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A Clinical Study of Patients With Symptomatic NOH to Assess Sustained Effects of Droxidopa Therapy

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ClinicalTrials.gov Identifier: NCT01927055
Recruitment Status : Terminated (Due to potential competition with a post-marketing study requested by FDA)
First Posted : August 22, 2013
Results First Posted : January 8, 2016
Last Update Posted : January 8, 2016
Sponsor:
Information provided by (Responsible Party):
Chelsea Therapeutics

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Conditions Symptomatic Neurogenic Orthostatic Hypotension
Parkinson's Disease
Multiple Systems Atrophy
Pure Autonomic Failure
Dopamine Beta Hydroxylase Deficiency
Interventions Drug: Droxidopa
Drug: Placebo
Enrollment 61

Recruitment Details 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.
Pre-assignment Details  
Arm/Group Title Open-label Titration Droxidopa Placebo
Hide Arm/Group Description

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 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 to match droxidopa capsules and strength designations

Period Title: Open-label
Started 61 0 0
Completed 45 0 0
Not Completed 16 0 0
Reason Not Completed
Adverse Event             6             0             0
Withdrawal by Subject             1             0             0
Study Stopped             4             0             0
Supine Hypertension             4             0             0
Difficulty attending study visits             1             0             0
Period Title: Double-Blind
Started 0 22 23
Completed 0 11 10
Not Completed 0 11 13
Reason Not Completed
Adverse Event             0             1             1
Protocol Violation             0             0             1
Withdrawal by Subject             0             2             3
Study Stopped             0             7             7
Supine Hypertension             0             0             1
Extended Vacation             0             1             0
Arm/Group Title Open-Label Droxidopa Placebo Total
Hide Arm/Group Description Patients entered open label droxidopa dose titration, but did not proceed into the randomization phase.

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 to match droxidopa capsules and strength designations. 100, 200, 300, 400, 500, 600mg TID dosing for up to 12 weeks of treatment

Total of all reporting groups
Overall Number of Baseline Participants 16 22 23 61
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 16 participants 22 participants 23 participants 61 participants
74.3  (6.99) 70.2  (7.08) 70.8  (9.00) 70.5  (8.03)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 22 participants 23 participants 61 participants
Female
2
  12.5%
9
  40.9%
7
  30.4%
18
  29.5%
Male
14
  87.5%
13
  59.1%
16
  69.6%
43
  70.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 22 participants 23 participants 61 participants
Hispanic or Latino
0
   0.0%
1
   4.5%
0
   0.0%
1
   1.6%
Not Hispanic or Latino
16
 100.0%
21
  95.5%
23
 100.0%
60
  98.4%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 22 participants 23 participants 61 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
2
  12.5%
0
   0.0%
2
   8.7%
4
   6.6%
White
13
  81.3%
22
 100.0%
21
  91.3%
56
  91.8%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
1
   6.3%
0
   0.0%
0
   0.0%
1
   1.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 22 participants 23 participants 61 participants
United States 12 19 22 53
Canada 4 3 1 8
Primary Diagnosis  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 22 participants 23 participants 61 participants
Parkinson's Disease 14 15 18 47
Pure Autonomic Failure 1 3 3 7
Multiple System Atrophy 1 4 2 7
DBH Deficiency 0 0 0 0
1.Primary Outcome
Title Change in Dizziness/ Lightheadedness/ Feeling Faint/ or Feeling Like You Might Blackout (OHSA Item 1)
Hide Description 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.
Time Frame Change from Randomization to Week 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description

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.

Arm/Group Title Droxidopa Placebo
Hide Arm/Group Description:

Droxidopa 100 mg, 200 mg, 300 mg

Droxidopa: Droxidopa at 100 mg, 200 mg, 300 mg

Placebo

Placebo: Placebo to match droxidopa capsules and strength designations

Overall Number of Participants Analyzed 22 20
Mean (Standard Deviation)
Unit of Measure: units on a scale
-1.1  (3.2) -0.5  (1.8)
Time Frame From end of screening to end of study (up to 17 weeks)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Open-label Titration Droxidopa Placebo
Hide Arm/Group Description All patients treated with study drug during dose titration (1-14 days)

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 to match droxidopa capsules and strength designations

All-Cause Mortality
Open-label Titration Droxidopa Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Open-label Titration Droxidopa Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/61 (4.92%)      1/22 (4.55%)      3/23 (13.04%)    
Gastrointestinal disorders       
Cyclic vomiting syndrome  1  0/61 (0.00%)  0 0/22 (0.00%)  0 1/23 (4.35%)  1
Pancreatitis, acute  1  1/61 (1.64%)  1 0/22 (0.00%)  0 0/23 (0.00%)  0
Infections and infestations       
Urinary tract infection  1  0/61 (0.00%)  0 0/22 (0.00%)  0 1/23 (4.35%)  1
Nervous system disorders       
Mental Impairment  1  0/61 (0.00%)  0 1/22 (4.55%)  1 0/23 (0.00%)  0
Parkinson's Disease  1  0/61 (0.00%)  0 1/22 (4.55%)  1 0/23 (0.00%)  0
Haemorrhage intracranial  1  1/61 (1.64%)  1 0/22 (0.00%)  0 0/23 (0.00%)  0
Presyncope  1  1/61 (1.64%)  1 0/22 (0.00%)  0 0/23 (0.00%)  0
Renal and urinary disorders       
Renal failure acute  1  0/61 (0.00%)  0 0/22 (0.00%)  0 1/23 (4.35%)  1
Vascular disorders       
Orthostatic hypotension  1  0/61 (0.00%)  0 0/22 (0.00%)  0 1/23 (4.35%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (16.1)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Open-label Titration Droxidopa Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   17/61 (27.87%)      7/22 (31.82%)      5/23 (21.74%)    
Gastrointestinal disorders       
Nausea  1  4/61 (6.56%)  5 0/22 (0.00%)  0 0/23 (0.00%)  0
General disorders       
Fatigue  1  0/61 (0.00%)  0 2/22 (9.09%)  2 0/23 (0.00%)  0
Infections and infestations       
Urinary tract infection  1  1/61 (1.64%)  1 1/22 (4.55%)  1 2/23 (8.70%)  2
Injury, poisoning and procedural complications       
Laceration  1  1/61 (1.64%)  1 2/22 (9.09%)  6 2/23 (8.70%)  2
Nervous system disorders       
Headache  1  5/61 (8.20%)  5 1/22 (4.55%)  1 1/23 (4.35%)  1
Dizziness  1  3/61 (4.92%)  3 2/22 (9.09%)  2 0/23 (0.00%)  0
Vascular disorders       
Hypertension  1  6/61 (9.84%)  6 3/22 (13.64%)  3 0/23 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (16.1)
The study was prematurely terminated; the limited sample size precludes meaningful conclusions on the efficacy or safety of droxidopa in this study.
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: Study director
Organization: H. Lundbeck A/S
Responsible Party: Chelsea Therapeutics
ClinicalTrials.gov Identifier: NCT01927055     History of Changes
Other Study ID Numbers: NOH401
First Submitted: August 16, 2013
First Posted: August 22, 2013
Results First Submitted: November 2, 2015
Results First Posted: January 8, 2016
Last Update Posted: January 8, 2016