Clinical trial NCT01232738: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis (ALS)
Lead sponsor: University of Kansas Medical Center
Sponsor class: OTHER
Collaborators: name: Western ALS Study Group
First posted: 2010-11-02
Enrollment: 36
Sex: ALL
Minimum age (years): 21
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Eligibility criteria: 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 3 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 propoxyphen, 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. History of renal disease.
9. History of liver disease.
10. Current pregnancy or lactation.
11. Limited mental capacity such that the patient cannot provide written informed consent or comply with evaluation procedures.
12. History of recent alcohol or drug abuse or noncompliance with treatment or other experimental protocols.
13. VC \< 75% of predicted.
14. Receipt of any investigational drug within the past 30 days.
15. Women with the potential to become pregnant who are not practicing effective birth control.

Primary outcomes:
measure: Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R)
timeFrame: up to 12 months
Secondary outcomes:
measure: Difference in Time to Treatment Failure
timeFrame: up to 12 months
Other outcomes:
measure: Change in JC-1 Mitochondrial Biomarkers
timeFrame: Baseline, 6 months, 12 months
measure: Change in Mitotracker Mitochondrial Biomarkers
timeFrame: Baseline, 6 months, 12 months
measure: Change in Percent Annexin V Mitochondrial Biomarkers
timeFrame: Baseline, 6 months, 12 months
measure: Change in BCL2/BAX Mitochondrial Biomarkers
timeFrame: Baseline, 6 months, 12 months
measure: Change in ORAC Mitochondrial Biomarkers
timeFrame: Baseline, 6 months, 12 months
Locations:
city: Phoenix
country: United States
facility: Phoenix Neurological Institute
state: Arizona
city: San Francisco
country: United States
facility: California Pacific Medical Center
state: California
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Kansas City
country: United States
facility: University Of Kansas Medical Center
state: Kansas
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Memphis
country: United States
facility: University of Tennessee
state: Tennessee
city: Houston
country: United States
facility: The Methodist Hospital System
state: Texas
city: Montreal
country: Canada
facility: McGill University
state: Quebec
Study officials:
affiliation: University of Kansas Medical Center
name: Yunxia Wang, MD
role: PRINCIPAL_INVESTIGATOR
References: