Trial Outcomes & Findings for Mirabegron in Parkinson Disease and Impaired Cognition (NCT NCT02536976)

NCT ID: NCT02536976

Last Updated: 2019-05-08

Results Overview

Change in Montreal Cognitive Assessment Total Score between week 2 and week 14. The Montreal Cognitive Assessment is a screening tool for global cognitive function with a total score ranging from 0 to 30 units on a scale, with higher score indicating better cognitive global function. Normal range is 26 thru 30.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

7 participants

Primary outcome timeframe

From Week 2 to Week 14

Results posted on

2019-05-08

Participant Flow

Participants were recruited in a single site from an ambulatory patient population of a dedicated Parkinson's Disease Center. First participant was screened in 12/2015 and the last participant completed the study in January 2018

17 participants signed the ICF. There were 10 screen failures. 7 participants enrolled. 2 participants withdrew consent before starting study drug, and 1 was terminated during the placebo run-in phase due to unrelated failing health. 4 participants completed all study activities.

Participant milestones

Participant milestones
Measure
Active Treatment
Participants received 1 daily placebo tablet matching 25 mg tablets of mirabegron for 2 weeks , followed by 1 daily 25 mg tablet of mirabegron for 2 weeks, followed by 2 daily 25 mg tablets of mirabegron for 10 weeks.
Placebo
Participants received 1 daily placebo tablet matching 25 mg tablets of mirabegron for 4 weeks , followed by 2 daily placebo tablets matching 25 mg tablets of mirabegron for 10 weeks.
Overall Study
STARTED
3
2
Overall Study
Randomization
3
1
Overall Study
Dose Escalation and Maintenance
3
1
Overall Study
Washout
3
1
Overall Study
COMPLETED
3
1
Overall Study
NOT COMPLETED
0
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Active Treatment
Participants received 1 daily placebo tablet matching 25 mg tablets of mirabegron for 2 weeks , followed by 1 daily 25 mg tablet of mirabegron for 2 weeks, followed by 2 daily 25 mg tablets of mirabegron for 10 weeks.
Placebo
Participants received 1 daily placebo tablet matching 25 mg tablets of mirabegron for 4 weeks , followed by 2 daily placebo tablets matching 25 mg tablets of mirabegron for 10 weeks.
Overall Study
Physician Decision
0
1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Active Treatment
n=3 Participants
2 weeks single blind placebo followed by 2 weeks double blind 25 mg mirabegron, followed by 10 weeks double blind 50 mg mirabegron
Placebo
n=2 Participants
2 weeks single blind placebo followed by 2 weeks double blind placebo matching 25 mg mirabegron, followed by 10 weeks double blind 50 mg mirabegron
Total
n=5 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=3 Participants
0 Participants
n=2 Participants
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
2 Participants
n=3 Participants
0 Participants
n=2 Participants
2 Participants
n=5 Participants
Age, Categorical
>=65 years
1 Participants
n=3 Participants
2 Participants
n=2 Participants
3 Participants
n=5 Participants
Sex: Female, Male
Female
2 Participants
n=3 Participants
2 Participants
n=2 Participants
4 Participants
n=5 Participants
Sex: Female, Male
Male
1 Participants
n=3 Participants
0 Participants
n=2 Participants
1 Participants
n=5 Participants
Race and Ethnicity Not Collected
0 Participants
Race and Ethnicity were not collected from any participant.
Region of Enrollment
United States
3 participants
n=3 Participants
2 participants
n=2 Participants
5 participants
n=5 Participants

PRIMARY outcome

Timeframe: From Week 2 to Week 14

Change in Montreal Cognitive Assessment Total Score between week 2 and week 14. The Montreal Cognitive Assessment is a screening tool for global cognitive function with a total score ranging from 0 to 30 units on a scale, with higher score indicating better cognitive global function. Normal range is 26 thru 30.

Outcome measures

Outcome measures
Measure
Active Treatment
n=3 Participants
Due to sample size formal statistics were not performed. Observations tabulated below: Subject ID 8801 8814 8816 Change +2 +2 -1
Placebo
n=1 Participants
Due to sample size formal statistics were not performed. Observations tabulated below: Subject ID 8805 Change +2
Change in Montreal Cognitive Assessment Total Score
1.5 units on a scale
Interval -1.0 to 2.0
2 units on a scale
Interval 2.0 to 2.0

SECONDARY outcome

Timeframe: From Week 2 to Week 14

The Overactive Bladder Questionnaire (OABQ) is a 33-item, self-administered instrument that contains a symptom bother scale (8 items) and a health-related quality of life (HRQL) scale (25 items), pertaining to OAB symptoms impact on HRQL. Symptom bother score ranges from 0-100 with higher scores indicating greater severity of symptoms. The HRQL score ranges from 0-100 with higher scores indicating better quality of life.

Outcome measures

Outcome measures
Measure
Active Treatment
n=3 Participants
Due to sample size formal statistics were not performed. Observations tabulated below: Subject ID 8801 8814 8816 Change +2 +2 -1
Placebo
n=1 Participants
Due to sample size formal statistics were not performed. Observations tabulated below: Subject ID 8805 Change +2
Change in Overactive Bladder Questionnaire Subscale Scores
Symptom Severity
-13.3 units on a scale
Interval -25.0 to 5.0
12.5 units on a scale
Interval 12.5 to 12.5
Change in Overactive Bladder Questionnaire Subscale Scores
HRQL
13.7 units on a scale
Interval 0.0 to 25.0
5 units on a scale
Interval 5.0 to 5.0

SECONDARY outcome

Timeframe: From Week 2 to Week 14

The UPDRS assesses motor and functional abilities of the subjects as it pertains to Parkinson's disease. The total UPDRS score (range 0-199; defined for this study as the sum of Parts I, II, III, and IV (I-Mentation, behavior, and mood section (4 items; range 0-16); II-Activities of Daily Living (ADL; 13 items; range 0-52); III-motor section (27 items; range 0-108) and IV-complications section; 11 items; range 0-23) will be completed by history and examination. Higher scores indicate greater severity of Parkinson's disease symptoms.

Outcome measures

Outcome measures
Measure
Active Treatment
n=3 Participants
Due to sample size formal statistics were not performed. Observations tabulated below: Subject ID 8801 8814 8816 Change +2 +2 -1
Placebo
n=1 Participants
Due to sample size formal statistics were not performed. Observations tabulated below: Subject ID 8805 Change +2
Change in Unified Parkinson's Disease Rating Scale
-5.3 units on a scale
Interval -16.0 to 6.0
-9 units on a scale
Interval -9.0 to -9.0

Adverse Events

Active Treatment

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Placebo

Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Active Treatment
n=3 participants at risk
Participants received 1 daily placebo tablet matching 25 mg tablets of mirabegron for 2 weeks , followed by 1 daily 25 mg tablet of mirabegron for 2 weeks, followed by 2 daily 25 mg tablets of mirabegron for 10 weeks.
Placebo
n=2 participants at risk
Participants received 1 daily placebo tablet matching 25 mg tablets of mirabegron for 4 weeks , followed by 2 daily placebo tablets matching 25 mg tablets of mirabegron for 10 weeks.
Respiratory, thoracic and mediastinal disorders
Pneumonia
0.00%
0/3 • 16 weeks
50.0%
1/2 • Number of events 1 • 16 weeks
Metabolism and nutrition disorders
hyponatremia
0.00%
0/3 • 16 weeks
50.0%
1/2 • Number of events 1 • 16 weeks
Gastrointestinal disorders
Diarrhea
0.00%
0/3 • 16 weeks
50.0%
1/2 • Number of events 1 • 16 weeks
Gastrointestinal disorders
constipation
0.00%
0/3 • 16 weeks
50.0%
1/2 • Number of events 1 • 16 weeks
Nervous system disorders
headache
0.00%
0/3 • 16 weeks
50.0%
1/2 • Number of events 1 • 16 weeks
Respiratory, thoracic and mediastinal disorders
upper respiratory infection
0.00%
0/3 • 16 weeks
50.0%
1/2 • Number of events 1 • 16 weeks
Nervous system disorders
cognitive dysfunction
0.00%
0/3 • 16 weeks
50.0%
1/2 • Number of events 1 • 16 weeks

Additional Information

Dr. Sotirios Parashos

Struthers Parkinson's Center

Phone: 001-952-993-5495

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place