E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated | Idiopathic pulmonary fibrosis | |
E.1.1.1 | Medical condition in easily understood language | Lung damage with tissue scarring and thickening due to unknown causes | |
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 | |
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 assess the efficacy of the investigational products compared to placebo in participants with IPF | |
E.2.2 | Secondary objectives of the trial | -To assess the efficacy of the investigational products compared to placebo in participants with IPF -Time to progression -To assess the incidence of absolute decline in FVC over 10% predicted -To assess the impact of the investigational products on pulmonary physiology -To assess the impact of the investigational products on exercise capacity -To assess the patient reported impacts of cough of the investigational products compared to placebo in K-Bild Scores -To assess the patient reported impacts of cough of the investigational products compared to placebo in Leicester Cough Scores -To assess the patient reported impacts IPF on quality of life of the investigational products compared to placebo in R-Scale Scores -To assess the patient reported impacts of Living with IPF of the investigational products compared to placebo in L-IPF Scores -To assess the patient reported impacts of Living with IPF of the investigational products compared to placebo in L-IPF Scores and Symptoms | |
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria | -Male and female participants at least 40 years of age -IPF diagnosed based on ATS/ERS/JRS/ALAT IPF 2018 modified guidelines -FVC ≥45% predicted -DLCO, corrected for hemoglobin, ≥25% predicted (inclusive) -Unlikely to undergo lung transplantation during this trial in the opinion of the investigator -If a participant is taking nintedanib or pirfenidone, they must be on a stable regimen for at least 8 weeks prior to randomization Additional protocol-defined inclusion criteria may apply. | |
E.4 | Principal exclusion criteria | -Airway obstruction (i.e. prebronchodilator FEV1/ FVC < 0.7) or evidence of a bronchodilator response at screening -Emphysema >20% on screening HRCT -Fibrosis <10% on screening HRCT -Clinical diagnosis of any connective tissue disease -Clinically diagnosed acute exacerbation of IPF (AE-IPF) or other significant clinical worsening within 3 months of randomization Additional protocol-defined exclusion criteria may apply. | |
E.5 End points |
E.5.1 | Primary end point(s) | Change from baseline to end of treatment in Forced Vital Capacity (FVC) expressed in percent predicted | |
E.5.1.1 | Timepoint(s) of evaluation of this end point | Baseline, Weeks 4, 8, 12, 16, 20, 26 | |
E.5.2 | Secondary end point(s) | 1. Change from baseline to end of treatment epoch in Forced Vital Capacity (FVC) 2. Time to progression 3. Number of participants with absolute decline of ≥10% predicted in FVC 4. Change from baseline to the end of treatment epoch in DLCO 5. Change from baseline to the end of treatment epoch in 6-minute walk distance 6. Change from baseline to the end of treatment epoch in scores from the k-BILD questionnaire 7. Change from baseline to the end of treatment epoch in scores from Leicester Cough questionnaire 8. Change from baseline to the end of treatment epoch in scores from the the R-Scale for IPF questionnaire 9. Change from baseline to the end of treatment epoch in scores from the Living with IPF questionnaire (Impacts) 10. Change from baseline to the end of treatment epoch in scores from the Living with IPF questionnaire (Symptoms) | |
E.5.2.1 | Timepoint(s) of evaluation of this end point | 1. Baseline, Weeks 4, 8, 12, 16, 20, 26 2. Baseline, Weeks 4, 8, 12, 16, 20, 26 3. Baseline, Weeks 4, 8, 12, 16, 20, 26 4. Baseline, Weeks 12 and 26 5. Baseline, Weeks 12 and 26 6. Baseline, Weeks 12 and 26 7. Baseline, Weeks 12 and 26 8. Baseline, Weeks 12 and 26 9. Baseline, Weeks 12 and 26 10. Baseline, Weeks 12 and 26 | |
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 | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description | Tolerability Immunogenicity | |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 10 |
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 | Australia | United Kingdom | United States | Czechia | Germany | Netherlands | Poland | |
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 | |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | 14 |
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 | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |