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Summary

for people ages 18 years and up (full criteria)
at Fresno, California and other locations
study started
estimated completion:

Description

Summary

SYMPHONY is prospective, multi-center, open-label, single-arm, Phase 3b psychometric validation study of the PAH-SYMPACT, a new quality of life questionnaire for patients with pulmonary arterial hypertension. Patients will be in the study for 5 1/2 months, 4 months of which they will receive macitentan, 10 mg, once daily. The primary objectives are to demonstrate the final content validity of the PAH SYMPACT instrument, to demonstrate the psychometric characteristics of reliability and construct validity of the PAH-SYMPACT instrument, and to demonstrate the ability of the PAH SYMPACT instrument to detect change. The secondary objective is to assess the safety of macitentan in patients with pulmonary arterial hypertension. The exploratory objective is to explore the effects of macitentan on PAH symptoms and their impact (as measured by the PAH-SYMPACT) in patients with pulmonary arterial hypertension.

Official Title

A Multi-center, Open-label, Single-arm, Phase 3b Study of Macitentan in Patients With Pulmonary Arterial Hypertension to Psychometrically Validate the PAH-SYMPACT Instrument

Keywords

Pulmonary Arterial Hypertension PAH-SYMPACT psychometric instrument Hypertension Familial Primary Pulmonary Hypertension Macitentan

Eligibility

You can join if…

Open to people ages 18 years and up

  1. Signed informed consent prior to initiation of any study mandated procedure
  2. Patients with symptomatic PAH in World Health Organization (WHO) Functional Class (FC)II to IV
  3. Patients with PAH belonging to one of the following subgroups of the Dana Point

Clinical Classification Group 1:

  1. Idiopathic, or
  2. Heritable, or
  3. Drug or toxin induced, or
  4. Associated with one of the following:
  5. Connective tissue disease ii. Congenital heart disease with simple systemic-to-pulmonary shunt at least one year after surgical repair iii. HIV infection
  6. Documented hemodynamic diagnosis of PAH by right heart catheterization - performed at any time prior to Screening showing:
  7. Resting mean pulmonary arterial pressure (mPAP) ≥ 25 mmHg and
  8. Resting pulmonary vascular resistance (PVR) > 240 dyn•s•cm-5 and
  9. Pulmonary capillary wedge pressure (PCWP) or left ventricular end diastolic pressure (LVEDP) ≤ 15 mmHg
  10. 6-minute walk distance (6MWD) ≥ 150 m at Screening
  11. Able to fluently speak and read English
  12. For patients on phosphodiesterase type-5 inhibitors (PDE5i), inhaled prostacyclin analogues, or calcium channel blockers, stable doses for at least 3 months prior to Visit 2
  13. For patients on oral diuretics, stable doses for at least 4 weeks prior to Visit 2
  14. Men or women aged 18 or older
  15. A woman is considered to be of childbearing potential unless she:
  16. Has not yet entered puberty, or
  17. Does not have a uterus, or
  18. Has gone through menopause (has not had a period for at least 12 months for natural reasons, or who has had their ovaries removed)
  19. A women of childbearing potential is eligible only if she meets both criteria below:
  20. Has a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline and agree to perform monthly urine pregnancy tests, and
  21. Agrees to use two methods of contraception (one method for patients with a progesterone implant or an intrauterine device or tubal sterilization) from the Screening Visit 1 until one month after study drug discontinuation

You CAN'T join if...

  1. Moderate to severe obstructive lung disease: forced expiratory volume in one second(FEV1) / forced vital capacity < 70% and FEV1 < 65% of predicted value after bronchodilator administration
  2. Moderate to severe restrictive lung disease: total lung capacity < 60% of predicted value
  3. Hemoglobin < 75% of the lower limit of the normal range at screening
  4. Serum aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 3 times the upper limit of normal (ULN) at screening
  5. Estimated creatinine clearance < 30 mL/min at screening
  6. Systolic blood pressure (SBP) < 90 mmHg at screening
  7. Body weight < 40 kg at screening
  8. Known concomitant life-threatening diseases with a life expectancy of < 12 months
  9. Any condition that prevents compliance with the protocol or adherence to therapy
  10. . Treatment with endothelin receptor antagonists (ERAs) within 3 months prior to Visit 2, or scheduled to receive any of these compounds, other than macitentan, during the trial
  11. . Treatment with intravenous or subcutaneous prostacyclin or prostacyclin analogs within 3 months prior to Visit 2, or scheduled to receive any of these compounds during the trial
  12. . Treatment with riociguat within 3 months prior to Visit 2, or scheduled to receive riociguat during the trial
  13. . Treatment with strong cytochrome P450 (CYP) 3A4 inducers or inhibitors within 4 weeks prior to Visit 2
  14. . Recently started (< 8 weeks prior to Visit 2) or planned cardio-pulmonary rehabilitation program based on exercise
  15. . Females who are lactating or pregnant (positive Screening or Baseline pregnancy test)or plan to become pregnant during the study
  16. . Known hypersensitivity to macitentan or its excipients or drugs of the same class
  17. . Treatment with another investigational drug within 3 months prior to Visit 2
  18. . Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results such as drug or alcohol dependence or psychiatric disease

Locations

  • UCSF Fresno
    Fresno, California, 93701, United States
  • University of California San Francisco Medical Center
    San Francisco, California, 94143, United States
  • Stanford University
    Stanford, California, 94305-2200, United States
  • Cedars-Sinai Medical Center
    Beverly Hills, California, 90211, United States
  • Los Angeles Biomedical Research Institute
    Torrance, California, 90502, United States
  • VAGLAHS, VA Greater LA Healthcare System
    Los Angeles, California, 90073, United States
  • University of California Los Angeles
    Los Angeles, California, 90095, United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Actelion
ID
NCT01841762
Phase
Phase 3
Study Type
Interventional
Last Updated
June 2016