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) (ROAR)

• ClinicalTrials.gov Identifier: NCT04813926
• Recruitment Status: Recruiting
• First Posted: March 24, 2021
• Last Update Posted: February 23, 2023
• Sponsor: Bayer
• Collaborator: Xcenda, LLC
• Information provided by (Responsible Party): Bayer

Tracking Information

• First Submitted Date: March 22, 2021
• First Posted Date: March 24, 2021
• Last Update Posted Date: February 23, 2023
• Actual Study Start Date: July 16, 2021
• Estimated Primary Completion Date: July 11, 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 22, 2021)

• Incidence of adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: Up to 24 months ]
• Change of 6-minute walk distance (6MWD) from baseline to Months 6, 12, and 24 [ Time Frame: At baseline, Month 6, 12, and 24 ]
• Change of NT-proBNP from baseline to Months 6, 12, and 24 [ Time Frame: At baseline, Month 6, 12, and 24 ]
  NT-proBNP: N-terminal pro-hormone B-type natriuretic peptide
• Change of BNP from baseline to Months 6, 12, and 24 [ Time Frame: At baseline, Month 6, 12, and 24 ]
  BNP: B-type natriuretic peptide
• Change of clinical PAH scores from baseline to Months 6, 12, and 24 [ Time Frame: At baseline, Month 6, 12, and 24 ]
• Change of hemodynamic measurements from RHC from baseline to Months 6, 12, and 24 [ Time Frame: At baseline, Month 6, 12, and 24 ]
  RHC: Right-heart catheterization
• Change of ECHO measurements from baseline to Months 6, 12, and 24 [ Time Frame: At baseline, Month 6, 12, and 24 ]
  ECHO: Echocardiogram
• Change of laboratory tests from baseline to Months 6, 12, and 24 [ Time Frame: At baseline, Month 6, 12, and 24 ]
• Change of NYHA/WHO PH functional class from baseline to Months 6, 12, and 24 [ Time Frame: At baseline, Month 6, 12, and 24 ]
  NYHA: New York Heart Association WHO: World Health Organization

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: March 22, 2021)

• Change from baseline to Month 6 and change from baseline to Month 12 in Borg Dysponea Index [ Time Frame: At baseline, Month 6 and Month 12 ]
• Change from baseline to Month 6 and change from baseline to Month 12 in emPHasis-10 [ Time Frame: At baseline, Month 6 and Month 12 ]
• Persistence/discontinuation rates for riociguat [ Time Frame: Up to 24 months ]
• Reasons for discontinuation of riociguat [ Time Frame: At Month 6, 12, and 24 post-baseline ]
• Real-world treatment patterns for riociguat for PAH [ Time Frame: Up to 24 months ]
• Demographic of patients treated with riociguat [ Time Frame: Up to 24 months ]
• Clinical characteristics of patients treated with riociguat [ Time Frame: Up to 24 months ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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)
• Official Title: RiOciguAt UseRs Registry

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 Provided
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: US patients aged ≥18 years, diagnosed with PAH, who are naïve 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.
• Condition: Pulmonary Arterial Hypertension

Intervention

Drug: Riociguat (Adempas, BAY63-2521)

Follow clinical practice.

Study Groups/Cohorts

Patients diagnosed with PAH

Intervention: Drug: Riociguat (Adempas, BAY63-2521)

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: March 22, 2021): 500
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: July 11, 2025
• Estimated Primary Completion Date: July 11, 2025 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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:

  1. Blinded clinical trial
  2. Clinical trial involving an unapproved drug
  3. 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Bayer Clinical Trials Contact; (+)1-888-84 22937; clinical-trials-contact@bayer.com

• Listed Location Countries: Puerto Rico, United States

Administrative Information

• NCT Number: NCT04813926
• Other Study ID Numbers: 21427
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

Plan Description

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.

• Current Responsible Party: Bayer
• Original Responsible Party: Same as current
• Current Study Sponsor: Bayer
• Original Study Sponsor: Same as current
• Collaborators: Xcenda, LLC
• Investigators: Not Provided
• PRS Account: Bayer
• Verification Date: February 2023