E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Idiopathic Pulmonary Fibrosis (IPF) |
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E.1.1.1 | Medical condition in easily understood language |
Fibrosis of the lungs of unidentified origin |
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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 | 21.1 |
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 |
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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 |
The primary objective is to demonstrate a reduction in lung function decline as measured by the change from baseline in FVC for BI 1015550 when compared to placebo in patients with IPF. |
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E.2.2 | Secondary objectives of the trial |
The main secondary objective of the trial is to demonstrate BI 1015550’s ability in reducing the occurrence of clinically meaningful events such as acute IPF exacerbation, hospitalization for respiratory cause or death over the duration of the trial when compared to placebo in patients with IPF. An additional secondary objective of the trial is to show an effect of BI 1015550 on symptoms and lung function. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients ≥40 years old at the time of signed consent IPF diagnosis based on 2022 ATS/ERS/JRS/ALAT Guidelines FVC ≥45% of predicted normal DLCO ≥25% predicted of normal corrected for hemoglobin On stable treatment with nintedanib or pirfenidone for at least 12 weeks or not on treatment with either nintedanib or pirfenidone for at least 8 weeks |
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E.4 | Principal exclusion criteria |
Prebronchodilator FEV1/FVC <0.7 Acute IPF exacerbation within 3 months and/or during the screening period Treated with immunomodulatory medications (other than oral corticosteroids) or prednisone >15 mg/day or equivalent for respiratory or pulmonary reasons Active, unstable or uncontrolled vasculitis within 8 weeks Any suicidal behaviour in the past 2 years Any suicidal ideation of type 4 or 5 on the C-SSRS in the past 3 months |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the absolute change from baseline in Force Vital Capacity (FVC) [mL] at Week 52. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1) key secondary endpoint: time to the first occurrence of any of the components of the composite endpoint: time to first acute IPF exacerbation, first hospitalization for respiratory cause, or death (whichever occurs first) over the duration of the trial 2) Time to first acute IPF exacerbation or death over the duration of the trial 3) Time to hospitalization for respiratory cause or death over the duration of the trial 4) Time to absolute decline in FVC % predicted of >10% from baseline or death over the duration of the trial 5) Time to absolute decline in (DLCO) % predicted of >15% from baseline or death over the duration of the trial 6) Time to death over the duration of the trial 7) Absolute change from baseline in Living with Pulmonary Fibrosis (LPF) Symptoms Dyspnea domain score at Week 52 8) Absolute change from baseline in Living with Pulmonary Fibrosis (LPF) Symptoms Cough domain score at Week 52 9) Absolute change from baseline in Living with Pulmonary Fibrosis (LPF) Symptoms Fatigue domain score at Week 52 10) Absolute change from baseline in FVC % predicted at Week 52 11) Absolute change from baseline in DLCO % predicted at Week 52 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1) 30 months 2) 30 months 3) 30 months 4) 30 months 5) 30 months 6) 30 months 7) 52 weeks 8) 52 weeks 9) 52 weeks 10) 52 weeks 11) 52 weeks |
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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 | Yes |
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 | No |
E.6.12 | Pharmacoeconomic | No |
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 | 3 |
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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 164 |
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 |
Argentina |
Chile |
Egypt |
Malaysia |
New Zealand |
Singapore |
Switzerland |
Ireland |
Taiwan |
Australia |
Austria |
Belgium |
Brazil |
Canada |
China |
Croatia |
Czechia |
Denmark |
Estonia |
Finland |
France |
Georgia |
Germany |
Greece |
Hungary |
India |
Israel |
Italy |
Japan |
Korea, Republic of |
Mexico |
Netherlands |
Norway |
Poland |
Portugal |
Saudi Arabia |
Serbia |
Slovenia |
South Africa |
Spain |
Sweden |
Thailand |
Turkey |
United Kingdom |
United States |
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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
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The end of the trial is defined as the date of the last visit of the last patient in the whole trial (“Last Patient Completed”). |
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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 | 2 |
E.8.9.1 | In the Member State concerned days | 4 |
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 | 2 |
E.8.9.2 | In all countries concerned by the trial days | 4 |