Trial of Safety and Efficacy of Rasagiline in Patients With Amyotrophic Lateral Sclerosis (ALS)
Primary Purpose
Amyotrophic Lateral Sclerosis (ALS)
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
rasagiline
Sponsored by
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis (ALS)
Eligibility Criteria
Inclusion Criteria:
- A clinical diagnosis of laboratory-supported probable, probable, or definite ALS, according to a modified El Escorial criteria, by the study investigator (Appendix IV).
- 21 to 80 years of age inclusive.
- VC greater or equal to 75% of predicted at screening and baseline.
- Onset of weakness within 3 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).
Exclusion criteria
- 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 propoxyphen, flexeril.
- Patients on fluoxetine or fluvoxamine.
- Patients taking amitriptyline > 50 mg/d, trazodone and sertraline > 100 mg/d, citalogram > 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.
- 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.
- VC < 75% of predicted.
- Receipt of any investigational drug within the past 30 days.
- Women with the potential to become pregnant who are not practicing effective birth control.
Sites / Locations
- Phoenix Neurological Institute
- California Pacific Medical Center
- University of Iowa
- University Of Kansas Medical Center
- University of Minnesota
- University of Nebraska Medical Center
- University of Pennsylvania
- University of Tennessee
- The Methodist Hospital System
- McGill University
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
rasagiline
Arm Description
Treated for 12 months with rasagiline 2mg orally, once daily.
Outcomes
Primary Outcome Measures
Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R)
The primary outcome measure is the difference in the rate of decline in function, as detected by the ALS Functional Rating Scale - Revised (ALSFRS-R) in patients taking rasagiline compared to a database of patients from randomized clinical trials conducted during 1997-2007. Minimum score is 0 (no function) to Maximum score is 48 (normal function)
Secondary Outcome Measures
Difference in Time to Treatment Failure
This group is defined as death, endotracheal intubation, tracheostomy-assisted ventilation or use of noninvasive ventilation >= 23 hours/day for 14 days or more.
Full Information
NCT ID
NCT01232738
First Posted
October 22, 2010
Last Updated
April 17, 2018
Sponsor
Yunxia Wang, MD
Collaborators
Western ALS Study Group
1. Study Identification
Unique Protocol Identification Number
NCT01232738
Brief Title
Trial of Safety and Efficacy of Rasagiline in Patients With Amyotrophic Lateral Sclerosis (ALS)
Official Title
A Multi-Center Controlled Screening Trial of Safety and Efficacy of Rasagiline in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Study Type
Interventional
2. Study Status
Record Verification Date
April 2018
Overall Recruitment Status
Completed
Study Start Date
December 2011 (undefined)
Primary Completion Date
May 2013 (Actual)
Study Completion Date
May 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Yunxia Wang, MD
Collaborators
Western ALS Study Group
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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. 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 and it is approved for use for patients with another disorder, the effectiveness of rasagiline for patients with ALS has not been tested.
Detailed Description
The specific aim of this screen study is to determine whether rasagiline is safe in this patient population and if the drug has the potential to slow ALS disease progression
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis (ALS)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
36 (Actual)
8. Arms, Groups, and Interventions
Arm Title
rasagiline
Arm Type
Experimental
Arm Description
Treated for 12 months with rasagiline 2mg orally, once daily.
Intervention Type
Drug
Intervention Name(s)
rasagiline
Intervention Description
rasagiline 2 mg daily for 12 months
Primary Outcome Measure Information:
Title
Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R)
Description
The primary outcome measure is the difference in the rate of decline in function, as detected by the ALS Functional Rating Scale - Revised (ALSFRS-R) in patients taking rasagiline compared to a database of patients from randomized clinical trials conducted during 1997-2007. Minimum score is 0 (no function) to Maximum score is 48 (normal function)
Time Frame
up to 12 months
Secondary Outcome Measure Information:
Title
Difference in Time to Treatment Failure
Description
This group is defined as death, endotracheal intubation, tracheostomy-assisted ventilation or use of noninvasive ventilation >= 23 hours/day for 14 days or more.
Time Frame
up to 12 months
Other Pre-specified Outcome Measures:
Title
Change in JC-1 Mitochondrial Biomarkers
Description
The 12 month change in mitochondrial biomarkerJC-1 red/green fluorescence ratio. We measured at baseline, 6 months and 12 months.
Time Frame
Baseline, 6 months, 12 months
Title
Change in Mitotracker Mitochondrial Biomarkers
Description
The 12 month change in mitochondrial biomarkerMitotracker. We measured at baseline, 6 months and 12 months.
Time Frame
Baseline, 6 months, 12 months
Title
Change in Percent Annexin V Mitochondrial Biomarkers
Description
The 12 month change in mitochondrial biomarker Annexin V %. We measured at baseline, 6 months and 12 months.
Time Frame
Baseline, 6 months, 12 months
Title
Change in BCL2/BAX Mitochondrial Biomarkers
Description
The 12 month change in mitochondrial biomarker BCL2/BAX. We measured at baseline, 6 months and 12 months.
Time Frame
Baseline, 6 months, 12 months
Title
Change in ORAC Mitochondrial Biomarkers
Description
The 12 month change in mitochondrial biomarker Oxygen Radical Antioxidant Capacity. We measured at baseline, 6 months and 12 months.
Time Frame
Baseline, 6 months, 12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
21 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
A clinical diagnosis of laboratory-supported probable, probable, or definite ALS, according to a modified El Escorial criteria, by the study investigator (Appendix IV).
21 to 80 years of age inclusive.
VC greater or equal to 75% of predicted at screening and baseline.
Onset of weakness within 3 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).
Exclusion criteria
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 propoxyphen, flexeril.
Patients on fluoxetine or fluvoxamine.
Patients taking amitriptyline > 50 mg/d, trazodone and sertraline > 100 mg/d, citalogram > 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.
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.
VC < 75% of predicted.
Receipt of any investigational drug within the past 30 days.
Women with the potential to become pregnant who are not practicing effective birth control.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yunxia Wang, MD
Organizational Affiliation
University of Kansas Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Phoenix Neurological Institute
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85018
Country
United States
Facility Name
California Pacific Medical Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94118
Country
United States
Facility Name
University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University Of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55414
Country
United States
Facility Name
University of Nebraska Medical Center
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198
Country
United States
Facility Name
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
University of Tennessee
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38104
Country
United States
Facility Name
The Methodist Hospital System
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
McGill University
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3A 2B4
Country
Canada
12. IPD Sharing Statement
Learn more about this trial
Trial of Safety and Efficacy of Rasagiline in Patients With Amyotrophic Lateral Sclerosis (ALS)
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