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Impact of Multiple Doses of BAY63-2521 on Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Patients With Interstitial Lung Disease (ILD) Associated Pulmonary Hypertension (PH)

Phase 2
Active, not recruiting
Conditions
Hypertension, Pulmonary
Interventions
Drug: Riociguat (Adempas, BAY63-2521)
Registration Number
NCT00694850
Lead Sponsor
Bayer
Brief Summary

The purpose of this study is to assess multiple ascending doses of a new drug (BAY63-2521) given orally, to evaluate if it is safe and can help to improve the well-being, symptoms (e.g. disturbed breathing) and outcome of pulmonary hypertension associated with lung fibrosis. Patients living with pulmonary hypertension associated with interstitial lung disease have a risk of increased number of hospitalisations because of worsening of their condition. Until now there is no approved medication for this disease. The current treatment of pulmonary hypertension associated with interstitial lung disease consists: of oxygen and medical treatment with vasodilators, e.g. so-called Calcium-antagonists. Therefore, there is a need for new drugs in the treatment of pulmonary hypertension associated with interstitial lung disease.

Detailed Description

Not available

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
22
Inclusion Criteria
  • Diagnosis of an interstitial lung disease (usual interstitial pneumonia [UIP], nonspecific interstitial pneumonia [NSIP] or sarcoidosis) with high resolution CT and a total lung capacity (TLC) ≤ 90% or scleroderma associated pulmonary arterial hypertension (PAH) with total lung capacity (TLC) ≤ 80%.
  • Interstitial lung disease (ILD) must have been stable for at least 3 months (decrease in forced vital capacity (FVC)< 10% and diffusing capacity of lung for carbon monoxide (DLco) < 15 % in 3 months), i.e. no significant changes in pulmonary function testing and stable medication in terms of ILD (e.g., corticosteroids, immunosuppressants)
  • Mean pulmonary vascular resistance (PVR) > 400 dyne sec cm-5 or mean pulmonary arterial pressure (PAP mean) > 30 mmHg
  • Pulmonary capillary wedge pressure (PCWP) < 15 mmHg
  • Hemodynamic parameters at baseline (PAP, PCWP, cardiac output [CO], systemic mean arterial pressure [SAP])
  • High resolution computer tomography (HRCT) (should not be older than 12 months prior start of the study)
  • Heart rate > 55 beats per minute (BPM) and < 105 BPM at rest
  • Systolic blood pressure (SBP) > 90 mmHg
  • World Health Organisation (WHO) functional class II, III and IV
  • 6 Minute Walking Test (6MWT) > 100m and < 450 m
  • Stable controlled arterial hypertension according to current guidelines
  • Women of childbearing potential will be included in the study if the pregnancy test is negative and combination of condoms with a safe and highly effective contraception method (hormonal contraception with implants or combined oral contraceptives, certain intra-uterine devices [IUDs]) is granted.
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Exclusion Criteria
  • Co-medication:

    • Patients pretreated with specific medication for pulmonary arterial hypertension (PAH) like endothelin receptor antagonists, prostaglandins or phosphodiesterase type 5 (PDE 5) blockers are excluded from the trial.
    • Requirement for concomitant use of nitrates are contraindicated.
  • Pre-existing clinically relevant lung disease other than ILD including.

    • Bronchial asthma and Chronic Obstructive Pulmonary Disease (COPD) with a forced expiratory volume in one second (FEV1)/FVC <60% pred., active tuberculosis
    • Pulmonary hypertension of another WHO group (I, II, IV and V)
    • Severe congenital abnormalities of the lungs, thorax and diaphragm
    • Clinical or radiological evidence of a pulmovenoocclusive disease (PVOD)
  • Systemic hemodynamics

    • Acute or severe chronic left heart failure (ejection fraction (EF) < 50%)
    • Severe coronary artery disease (CAD; EF < 50%); CAD patients must be asymptomatic and stable
    • Congenital or acquired valvular or myocardial disease if clinically significant apart from tricuspid valvular insufficiency due to pulmonary hypertension
  • Pulmonary function

    • TLC predicted < 30%
    • FEV1 (related to FVC) < 60% predicted
  • Blood gases at room air

    • Arterial partial carbon dioxide pressure (Pa CO2) > 45 mmHg
    • Arterial partial oxygen pressure (Pa O2) < 50 mmHg at O2 supply >/= 4 L/min
  • Peripheral organ function

    • Moderate or severe hepatic insufficiency (Child-Pugh Class Band C and/or total bilirubin > 2.5 mg/dl (0.043 mmol/L); and/or hepatic transaminases >3 upper limit normal [ULN])
    • Moderate or severe renal insufficiency (creatinine > 2 mg/dl) or creatinine clearance according to Cockroft-Gault formula < 35 mL/ min
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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Arm 1Riociguat (Adempas, BAY63-2521)-
Primary Outcome Measures
NameTimeMethod
Safety and tolerability12 weeks treatment
Secondary Outcome Measures
NameTimeMethod
Quality of life assessmentsat baseline, after 6 weeks, after 12 weeks, Follow-up and at each visit during long term extension phase

The assessment will be stopped after protocol amendment 4, which was effective since Jan 06, 2014

Modified borg scaleat every study visit except at Follow-up

The assessment will be stopped after protocol amendment 4, which was effective since Jan 06, 2014

Hemodynamic parametersoptional after 12weeks
Blood pressure and heart rateat each study visit during run-in and treatment phase and long term extension
Pharmacokineticsat every study visit except at run-in and Follow-up

The assessment will be stopped after protocol amendment 4, which was effective since Jan 06, 2014

6-Minute Walk Testat every study visit except at Follow-up
Electrocardiogram (ECG)at each study visit during run-in and treatment phase and long term extension

The assessment will be stopped after protocol amendment 4, which was effective since Jan 06, 2014

Laboratory Parametersat each study visit during run-in and treatment phase and long term extension

The assessment will be stopped after protocol amendment 4, which was effective since Jan 06, 2014

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