A Study to Learn About How Well Riociguat Works, How Safe it is and How it is Used Under Real World Conditions in Patients in the United States Who Are Receiving Riociguat for High Blood Pressure in the Arteries That Carry Blood From the Heart to the Lungs (Pulmonary Arterial Hypertension, PAH)
- Conditions
- Pulmonary Arterial Hypertension
- Interventions
- Registration Number
- NCT04813926
- Lead Sponsor
- Bayer
- Brief Summary
Pulmonary arterial hypertension (PAH) is a type of high blood pressure in the arteries that carry blood from the heart to the lungs. PAH occurs when the openings in the blood vessels of the lungs get smaller and smaller. These smaller openings can be caused by the following:
* The walls of the arteries tightening
* The walls of the arteries becoming stiff and narrow from an overgrowth of cells The increased pressure in the pulmonary arteries strains the right side of the heart and it begins to fail, causing difficulty breathing and other symptoms. As PAH progresses, symptoms get worse.
There is no cure for PAH, but several medications like endothelin receptor antagonists (ERAs), prostacyclin analogues (PCAs) and riociguat, a soluable guanylate cyclase stimulator, are available to help slow the progression of changes in the pulmonary arteries and help reduce symptoms. Riociguat can be taken together with ERAs and PCAs.
In this study, the researchers want to learn about how well riociguat works, how safe it is when patients take it in 1 of these ways:
* alone
* with ERA
* with PCA
* with ERA and PCA The dosage for each patient will be decided by their doctor. The researchers will review information collected from the patients who have decided with their doctor to start riociguat treatment for their PAH. The study will include about 500 patients in the United States who are at least 18 years old. All of the patients will have either just started taking riociguat or will have been taking it for less than 3 months No investigational products will be administered in this study. Patients will be treated with the Standard of Care (SOC) for PAH. The SOC is the currently appropriate treatment in accordance with scientific evidence and agreed upon in collaboration between medical experts for PAH. There will be no study-mandated visits or treatments.
The patients will be in the study for up to 2 years. During this time, they will visit their doctor every 3 to 6 months as part of the Standard of Care. At these visits, the patients will answer questions about their PAH symptoms and whether they have any medical problems. They will also do exercise tests to see how well they are able to breathe and how tired they get while exercising. The doctors will perform other usual examinations which are part of the Standard of Care such as echocardiograms (images of the heart to show how the heart is working) and a right heart catheters (to measure the pressures in the heart) and will take the usual blood and urine samples.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 500
-
Patients aged ≥18 years at the time of riociguat treatment initiation
-
Diagnosis of PAH per National Institute for Health and Care Excellence (NICE) 2018 classification
-
Decision to initiate treatment with riociguat as per investigator's routine treatment practice made prior to enrollment in the study
-
Initiation of riociguat, as per the FDA-approved US label:
- At enrollment OR
- ≤90 days prior to enrollment, with a documented titration regimen (defined as all documented dose changes including, but not limited to: starting dose and dates and highest tolerated dose and dates)
-
Signed informed consent
-
Previously treated with and discontinued use of riociguat for any reason prior to study enrollment (discontinuation defined as an interruption of therapy ≥30 days)
-
Participating in any of the following:
- Blinded clinical trial
- Clinical trial involving an unapproved drug
- Investigational program with interventions outside of routine clinical practice
-
Life expectancy <12 months
-
Contraindicated to receive riociguat per the FDA approved US label
-
Use of nitrates or NO donors in any form
-
Use of PDE5 inhibitors
-
PH associated with idiopathic interstitial pneumonias
-
Unable or unwilling to provide informed consent
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Patients diagnosed with PAH Riociguat (Adempas, BAY63-2521) -
- Primary Outcome Measures
Name Time Method Change of NT-proBNP from baseline to Months 6, 12, and 24 At baseline, Month 6, 12, and 24 NT-proBNP: N-terminal pro-hormone B-type natriuretic peptide
Change of hemodynamic measurements from RHC from baseline to Months 6, 12, and 24 At baseline, Month 6, 12, and 24 RHC: Right-heart catheterization
Change of 6-minute walk distance (6MWD) from baseline to Months 6, 12, and 24 At baseline, Month 6, 12, and 24 Change of clinical PAH scores from baseline to Months 6, 12, and 24 At baseline, Month 6, 12, and 24 Incidence of adverse events (AEs) and serious adverse events (SAEs) Up to 24 months Change of BNP from baseline to Months 6, 12, and 24 At baseline, Month 6, 12, and 24 BNP: B-type natriuretic peptide
Change of ECHO measurements from baseline to Months 6, 12, and 24 At baseline, Month 6, 12, and 24 ECHO: Echocardiogram
Change of laboratory tests from baseline to Months 6, 12, and 24 At baseline, Month 6, 12, and 24 Change of NYHA/WHO PH functional class from baseline to Months 6, 12, and 24 At baseline, Month 6, 12, and 24 NYHA: New York Heart Association WHO: World Health Organization
- Secondary Outcome Measures
Name Time Method Reasons for discontinuation of riociguat At Month 6, 12, and 24 post-baseline Demographic of patients treated with riociguat Up to 24 months Clinical characteristics of patients treated with riociguat Up to 24 months Change from baseline to Month 6 and change from baseline to Month 12 in Borg Dysponea Index At baseline, Month 6 and Month 12 Change from baseline to Month 6 and change from baseline to Month 12 in emPHasis-10 At baseline, Month 6 and Month 12 Persistence/discontinuation rates for riociguat Up to 24 months Real-world treatment patterns for riociguat for PAH Up to 24 months
Trial Locations
- Locations (50)
Cedar Sinai
🇺🇸Los Angeles, California, United States
Northwestern
🇺🇸Chicago, Illinois, United States
UCSF
🇺🇸Fresno, California, United States
Santa Barbara Cottage Hospital
🇺🇸Santa Barbara, California, United States
St Francis Medical Ctr
🇺🇸Columbus, Georgia, United States
UCSD
🇺🇸San Diego, California, United States
UC Irvine
🇺🇸Irvine, California, United States
Temple University
🇺🇸Philadelphia, Pennsylvania, United States
Houston Methodist
🇺🇸Houston, Texas, United States
Baylor Scott and White
🇺🇸Plano, Texas, United States
USC
🇺🇸Los Angeles, California, United States
Allegheny General Hospital
🇺🇸Pittsburgh, Pennsylvania, United States
UT Southwestern
🇺🇸Dallas, Texas, United States
AnMed Health Medical Center
🇺🇸Anderson, South Carolina, United States
Premier Pulmonary
🇺🇸Denison, Texas, United States
Mass General
🇺🇸Boston, Massachusetts, United States
Providence
🇺🇸Spokane, Washington, United States
University of Missouri
🇺🇸Columbia, Missouri, United States
UNMH
🇺🇸Albuquerque, New Mexico, United States
University of Rochester
🇺🇸Rochester, New York, United States
Banner University Medical Center- Phoenix
🇺🇸Phoenix, Arizona, United States
Honor Health
🇺🇸Phoenix, Arizona, United States
University of Cincinnati
🇺🇸Cincinnati, Ohio, United States
Integris
🇺🇸Oklahoma City, Oklahoma, United States
Legacy Health
🇺🇸Portland, Oregon, United States
Advocate Aurora
🇺🇸Milwaukee, Wisconsin, United States
Froedtert/Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Seton Heart
🇺🇸Austin, Texas, United States
Richmond Pulmonary Associates
🇺🇸Richmond, Virginia, United States
VCU/MCV
🇺🇸Richmond, Virginia, United States
Univ of Arizona College of Medicine, Tucson
🇺🇸Tucson, Arizona, United States
CCF (Cleveland Clinic Florida)
🇺🇸Weston, Florida, United States
Loyola
🇺🇸Maywood, Illinois, United States
KUMC
🇺🇸Kansas City, Kansas, United States
Advocate Christ
🇺🇸Oak Lawn, Illinois, United States
Boston University
🇺🇸Boston, Massachusetts, United States
Beaumont Hospital
🇺🇸Troy, Michigan, United States
St. Louis University
🇺🇸Saint Louis, Missouri, United States
Barnes / Wash U
🇺🇸Saint Louis, Missouri, United States
UNMC
🇺🇸Omaha, Nebraska, United States
Mount Sinai
🇺🇸New York, New York, United States
Winthrop
🇺🇸Mineola, New York, United States
Northwell Health
🇺🇸New Hyde Park, New York, United States
NYU Langone
🇺🇸New York, New York, United States
Columbia
🇺🇸New York, New York, United States
Alliance Pulmonary
🇵🇷Guaynabo, Puerto Rico
UC Davis
🇺🇸Sacramento, California, United States
Advent Health
🇺🇸Orlando, Florida, United States
Tampa General Hospital USF
🇺🇸Tampa, Florida, United States
Norton Pulmonary Specialists
🇺🇸Louisville, Kentucky, United States