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

Phase 3

Terminated

• Conditions: Multiple Systems Atrophy; Pure Autonomic Failure; Parkinson's Disease; Symptomatic Neurogenic Orthostatic Hypotension; Dopamine Beta Hydroxylase Deficiency
• Interventions: Drug: Placebo; Drug: Droxidopa

• Registration Number: NCT01927055
• Lead Sponsor: Chelsea Therapeutics

Brief Summary

Evaluate the clinical efficacy and safety of droxidopa versus placebo over a 17 week (maximum) treatment period in patients with symptomatic NOH.

Detailed Description

This is a multi-center, multi-national, randomized, parallel-group, placebo-controlled, double-blind study with a 17 week (maximum) treatment period consisting of an initial, open-label dose titration (up to 2 weeks), followed by a washout period (up to 3 weeks), followed by a 12 week treatment period on a stable dose.

Study Timeline

• Study First Submitted: 2013-08-16
• Study First Submitted QC: 2013-08-21
• Study First Posted: 2013-08-22
• Study Start: 2013-11
• Primary Completion: 2015-02
• Study Completion: 2015-02
• Results First Submitted: 2015-11-02
• Status Verified: 2015-12
• Results First Submitted QC: 2015-12-04
• Last Update Submitted: 2015-12-04
• Results First Posted: 2016-01-08
• Last Update Posted: 2016-01-08

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 61

Inclusion Criteria

• 1. 18 years and older and ambulatory (defined as able to walk at least 10 meters);

  2. Clinical diagnosis of symptomatic orthostatic hypotension associated with Primary Autonomic Failure (PD, MSA and PAF), Dopamine Beta Hydroxylase Deficiency;

  3. At the Baseline visit (Visit 2), patients must demonstrate:

  4. a score of at least 4 or greater on the Orthostatic Hypotension Symptom Assessment (OHSA) Item #1;

  5. a fall of at least 20 mmHg in their systolic blood pressure, within 3 minutes of standing;

     4. Provide written informed consent to participate in the study and understand that they may withdraw their consent at any time without prejudice to their future medical care;

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Exclusion Criteria

• 1. Score of 23 or lower on the mini-mental state examination (MMSE);

  2. Concomitant use of vasoconstricting agents for the purpose of increasing blood pressure;

  3. Patients taking vasoconstricting agents such as ephedrine, dihydroergotamine, or midodrine must stop taking these drugs at least 2 days or 5 half-lives (whichever is longer) prior to their baseline visit (Visit 2) and throughout the duration of the study;

     3. Known or suspected alcohol or substance abuse within the past 12 months (DSM-IV definition of alcohol or substance abuse);

     4. Women who are pregnant or breastfeeding;

     5. Women of child bearing potential (WOCP) who are not using at least one method of contraception with their partner;

     6. Male patients who are sexually active with a woman of child bearing potential (WOCP) and not using at least one method of contraception;

     7. Untreated closed angle glaucoma;

     8. Diagnosis of hypertension that requires treatment with antihypertensive medications (short-acting antihypertensives to treat nocturnal supine HTN are allowed in this study) Any significant uncontrolled cardiac arrhythmia;

     9. History of myocardial infarction, within the past 2 years;

     10. Current unstable angina;

     11. Congestive heart failure (NYHA Class 3 or 4);

     12. History of cancer within the past 2 years other than a successfully treated, non-metastatic cutaneous squamous cell or basal cell carcinoma or cervical cancer in situ;

     13. Gastrointestinal condition that may affect the absorption of study drug (e.g. ulcerative colitis, gastric bypass);

     14. Any major surgical procedure within 30 days prior to the Baseline visit (Visit 2);

     15. Previously treated with droxidopa within 30 days prior to the Baseline visit (Visit 2);

     16. Currently receiving any other investigational drug or have received an investigational drug within 30 days prior to the Baseline visit (Visit 2);

     17. Any condition or laboratory test result, which in the Investigator's judgment, might result in an increased risk to the patient, or would affect their participation in the study;

     18. The Investigator has the ability to exclude a patient if for any reason they feel the subject is not a good candidate for the study or will not be able to follow study procedures.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Placebo
Droxidopa	Droxidopa	Droxidopa 100 mg, 200 mg, 300 mg

Primary Outcome Measures

Name	Time	Method
Change in Dizziness/ Lightheadedness/ Feeling Faint/ or Feeling Like You Might Blackout (OHSA Item 1)	Change from Randomization to Week 1	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.

Trial Locations

Locations (3)

Information on additional locations involved in this clinical trial contact Chelsea Therapeutics; 🇺🇸 Charlotte, North Carolina, United States

NYU Langone Medical Center; 🇺🇸 New York, New York, United States

Wisconsin Institute for Neurology and Sleep Disorders; 🇺🇸 Milwaukee, Wisconsin, United States