Rasagiline in Subjects With Amyotrophic Lateral Sclerosis (ALS)

NCT01786603 | Completed Phase 2 Results DMC

• 2 other identifiers: 12312R01FD003739
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 10

Brief Summary

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).

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Outcome Measures

Primary Outcomes (1)

• 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).

  ALS Functional Rating Scale-Revised (ALSFRS-R) Difference from Baseline to Month 12

Secondary Outcomes (6)

• Change in Vital Capacity (VC)

  Vital Capacity Change from Baseline to Month 12

• Change in Quality of Life

  Quality of Life Change from Baseline to Month 12

• Number of Participants With Adverse Events

  Adverse Events from Baseline to Month 12

• Difference in Survival Status Between Study Groups

  Survival status at Month 12

• Effect of Study Drug on Apoptosis Markers

  Apoptosis Marker change from Baseline to Month 12

Study Arms (2)

Rasagiline

EXPERIMENTAL

Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.

Drug: Rasagiline

Placebo

PLACEBO COMPARATOR

Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.

Drug: Placebo

Interventions

Rasagiline DRUG

Rasagiline 2mg once a day for 12 months.

Also known as: Azilect

Rasagiline

Placebo DRUG

Placebo (looks like study drug but has no active ingredients) once a day for 12 months.

Placebo

Eligibility Criteria

• Age: 21 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• A clinical diagnosis of laboratory-supported probable, probable, or definite ALS, according to a modified El Escorial criteria, by the study investigator (Appendix IV).
• to 80 years of age inclusive.
• VC greater or equal to 75% of predicted at screening and baseline.
• Onset of weakness within 2 years prior to enrollment.
• If patients are taking riluzole for ALS, they must be on a stable dose for at least thirty days prior to the baseline visit.
• 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.
• Willing and able to give signed informed consent that has been approved by the Institutional Review Board (IRB).

You may not qualify if:

• Requirement for tracheotomy ventilation or non-invasive ventilation for \> 23 hours per day.
• Patients on sympathomimetic agents. This includes pseudoephedrine, phenylephrine, phenylpropanolamine, and ephedrine.
• Patients on analgesics with serotoninergic properties such as meperidine, tramadol, methadone and propoxyphene, flexeril.
• Patients on fluoxetine or fluvoxamine.
• Patients taking amitriptyline \> 50 mg/d, trazodone and sertraline \> 100 mg/d, citalopram \> 20 mg/d or paroxetine \> 30 mg/d.
• Diagnosis of other neurodegenerative diseases (Parkinson disease, Alzheimer disease, etc).
• Clinically significant history of unstable medical illness (unstable angina, advanced cancer, etc) over the last 30 days.
• Has a diaphragm pacing device or plan on obtaining a diaphragm pacing device during the course of the study.
• History of renal disease.
• History of liver disease.
• Current pregnancy or lactation.
• Limited mental capacity such that the patient cannot provide written informed consent or comply with evaluation procedures.
• History of recent alcohol or drug abuse or noncompliance with treatment or other experimental protocols.
• Vital Capacity (VC) \< 75% of predicted.
• Receipt of any investigational drug within the past 30 days.

Sponsors & Collaborators

• Richard Barohn, MD: lead

Study Sites (10)

Phoenix Neurological Associates

Phoenix, Arizona, 85018, United States

Location

University of California - Irvine

Irvine, California, 92868, United States

Location

California Pacific Medical Center

San Francisco, California, 94115, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

St. Louis University

St Louis, Missouri, 63104, United States

Location

University of Nebraska

Omaha, Nebraska, 68198, United States

Location

Columbia University

New York, New York, 10032, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19107, United States

Location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

rasagiline

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Dr. Richard Barohn
• Organization: University of Kansas Medical Center

rbarohn@kumc.edu

913-945-9944

Study Officials

• Richard Barohn, MD

  University of Kansas Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Gertrude and Dewey Zeigler Professor of Neurology and Chair

Study Record Dates

• First Submitted: November 28, 2012
• First Posted: February 8, 2013
• Study Start: November 21, 2013
• Primary Completion: July 27, 2016
• Study Completion: July 27, 2016
• Last Updated: January 27, 2020
• Results First Posted: January 27, 2020

Record last verified: 2020-01

Locations

US (10)