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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00810693




Registration number
NCT00810693
Ethics application status
Date submitted
17/12/2008
Date registered
18/12/2008
Date last updated
28/11/2016

Titles & IDs
Public title
A Study to Evaluate Efficacy and Safety of Oral BAY63-2521 in Patients With Pulmonary Arterial Hypertension (PAH)
Scientific 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)
Secondary ID [1] 0 0
2008-003482-68
Secondary ID [2] 0 0
12934
Universal Trial Number (UTN)
Trial acronym
PATENT-1
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pulmonary Hypertension 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Cardiovascular 0 0 0 0
Hypertension

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Riociguat (Adempas, BAY63-2521)
Treatment: Drugs - Riociguat (Adempas, BAY63-2521)
Treatment: Drugs - Placebo

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

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

Placebo Comparator: Placebo - Participants received Placebo orally as a film-coated tablet three times daily (tid) for 12 weeks


Treatment: Drugs: Riociguat (Adempas, BAY63-2521)
BAY63-2521: 1mg tid - 2.5mg tid orally for 12 weeks

Treatment: Drugs: Riociguat (Adempas, BAY63-2521)
BAY63-2521: 1.5mg tid orally for 12 weeks

Treatment: Drugs: Placebo
Matching Placebo tid orally for 12 weeks

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
6 Minutes Walking Distance (6MWD) - Change From Baseline to Week 12 - 6-minute walking distance (6MWD) is a measure for the objective evaluation of a patient's functional exercise capacity.
Timepoint [1] 0 0
Baseline and week 12
Secondary outcome [1] 0 0
Pulmonary Vascular Resistance (PVR) - Change From Baseline to 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
Timepoint [1] 0 0
Baseline and week 12
Secondary outcome [2] 0 0
N-terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP) - Change From Baseline to 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.
Timepoint [2] 0 0
Baseline and week 12
Secondary outcome [3] 0 0
World Health Organization (WHO) Functional Class - Change From Baseline to 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.
Timepoint [3] 0 0
Baseline and week 12
Secondary outcome [4] 0 0
Percentage of Participants With Clinical Worsening - 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 .
Timepoint [4] 0 0
At week 12
Secondary outcome [5] 0 0
Borg CR 10 Scale - Change From Baseline to 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").
Timepoint [5] 0 0
Baseline and week 12
Secondary outcome [6] 0 0
EQ-5D Utility Score - Change From Baseline to 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).
Timepoint [6] 0 0
Baseline and week 12
Secondary outcome [7] 0 0
Living With Pulmonary Hypertension (LPH) Questionnaire - Change From Baseline to 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).
Timepoint [7] 0 0
Baseline and week 12

Eligibility
Key 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.).
Minimum age
18 Years
Maximum age
80 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s

The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,TAS,VIC
Recruitment hospital [1] 0 0
- Darlinghurst
Recruitment hospital [2] 0 0
- New Lambton Heights
Recruitment hospital [3] 0 0
- Auchenflower
Recruitment hospital [4] 0 0
- Chermside
Recruitment hospital [5] 0 0
Royal Hobart Hospital - Hobart
Recruitment hospital [6] 0 0
- Heidelberg
Recruitment hospital [7] 0 0
- Prahran
Recruitment postcode(s) [1] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [2] 0 0
2305 - New Lambton Heights
Recruitment postcode(s) [3] 0 0
4066 - Auchenflower
Recruitment postcode(s) [4] 0 0
4032 - Chermside
Recruitment postcode(s) [5] 0 0
7000 - Hobart
Recruitment postcode(s) [6] 0 0
3084 - Heidelberg
Recruitment postcode(s) [7] 0 0
3181 - Prahran
Recruitment outside Australia
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United States of America
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Alabama
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Kentucky
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Lisboa
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Linköping
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Lund
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Istanbul
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Izmir
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United Kingdom
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Cambridgeshire
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West Dunbartonshire
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Newcastle
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Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Bayer
Address
Country

Ethics approval
Ethics application status

Summary
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).
Trial website
https://clinicaltrials.gov/show/NCT00810693
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Bayer Study Director
Address 0 0
Bayer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications