Amyotrophic Lateral Sclerosis (ALS)Phase 2NCT01786603
Rasagiline in Subjects With Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing Rasagiline in people with Amyotrophic Lateral Sclerosis (ALS), run by Richard Barohn, MD at 10 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 21 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- Rasagiline
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 10 sitesUnited States
- Study dates
- 21 Nov 2013to 27 Jul 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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).
Show all 18Show fewer
- 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.
- Women with the potential to become pregnant who are not practicing effective birth control.
- Poorly controlled hypertensive subjects or resting systolic blood pressure (SBP) > 160 mmHg and/or diastolic (DBP) > 95 mmHg.
- Use of BiPAP at screening.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
UT Southwestern Medical Center
Dallas, Texas, 75390
University of California - Irvine
Irvine, California, 92868
University of Kansas Medical Center
Kansas City, Kansas, 66160
Columbia University
New York, New York, 10032
University of Nebraska
Omaha, Nebraska, 68198
University of Pennsylvania
Philadelphia, Pennsylvania, 19107
Phoenix Neurological Associates
Phoenix, Arizona, 85018
Oregon Health and Science University
Portland, Oregon, 97239
California Pacific Medical Center
San Francisco, California, 94115
St. Louis University
St Louis, Missouri, 63104
About this study
Official title: 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.
Read more
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).
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- Rasagiline, Placebo
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RasagilineDrug
Also called Azilect
Rasagiline 2mg once a day for 12 months.
PlaceboDrug
Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
| Study group | Role | What they receive |
|---|---|---|
| Rasagiline | Experimental | Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months. Receives: Drug: Rasagiline |
| Placebo | Placebo comparator | Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: ALS Functional Rating Scale-Revised (ALSFRS-R) Difference from Baseline to Month 12
Secondary outcomes (6)
Change in Vital Capacity (VC)
Determine if decline in vital capacity is slower in participants taking 2 mg rasagiline than controls.
Measured: Vital Capacity Change from Baseline to Month 12
Change in Quality of Life
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).
Measured: Quality of Life Change from Baseline to Month 12
Number of Participants With Adverse Events
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.
Measured: Adverse Events from Baseline to Month 12
Difference in Survival Status Between Study Groups
Determine if there is a difference in survival between participants on rasagiline than patients not on rasagiline
Measured: Survival status at Month 12
Effect of Study Drug on Apoptosis Markers
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.
Measured: Apoptosis Marker change from Baseline to Month 12
Effect of Study Drug on Oxidative Stress
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.
Measured: Oxidative Stress change from Baseline to Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2013 (estimated)
- Study start21 Nov 2013
- Primary completion27 Jul 2016
- Study completion27 Jul 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- Richard Barohn, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Richard Barohn, MD, University of Kansas Medical Center
- Investigators
- Richard Barohn, MD, Principal investigator, University of Kansas Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01786603
- Sponsor's protocol ID
- 12312
- Other IDs
- R01FD003739
- Status last verified
- Jan 2020
- First submitted
- 28 Nov 2012
- Last updated
- 27 Jan 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Witzel S, Statland JM, Steinacker P, Otto M, Dorst J, Schuster J, Barohn RJ, Ludolph AC. Longitudinal course of neurofilament light chain levels in amyotrophic lateral sclerosis-insights from a completed randomized controlled trial with rasagiline. Eur J Neurol. 2024 Mar;31(3):e16154. doi: 10.1111/ene.16154. Epub 2023 Nov 17.
PubMed 37975796
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study of Rasagiline for Treatment of Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT01786603
- Acronym / sponsor ID
- 12312
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 80 participants (actual)
- Lead sponsor
- Richard Barohn, MDUniversity, hospital or other
- Study start
- 21 Nov 2013 (actual)
- Primary completion
- 27 Jul 2016 (actual)
- Study completion
- 27 Jul 2016 (actual)
- First posted
- 8 Feb 2013 (estimated)
- Results
- Posted 27 Jan 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Rasagiline | Experimental | Drug: RasagilineRasagiline 1mg administered orally as a 2mg single dose once daily for 12 months. |
| Placebo | Placebo comparator | Drug: PlaceboInactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months. |
Who may qualify
- Ages
- 21 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (18 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RasagilineAlso: AzilectRasagiline 2mg once a day for 12 months. | Drug | Rasagiline |
| PlaceboPlacebo (looks like study drug but has no active ingredients) once a day for 12 months. | Drug | Placebo |
Primary outcome
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).
6 secondary outcomes
Change in Vital Capacity (VC)
Time frame: Vital Capacity Change from Baseline to Month 12
Change in Quality of Life
Time frame: Quality of Life Change from Baseline to Month 12
Number of Participants With Adverse Events
Time frame: Adverse Events from Baseline to Month 12
Difference in Survival Status Between Study Groups
Time frame: Survival status at Month 12
Effect of Study Drug on Apoptosis Markers
Time frame: 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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37975796Witzel S, Statland JM, Steinacker P, Otto M, Dorst J, Schuster J, Barohn RJ, Ludolph AC. Longitudinal course of neurofilament light chain levels in amyotrophic lateral sclerosis-insights from a completed randomized controlled trial with rasagiline. Eur J Neurol. 2024 Mar;31(3):e16154. doi: 10.1111/ene.16154. Epub 2023 Nov 17.
Related trials for Amyotrophic Lateral Sclerosis (ALS)
RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), Bacillus Coagulans
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Not yet recruitingPhase 1
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Recruiting
Clinical Outcome Assessment for AT & BCI
Spinal Cord Injury, ALS (Amyotrophic Lateral Sclerosis)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Richard Barohn, MD principal investigator
University of Kansas Medical Center
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rasagiline in people with Amyotrophic Lateral Sclerosis (ALS), run by Richard Barohn, MD at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Jan 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ALS Functional Rating Scale-Revised (ALSFRS-R) (time frame: ALS Functional Rating Scale-Revised (ALSFRS-R) Difference from Baseline to Month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01786603, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jan 2020.