Trial Outcomes & Findings for Rasagiline in Subjects With Amyotrophic Lateral Sclerosis (ALS) (NCT NCT01786603)
NCT ID: NCT01786603
Last Updated: 2020-01-27
Results Overview
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).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
80 participants
Primary outcome timeframe
ALS Functional Rating Scale-Revised (ALSFRS-R) Difference from Baseline to Month 12
Results posted on
2020-01-27
Participant Flow
Participant milestones
| Measure |
Rasagiline
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Rasagiline: Rasagiline 2mg once a day for 12 months.
|
Placebo
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Placebo: Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
|
|---|---|---|
|
Overall Study
STARTED
|
60
|
20
|
|
Overall Study
COMPLETED
|
42
|
8
|
|
Overall Study
NOT COMPLETED
|
18
|
12
|
Reasons for withdrawal
| Measure |
Rasagiline
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Rasagiline: Rasagiline 2mg once a day for 12 months.
|
Placebo
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Placebo: Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
|
|---|---|---|
|
Overall Study
Death
|
5
|
1
|
|
Overall Study
Adverse Event
|
6
|
2
|
|
Overall Study
Physician decision to withdraw
|
6
|
7
|
|
Overall Study
Lost to Follow-up
|
1
|
2
|
Baseline Characteristics
Rasagiline in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Baseline characteristics by cohort
| Measure |
Rasagiline
n=60 Participants
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Rasagiline: Rasagiline 2mg once a day for 12 months.
|
Placebo
n=20 Participants
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Placebo: Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
|
Total
n=80 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
58.4 years
STANDARD_DEVIATION 10.2 • n=99 Participants
|
57.5 years
STANDARD_DEVIATION 8.5 • n=107 Participants
|
57.95 years
STANDARD_DEVIATION 9.35 • n=206 Participants
|
|
Sex: Female, Male
Female
|
20 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
27 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
40 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
53 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: ALS Functional Rating Scale-Revised (ALSFRS-R) Difference from Baseline to Month 12Difference 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).
Outcome measures
| Measure |
Rasagiline
n=60 Participants
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Rasagiline: Rasagiline 2mg once a day for 12 months.
|
Placebo
n=20 Participants
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Placebo: Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
|
|---|---|---|
|
ALS Functional Rating Scale-Revised (ALSFRS-R)
|
-1.00 score on a scale
Interval -1.22 to -0.77
|
-1.26 score on a scale
Interval -1.64 to -0.87
|
SECONDARY outcome
Timeframe: Vital Capacity Change from Baseline to Month 12Determine if decline in vital capacity is slower in participants taking 2 mg rasagiline than controls.
Outcome measures
| Measure |
Rasagiline
n=60 Participants
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Rasagiline: Rasagiline 2mg once a day for 12 months.
|
Placebo
n=20 Participants
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Placebo: Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
|
|---|---|---|
|
Change in Vital Capacity (VC)
|
-2.24 Liters
Interval -2.86 to -1.64
|
-2.48 Liters
Interval -3.54 to -1.43
|
SECONDARY outcome
Timeframe: Quality of Life Change from Baseline to Month 12Participants 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).
Outcome measures
| Measure |
Rasagiline
n=60 Participants
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Rasagiline: Rasagiline 2mg once a day for 12 months.
|
Placebo
n=20 Participants
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Placebo: Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
|
|---|---|---|
|
Change in Quality of Life
|
-0.09 Score on a scale
Interval -0.13 to -0.06
|
-0.12 Score on a scale
Interval -0.18 to -0.05
|
SECONDARY outcome
Timeframe: Adverse Events from Baseline to Month 12Determine 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.
Outcome measures
| Measure |
Rasagiline
n=60 Participants
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Rasagiline: Rasagiline 2mg once a day for 12 months.
|
Placebo
n=20 Participants
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Placebo: Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
|
|---|---|---|
|
Number of Participants With Adverse Events
|
28 Participants
|
15 Participants
|
SECONDARY outcome
Timeframe: Survival status at Month 12Determine if there is a difference in survival between participants on rasagiline than patients not on rasagiline
Outcome measures
| Measure |
Rasagiline
n=60 Participants
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Rasagiline: Rasagiline 2mg once a day for 12 months.
|
Placebo
n=20 Participants
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Placebo: Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
|
|---|---|---|
|
Difference in Survival Status Between Study Groups
|
55 Participants
|
19 Participants
|
SECONDARY outcome
Timeframe: Apoptosis Marker change from Baseline to Month 12Effect 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.
Outcome measures
| Measure |
Rasagiline
n=60 Participants
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Rasagiline: Rasagiline 2mg once a day for 12 months.
|
Placebo
n=20 Participants
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Placebo: Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
|
|---|---|---|
|
Effect of Study Drug on Apoptosis Markers
|
0.019 percentage of change
Standard Deviation 0.023
|
0.02 percentage of change
Standard Deviation 0.019
|
SECONDARY outcome
Timeframe: Oxidative Stress change from Baseline to Month 12Determine 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.
Outcome measures
| Measure |
Rasagiline
n=60 Participants
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Rasagiline: Rasagiline 2mg once a day for 12 months.
|
Placebo
n=20 Participants
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Placebo: Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
|
|---|---|---|
|
Effect of Study Drug on Oxidative Stress
|
0.14 pmole/ml
Standard Deviation 0.73
|
1.21 pmole/ml
Standard Deviation 2.34
|
Adverse Events
Rasagiline
Serious events: 0 serious events
Other events: 28 other events
Deaths: 5 deaths
Placebo
Serious events: 0 serious events
Other events: 15 other events
Deaths: 1 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Rasagiline
n=60 participants at risk
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
Rasagiline: Rasagiline 2mg once a day for 12 months.
|
Placebo
n=20 participants at risk
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
Placebo: Placebo (looks like study drug but has no active ingredients) once a day for 12 months.
|
|---|---|---|
|
Cardiac disorders
Cardiac
|
0.00%
0/60
|
5.0%
1/20 • Number of events 1
|
|
Ear and labyrinth disorders
Ear and Labyrinth
|
0.00%
0/60
|
5.0%
1/20 • Number of events 1
|
|
Eye disorders
Eye
|
5.0%
3/60 • Number of events 5
|
10.0%
2/20 • Number of events 2
|
|
Gastrointestinal disorders
Gastrointestinal
|
36.7%
22/60 • Number of events 49
|
60.0%
12/20 • Number of events 21
|
|
General disorders
General Disorders
|
20.0%
12/60 • Number of events 20
|
15.0%
3/20 • Number of events 3
|
|
Infections and infestations
Infections
|
18.3%
11/60 • Number of events 15
|
15.0%
3/20 • Number of events 4
|
|
Injury, poisoning and procedural complications
Injury, Poisoning, Procedural Complications
|
18.3%
11/60 • Number of events 25
|
15.0%
3/20 • Number of events 4
|
|
Blood and lymphatic system disorders
Laboratory Abnormality
|
10.0%
6/60 • Number of events 6
|
5.0%
1/20 • Number of events 1
|
|
Metabolism and nutrition disorders
Metabolism and Nutrition
|
16.7%
10/60 • Number of events 11
|
15.0%
3/20 • Number of events 3
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal
|
25.0%
15/60 • Number of events 25
|
45.0%
9/20 • Number of events 20
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasm
|
1.7%
1/60 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
Nervous system disorders
Nervous System
|
26.7%
16/60 • Number of events 20
|
30.0%
6/20 • Number of events 9
|
|
Psychiatric disorders
Psychiatric
|
18.3%
11/60 • Number of events 19
|
30.0%
6/20 • Number of events 11
|
|
Renal and urinary disorders
Renal and Urinary
|
13.3%
8/60 • Number of events 8
|
15.0%
3/20 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory
|
25.0%
15/60 • Number of events 25
|
35.0%
7/20 • Number of events 10
|
|
Skin and subcutaneous tissue disorders
Skin
|
8.3%
5/60 • Number of events 6
|
25.0%
5/20 • Number of events 7
|
|
Surgical and medical procedures
Surgical/Medical
|
3.3%
2/60 • Number of events 2
|
10.0%
2/20 • Number of events 2
|
|
Vascular disorders
Vascular
|
11.7%
7/60 • Number of events 10
|
40.0%
8/20 • Number of events 10
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place