Prospective, Non-interventional, Multi-center, Post-authorization Safety Study of Riociguat for Pulmonary Arterial Hypertension (PAH) (JPMS-PAH)

Drug Use Investigation of Riociguat for Pulmonary Arterial Hypertension (PAH)

The primary objective in this study is to collect post-marketing information on Riociguat safety.Thus, the information on adverse events (AEs) and adverse drug reactions (ADRs) that occur within the first 12 months and in the following 6 -year observation at most, after starting Riociguat treatment under the routine clinical practice will be collected.

The secondary objectives are collecting information on Riociguat effectiveness, such as 6-Minute Walking Distance (6MWD) Test. Since it is assumed that Riociguat is for long-term use, information on Pulmonary Arterial Hypertension (PAH) clinical worsening will also be surveyed once a year.

Study Overview

• Status: Completed
• Conditions: Hypertension, Pulmonary
• Intervention / Treatment: Drug: Riociguat (ADEMPAS, BAY63-2521)

Detailed Description

This local, prospective, non-interventional, multi-center study includes patients treated with Riociguat for PAH. A total of 600 patients (valid for safety analysis) is planned to be included in 4 years. This study is performed as an "all-case investigation" therefore; all patients who receive Riociguat treatment for PAH need to be registered, in principle. The treatment should be performed based on the product label in Japan. The standard observation period will last for 12 months from starting Riociguat treatment. Safety and effectiveness will be evaluated at the 3rd and 12th month of treatment. In addition, the extension observation period will be carried out as long as Riociguat treatment continues or at most for more 6 years. The purpose is to collect information on safety and effectiveness, such as adverse events and clinical worsening of PAH, once a year. Combining the standard observation and the extension observation period the patient could be followed for at most 7 years. When Riociguat treatment is terminated, the observation of the patient ends. The investigator should record data for each patient, as defined in the protocol, using the Electronic Data Capture (EDC) system. The duration of the study is of approximately 8 years from PAH indication approval.

• Study Type: Observational
• Enrollment (Actual): 882

Contacts and Locations

• Study Contact: Name: Bayer Clinical Trials Contact; Phone Number: (+) 1-888-8422937; Email: clinical-trials-contact@bayer.com

Study Locations

• Japan
  • Multiple Locations, Japan
    • Many Locations

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

Study population includes patients treated with Riociguat for PAH. This study is performed as an all-case investigation. Therefore, all patients who have been treated with Riociguat for PAH need to be registered in principle, until the target number of patients reached.

Description

Inclusion Criteria:

• Patients who are treated with Riociguat for PAH

Exclusion Criteria:

• N/A

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Riociguat; Riociguat treatment group	Drug: Riociguat (ADEMPAS, BAY63-2521); The treatment of Riociguat should comply with the local product information

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of participants with treatment emergent adverse events. Adverse events of special interest:Hypotension,Gastrointestinal disorder,Hemoptysis, pulmonary hemorrhage and Pulmonary congestion, pulmonary edema	Up to 7 years
Number of participants with adverse drug reaction	Up to 7 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in 6-Minute Walking Distance at 3 months and 12 months		Baseline and 3 months,Baseline and 12 months
Change from baseline in Pulmonary Vascular Resistance (PVR) after 3 and 12 months		Baseline and 3 months,Baseline and 12 months
Change from baseline in tricuspid regurgitation pressure gradient (TRPG) after 3 and 12 months		Baseline and 3 months,Baseline and 12 months
Change in blood concentration from baseline in brain natriuretic peptide / N-terminal pro-brain natriuretic peptide (BPN/NT-pro BNP) after 3 and 12 months.		Baseline and 3 months,Baseline and 12 months
Change from baseline in WHO functional class after 3 and 12 months		Baseline and 3 months,Baseline and 12 months
Time to Clinical Worsening	The 1st occurrence of the following events is recorded & will considered for the calculation of the combined endpoint : -Death (all-cause mortality)-Heart/lung transplantation -Atrial Septostomy -Hospitalization due to persistent worsening of Pulmonary Hypertension -Start of new PH specific treatment (endothelin receptor antagonists, prostacycline analogues)or modification of a preexisting Prostacycline analogues treatment)due to worsening Pulmonary Hypertension (WPH) -Persistent decrease in 6MWD due to WPH (e.g. more than 15 % from baseline or more than 30% compared to the last measurement)	Up to 7 years

Collaborators and Investigators

• Sponsor: Bayer
• Investigators: Study Director: Bayer Study Director, Bayer

Study record dates

Study Major Dates

• Study Start (Actual): June 29, 2015
• Primary Completion (Actual): April 17, 2023
• Study Completion (Actual): September 20, 2023

Study Registration Dates

• First Submitted: April 21, 2015
• First Submitted That Met QC Criteria: April 24, 2015
• First Posted (Estimated): April 29, 2015

Study Record Updates

• Last Update Posted (Estimated): January 29, 2024
• Last Update Submitted That Met QC Criteria: January 26, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: Pulmonary arterial hypertension; Japan; Post-marketing surveillance; Adempas; Riociguat; soluble guanylate cyclase (sGC) stimulator
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Respiratory Tract Diseases; Lung Diseases; Hypertension; Pulmonary Arterial Hypertension; Hypertension, Pulmonary; Molecular Mechanisms of Pharmacological Action; Enzyme Activators; Riociguat
• Other Study ID Numbers: 17519; AD1510JP (Other Identifier: Company Internal)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

IPD 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No