Prospective, Non-interventional, Multi-center Post-authorization Safety Study of Riociguat for Chronic Thromboembolic Pulmonary Hypertension (CTEPH ) (JPMS-CTEPH)

Drug Use Investigation of Riociguat for ChronicThromboembolic Pulmonary Hypertension (CTEPH)

This local, prospective, non-interventional, multi-center study includes patients treated with Riociguat for inoperable CTEPH(Chronic thromboembolic pulmonary hypertension)/ persistent or recurrent CTEPH after surgical treatment. It is planned to include a total of 400 patients (valid for safety analysis). This study is performed as an all-case investigation. The treatment of Riociguat is performed based on the product label in Japan. The standard observation period is 12 months from the 1st treatment of Riociguat. Safety and effectiveness are evaluated at 4th and 12th month. In addition, the extension observation is carried out once a year for 7 years at the longest to collect information on safety and effectiveness as long as Riociguat treatment continues. When the treatment of Riociguat is terminated, observation of a patient ends. For each patient, the investigator records data as defined in the protocol at each evaluation point by using the Electronic Data Capture (EDC) system. The duration of the study is approximately 9 years from launch.

Study Overview

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

• Study Type: Observational
• Enrollment (Actual): 1298

Contacts and Locations

Study Locations

• Japan
  • Multiple Locations, Japan

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 inoperable CTEPH/ persistent or recurrent CTEPH after surgical treatment. This study is performed as an all-case investigation. Therefore, all patients who have been treated with Riociguat for CTEPH need to be registered in principle, until the target number of patients reached.

Description

Inclusion Criteria:

• Patients who are treated with Riociguat for CTEPH

Exclusion Criteria:

• Patients who are contraindicated based on the product label

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
Group 1; Riociguat treatment group	Drug: Riociguat (ADEMPAS, BAY63-2521); The treatment of Riociguat should comply with the local product information (start dose: 3 mg/day, maximum dose: 7.5 mg/day)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of participants with treatment emergent adverse events and adverse drug reactions	up to 8 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in 6MWD (6-Minute Walking Distance) after 4 and 12 months		baseline and 4 months, and 12 months
Change from baseline in Pulmonary Vascular Resistance (PVR) after 4 and 12 months		baseline and 4 months, and 12 months
Change from baseline in TRPG (Tricuspid Regurgitation Pressure Gradient) after 4 and 12 months		baseline and 4 months, and 12 months
Change from baseline in BNP/NT-pro BNP after 4 and 12 months		baseline and 4 months, and 12 months
Change from baseline in WHO (World Health Organization) functional class after 4 and 12 months		baseline and 4 months, and 12 months
Time to Clinical Worsening	The first occurrence of the following events is recorded and will be considered for the calculation of the combined endpoint: • Death (all-cause mortality) • Heart/lung transplantation • Rescue Pulmonary Endarterectomy, Rescue Balloon Pulmonary Angioplasty or Hospitalization due to persistent worsening of Pulmonary Hypertension • Start of new PH specific treatment due to worsening Pulmonary Hypertension. • Persistent decrease in 6MWD due to worsening pulmonary hypertension • Persistent worsening of functional class due to deterioration of Pulmonary Hypertension.	up to 8 years

Collaborators and Investigators

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

Publications and helpful links

General Publications

• Tanabe N, Ogo T, Hatano M, Kigawa A, Sunaya T, Sato S. Safety and effectiveness of riociguat for chronic thromboembolic pulmonary hypertension in real-world clinical practice: interim data from post-marketing surveillance in Japan. Pulm Circ. 2020 Jul 23;10(3):2045894020938986. doi: 10.1177/2045894020938986. eCollection 2020 Jul-Sep.

Study record dates

Study Major Dates

• Study Start (Actual): July 16, 2014
• Primary Completion (Actual): April 3, 2023
• Study Completion (Actual): September 20, 2023

Study Registration Dates

• First Submitted: April 16, 2014
• First Submitted That Met QC Criteria: April 16, 2014
• First Posted (Estimated): April 21, 2014

Study Record Updates

• Last Update Posted (Actual): April 11, 2024
• Last Update Submitted That Met QC Criteria: April 9, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Riociguat, Adempas, sGC stimulator, CTEPH, Japan, PMS
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Respiratory Tract Diseases; Lung Diseases; Hypertension; Hypertension, Pulmonary; Molecular Mechanisms of Pharmacological Action; Enzyme Activators; Riociguat
• Other Study ID Numbers: 16843; ADEMPAS-CTEPH (Other Identifier: company internal)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: Availability of this study's data will 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.clinicalstudydatarequest.com 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 Study sponsors 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
• product manufactured in and exported from the U.S.: No