NCT04813926

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
500

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jul 2021

Typical duration for all trials

Geographic Reach
2 countries

50 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 22, 2021

Completed
2 days until next milestone

First Posted

Study publicly available on registry

March 24, 2021

Completed
4 months until next milestone

Study Start

First participant enrolled

July 16, 2021

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 19, 2024

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

February 3, 2025

Completed
Last Updated

February 10, 2025

Status Verified

February 1, 2025

Enrollment Period

3.3 years

First QC Date

March 22, 2021

Last Update Submit

February 7, 2025

Conditions

Outcome Measures

Primary Outcomes (9)

  • Incidence of adverse events (AEs) and serious adverse events (SAEs)

    Up to 24 months

  • Change of 6-minute walk distance (6MWD) from baseline to Months 6, 12, and 24

    At baseline, Month 6, 12, and 24

  • Change of NT-proBNP from baseline to Months 6, 12, and 24

    NT-proBNP: N-terminal pro-hormone B-type natriuretic peptide

    At baseline, Month 6, 12, and 24

  • Change of BNP from baseline to Months 6, 12, and 24

    BNP: B-type natriuretic peptide

    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

  • Change of hemodynamic measurements from RHC from baseline to Months 6, 12, and 24

    RHC: Right-heart catheterization

    At baseline, Month 6, 12, and 24

  • Change of ECHO measurements from baseline to Months 6, 12, and 24

    ECHO: Echocardiogram

    At baseline, Month 6, 12, and 24

  • 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

    NYHA: New York Heart Association WHO: World Health Organization

    At baseline, Month 6, 12, and 24

Secondary Outcomes (7)

  • 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

  • Reasons for discontinuation of riociguat

    At Month 6, 12, and 24 post-baseline

  • Real-world treatment patterns for riociguat for PAH

    Up to 24 months

  • +2 more secondary outcomes

Study Arms (1)

Patients diagnosed with PAH

Drug: Riociguat (Adempas, BAY63-2521)

Interventions

Follow clinical practice.

Patients diagnosed with PAH

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

US patients aged ≥18 years, diagnosed with PAH, who are naĂ¯ve to and initiating treatment with riociguat as per the FDA approved US label for PAH - either at study enrollment or have initiated riociguat within 90 days prior to study enrollment.

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (50)

Banner University Medical Center- Phoenix

Phoenix, Arizona, 85006, United States

Location

Honor Health

Phoenix, Arizona, 85020, United States

Location

Univ of Arizona College of Medicine, Tucson

Tucson, Arizona, 85719, United States

Location

UCSF

Fresno, California, 93710, United States

Location

UC Irvine

Irvine, California, 92697, United States

Location

Cedar Sinai

Los Angeles, California, 90048, United States

Location

USC

Los Angeles, California, 90089, United States

Location

UC Davis

Sacramento, California, 95817, United States

Location

UCSD

San Diego, California, 92103, United States

Location

Santa Barbara Cottage Hospital

Santa Barbara, California, 93105, United States

Location

Advent Health

Orlando, Florida, 32808, United States

Location

Tampa General Hospital USF

Tampa, Florida, 33606, United States

Location

CCF (Cleveland Clinic Florida)

Weston, Florida, 33331, United States

Location

St Francis Medical Ctr

Columbus, Georgia, 31904, United States

Location

Northwestern

Chicago, Illinois, 60611, United States

Location

Loyola

Maywood, Illinois, 60153, United States

Location

Advocate Christ

Oak Lawn, Illinois, 60453, United States

Location

KUMC

Kansas City, Kansas, 66160, United States

Location

Norton Pulmonary Specialists

Louisville, Kentucky, 40202, United States

Location

Mass General

Boston, Massachusetts, 02210, United States

Location

Boston University

Boston, Massachusetts, 02215, United States

Location

Beaumont Hospital

Troy, Michigan, 48085, United States

Location

University of Missouri

Columbia, Missouri, 30309, United States

Location

St. Louis University

St Louis, Missouri, 63103, United States

Location

Barnes / Wash U

St Louis, Missouri, 63108, United States

Location

UNMC

Omaha, Nebraska, 68198, United States

Location

UNMH

Albuquerque, New Mexico, 87114, United States

Location

Winthrop

Mineola, New York, 11501, United States

Location

Northwell Health

New Hyde Park, New York, 11042, United States

Location

Mount Sinai

New York, New York, 10003, United States

Location

NYU Langone

New York, New York, 10016, United States

Location

Columbia

New York, New York, 10032, United States

Location

University of Rochester

Rochester, New York, 14642, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45220, United States

Location

Integris

Oklahoma City, Oklahoma, 73112, United States

Location

Legacy Health

Portland, Oregon, 97232, United States

Location

Temple University

Philadelphia, Pennsylvania, 19140, United States

Location

Allegheny General Hospital

Pittsburgh, Pennsylvania, 15212, United States

Location

AnMed Health Medical Center

Anderson, South Carolina, 29621, United States

Location

Seton Heart

Austin, Texas, 78759, United States

Location

UT Southwestern

Dallas, Texas, 75390, United States

Location

Premier Pulmonary

Denison, Texas, 75020, United States

Location

Houston Methodist

Houston, Texas, 77030, United States

Location

Baylor Scott and White

Plano, Texas, 75093, United States

Location

Richmond Pulmonary Associates

Richmond, Virginia, 23219, United States

Location

VCU/MCV

Richmond, Virginia, 23219, United States

Location

Providence

Spokane, Washington, 99208, United States

Location

Advocate Aurora

Milwaukee, Wisconsin, 53215, United States

Location

Froedtert/Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Alliance Pulmonary

Guaynabo, 00968, Puerto Rico

Location

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Interventions

riociguat

Condition Hierarchy (Ancestors)

Hypertension, PulmonaryLung DiseasesRespiratory Tract Diseases

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 22, 2021

First Posted

March 24, 2021

Study Start

July 16, 2021

Primary Completion

October 19, 2024

Study Completion

February 3, 2025

Last Updated

February 10, 2025

Record last verified: 2025-02

Data Sharing

IPD Sharing
Will not share

Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

Locations