Trial Outcomes & Findings for Norepinephrine-targeted Therapy for Action Control in Parkinson Disease (NCT NCT03115827)

NCT ID: NCT03115827

Last Updated: 2020-01-27

Results Overview

Safety will be defined by the percent of subjects who develop an adverse event during the 7-week treatment period that is determined to be likely related to the study medications.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 15 participants
• Primary outcome timeframe: 7 weeks
• Results posted on: 2020-01-27

Participant Flow

Participant milestones

Measure	Arm 1 Droxidopa 600mg by mouth twice a day and carbidopa 200mg by mouth twice a day for 4 weeks Droxidopa: Droxidopa will be started at 100mg twice a day and titrated up to a maximum of 600mg twice a day Carbidopa: Carbidopa 200mg twice a day
Overall Study STARTED	15
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Norepinephrine-targeted Therapy for Action Control in Parkinson Disease

Baseline characteristics by cohort

Measure	Arm 1 n=15 Participants Droxidopa 600mg by mouth twice a day and carbidopa 200mg by mouth twice a day for 4 weeks Droxidopa: Droxidopa will be started at 100mg twice a day and titrated up to a maximum of 600mg twice a day Carbidopa: Carbidopa 200mg twice a day
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	11 Participants n=5 Participants
Age, Continuous	69.6 years STANDARD_DEVIATION 10 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	15 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	15 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants

PRIMARY outcome

Timeframe: 7 weeks

Safety will be defined by the percent of subjects who develop an adverse event during the 7-week treatment period that is determined to be likely related to the study medications.

Outcome measures

Measure	Arm 1 n=15 Participants Droxidopa 600mg by mouth twice a day and carbidopa 200mg by mouth twice a day for 4 weeks Droxidopa: Droxidopa will be started at 100mg twice a day and titrated up to a maximum of 600mg twice a day Carbidopa: Carbidopa 200mg twice a day
Number of Subjects Who Develop an Adverse Event During the 7-week Treatment Period That is Determined to be Likely Related to the Study Medications.	8 Participants

PRIMARY outcome

Timeframe: 7 weeks

Tolerability will be defined by the number of patients who discontinue the study drug due to adverse effects.

Outcome measures

Measure	Arm 1 n=15 Participants Droxidopa 600mg by mouth twice a day and carbidopa 200mg by mouth twice a day for 4 weeks Droxidopa: Droxidopa will be started at 100mg twice a day and titrated up to a maximum of 600mg twice a day Carbidopa: Carbidopa 200mg twice a day
Number of Participants Who Discontinue the Study Drug Due to Adverse Effects During the 7-week Treatment Period. Participants that discontinued due to SAEs	2 Participants
Number of Participants Who Discontinue the Study Drug Due to Adverse Effects During the 7-week Treatment Period. Participants that discontinued due to mild AEs	1 Participants
Number of Participants Who Discontinue the Study Drug Due to Adverse Effects During the 7-week Treatment Period. Participants that did not discontinue	12 Participants

SECONDARY outcome

Timeframe: Week 4 to Week 7

The mean maximum tolerated dose of droxidopa reached by the study participants

Outcome measures

Measure	Arm 1 n=15 Participants Droxidopa 600mg by mouth twice a day and carbidopa 200mg by mouth twice a day for 4 weeks Droxidopa: Droxidopa will be started at 100mg twice a day and titrated up to a maximum of 600mg twice a day Carbidopa: Carbidopa 200mg twice a day
Maximum Tolerated Dose	1053 mg/day Standard Deviation 267

SECONDARY outcome

Timeframe: 7 weeks

Percent compliance is defined as the percent of study participants who take greater than or equal to 70% of the assigned dosage

Outcome measures

Measure	Arm 1 n=15 Participants Droxidopa 600mg by mouth twice a day and carbidopa 200mg by mouth twice a day for 4 weeks Droxidopa: Droxidopa will be started at 100mg twice a day and titrated up to a maximum of 600mg twice a day Carbidopa: Carbidopa 200mg twice a day
Percent Compliance	11 Participants

SECONDARY outcome

Timeframe: baseline and week 7

The Stop-Signal reaction time is a computerized test that assesses reaction time and response inhibition

Outcome measures

Measure	Arm 1 n=15 Participants Droxidopa 600mg by mouth twice a day and carbidopa 200mg by mouth twice a day for 4 weeks Droxidopa: Droxidopa will be started at 100mg twice a day and titrated up to a maximum of 600mg twice a day Carbidopa: Carbidopa 200mg twice a day
Change in Stop-Signal Reaction Time From Baseline to Week 7	-14.38 change in SSRT in seconds Standard Deviation 40.42

Adverse Events

Arm 1

Serious events: 2 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Measure	Arm 1 n=15 participants at risk Droxidopa 600mg by mouth twice a day and carbidopa 200mg by mouth twice a day for 4 weeks Droxidopa: Droxidopa will be started at 100mg twice a day and titrated up to a maximum of 600mg twice a day Carbidopa: Carbidopa 200mg twice a day
Gastrointestinal disorders transient cholecystitis	6.7% 1/15 • Number of events 1 • 11 weeks The definitions used to collect the adverse event information does not differ from the clinicaltrials.gov definitions. Serious adverse event information was collected over the phone at the time of onset. Adverse Event information was collected at each study visit including baseline, visit 1, visit 2, visit 3, and the follow-up visit.
Gastrointestinal disorders ventral hernia	6.7% 1/15 • Number of events 1 • 11 weeks The definitions used to collect the adverse event information does not differ from the clinicaltrials.gov definitions. Serious adverse event information was collected over the phone at the time of onset. Adverse Event information was collected at each study visit including baseline, visit 1, visit 2, visit 3, and the follow-up visit.

Other adverse events

Measure	Arm 1 n=15 participants at risk Droxidopa 600mg by mouth twice a day and carbidopa 200mg by mouth twice a day for 4 weeks Droxidopa: Droxidopa will be started at 100mg twice a day and titrated up to a maximum of 600mg twice a day Carbidopa: Carbidopa 200mg twice a day
Gastrointestinal disorders Nausea	13.3% 2/15 • Number of events 2 • 11 weeks The definitions used to collect the adverse event information does not differ from the clinicaltrials.gov definitions. Serious adverse event information was collected over the phone at the time of onset. Adverse Event information was collected at each study visit including baseline, visit 1, visit 2, visit 3, and the follow-up visit.
General disorders Dry Mouth	6.7% 1/15 • Number of events 1 • 11 weeks The definitions used to collect the adverse event information does not differ from the clinicaltrials.gov definitions. Serious adverse event information was collected over the phone at the time of onset. Adverse Event information was collected at each study visit including baseline, visit 1, visit 2, visit 3, and the follow-up visit.
Nervous system disorders Increased Freezing	6.7% 1/15 • Number of events 1 • 11 weeks The definitions used to collect the adverse event information does not differ from the clinicaltrials.gov definitions. Serious adverse event information was collected over the phone at the time of onset. Adverse Event information was collected at each study visit including baseline, visit 1, visit 2, visit 3, and the follow-up visit.
Eye disorders Conjunctival Hemhorrage	6.7% 1/15 • Number of events 1 • 11 weeks The definitions used to collect the adverse event information does not differ from the clinicaltrials.gov definitions. Serious adverse event information was collected over the phone at the time of onset. Adverse Event information was collected at each study visit including baseline, visit 1, visit 2, visit 3, and the follow-up visit.
Cardiac disorders Palpitations	6.7% 1/15 • Number of events 1 • 11 weeks The definitions used to collect the adverse event information does not differ from the clinicaltrials.gov definitions. Serious adverse event information was collected over the phone at the time of onset. Adverse Event information was collected at each study visit including baseline, visit 1, visit 2, visit 3, and the follow-up visit.
Psychiatric disorders Brief Confusional Episode	6.7% 1/15 • Number of events 1 • 11 weeks The definitions used to collect the adverse event information does not differ from the clinicaltrials.gov definitions. Serious adverse event information was collected over the phone at the time of onset. Adverse Event information was collected at each study visit including baseline, visit 1, visit 2, visit 3, and the follow-up visit.
General disorders Influenza-like symptoms	6.7% 1/15 • Number of events 1 • 11 weeks The definitions used to collect the adverse event information does not differ from the clinicaltrials.gov definitions. Serious adverse event information was collected over the phone at the time of onset. Adverse Event information was collected at each study visit including baseline, visit 1, visit 2, visit 3, and the follow-up visit.
Nervous system disorders Increased Uncoordinated Movements	6.7% 1/15 • Number of events 1 • 11 weeks The definitions used to collect the adverse event information does not differ from the clinicaltrials.gov definitions. Serious adverse event information was collected over the phone at the time of onset. Adverse Event information was collected at each study visit including baseline, visit 1, visit 2, visit 3, and the follow-up visit.
Nervous system disorders Increased Foot Dystonia	6.7% 1/15 • Number of events 1 • 11 weeks The definitions used to collect the adverse event information does not differ from the clinicaltrials.gov definitions. Serious adverse event information was collected over the phone at the time of onset. Adverse Event information was collected at each study visit including baseline, visit 1, visit 2, visit 3, and the follow-up visit.

Additional Information

Dr. Katherine McDonell

Vanderbilt University Medical Center

Phone: 615-875-7987

Email: katherine.mcdonell@vumc.org

Results disclosure agreements

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