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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.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00810693
First Posted: December 18, 2008
Last Update Posted: November 28, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Bayer
Results First Submitted: November 5, 2013  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Pulmonary Hypertension
Interventions: Drug: Riociguat (Adempas, BAY63-2521)
Drug: Placebo

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Only participants with symptomatic Pulmonary arterial hypertension (PAH) could participate in this study. Both treatment-naïve participants and participants pre-treated with an endothelin receptor antagonist or a non-intravenous prostacyclin analogue could be included.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
586 participants were enrolled in 124 study centers in 30 countries worldwide. 141 of the 586 enrolled participants were not randomized (adverse event [4], protocol violation [129], withdrawal by subject [8]). 445 of the 586 participants were randomized. 443 of the 445 randomized participants received study medication.

Reporting Groups
  Description
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
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 Participants received Placebo orally as a film-coated tablet three times daily (tid) for 12 weeks

Participant Flow for 2 periods

Period 1:   Treatment Period
    Riociguat (Adempas, BAY63-2521) up to 2.5 mg_IDT   Riociguat (Adempas, BAY63-2521) up to 1.5 mg_IDT   Placebo
STARTED   254   64 [1]   127 [1] 
Participants Received Treatment   254   63   126 
COMPLETED   237   57   111 
NOT COMPLETED   17   7   16 
Adverse Event                8                1                7 
Death                0                1                2 
Lack of Efficacy                0                0                1 
Lost to Follow-up                1                0                0 
Non-compliance                1                0                0 
Protocol Violation                1                2                2 
Withdrawal by Subject                6                2                3 
Not treated                0                1                1 
[1] 1 randomized but not treated

Period 2:   Follow-up Period (FUP)
    Riociguat (Adempas, BAY63-2521) up to 2.5 mg_IDT   Riociguat (Adempas, BAY63-2521) up to 1.5 mg_IDT   Placebo
STARTED   23 [1]   7 [1]   16 [1] 
COMPLETED   15   4   12 
NOT COMPLETED   8   3   4 
Adverse Event                3                1                1 
Death                2                0                1 
Lost to Follow-up                1                0                2 
Protocol Violation                0                1                0 
Withdrawal by Subject                2                1                0 
[1] Started FUP only if prematurely withdrawn from trt period or if not entering LTE study.



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
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
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 Participants received Placebo orally as a film-coated tablet three times daily (tid) for 12 weeks
Total Total of all reporting groups

Baseline Measures
   Riociguat (Adempas, BAY63-2521) up to 2.5 mg_IDT   Riociguat (Adempas, BAY63-2521) up to 1.5 mg_IDT   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 254   63   126   443 
Age 
[Units: Years]
Mean (Standard Deviation)
 51.1  (16.6)   48.8  (16.1)   50.7  (16.5)   50.6  (16.5) 
Gender 
[Units: Participants]
       
Female   203   49   98   350 
Male   51   14   28   93 
Race/Ethnicity, Customized 
[Units: Participants]
       
White   161   33   78   272 
Black or African American   4   1   1   6 
Asian   79   22   38   139 
Mixed   1   0   1   2 
Not reported   9   7   8   24 
Prior PH therapy 
[Units: Participants]
       
Therapy-Naive   123   32   66   221 
Pre-Treated   131   31   60   222 
pre-treated with endothelin receptor antagonist [1] 
[Units: Participants]
       
Yes   113   27   54   194 
No   141   36   72   249 
[1] Subjects pre-treated with an endothelin receptor antagonist.
pre-treated with prostacyclin analogue [1] 
[Units: Participants]
       
Yes   20   4   7   31 
No   234   59   119   412 
[1] Subjects pre-treated with a prostacyclin analogue.
PAH subtype [1] 
[Units: Participants]
       
Idiopathic PAH   149   39   84   272 
Familial PAH   7   1   1   9 
Connective tissue disease assoc. PAH   71   15   25   111 
Congenital heart disease (operated) assoc. PAH   15   8   12   35 
Portal Pulmonary hypertension   11   0   2   13 
Anorexigen or Amphtamin assoc: PAH   1   0   2   3 
[1] Subtypes acc. to the Venice Clinical Classification of PH, Group 1 (idiopathic PAH, familial PAH, associated PAH due to [1] connective tissue disease, [2] congenital heart disease, [3] portal hypertension with liver cirrhosis, or [4] anorexigen or amphetamine use)
BMI 
[Units: Kg/m^2]
Mean (Standard Deviation)
 25.91  (5.48)   26.85  (5.35)   26.26  (5.92)   26.14  (5.59) 
Baseline 6MWD [1] 
[Units: Meters]
Mean (Standard Deviation)
 361.4  (67.7)   363.2  (66.6)   367.8  (74.6)   363.5  (69.5) 
[1] 6-minute walking distance (6MWD) is a measure for the objective evaluation of a patient's functional exercise capacity.
WHO (World Health Organization) functional class [1] 
[Units: Participants]
       
 5   5   4   14 
II   108   19   60   187 
III   140   39   58   237 
IV   1   0   3   4 
missing   0   0   1   1 
[1] 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.
Pulmonary vascular resistance [1] 
[Units: Dn*s*cm^-5]
Mean (Standard Deviation)
 790.96  (452.60)   847.81  (548.17)   834.06  (476.71)   810.89  (473.45) 
[1] 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


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   6 Minutes Walking Distance (6MWD) - Change From Baseline to Week 12   [ Time Frame: Baseline and week 12 ]

2.  Secondary:   Pulmonary Vascular Resistance (PVR) - Change From Baseline to Week 12   [ Time Frame: Baseline and week 12 ]

3.  Secondary:   N-terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP) - Change From Baseline to Week 12   [ Time Frame: Baseline and week 12 ]

4.  Secondary:   World Health Organization (WHO) Functional Class - Change From Baseline to Week 12   [ Time Frame: Baseline and week 12 ]

5.  Secondary:   Percentage of Participants With Clinical Worsening   [ Time Frame: At week 12 ]

6.  Secondary:   Borg CR 10 Scale - Change From Baseline to Week 12   [ Time Frame: Baseline and week 12 ]

7.  Secondary:   EQ-5D Utility Score - Change From Baseline to Week 12   [ Time Frame: Baseline and week 12 ]

8.  Secondary:   Living With Pulmonary Hypertension (LPH) Questionnaire - Change From Baseline to Week 12   [ Time Frame: Baseline and week 12 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Therapeutic Area Head
Organization: Bayer
e-mail: clinical-trials-contact@bayerhealthcare.com


Publications of Results:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Bayer
ClinicalTrials.gov Identifier: NCT00810693     History of Changes
Other Study ID Numbers: 12934
2008-003482-68 ( EudraCT Number )
First Submitted: December 17, 2008
First Posted: December 18, 2008
Results First Submitted: November 5, 2013
Results First Posted: February 26, 2014
Last Update Posted: November 28, 2016