Trial Outcomes & Findings for A Clinical Study of Patients With Symptomatic NOH to Assess Sustained Effects of Droxidopa Therapy (NCT NCT01927055)
NCT ID: NCT01927055
Last Updated: 2016-01-08
Results Overview
OHSA item 1 scale range: 0 (none) -10 (worst), likert scale. Change: score at end of study minus score at randomization. A positive score indicates worsening during the double-blind randomized phase relative to value at randomization, while a negative score indicates an improvement in symptom severity.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
61 participants
Primary outcome timeframe
Change from Randomization to Week 1
Results posted on
2016-01-08
Participant Flow
61 patients enrolled in the open-label dose titration, 16 patients discontinued in the open-label period and 45 patients continued on to the randomized double-blind treatment period.
Participant milestones
| Measure |
Open-label Titration
Droxidopa 100 mg, 200 mg
Droxidopa: 100 mg and 200 mg capsules 100, 200, 300, 400, 500, 600mg TID dosing for up to 2 weeks of treatment
|
Droxidopa
Droxidopa 100 mg, 200 mg, 300 mg
Droxidopa: 100 mg, 200 mg and 300 mg capsules 100, 200, 300, 400, 500, 600mg TID dosing for up to 12 weeks of treatment
|
Placebo
Placebo
Placebo: Placebo to match droxidopa capsules and strength designations
|
|---|---|---|---|
|
Open-label
STARTED
|
61
|
0
|
0
|
|
Open-label
COMPLETED
|
45
|
0
|
0
|
|
Open-label
NOT COMPLETED
|
16
|
0
|
0
|
|
Double-Blind
STARTED
|
0
|
22
|
23
|
|
Double-Blind
COMPLETED
|
0
|
11
|
10
|
|
Double-Blind
NOT COMPLETED
|
0
|
11
|
13
|
Reasons for withdrawal
| Measure |
Open-label Titration
Droxidopa 100 mg, 200 mg
Droxidopa: 100 mg and 200 mg capsules 100, 200, 300, 400, 500, 600mg TID dosing for up to 2 weeks of treatment
|
Droxidopa
Droxidopa 100 mg, 200 mg, 300 mg
Droxidopa: 100 mg, 200 mg and 300 mg capsules 100, 200, 300, 400, 500, 600mg TID dosing for up to 12 weeks of treatment
|
Placebo
Placebo
Placebo: Placebo to match droxidopa capsules and strength designations
|
|---|---|---|---|
|
Open-label
Adverse Event
|
6
|
0
|
0
|
|
Open-label
Withdrawal by Subject
|
1
|
0
|
0
|
|
Open-label
Study Stopped
|
4
|
0
|
0
|
|
Open-label
Supine Hypertension
|
4
|
0
|
0
|
|
Open-label
Difficulty attending study visits
|
1
|
0
|
0
|
|
Double-Blind
Adverse Event
|
0
|
1
|
1
|
|
Double-Blind
Protocol Violation
|
0
|
0
|
1
|
|
Double-Blind
Withdrawal by Subject
|
0
|
2
|
3
|
|
Double-Blind
Study Stopped
|
0
|
7
|
7
|
|
Double-Blind
Supine Hypertension
|
0
|
0
|
1
|
|
Double-Blind
Extended Vacation
|
0
|
1
|
0
|
Baseline Characteristics
A Clinical Study of Patients With Symptomatic NOH to Assess Sustained Effects of Droxidopa Therapy
Baseline characteristics by cohort
| Measure |
Open-Label
n=16 Participants
Patients entered open label droxidopa dose titration, but did not proceed into the randomization phase.
|
Droxidopa
n=22 Participants
Droxidopa 100 mg, 200 mg, 300 mg
Droxidopa: 100 mg, 200 mg and 300 mg capsules 100, 200, 300, 400, 500, 600mg TID dosing for up to 12 weeks of treatment
|
Placebo
n=23 Participants
Placebo
Placebo: Placebo to match droxidopa capsules and strength designations. 100, 200, 300, 400, 500, 600mg TID dosing for up to 12 weeks of treatment
|
Total
n=61 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
74.3 years
STANDARD_DEVIATION 6.99 • n=5 Participants
|
70.2 years
STANDARD_DEVIATION 7.08 • n=7 Participants
|
70.8 years
STANDARD_DEVIATION 9.00 • n=5 Participants
|
70.5 years
STANDARD_DEVIATION 8.03 • n=4 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
18 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
43 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
16 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
60 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
13 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
56 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=5 Participants
|
19 participants
n=7 Participants
|
22 participants
n=5 Participants
|
53 participants
n=4 Participants
|
|
Region of Enrollment
Canada
|
4 participants
n=5 Participants
|
3 participants
n=7 Participants
|
1 participants
n=5 Participants
|
8 participants
n=4 Participants
|
|
Primary Diagnosis
Parkinson's Disease
|
14 participants
n=5 Participants
|
15 participants
n=7 Participants
|
18 participants
n=5 Participants
|
47 participants
n=4 Participants
|
|
Primary Diagnosis
Pure Autonomic Failure
|
1 participants
n=5 Participants
|
3 participants
n=7 Participants
|
3 participants
n=5 Participants
|
7 participants
n=4 Participants
|
|
Primary Diagnosis
Multiple System Atrophy
|
1 participants
n=5 Participants
|
4 participants
n=7 Participants
|
2 participants
n=5 Participants
|
7 participants
n=4 Participants
|
|
Primary Diagnosis
DBH Deficiency
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
0 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Change from Randomization to Week 1Population: Patients entering the double-blind, randomized phase and having a visit at week 1 of the double-blind phase were analyzed. Study was stopped when only 5% of planned participants had completed the study to prevent competition with FDA mandated post-marketing requirement study.
OHSA item 1 scale range: 0 (none) -10 (worst), likert scale. Change: score at end of study minus score at randomization. A positive score indicates worsening during the double-blind randomized phase relative to value at randomization, while a negative score indicates an improvement in symptom severity.
Outcome measures
| Measure |
Droxidopa
n=22 Participants
Droxidopa 100 mg, 200 mg, 300 mg
Droxidopa: Droxidopa at 100 mg, 200 mg, 300 mg
|
Placebo
n=20 Participants
Placebo
Placebo: Placebo to match droxidopa capsules and strength designations
|
|---|---|---|
|
Change in Dizziness/ Lightheadedness/ Feeling Faint/ or Feeling Like You Might Blackout (OHSA Item 1)
|
-1.1 units on a scale
Standard Deviation 3.2
|
-0.5 units on a scale
Standard Deviation 1.8
|
Adverse Events
Open-label Titration
Serious events: 3 serious events
Other events: 17 other events
Deaths: 0 deaths
Droxidopa
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
Placebo
Serious events: 3 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Open-label Titration
n=61 participants at risk
All patients treated with study drug during dose titration (1-14 days)
|
Droxidopa
n=22 participants at risk
Droxidopa 100 mg, 200 mg, 300 mg
Droxidopa: 100 mg, 200 mg and 300 mg capsules 100, 200, 300, 400, 500, 600mg TID dosing for up to 12 weeks of treatment
|
Placebo
n=23 participants at risk
Placebo
Placebo: Placebo to match droxidopa capsules and strength designations
|
|---|---|---|---|
|
Nervous system disorders
Mental Impairment
|
0.00%
0/61 • From end of screening to end of study (up to 17 weeks)
|
4.5%
1/22 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/23 • From end of screening to end of study (up to 17 weeks)
|
|
Nervous system disorders
Parkinson's Disease
|
0.00%
0/61 • From end of screening to end of study (up to 17 weeks)
|
4.5%
1/22 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/23 • From end of screening to end of study (up to 17 weeks)
|
|
Nervous system disorders
Haemorrhage intracranial
|
1.6%
1/61 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/22 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/23 • From end of screening to end of study (up to 17 weeks)
|
|
Nervous system disorders
Presyncope
|
1.6%
1/61 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/22 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/23 • From end of screening to end of study (up to 17 weeks)
|
|
Gastrointestinal disorders
Cyclic vomiting syndrome
|
0.00%
0/61 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/22 • From end of screening to end of study (up to 17 weeks)
|
4.3%
1/23 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
|
Gastrointestinal disorders
Pancreatitis, acute
|
1.6%
1/61 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/22 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/23 • From end of screening to end of study (up to 17 weeks)
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/61 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/22 • From end of screening to end of study (up to 17 weeks)
|
4.3%
1/23 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
|
Renal and urinary disorders
Renal failure acute
|
0.00%
0/61 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/22 • From end of screening to end of study (up to 17 weeks)
|
4.3%
1/23 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
|
Vascular disorders
Orthostatic hypotension
|
0.00%
0/61 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/22 • From end of screening to end of study (up to 17 weeks)
|
4.3%
1/23 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
Other adverse events
| Measure |
Open-label Titration
n=61 participants at risk
All patients treated with study drug during dose titration (1-14 days)
|
Droxidopa
n=22 participants at risk
Droxidopa 100 mg, 200 mg, 300 mg
Droxidopa: 100 mg, 200 mg and 300 mg capsules 100, 200, 300, 400, 500, 600mg TID dosing for up to 12 weeks of treatment
|
Placebo
n=23 participants at risk
Placebo
Placebo: Placebo to match droxidopa capsules and strength designations
|
|---|---|---|---|
|
Nervous system disorders
Headache
|
8.2%
5/61 • Number of events 5 • From end of screening to end of study (up to 17 weeks)
|
4.5%
1/22 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
4.3%
1/23 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
|
Nervous system disorders
Dizziness
|
4.9%
3/61 • Number of events 3 • From end of screening to end of study (up to 17 weeks)
|
9.1%
2/22 • Number of events 2 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/23 • From end of screening to end of study (up to 17 weeks)
|
|
Injury, poisoning and procedural complications
Laceration
|
1.6%
1/61 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
9.1%
2/22 • Number of events 6 • From end of screening to end of study (up to 17 weeks)
|
8.7%
2/23 • Number of events 2 • From end of screening to end of study (up to 17 weeks)
|
|
General disorders
Fatigue
|
0.00%
0/61 • From end of screening to end of study (up to 17 weeks)
|
9.1%
2/22 • Number of events 2 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/23 • From end of screening to end of study (up to 17 weeks)
|
|
Vascular disorders
Hypertension
|
9.8%
6/61 • Number of events 6 • From end of screening to end of study (up to 17 weeks)
|
13.6%
3/22 • Number of events 3 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/23 • From end of screening to end of study (up to 17 weeks)
|
|
Infections and infestations
Urinary tract infection
|
1.6%
1/61 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
4.5%
1/22 • Number of events 1 • From end of screening to end of study (up to 17 weeks)
|
8.7%
2/23 • Number of events 2 • From end of screening to end of study (up to 17 weeks)
|
|
Gastrointestinal disorders
Nausea
|
6.6%
4/61 • Number of events 5 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/22 • From end of screening to end of study (up to 17 weeks)
|
0.00%
0/23 • From end of screening to end of study (up to 17 weeks)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place