Rasagiline in Subjects With Amyotrophic Lateral Sclerosis (ALS)

January 14, 2020 updated by: Richard Barohn, MD

Phase 2 Study of Rasagiline for Treatment of Amyotrophic Lateral Sclerosis

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 Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Interventional

Enrollment (Actual)

80

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85018
        • Phoenix Neurological Associates
    • California
      • Irvine, California, United States, 92868
        • University of California - Irvine
      • San Francisco, California, United States, 94115
        • California Pacific Medical Center
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Missouri
      • Saint Louis, Missouri, United States, 63104
        • St. Louis University
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • University of Nebraska
    • New York
      • New York, New York, United States, 10032
        • Columbia University
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • University of Pennsylvania
    • Texas
      • Dallas, Texas, United States, 75390
        • UT Southwestern Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

19 years to 78 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Rasagiline
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Drug: Rasagiline
Rasagiline 2mg once a day for 12 months.
Other Names:
  • Azilect
Placebo Comparator: Placebo
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Drug: Placebo
Placebo (looks like study drug but has no active ingredients) once a day for 12 months.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
ALS Functional Rating Scale-Revised (ALSFRS-R)
Time Frame: ALS Functional Rating Scale-Revised (ALSFRS-R) Difference from Baseline to Month 12
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).
ALS Functional Rating Scale-Revised (ALSFRS-R) Difference from Baseline to Month 12

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Vital Capacity (VC)
Time Frame: Vital Capacity Change from Baseline to Month 12
Determine if decline in vital capacity is slower in participants taking 2 mg rasagiline than controls.
Vital Capacity Change from Baseline to Month 12
Change in Quality of Life
Time Frame: Quality of Life Change from Baseline to Month 12
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).
Quality of Life Change from Baseline to Month 12
Number of Participants With Adverse Events
Time Frame: Adverse Events from Baseline to Month 12
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.
Adverse Events from Baseline to Month 12
Difference in Survival Status Between Study Groups
Time Frame: Survival status at Month 12
Determine if there is a difference in survival between participants on rasagiline than patients not on rasagiline
Survival status at Month 12
Effect of Study Drug on Apoptosis Markers
Time Frame: Apoptosis Marker change from Baseline to Month 12
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.
Apoptosis Marker change from Baseline to Month 12
Effect of Study Drug on Oxidative Stress
Time Frame: Oxidative Stress change from Baseline to Month 12
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.
Oxidative Stress change from Baseline to Month 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Richard Barohn, MD

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 21, 2013

Primary Completion (Actual)

July 27, 2016

Study Completion (Actual)

July 27, 2016

Study Registration Dates

First Submitted

November 28, 2012

First Submitted That Met QC Criteria

February 5, 2013

First Posted (Estimate)

February 8, 2013

Study Record Updates

Last Update Posted (Actual)

January 27, 2020

Last Update Submitted That Met QC Criteria

January 14, 2020

Last Verified

January 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 12312
  • R01FD003739 (U.S. FDA Grant/Contract)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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