A Study to Evaluate Efficacy and Safety of Oral BAY63-2521 in Patients With Pulmonary Arterial Hypertension (PAH)

Phase 3

Completed

• Conditions: Pulmonary Hypertension
• Interventions: Drug: Riociguat (Adempas, BAY63-2521); Drug: Placebo

• Registration Number: NCT00810693
• Lead Sponsor: Bayer

Brief Summary

The aim of the study is to assess the efficacy and safety of different doses of BAY63-2521 given orally for 12 weeks, in patients with symptomatic Pulmonary Arterial Hypertension (PAH).

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study Start: 2008-12-17
• Study First Submitted: 2008-12-17
• Study First Submitted QC: 2008-12-17
• Study First Posted: 2008-12-18
• Primary Completion: 2012-05-14
• Study Completion: 2012-05-14
• Results First Submitted: 2013-11-05
• Results First Submitted QC: 2014-01-24
• Results First Posted: 2014-02-26
• Status Verified: 2023-11
• Last Update Submitted: 2023-11-03
• Last Update Posted: 2023-11-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 445

Inclusion Criteria

• Male and female patients with symptomatic PAH (Idiopathic, Familial, Associated PAH due to connective tissue disease, congenital heart disease, portal hypertension with liver cirrhosis, or due to anorexigen or amphetamine use)
• Treatment naive patients and patients pre-treated with an Endothelin Antagonist or a Prostacyclinanalogue (except I.V.).

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Exclusion Criteria

• All types of pulmonary hypertension except subtypes of Venice Group I specified in the inclusion criteria, severe COPD (chronic obstructive pulmonary disease), uncontrolled arterial hypertension, left heart failure.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Riociguat (Adempas, BAY63-2521) up to 2.5 mg_IDT	Riociguat (Adempas, BAY63-2521)	Participants received Riociguat orally as a film-coated tablet up to 2.5mg three times daily (tid) (titration between 1.0 mg and 2.5 mg tid based on an individual dose titration (IDT) scheme) for 12 weeks
Riociguat (Adempas, BAY63-2521) up to 1.5 mg_IDT	Riociguat (Adempas, BAY63-2521)	Participants received Riociguat orally as a film-coated tablet up to 1.5mg three times daily (tid) (titration between 1.0 mg and 1.5 mg tid based on an individual dose titration (IDT) scheme) for 12 weeks
Placebo	Placebo	Participants received Placebo orally as a film-coated tablet three times daily (tid) for 12 weeks

Primary Outcome Measures

Name	Time	Method
6 Minutes Walking Distance (6MWD) - Change From Baseline to Week 12	Baseline and week 12	6-minute walking distance (6MWD) is a measure for the objective evaluation of a patient's functional exercise capacity.

Secondary Outcome Measures

Name	Time	Method
Pulmonary Vascular Resistance (PVR) - Change From Baseline to Week 12	Baseline and week 12	The pulmonary vascular resistance (PVR) is a calculated hemodynamic parameter. PVR is derived from the directly measured parameters mean pulmonary arterial pressure (PAPmean) and pulmonary capillary wedge pressure (PCWP), divided by the cardiac output (CO). PVR and PAPmean are acquired during a right heart catheterization. CO is a calculated hemodynamic parameter, too. Formula: PVR = 80\*(PAPmean - PCWP)/CO
N-terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP) - Change From Baseline to Week 12	Baseline and week 12	N-terminal pro-brain natriuretic peptide (NT-proBNP) levels in the blood are used for screening, diagnosis of acute congestive heart failure (CHF) and may be useful to establish prognosis in heart failure.
World Health Organization (WHO) Functional Class - Change From Baseline to Week 12	Baseline and week 12	The WHO functional assessment of pulmonary arterial hypertension ranged from functional class I (participants with PH but without resulting limitation of physical activity) to class IV (participants with PH with inability to carry out any physical activity without symptoms. These participants manifest signs of right-heart failure.). Changes to a lower WHO functional class resemble improvement; changes to a higher functional class resemble deterioration of PAH.
Percentage of Participants With Clinical Worsening	At week 12	The combined endpoint "time to clinical worsening", made up of the following components, defined by the first occurrence: all-cause mortality; heart/lung transplantation; atrial septostomy; first hospitalization due to pulmonary hypertension; start of a new pulmonary hypertension treatment; persistent worsening of 6MWD or WHO functional class due to deterioration of PH .
Living With Pulmonary Hypertension (LPH) Questionnaire - Change From Baseline to Week 12	Baseline and week 12	The self-reported Living with Pulmonary Hypertension (LPH) questionnaire is designed to measure the effects of PH and PH-specific treatments on an individual's quality of life. The LPH total score can range from 0 (best) to 105 (worst).
EQ-5D Utility Score - Change From Baseline to Week 12	Baseline and week 12	EQ-5D utility score is a Quality-of-Life participant reported outcome measure. The utility score is calculated based on five questions concerning problems with mobility, self-care, usual activities, pain/discomfort and anxiety/depression. An increase in the utility score represents an improvement in quality of life. The score ranges from -0.594 (worst answer in all five questions) to 1 (best answer in all five questions).
Borg CR 10 Scale - Change From Baseline to Week 12	Baseline and week 12	The Borg CR10 Scale is a participant reported outcome measure used in clinical diagnosis of e.g. breathlessness and dyspnea. It documents the participant's exertion during a physical test. Low values indicate low levels of exertion; high values indicate more intense exertion reported by the participant. The score ranges from 0 ("Nothing at all") to 10 ("Extremely strong - Maximal").

Trial Locations

Locations (1)

Royal Hobart Hospital; 🇦🇺 Hobart, Tasmania, Australia