Rasagiline in Subjects With Amyotrophic Lateral Sclerosis (ALS)

• ClinicalTrials.gov Identifier: NCT01786603
• Recruitment Status: Completed
• First Posted: February 8, 2013
• Results First Posted: January 27, 2020
• Last Update Posted: January 27, 2020
• Sponsor: Richard Barohn, MD
• Information provided by (Responsible Party): Richard Barohn, MD, University of Kansas Medical Center

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment
• Condition: Amyotrophic Lateral Sclerosis (ALS)
• Interventions: Drug: Rasagiline; Drug: Placebo
• Enrollment: 80

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Rasagiline	Placebo
Arm/Group Description	Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months. Rasagiline: Rasagiline 2mg once a day for 12 months.	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.
Period Title: Overall Study
Started	60	20
Completed	42	8
Not Completed	18	12
Reason Not Completed
Death	5	1
Adverse Event	6	2
Physician decision to withdraw	6	7
Lost to Follow-up	1	2

Baseline Characteristics

Arm/Group Title		Rasagiline	Placebo	Total
Arm/Group Description		Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months. Rasagiline: Rasagiline 2mg once a day for 12 months.	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 of all reporting groups
Overall Number of Baseline Participants		60	20	80
Baseline Analysis Population Description		[Not Specified]
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	60 participants	20 participants	80 participants
		58.4 (10.2)	57.5 (8.5)	57.95 (9.35)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	60 participants	20 participants	80 participants
	Female	20 33.3%	7 35.0%	27 33.8%
	Male	40 66.7%	13 65.0%	53 66.3%

Outcome Measures

1. Primary Outcome

Title	ALS Functional Rating Scale-Revised (ALSFRS-R)
Description	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).
Time Frame	ALS Functional Rating Scale-Revised (ALSFRS-R) Difference from Baseline to Month 12

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Rasagiline	Placebo
Arm/Group Description:	Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months. Rasagiline: Rasagiline 2mg once a day for 12 months.	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 Number of Participants Analyzed	60	20
Mean (95% Confidence Interval); Unit of Measure: score on a scale
	-1.00; (-1.22 to -0.77)	-1.26; (-1.64 to -0.87)

2. Secondary Outcome

Title	Change in Vital Capacity (VC)
Description	Determine if decline in vital capacity is slower in participants taking 2 mg rasagiline than controls.
Time Frame	Vital Capacity Change from Baseline to Month 12

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Rasagiline	Placebo
Arm/Group Description:	Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months. Rasagiline: Rasagiline 2mg once a day for 12 months.	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 Number of Participants Analyzed	60	20
Mean (95% Confidence Interval); Unit of Measure: Liters
	-2.24; (-2.86 to -1.64)	-2.48; (-3.54 to -1.43)

3. Secondary Outcome

Title	Change in Quality of Life
Description	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).
Time Frame	Quality of Life Change from Baseline to Month 12

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Rasagiline	Placebo
Arm/Group Description:	Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months. Rasagiline: Rasagiline 2mg once a day for 12 months.	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 Number of Participants Analyzed	60	20
Mean (95% Confidence Interval); Unit of Measure: Score on a scale
	-0.09; (-0.13 to -0.06)	-0.12; (-0.18 to -0.05)

4. Secondary Outcome

Title	Number of Participants With Adverse Events
Description	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.
Time Frame	Adverse Events from Baseline to Month 12

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Rasagiline	Placebo
Arm/Group Description:	Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months. Rasagiline: Rasagiline 2mg once a day for 12 months.	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 Number of Participants Analyzed	60	20
Measure Type: Count of Participants; Unit of Measure: Participants
	28 46.7%	15 75.0%

5. Secondary Outcome

Title	Difference in Survival Status Between Study Groups
Description	Determine if there is a difference in survival between participants on rasagiline than patients not on rasagiline
Time Frame	Survival status at Month 12

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Rasagiline	Placebo
Arm/Group Description:	Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months. Rasagiline: Rasagiline 2mg once a day for 12 months.	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 Number of Participants Analyzed	60	20
Measure Type: Count of Participants; Unit of Measure: Participants
	55 91.7%	19 95.0%

6. Secondary Outcome

Title	Effect of Study Drug on Apoptosis Markers
Description	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.
Time Frame	Apoptosis Marker change from Baseline to Month 12

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Rasagiline	Placebo
Arm/Group Description:	Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months. Rasagiline: Rasagiline 2mg once a day for 12 months.	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 Number of Participants Analyzed	60	20
Mean (Standard Deviation); Unit of Measure: percentage of change
	0.019 (0.023)	0.02 (0.019)

7. Secondary Outcome

Title	Effect of Study Drug on Oxidative Stress
Description	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.
Time Frame	Oxidative Stress change from Baseline to Month 12

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Rasagiline	Placebo
Arm/Group Description:	Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months. Rasagiline: Rasagiline 2mg once a day for 12 months.	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 Number of Participants Analyzed	60	20
Mean (Standard Deviation); Unit of Measure: pmole/ml
	0.14 (0.73)	1.21 (2.34)

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Rasagiline		Placebo
Arm/Group Description	Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months. Rasagiline: Rasagiline 2mg once a day for 12 months.		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.
All-Cause Mortality
	Rasagiline		Placebo
	Affected / at Risk (%)		Affected / at Risk (%)
Total	5/60 (8.33%)		1/20 (5.00%)
Serious Adverse Events
	Rasagiline		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/60 (0.00%)		0/20 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Rasagiline		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	28/60 (46.67%)		15/20 (75.00%)
Blood and lymphatic system disorders
Laboratory Abnormality †	6/60 (10.00%)	6	1/20 (5.00%)	1
Cardiac disorders
Cardiac †	0/60 (0.00%)	0	1/20 (5.00%)	1
Ear and labyrinth disorders
Ear and Labyrinth †	0/60 (0.00%)	0	1/20 (5.00%)	1
Eye disorders
Eye †	3/60 (5.00%)	5	2/20 (10.00%)	2
Gastrointestinal disorders
Gastrointestinal †	22/60 (36.67%)	49	12/20 (60.00%)	21
General disorders
General Disorders †	12/60 (20.00%)	20	3/20 (15.00%)	3
Infections and infestations
Infections †	11/60 (18.33%)	15	3/20 (15.00%)	4
Injury, poisoning and procedural complications
Injury, Poisoning, Procedural Complications †	11/60 (18.33%)	25	3/20 (15.00%)	4
Metabolism and nutrition disorders
Metabolism and Nutrition †	10/60 (16.67%)	11	3/20 (15.00%)	3
Musculoskeletal and connective tissue disorders
Musculoskeletal †	15/60 (25.00%)	25	9/20 (45.00%)	20
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasm †	1/60 (1.67%)	1	1/20 (5.00%)	1
Nervous system disorders
Nervous System †	16/60 (26.67%)	20	6/20 (30.00%)	9
Psychiatric disorders
Psychiatric †	11/60 (18.33%)	19	6/20 (30.00%)	11
Renal and urinary disorders
Renal and Urinary †	8/60 (13.33%)	8	3/20 (15.00%)	3
Respiratory, thoracic and mediastinal disorders
Respiratory †	15/60 (25.00%)	25	7/20 (35.00%)	10
Skin and subcutaneous tissue disorders
Skin †	5/60 (8.33%)	6	5/20 (25.00%)	7
Surgical and medical procedures
Surgical/Medical †	2/60 (3.33%)	2	2/20 (10.00%)	2
Vascular disorders
Vascular †	7/60 (11.67%)	10	8/20 (40.00%)	10
† Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Dr. Richard Barohn
• Organization: University of Kansas Medical Center
• Phone: 913-945-9944
• EMail: rbarohn@kumc.edu

• Responsible Party: Richard Barohn, MD, University of Kansas Medical Center
• ClinicalTrials.gov Identifier: NCT01786603
• Other Study ID Numbers: 12312; R01FD003739 ( U.S. FDA Grant/Contract )
• First Submitted: November 28, 2012
• First Posted: February 8, 2013
• Results First Submitted: October 15, 2019
• Results First Posted: January 27, 2020
• Last Update Posted: January 27, 2020