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

Chelsea Therapeutics

Status and phase

Terminated

Phase 3

Conditions

Parkinson's Disease

Symptomatic Neurogenic Orthostatic Hypotension

Multiple Systems Atrophy

Dopamine Beta Hydroxylase Deficiency

Pure Autonomic Failure

Treatments

Drug: Placebo

Drug: Droxidopa

Study type

Interventional

Funder types

Industry

Identifiers

NCT01927055

NOH401

Details and patient eligibility

About

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

Full 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.

Enrollment

61 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

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;
  1. 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;
  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;
  1. Known or suspected alcohol or substance abuse within the past 12 months (DSM-IV definition of alcohol or substance abuse);
  2. Women who are pregnant or breastfeeding;
  3. Women of child bearing potential (WOCP) who are not using at least one method of contraception with their partner;
  4. Male patients who are sexually active with a woman of child bearing potential (WOCP) and not using at least one method of contraception;
  5. Untreated closed angle glaucoma;
  6. 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;
  7. History of myocardial infarction, within the past 2 years;
  8. Current unstable angina;
  9. Congestive heart failure (NYHA Class 3 or 4);
  10. 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;
  11. Gastrointestinal condition that may affect the absorption of study drug (e.g. ulcerative colitis, gastric bypass);
  12. Any major surgical procedure within 30 days prior to the Baseline visit (Visit 2);
  13. Previously treated with droxidopa within 30 days prior to the Baseline visit (Visit 2);
  14. Currently receiving any other investigational drug or have received an investigational drug within 30 days prior to the Baseline visit (Visit 2);
  15. 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;
  16. 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.