Trial record 3 of 10 for:    PATENT PAH

A Study to Evaluate Efficacy and Safety of Oral BAY63-2521 in Patients With Pulmonary Arterial Hypertension (PAH) (PATENT-1)

This study has been completed.
Information provided by (Responsible Party):
Bayer Identifier:
First received: December 17, 2008
Last updated: October 27, 2014
Last verified: October 2014
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).

Condition Intervention Phase
Pulmonary Hypertension
Drug: Riociguat (Adempas, BAY63-2521)
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Randomized, Double-blind, Placebo-controlled, Multi-centre, Multi-national Study to Evaluate the Efficacy and Safety of Oral BAY63-2521 (1 mg, 1.5 mg, 2 mg, or 2.5 mg Tid) in Patients With Symptomatic Pulmonary Arterial Hypertension (PAH)

Resource links provided by NLM:

Further study details as provided by Bayer:

Primary Outcome Measures:
  • 6 Minutes Walking Distance (6MWD) - Change From Baseline to Week 12 [ Time Frame: Baseline and week 12 ] [ Designated as safety issue: No ]
    6-minute walking distance (6MWD) is a measure for the objective evaluation of a patient's functional exercise capacity.

Secondary Outcome Measures:
  • Pulmonary Vascular Resistance (PVR) - Change From Baseline to Week 12 [ Time Frame: Baseline and week 12 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline and week 12 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline and week 12 ] [ Designated as safety issue: No ]
    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 [ Time Frame: At week 12 ] [ Designated as safety issue: No ]
    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 .

  • Borg CR 10 Scale - Change From Baseline to Week 12 [ Time Frame: Baseline and week 12 ] [ Designated as safety issue: No ]
    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").

  • EQ-5D Utility Score - Change From Baseline to Week 12 [ Time Frame: Baseline and week 12 ] [ Designated as safety issue: No ]
    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).

  • Living With Pulmonary Hypertension (LPH) Questionnaire - Change From Baseline to Week 12 [ Time Frame: Baseline and week 12 ] [ Designated as safety issue: No ]
    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).

Enrollment: 445
Study Start Date: December 2008
Study Completion Date: May 2012
Primary Completion Date: May 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Riociguat (Adempas, BAY63-2521) up to 2.5 mg_IDT
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
Drug: Riociguat (Adempas, BAY63-2521)
BAY63-2521: 1mg tid - 2.5mg tid orally for 12 weeks
Experimental: Riociguat (Adempas, BAY63-2521) up to 1.5 mg_IDT
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
Drug: Riociguat (Adempas, BAY63-2521)
BAY63-2521: 1.5mg tid orally for 12 weeks
Placebo Comparator: Placebo
Participants received Placebo orally as a film-coated tablet three times daily (tid) for 12 weeks
Drug: Placebo
Matching Placebo tid orally for 12 weeks


Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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.).

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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Please refer to this study by its identifier: NCT00810693

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Sponsors and Collaborators
Study Director: Bayer Study Director Bayer
  More Information

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Bayer Identifier: NCT00810693     History of Changes
Other Study ID Numbers: 12934  2008-003482-68 
Study First Received: December 17, 2008
Results First Received: November 5, 2013
Last Updated: October 27, 2014
Health Authority: Argentina: National Administration of Drugs, Foods and Medical Technology
Australia: Department of Health
Austria: Ministry of Labor, Health and Social Affairs
Belgium: Ministry of Social Affairs, Public Health and the Environment
Brazil: Ministry of Health
Canada: Health Protection Branch
China: Ministry of Health- State Food and Drug Administration
Czech Republic: Ministry of Health
Denmark: Danish Medicines Agency
France: Ministry of Health
Germany: Federal Institute for Drugs and Medical Devices
Greece: Ministry of Health and Welfare
Ireland: Irisch Medicines Board
Israel: Ministry of Health
Italy: Ministry of Health
Japan: Ministry of Health and Welfare
Korea: Food and Drug Administration
Mexico: Ministry of Health
Netherlands: Medicines Evaluetion Board
New Zealand: Medicines and Medical Devices Safety Authority
Poland: Ministry of Health and Social Security - Drug Institute
Portugal: Ministry of Health
Russia: Ministry of Health
Singapore: Ministry of Health
Spain: Ministry of Health and Consumption
Sweden: Medical Products Agency
Switzerland: Federal Office of Public Health
Thailand: Ministry of Public Health
Taiwan: Department of Health
Turkey: Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration

Keywords provided by Bayer:
Pulmonary arterial hypertension

Additional relevant MeSH terms:
Familial Primary Pulmonary Hypertension
Hypertension, Pulmonary
Cardiovascular Diseases
Lung Diseases
Respiratory Tract Diseases
Vascular Diseases processed this record on May 26, 2016