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Pulmonary Arterial HypertensionPhase 3NCT01841762

Clinical Study of Macitentan in Patients With Pulmonary Arterial Hypertension to Psychometrically Validate the PAH-SYMPACT Instrument

A Phase 3 study testing Macitentan in people with Pulmonary Arterial Hypertension, run by Actelion at 81 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Pulmonary Arterial Hypertension
What is being tested?
Macitentan
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
284 participants
Where
81 sitesUnited States
Study dates
1 Apr 2013to 1 Nov 2015
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Signed informed consent prior to initiation of any study mandated procedure
  • Patients with symptomatic PAH in World Health Organization (WHO) Functional Class (FC) II to IV
  • Patients with PAH belonging to one of the following subgroups of the Dana Point Clinical Classification Group 1:
  • Idiopathic, or
  • Heritable, or
  • Drug or toxin induced, or
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  • Associated with one of the following:
  • Connective tissue disease ii. Congenital heart disease with simple systemic-to-pulmonary shunt at least one year after surgical repair iii. HIV infection
  • Documented hemodynamic diagnosis of PAH by right heart catheterization - performed at any time prior to Screening showing:
  • Resting mean pulmonary arterial pressure (mPAP) ≥ 25 mmHg and
  • Resting pulmonary vascular resistance (PVR) > 240 dyn•s•cm-5 and
  • Pulmonary capillary wedge pressure (PCWP) or left ventricular end diastolic pressure (LVEDP) ≤ 15 mmHg
  • 6-minute walk distance (6MWD) ≥ 150 m at Screening
  • Able to fluently speak and read English
  • 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
  • For patients on oral diuretics, stable doses for at least 4 weeks prior to Visit 2
  • Men or women aged 18 or older
  • 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)
  • 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

Who cannot join

  • 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
  • Moderate to severe restrictive lung disease: total lung capacity < 60% of predicted value
  • Hemoglobin < 75% of the lower limit of the normal range at screening
  • Serum aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 3 times the upper limit of normal (ULN) at screening
  • Estimated creatinine clearance < 30 mL/min at screening
  • Systolic blood pressure (SBP) < 90 mmHg at screening
Show all 18Show fewer
  • Body weight < 40 kg at screening
  • Known concomitant life-threatening diseases with a life expectancy of < 12 months
  • Any condition that prevents compliance with the protocol or adherence to therapy
  • 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
  • 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
  • Treatment with riociguat within 3 months prior to Visit 2, or scheduled to receive riociguat during the trial
  • Treatment with strong cytochrome P450 (CYP) 3A4 inducers or inhibitors within 4 weeks prior to Visit 2
  • Recently started (< 8 weeks prior to Visit 2) or planned cardio-pulmonary rehabilitation program based on exercise
  • Females who are lactating or pregnant (positive Screening or Baseline pregnancy test) or plan to become pregnant during the study
  • Known hypersensitivity to macitentan or its excipients or drugs of the same class
  • Treatment with another investigational drug within 3 months prior to Visit 2
  • 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

Trial locations and contacts

81 sites in 1 country.

81 sites in 1 country

United States81 sites

  • University of Michigan

    Ann Arbor, Michigan, 48109-5644

  • Georgia Regents University

    Augusta, Georgia, 30912

  • University of Colorado

    Aurora, Colorado, 80045

  • Georgia Clinical Research

    Austell, Georgia, 30106

  • University of Maryland Medical Center

    Baltimore, Maryland, 21201

  • Johns Hopkins University

    Baltimore, Maryland, 21205

  • Cedars-Sinai Medical Center

    Beverly Hills, California, 90211

  • Cardiovascular Associates of the Southeast, LLC

    Birmingham, Alabama, 35243

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35294

  • Tufts Medical Center

    Boston, Massachusetts, 02111

  • Boston University Medical Center

    Boston, Massachusetts, 02118

  • Bay Area Cardiology Associates, P.A.

    Brandon, Florida, 33511

  • Buffalo General Medical Center

    Buffalo, New York, 14203

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599

  • Northwestern University

    Chicago, Illinois, 60611

  • University of Chicago Medical

    Chicago, Illinois, 60637

  • The Christ Hospital

    Cincinnati, Ohio, 45219

  • UC Health/University of Cincinnati

    Cincinnati, Ohio, 45267

  • Cleveland Clinic

    Cleveland, Ohio, 45219

  • Davis Heart & Lung Research Institute

    Columbus, Ohio, 43210-1252

  • The Ohio State University

    Columbus, Ohio, 43221

  • Baylor Research Institute (BRI)

    Dallas, Texas, 75204

  • UT Southwestern Medical Center

    Dallas, Texas, 75390-8550

  • Chest Infectious Diseases and Critical Care Associates, PC

    Des Moines, Iowa, 50325-7046

  • Inova Heart and Vascular Institue / Inova Fairfax Hospital

    Falls Church, Virginia, 22042-3307

  • UCSF Fresno

    Fresno, California, 93701

  • University of Florida Academic Health Center

    Gainesville, Florida, 32610

  • Baylor College of Medicine

    Houston, Texas, 77030-2348

  • The University of Texas Health Science Center at Houston

    Houston, Texas, 77030

  • University of Iowa Hospitals & Clinics

    Iowa City, Iowa, 52242

  • Iowa City Heart Center

    Iowa City, Iowa, 52245

  • University of Florida College of Medicine, Jacksonville

    Jacksonville, Florida, 32209

  • Mayo Clinic

    Jacksonville, Florida, 32224-1865

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160-0001

  • Midwest Pulmonary Consultants

    Kansas City, Missouri, 64111

  • UCSD Medical Center, Pulmonary Department

    La Jolla, California, 92093

  • Nebraska Pulmonary Specialties

    Lincoln, Nebraska, 68506

  • VAGLAHS, VA Greater LA Healthcare System

    Los Angeles, California, 90073

  • University of California Los Angeles

    Los Angeles, California, 90095

  • Kentuckiana Pulmonary Associates

    Louisville, Kentucky, 40202

  • University of Louisville

    Louisville, Kentucky, 40202

  • University of Wisconsin School of Medicine and Public Health

    Madison, Wisconsin, 53792

  • Novant Health Pulmonary and Critical Care

    Matthews, North Carolina, 28105

  • Aurora Cardiovascular Services

    Milwaukee, Wisconsin, 53215

  • Winthrop University Hospital

    Mineola, New York, 11501

  • North Shore-LIJ/Advance Lung Disease Clinic

    New Hyde Park, New York, 11040

  • Beth Israel Medical Center

    New York, New York, 10003

  • Columbia University Medical Center

    New York, New York, 10032

  • Sentara Norfolk General Hospital

    Norfolk, Virginia, 23507

  • Advocate Health and Hospitals Corporation

    Oakbrook Terrace, Illinois, 60181

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198

  • Thomas Jefferson University, Division on Pulmonary and Critical Care

    Philadelphia, Pennsylvania, 19107-5109

  • Temple Lung Center

    Philadelphia, Pennsylvania, 19140

  • Pulmonary Associates, PA

    Phoenix, Arizona, 85006-2611

  • Mayo Clinic Arizona

    Phoenix, Arizona, 85054-4502

  • UPMC

    Pittsburgh, Pennsylvania, 15123

  • Allegheny General Hospital

    Pittsburgh, Pennsylvania, 15212-4756

  • The Oregon Clinic

    Portland, Oregon, 97220

  • CDA for Oregon Pulmonary Associate

    Portland, Oregon, 97225

  • Oregon Health and Science University

    Portland, Oregon, 97239

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • University of California San Francisco Medical Center

    San Francisco, California, 94143

  • Sioux Falls Cardiovascular, PC

    Sioux Falls, South Dakota, 57108

  • Pulmonary & Sleep Research

    Spokane, Washington, 99204

  • Ferrell-Duncan Clinic

    Springfield, Missouri, 65807

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • Mercy Clinic Pulmonology

    St Louis, Missouri, 63141

  • Clayton Sleep Institute

    St Louis, Missouri, 63143

  • Stanford University

    Stanford, California, 94305-2200

  • Scott & White Memorial Hospital

    Temple, Texas, 76508

  • Montefiore Medical Center, Weiler Division

    The Bronx, New York, 10461

  • Veritas Clinical Specialties

    Topeka, Kansas, 66606

  • Los Angeles Biomedical Research Institute

    Torrance, California, 90502

  • Beaumont Hospital

    Troy, Michigan, 48085

  • Pulmonary and Critical Care Associates

    Union, New Jersey, 07083

  • MedStar Georgetown University Hospital

    Washington D.C., District of Columbia, 20007

  • Medstar Washington Hospital Center

    Washington D.C., District of Columbia, 20010

  • Cleveland Clinic Florida

    Weston, Florida, 33331-3609

  • Berks Schuylkill Respiratory Specialists, Ltd.

    Wyomissing, Pennsylvania, 19610

  • Wellspan Lung, Sleep and Critical Care

    York, Pennsylvania, 17402-8200

Other trials in United States

About this study

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

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.

Being tested
Macitentan
Keywords
PAH-SYMPACT, Pulmonary Arterial Hypertension, psychometric instrument
Medical subject headings
Pulmonary Arterial Hypertension

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MacitentanDrug

    Also called Macitentan / ACT-064992

    Macitentan tablet, dose of 10 mg, once daily

Study groups in NCT01841762
Study groupRoleWhat they receive
MacitentanExperimentalMacitentan tablet, dose of 10 mg, once daily
Receives: Drug: Macitentan

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Development and Refinement of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT)

    Content validity of the PAH-SYMPACT was assessed using item performance, exploratory and confirmatory factor analysis. The final item content and domain structure of PAH-SYMPACT was determined based on these analyses from the Steering Committee (expert clinicians) and findings from the qualitative research done with patients previously.

    Measured: From Screening Visit (Day -14) to End of Treatment (EOT) Visit (Visit 4, Week 16)

  2. Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), With Reliability Assessed Via Test-retest Reliability.

    The reliability of the PAH-SYMPACT is assessed by test-retest reliability. Intra-class correlation coefficients (ICCs) assess test-retest reliability for the symptom and impact part scores as well as domains. ICCs equal to or greater than 0.70 are considered to demonstrate good test-retest reliability for total and domain scores.

    Measured: From ePRO period 1 (Days -14 to -8) to ePRO period 2 (Days -7 to -1) in screening period.

  3. Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), Assessing Internal Consistency Reliability.

    The reliability of the PAH-SYMPACT is assessed by internal consistency reliability. This was determined using Cronbach's alpha-a value on an internal level scale from 0 to 1.0 with higher scores indicating a more-reliable (precise) instrument.

    Measured: From ePRO period 1 (Days -14 to -8) to ePRO period 2 (Days -7 to -1) in screening period.

Secondary outcomes (1)

  1. Number of Participants With Treatment-emergent Adverse Events, Serious Adverse Events, and Adverse Events Resulting in Patient Study Drug Discontinuation Between Time Periods, BL to End of Study Visit (EoS, Week 16+30 Days for Follow-up Safety Visits)

    Safety events are reported and documented as defined in study protocol.

    Measured: From Day 1 (Baseline Visit) to End of Study visit (EoS).

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Apr 2013 (estimated)
  2. Study start1 Apr 2013
  3. Primary completion1 Nov 2015
  4. Study completion1 Nov 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
284 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01841762
Sponsor's protocol ID
AC-055-401
Status last verified
Feb 2019
First submitted
27 Mar 2013
Last updated
26 Feb 2019
Data monitoring committee
No

Publications

  • Chin KM, Gomberg-Maitland M, Channick RN, Cuttica MJ, Fischer A, Frantz RP, Hunsche E, Kleinman L, McConnell JW, McLaughlin VV, Miller CE, Zamanian RT, Zastrow MS, Badesch DB. Psychometric Validation of the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire: Results of the SYMPHONY Trial. Chest. 2018 Oct;154(4):848-861. doi: 10.1016/j.chest.2018.04.027. Epub 2018 Apr 26.

    PubMed 29705220

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Macitentan in people with Pulmonary Arterial Hypertension, run by Actelion at 81 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 24 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 26 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 81 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 31 Oct 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Development and Refinement of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT) (time frame: From Screening Visit (Day -14) to End of Treatment (EOT) Visit (Visit 4, Week 16)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01841762, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Feb 2019.

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