E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Idiopathic pulmonary fibrosis IPF ICD X J84.1other interstitial pulmonary |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 6.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10037383 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
OBJECTIVES To assess the efficacy of treatment with pirfenidone 2403 mg/d compared with placebo in patients with idiopathic pulmonary fibrosis IPF To assess the safety of treatment with pirfenidone 1197 and 2403 mg/d compared with placebo in patients with IPF To characterize the pharmacokinetic disposition of pirfenidone in patients with IPF |
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E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Clinical symptoms consistent with IPF of 3 months duration Diagnosis of IPF within 48 months of randomization Age 40 through 80 years, inclusive High-resolution computed tomographic HRCT scan showing a pattern of disease consistent with a confident definite radiographic diagnosis of usual interstitial pneumonia UIP /IPF. For patients with surgical lung biopsy showing definite or probable UIP, the HRCT criterion of probable IPF is sufficient. For patients aged 50 years open or video-assisted thoracoscopic VATS lung biopsy showing definite or probable UIP within 48 months of randomization. In addition, there are no features supporting an alternative diagnosis on transbronchial biopsy or bronchoalveolar lavage BAL if performed. For patients aged 50 years At least one of the following diagnostic findings, as well as the absence of any features on specimens resulting from any of these procedures that support an alternative diagnosis, within 48 months of randomization Open or VATS lung biopsy showing definite or probable UIP Transbronchial biopsy showing no features to support an alternative diagnosis BAL showing no features to support an alternative diagnosis |
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E.4 | Principal exclusion criteria |
Not a suitable candidate for enrollment or unlikely to comply with the requirements of this study, in the opinion of the investigator Premature withdrawal from a randomized IPF clinical trial in the previous 2 years for any reason other than Sponsor decision or current participation in a clinical drug trial Forced expiratory volume in the first second FEV1 /FVC ratio 0.7 after administration of bronchodilator Bronchodilator Response defined by an absolute increase in FEV1 or FVC of 8805;12 predicted or 200 mL after bronchodilator use The change in FVC between the Screen Visit and Day 1 Baseline cannot vary by more than 10 absolute difference and the Bronchodilator Response, Baseline FVC and FEV1/FVC ratio must all continue to meet the inclusion and exclusion criteria Residual volume RV 120 of predicted before administration of bronchodilator History of clinically significant environmental exposure known to cause pulmonary fibrosis including but not limited to drugs, asbestos, beryllium, radiation, domestic birds Known explanation for interstitial lung disease, including but not limited to radiation, sarcoidosis, hypersensitivity pneumonitis, bronchiolitis obliterans organizing pneumonia, human immunodeficiency virus HIV , viral hepatitis and cancer Diagnosis of any connective tissue disease, including but not limited to scleroderma, systemic lupus erythematosus, and rheumatoid arthritis Clinical evidence of active infection, including but not limited to bronchitis, pneumonia, sinusitis, urinary tract infection, or cellulitis On a lung transplantation waiting list at time of randomization Unable to undergo pulmonary function testing |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary Efficacy Outcome Variable The primary outcome variable is the absolute change in percent predicted FVC from Baseline to Week 60. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | Information not present in EudraCT |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 10 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 10 |