E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Idiopathic Pulmonary Fibrosis |
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E.1.1.1 | Medical condition in easily understood language |
Idiopathic Pulmonary Fibrosis (IPF) is a progressive chronic disease of unknown cause that results in scarring of the lung |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10021240 |
E.1.2 | Term | Idiopathic pulmonary fibrosis |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10037383 |
E.1.2 | Term | Pulmonary fibrosis |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
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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 |
To demonstrate a reduction of lung function decline, as measured by a change of the yearly rate of decline of forced vital capacity (FVC). |
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E.2.2 | Secondary objectives of the trial |
To assess the patient's perception of his/her disease, and the time to IPF exacerbation.
To investigate respiratory and overall survival, as well as causes of mortality.
To assess safety and tolerability. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Age >= 40 years;
IPF diagnosed, according to most recent ATS/ERS/JRS/ALAT IPF guideline for diagnosis and management, within 5 years;
Combination of HRCT pattern, and if available surgical lung biopsy pattern, as assessed by central reviewers, are consistent with diagnosis of IPF;
Dlco (corrected for Hb): 30%-79% predicted of normal;
FVC ≥ 50% predicted of normal |
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E.4 | Principal exclusion criteria |
Laboratory parameters (AST, ALT > 1.5 x ULN; Bilirubin > 1.5 x ULN);
Relevant airways obstruction (i.e. pre-bronchodilator FEV1/FVC < 0.7);
Patient likely to have lung transplantation during study (being on transplantation list is acceptable for participation);
Myocardial infarction within 6 months;
Unstable angina within 1 month;
Bleeding risk (genetic predisposition; fibrinolysis or full-dose therapeutic anticoagulation or high dose antiplatelet therapy; history of hemorrhagic CNS event within 12 months; haemoptysis or haematuria or active gastro-intestinal bleeding or ulcers or major injury or surgery within 3 months);
Thrombotic risk (inherited predisposition; history of thrombotic event (including stroke and transient ischemic attacks) within 12 months; International normalised ratio (INR) > 2; prolongation of prothrombin time (PT) and partial thromboplastin time
(PTT) by > 50% of institutional ULN);
N-Acetyl Cystein, prednisone > 15mg/day or equivalent received within 2 weeks of visit 1;
Pirfenidone, azathioprine, cyclophosphamide, cyclosporine A received within 8
weeks of visit 1. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Annual rate of decline in FVC (expressed in mL over 52 weeks). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
The data for the primary endpoint (FVC) are collected over the whole study period and are evaluated at the end of the trial. |
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E.5.2 | Secondary end point(s) |
Patient reported outcomes (SGRQ, SOBQ, CASA-Q(CD), EQ-5D, PGI-C); Time to first IPF exacerbation;
Further analyses regarding acute exacerbations; Further analyses on lung function (e.g. FVC, Dlco SPO2); Survival (on-treatment, overall); respiratory mortality. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
The data for the secondary endpoints are collected over the whole study period and are evaluated at the end of the trial |
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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 | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | Yes |
E.6.13 | Others | No |
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 | Yes |
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.8.2.4 | Number of treatment arms in the trial | 2 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 41 |
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 | No |
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 |
Canada |
Chile |
China |
Finland |
France |
Germany |
Greece |
India |
Japan |
Korea, Republic of |
Mexico |
Netherlands |
Portugal |
Russian Federation |
Spain |
Turkey |
United States |
|
E.8.7 | Trial has a data monitoring committee | Yes |
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
|
End of trial = when the last patient will have completed the treatment period (52 weeks) and the follow-up period of 28 days. |
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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 | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
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 | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |