Phase 2
N=80
Rasagiline in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Bottom Line
View on ClinicalTrials.gov: NCT01786603 ↗Enrolled (actual)
80
Serious AEs
0.0%
Results posted
Jan 2020
Primary outcome: Primary: ALS Functional Rating Scale-Revised (ALSFRS-R) — -1.00; -1.26 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Rasagiline (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 21+ yrs
- Sex
- All
- Sponsor
- Richard Barohn, MD
- Primary completion
- Jul 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY ALS Functional Rating Scale-Revised (ALSFRS-R) |
-1.00; -1.26 | — |
| SECONDARY Change in Vital Capacity (VC) |
-2.24; -2.48 | — |
| SECONDARY Change in Quality of Life |
-0.09; -0.12 | — |
| SECONDARY Number of Participants With Adverse Events |
28; 15 | — |
| SECONDARY Difference in Survival Status Between Study Groups |
55; 19 | — |
| SECONDARY Effect of Study Drug on Apoptosis Markers |
0.019; 0.02 | — |
| SECONDARY Effect of Study Drug on Oxidative Stress |
0.14; 1.21 | — |
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).
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 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).
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 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) 160 mmHg and/or diastolic (DBP) > 95 mmHg.
- Use of BiPAP at screening.
Data sourced from ClinicalTrials.gov (NCT01786603). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.