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SYMPHONY: A Study of Macitentan in Pulmonary Arterial Hypertension to Validate the PAH-SYMPACT

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by Actelion
Sponsor:
Information provided by (Responsible Party):
Actelion
ClinicalTrials.gov Identifier:
NCT01841762
First received: March 27, 2013
Last updated: September 18, 2014
Last verified: September 2014

March 27, 2013
September 18, 2014
April 2013
January 2015   (final data collection date for primary outcome measure)
Development of patient-reported outcome measure of symptoms and their impact in PAH (the PAH-SYMPACT) [ Time Frame: From Screening Visit (Visit 1) to End of Treatment (EOT) Visit (Visit 4, Week 16) ] [ Designated as safety issue: No ]
Assessed through item analyses, factor analyses, and Rasch analyses.
Same as current
Complete list of historical versions of study NCT01841762 on ClinicalTrials.gov Archive Site
To assess the frequency of treatment-emergent adverse events, serious adverse events, and adverse events leading to study drug discontinuation from Baseline to Week 16. [ Time Frame: From Baseline Visit (Visit 2, Day 1) to End of Treatment (EOT) Visit (Visit 4, Week 16) ] [ Designated as safety issue: Yes ]
Same as current
Change from Baseline to Week 16 in the symptom and impact scales of the PAH-SYMPACT. [ Time Frame: From Baseline Visit (Visit 2, Day 1) to End of Treatment (EOT) Visit (Visit 4, Week 16) ] [ Designated as safety issue: No ]
Assessed by the PAH-SYMPACT questionnaire.
Same as current
 
SYMPHONY: A Study of Macitentan in Pulmonary Arterial Hypertension to Validate the PAH-SYMPACT
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

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.

Not Provided
Interventional
Phase 3
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Pulmonary Arterial Hypertension
Drug: Macitentan
Macitentan tablet, dose of 10 mg, once daily
Other Name: Macitentan / ACT-064992
Experimental: Macitentan
Macitentan tablet, dose of 10 mg, once daily
Intervention: Drug: Macitentan
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
275
January 2015
January 2015   (final data collection date for primary outcome measure)

Inclusion Criteria:

  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:

    i. Connective tissue disease ii. Congenital heart disease with simple systemic-to-pulmonary shunt at least one year after surgical repair iii. HIV infection

  4. Documented hemodynamic diagnosis of PAH by right heart catheterization - performed at any time prior to Screening showing:

    1. Resting mean pulmonary arterial pressure (mPAP) ≥ 25 mmHg and
    2. Resting pulmonary vascular resistance (PVR) > 240 dyn•s•cm-5 and
    3. Pulmonary capillary wedge pressure (PCWP) or left ventricular end diastolic pressure (LVEDP) ≤ 15 mmHg
  5. 6-minute walk distance (6MWD) ≥ 150 m at Screening
  6. Able to fluently speak and read English
  7. 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
  8. For patients on oral diuretics, stable doses for at least 4 weeks prior to Visit 2
  9. Men or women aged 18 or older

    1. A woman is considered to be of childbearing potential unless she:

      • Has not yet entered puberty, or
      • Does not have a uterus, or
      • Has gone through menopause (has not had a period for at least 12 months for natural reasons, or who has had their ovaries removed)
    2. A women of childbearing potential is eligible only if she meets both criteria below:

      • 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
      • 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

Exclusion Criteria:

  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
Both
18 Years and older
No
Contact: Scott Tsurutani 650 808 - 6586 scott.tsurutani@actelion.com
United States
 
NCT01841762
AC-055-401
No
Actelion
Actelion
Not Provided
Study Chair: Rajiv Patni, MD Actelion Pharmaceuticals US, Inc
Actelion
September 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP