Retrospective Chart Review of First-time Opsumit® (Macitentan) Users in the United States (OrPHeUS)

Multicenter, Retrospective Chart Review of First-time Opsumit® (Macitentan) Users in the United States

The Opsumit Users registry (OPUS; NCT02126943) was developed to characterize the safety profile of Opsumit and to describe clinical characteristics and outcomes of patients newly treated with Opsumit in the post-marketing setting. It is expected that the recruitment target of the OPUS registry cannot be achieved within the planned time period (5000 Opsumit new users by October 2018). The OrPHeUS study is designed to supplement the OPUS registry with retrospectively identified first-time Opsumit users in order to achieve the desired sample size.

Study Overview

• Status: Completed
• Conditions: Pulmonary Arterial Hypertension
• Intervention / Treatment: Other: Notapplicable

• Study Type: Observational
• Enrollment (Actual): 2200

Contacts and Locations

Study Locations

• Puerto Rico
  • Guaynabo, Puerto Rico, 00968
    • CardioPulmonary Research Center
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85006
      • Pulmonary Associates
    • Phoenix, Arizona, United States, 85054
      • Mayo Clinic - Arizona
    • Phoenix, Arizona, United States, 85032
      • Cardiovascular Consultants
  • California
    • La Jolla, California, United States, 93093
      • University of California San Diego
    • Loma Linda, California, United States, 92354
      • Loma Linda University Medical Center
    • Los Angeles, California, United States, 90033
      • University of Southern California
    • Los Angeles, California, United States, 90095-1690
      • David Geffen School of Medicine at University of California Los Angeles
    • San Francisco, California, United States, 94143
      • UCSF School of Medicine
    • San Juan Capistrano, California, United States, 92675
      • Paloma Medical Group
    • Santa Barbara, California, United States, 93105
      • Santa Barbara Cottage Hospital
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado
    • Denver, Colorado, United States, 80206
      • National Jewish Health
  • Connecticut
    • New Haven, Connecticut, United States, 06520
      • Yale University
  • Delaware
    • Newark, Delaware, United States, 19718
      • Christiana Care Health Services
  • District of Columbia
    • Washington, District of Columbia, United States, 20007
      • MedStar Georgetown University Hospital
  • Florida
    • Brandon, Florida, United States, 33511
      • Bay Area Cardiology Associates
    • Celebration, Florida, United States, 34747
      • Florida Lung, Asthma and Sleep Specialists
    • Fort Lauderdale, Florida, United States, 33316
      • Frank Hull, MD, PA
    • Gainesville, Florida, United States, 32610
      • University of Florida
    • Hudson, Florida, United States, 34667
      • West Pasco Pulmonary Associates
    • Jacksonville, Florida, United States, 32209
      • Saint Vincent's Medical Center
    • Leesburg, Florida, United States, 34748
      • Medical Research of Central Florida
    • Melbourne, Florida, United States, 32901
      • Independent Lung Associates
    • Miami, Florida, United States, 33136
      • University of Miami
    • Miami Beach, Florida, United States, 33140
      • Mount Sinai Comprehensive Cancer Center
    • Orlando, Florida, United States, 32803
      • Central Florida Pulmonary Group, P.A. - Downtown Orlando Office
    • Saint Petersburg, Florida, United States, 33704
      • Coastal Pulmonary and Critical Care
    • Sebring, Florida, United States, 33870
      • Bassetti Medical Research Inc
    • South Miami, Florida, United States, 33143
      • Cardiovascular Medicine Associates
    • Tampa, Florida, United States, 33606
      • University of South Florida
    • Weston, Florida, United States, 33331
      • Cleveland Clinic Florida Hospital
    • Zephyrhills, Florida, United States, 33542
      • Florida Medical Clinic
  • Georgia
    • Austell, Georgia, United States, 30106
      • Piedmont Healthcare
    • Columbus, Georgia, United States, 31904
      • Southeastern Cardiology Associates
    • Marietta, Georgia, United States, 30060
      • Wellstar Marietta Pulmonary Medicine
  • Idaho
    • Shelley, Idaho, United States, 83274
      • Bingham Memorial Hospital
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern University
  • Indiana
    • Carmel, Indiana, United States, 46032
      • IU Health Physicians Pulmonary & Critical Care
    • Fort Wayne, Indiana, United States, 46804
      • Lutheran Hospital
    • Greenwood, Indiana, United States, 46143
      • Indiana Internal Medicine Consultants
    • Indianapolis, Indiana, United States, 46260
      • St. Vincent Medical Group, Inc.
  • Iowa
    • Clive, Iowa, United States, 50325
      • CIC Associates
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals and Clinics
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • The University of Kansas Physicians
  • Kentucky
    • Erlanger, Kentucky, United States, 41018
      • St. Elizabeth Healthcare Clinical Research Institute
    • Louisville, Kentucky, United States, 40202
      • Kentuckiana Pulmonary Associates
  • Louisiana
    • New Orleans, Louisiana, United States, 70121
      • Ochsner Medical Center
    • New Orleans, Louisiana, United States, 70112
      • LSU Health Sciences Center
    • Shreveport, Louisiana, United States, 71105
      • Pulmonary & Critical Care Specialists
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
    • Boston, Massachusetts, United States, 02111
      • Tufts Medical Center
    • Boston, Massachusetts, United States, 02118
      • Boston University Medical Center
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Medical Center
    • Detroit, Michigan, United States, 48202
      • Henry Ford Health System
    • Lansing, Michigan, United States, 48912
      • Sparrow Clinical Research Institute
    • Troy, Michigan, United States, 48085
      • Beaumont Medical Center
  • Minnesota
    • Minneapolis, Minnesota, United States, 55407-3799
      • Minneapolis Heart Institute
  • Mississippi
    • Jackson, Mississippi, United States, 39216
      • University of Mississippi Medical Center
  • Missouri
    • Saint Louis, Missouri, United States, 63141
      • Mercy Hospital Saint Louis
    • Saint Louis, Missouri, United States, 63104
      • Saint Louis University
    • Saint Louis, Missouri, United States, 63136
      • Pulmonary Consultants Inc-St. Louis
  • Nebraska
    • Lincoln, Nebraska, United States, 68506
      • Nebraska Pulmonary Specialties, LLC
    • Omaha, Nebraska, United States, 68198-5990
      • University of Nebraska Medical Center
  • New Jersey
    • Newark, New Jersey, United States, 07112
      • Barnabas Health Ambulatory Care Center
    • Union, New Jersey, United States, 07083
      • Pulmonary and Critical Care Associates
  • New Mexico
    • Albuquerque, New Mexico, United States, 87131-0001
      • University of New Mexico
  • New York
    • Albany, New York, United States, 12208
      • Albany Medical Center
    • Bronx, New York, United States, 10467
      • Montefiore Medical Center - Bronx
    • Fayetteville, New York, United States, 13066
      • Pulmonary Health Physicians
    • Hauppauge, New York, United States, 11788
      • Stony Brook University Hospital
    • Middletown, New York, United States, 10941
      • Temple University Hospital
    • Mineola, New York, United States, 11501
      • Winthrop University Hospital, Clinical Trials Center
    • New York, New York, United States, 10029
      • Mount Sinai
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • University of North Carolina Hospitals
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Ohio
    • Cincinnati, Ohio, United States, 45242
      • University of Cincinnati
    • Columbus, Ohio, United States, 43210
      • Ohio State University, Wexner Medical Center
    • Columbus, Ohio, United States, 43213
      • Mount Carmel Medical Group
    • Dayton, Ohio, United States, 45459
      • Midwest Pulmonary & Sleep Research
    • Middleburg Heights, Ohio, United States, 44130
      • Southwest General Health Center
    • Toledo, Ohio, United States, 43614
      • The University of Toledo Medical Center
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73120
      • Oklahoma Heart Hospital
  • Oregon
    • Portland, Oregon, United States, 97210
      • Legacy Research Institute
    • Portland, Oregon, United States, 97225
      • The Oregon Clinic
    • Portland, Oregon, United States, 97201-3098
      • Oregon Health & Science University
  • Pennsylvania
    • Doylestown, Pennsylvania, United States, 18907
      • Doylestown Health Cardiology, a division of Doylestown Health Physicians
    • Philadelphia, Pennsylvania, United States, 19107
      • Thomas Jefferson University
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania Health System
    • Pittsburgh, Pennsylvania, United States, 15212
      • Allegheny General Hospital
    • Wynnewood, Pennsylvania, United States, 19096
      • Lankenau Medical Center
  • South Carolina
    • Anderson, South Carolina, United States, 29621
      • AnMed Health Medical Center
    • Rock Hill, South Carolina, United States, 29732
      • Carolina Cardiology Associates
  • South Dakota
    • Sioux Falls, South Dakota, United States, 57108
      • Sioux Falls Cardiovascular PC
  • Tennessee
    • Chattanooga, Tennessee, United States, 37404
      • Memorial Research Center
    • Memphis, Tennessee, United States, 38157
      • Mid-South Pulmonary Specialists
  • Texas
    • Dallas, Texas, United States, 75390-8550
      • University of Texas Southwestern Medical Center at Dallas
    • Denison, Texas, United States, 75020
      • Premier Pulmonary CC and Sleep Medicine, PA
    • Houston, Texas, United States, 77030
      • Houston Methodist Hospital
    • Houston, Texas, United States, 77030
      • University of Texas Health Science Center at Houston
    • Plano, Texas, United States, 75093
      • Baylor Regional Medical Center at Plano
    • Temple, Texas, United States, 76508-0001
      • Scott and White Memorial Hospital
  • Washington
    • Seattle, Washington, United States, 98195
      • University of Washington
    • Spokane, Washington, United States, 99204-4800
      • Spokane Respiratory Consultants
    • Tacoma, Washington, United States, 98405
      • Cardiac Study Center
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53215
      • Aurora Health Care - Saint Luke's Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients who initiated Opsumit for the first time between 19 October 2013 and 31 December 2016 (inclusive) are eligible for OrPHeUS (i.e., eligibility period).

Description

Inclusion Criteria:

• Patients who initiated Opsumit for the first time between 19 October 2013 and 31 December 2016 (inclusive)
• Patients whose medical charts are available for data collection

Exclusion Criteria:

• Patient participation in the OPUS registry
• Patient participation in any clinical trial involving Opsumit treatment or macitentan investigational product

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Notapplicable; Not applicable as non-interventional study	Other: Notapplicable; Not applicable as non-interventional study

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Estimation of incidence rates of adverse events	Incidence rates of liver test abnormalities, Occurrence of Hepatic Adverse Events (HAEs), Occurrence of any other Adverse Event (AE), Occurrence and reason for discontinuation of Opsumit treatment, hospitalization and death	Observation period from 19 Oct 2013 to 31 March 2017, Data collection from 1 Apr 2017 to 30 Jun 2018

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Description of demographic characteristics of patients	To describe demographic characteristics of patients treated with Opsumit at beginning and during study time period	Observation period from 19 Oct 2013 to 31 March 2017, Data collection from 1 Apr 2017 to 30 Jun 2018
Description of clinical characteristics of patients	To describe clinical characteristics of patients treated with Opsumit at beginning and during study time period	Observation period from 19 Oct 2013 to 31 March 2017, Data collection from 1 Apr 2017 to 30 Jun 2018

Collaborators and Investigators

• Sponsor: Actelion

Study record dates

Study Major Dates

• Study Start (Actual): August 3, 2017
• Primary Completion (Actual): September 6, 2018
• Study Completion (Actual): September 6, 2018

Study Registration Dates

• First Submitted: May 12, 2017
• First Submitted That Met QC Criteria: June 21, 2017
• First Posted (Actual): June 23, 2017

Study Record Updates

• Last Update Posted (Actual): September 26, 2018
• Last Update Submitted That Met QC Criteria: September 25, 2018
• Last Verified: September 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Respiratory Tract Diseases; Lung Diseases; Hypertension, Pulmonary; Hypertension; Pulmonary Arterial Hypertension
• Other Study ID Numbers: AC-055-510

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No