A Study to Evaluate Efficacy and Safety of Oral BAY63-2521 in Patients With Pulmonary Arterial Hypertension (PAH) (PATENT-1)
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ClinicalTrials.gov Identifier: NCT00810693 |
Recruitment Status :
Completed
First Posted : December 18, 2008
Results First Posted : February 26, 2014
Last Update Posted : November 28, 2016
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Sponsor:
Bayer
Information provided by (Responsible Party):
Bayer
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Study Type | Interventional |
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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 |
Enrollment | 445 |
Participant Flow
Recruitment Details | 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 | 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. |
Arm/Group Title | Riociguat (Adempas, BAY63-2521) up to 2.5 mg_IDT | Riociguat (Adempas, BAY63-2521) up to 1.5 mg_IDT | Placebo |
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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 | 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 | Participants received Placebo orally as a film-coated tablet three times daily (tid) for 12 weeks |
Period Title: Treatment Period | |||
Started | 254 | 64 [1] | 127 [1] |
Participants Received Treatment | 254 | 63 | 126 |
Completed | 237 | 57 | 111 |
Not Completed | 17 | 7 | 16 |
Reason Not Completed | |||
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
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Period Title: Follow-up Period (FUP) | |||
Started | 23 [1] | 7 [1] | 16 [1] |
Completed | 15 | 4 | 12 |
Not Completed | 8 | 3 | 4 |
Reason Not Completed | |||
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.
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Baseline Characteristics
Arm/Group Title | Riociguat (Adempas, BAY63-2521) up to 2.5 mg_IDT | Riociguat (Adempas, BAY63-2521) up to 1.5 mg_IDT | Placebo | Total | |
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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 | 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 | Participants received Placebo orally as a film-coated tablet three times daily (tid) for 12 weeks | Total of all reporting groups | |
Overall Number of Baseline Participants | 254 | 63 | 126 | 443 | |
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[Not Specified]
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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Number Analyzed | 254 participants | 63 participants | 126 participants | 443 participants | |
51.1 (16.6) | 48.8 (16.1) | 50.7 (16.5) | 50.6 (16.5) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 254 participants | 63 participants | 126 participants | 443 participants | |
Female |
203 79.9%
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49 77.8%
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98 77.8%
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350 79.0%
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Male |
51 20.1%
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14 22.2%
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28 22.2%
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93 21.0%
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Race/Ethnicity, Customized
Measure Type: Number Unit of measure: Participants |
Number Analyzed | 254 participants | 63 participants | 126 participants | 443 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
Measure Type: Number Unit of measure: Participants |
Number Analyzed | 254 participants | 63 participants | 126 participants | 443 participants |
Therapy-Naive | 123 | 32 | 66 | 221 | |
Pre-Treated | 131 | 31 | 60 | 222 | |
pre-treated with endothelin receptor antagonist
[1] Measure Type: Number Unit of measure: Participants |
Number Analyzed | 254 participants | 63 participants | 126 participants | 443 participants |
Yes | 113 | 27 | 54 | 194 | |
No | 141 | 36 | 72 | 249 | |
[1]
Measure Description: Subjects pre-treated with an endothelin receptor antagonist.
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pre-treated with prostacyclin analogue
[1] Measure Type: Number Unit of measure: Participants |
Number Analyzed | 254 participants | 63 participants | 126 participants | 443 participants |
Yes | 20 | 4 | 7 | 31 | |
No | 234 | 59 | 119 | 412 | |
[1]
Measure Description: Subjects pre-treated with a prostacyclin analogue.
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PAH subtype
[1] Measure Type: Number Unit of measure: Participants |
Number Analyzed | 254 participants | 63 participants | 126 participants | 443 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]
Measure Description: 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)
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BMI
Mean (Standard Deviation) Unit of measure: Kg/m^2 |
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Number Analyzed | 254 participants | 63 participants | 126 participants | 443 participants | |
25.91 (5.48) | 26.85 (5.35) | 26.26 (5.92) | 26.14 (5.59) | ||
Baseline 6MWD
[1] Mean (Standard Deviation) Unit of measure: Meters |
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Number Analyzed | 254 participants | 63 participants | 126 participants | 443 participants | |
361.4 (67.7) | 363.2 (66.6) | 367.8 (74.6) | 363.5 (69.5) | ||
[1]
Measure Description: 6-minute walking distance (6MWD) is a measure for the objective evaluation of a patient's functional exercise capacity.
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WHO (World Health Organization) functional class
[1] Measure Type: Number Unit of measure: Participants |
Number Analyzed | 254 participants | 63 participants | 126 participants | 443 participants |
I | 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]
Measure Description: 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.
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Pulmonary vascular resistance
[1] Mean (Standard Deviation) Unit of measure: Dn*s*cm^-5 |
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Number Analyzed | 254 participants | 63 participants | 126 participants | 443 participants | |
790.96 (452.60) | 847.81 (548.17) | 834.06 (476.71) | 810.89 (473.45) | ||
[1]
Measure Description: 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
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Outcome Measures
Adverse Events
Limitations and Caveats
[Not Specified]
More Information
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 embargo can be up to 6 months (equal to the 180 days), moreover if it is necessary the embargo period can be prolonged to expiry of priority year.
Results Point of Contact
Name/Title: | Therapeutic Area Head |
Organization: | Bayer |
EMail: | 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 |
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 |