, Arizona 85018
United States

University of California - Irvine

Irvine, California 92868
United States

California Pacific Medical Center

San Francisco, California 94115
United States

University of Kansas Medical Center

Kansas City, Kansas 66160
United States

St. Louis University

Saint Louis, Missouri 63104
United States

University of Nebraska

Omaha, Nebraska 68198
United States

Columbia University

New York, New York 10032
United States

Oregon Health and Science University

Portland, Oregon 97239
United States

University of Pennsylvania

Philadelphia, Pennsylvania 19107
United States

UT Southwestern Medical Center

Dallas, Texas 75390
United States