E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
patients with idiopathic pulmonary fibrosis (IPF) |
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E.1.1.1 | Medical condition in easily understood language |
serious scarring disease of the lung |
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E.1.1.2 | Therapeutic area | Body processes [G] - Circulatory and Respiratory Physiological Phenomena [G09] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
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.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10067761 |
E.1.2 | Term | Exacerbation of 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 |
•Efficacy of TD139 by assessing the annual rate of decline in Forced Vital Capacity (FVC; expressed in mL over 52 weeks) |
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E.2.2 | Secondary objectives of the trial |
•Proportion of subjects with an absolute decline from baseline in FVC % pred of >10% at w52 •Time to first hospitalization (IPF related, including acute exacerbation of IPF) . •Time to death (all-causes, respiratory-related causes and/or caused by IPF) Additional secondary and exploratory efficacy variables with bearing on the efficacy of TD139 (e.g. QoL) are described in detail in the Main Criteria and Evaluation and Analyses ) |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Male and female subjects aged ≥ 40 years of age with a diagnosis of IPF established during the previous three years according to ATS/ERS/Fleischner criteria. A historical diagnostic HRCT scan assessed according to the ATS/ERS/Fleischner criteria must be available from within the 12 months prior to screening. Diagnostic HRCTs will be subject to central reading for confirmation. 2.Lung function parameters as follows: a.FVC > 45% of the predicted value at screening b.DLCO (corrected for Hb) of 30% to 79% of the predicted value at screening. 3.Any existing SoC treatment (e.g. pirfenidone or nintedanib in the SoC1 group but excluding prohibited medicines) must be deemed as stable in terms of dosing regimen, and tolerability by the PI/treating physician before randomization into the study. 4.Subjects must sign and date a written, informed consent form and any required authorization prior to initiation of any study procedures
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E.4 | Principal exclusion criteria |
Subjects meeting any of the following exclusion criteria are not to be enrolled in the study/randomized to treatment: 1.Currently has significant airways obstruction: FEV1/FVC ratio of < 0.7 at screening 2.Has clinical evidence of active infection, including, but not limited to, bronchitis, pneumonia, sinusitis, urinary tract infection, and cellulitis. 3.Has a history of malignancy within the last 2 years with the exception of basal cell carcinoma, chronic lymphocytic leukaemia (under observation) and prostate cancer requiring anti-androgens, localised treatment (minor surgery, radiotherapy) and/or managed by observation. 4.Has any condition other than IPF that, in the opinion of the investigator, is likely to result in the death of the subject within the next 2 years. 5.Presence of other disease that may interfere with testing procedures or in the judgement of the Investigator may interfere with trial participation or may put the patient at risk when participating in this trial 6.Is likely to receive lung transplantation within the next 12 months 7.Currently receiving high dose corticosteroid, cytotoxic (e.g., chlorambucil, azathioprine, cyclophosphamide, methotrexate), vasodilator therapy for pulmonary hypertension (e.g., bosentan), and or investigational therapy for IPF or administration of such therapeutics within 4 weeks of initial screening (or 5 half-lives, whichever is longer). Also see excluded concomitant medicines (Section 7.3). A current dose of less than or equal to 15 mg/day of prednisone or its equivalent is acceptable if the dose is anticipated to remain stable during the study. 8.Has a history of unstable or deteriorating cardiac or pulmonary disease (other than IPF) within the previous six months, including, but not limited to, the following: a.Unstable angina pectoris or myocardial infarction, or percutaneous coronary intervention within the last 6 months b.Congestive heart failure requiring hospitalization c.Uncontrolled clinically significant arrhythmias. 9.If female, the subject is pregnant or lactating or intending to become pregnant before participating in this study during the study and within (5 half- lives PLUS 30 days) after last dose of the study drug; or intending to donate ova during such time period. 10.Woman considered to be of childbearing potential who do not use highly effective birth control methods during the study.
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E.5 End points |
E.5.1 | Primary end point(s) |
•Efficacy of TD139 by assessing the annual rate of decline in Forced Vital Capacity (FVC; expressed in mL over 52 weeks)
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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) |
•Proportion of subjects with an absolute decline from baseline in FVC (% pred) of >10% at w52. •Time to first hospitalization (IPF related, including acute exacerbation of IPF). •Time to death (from all-causes, respiratory-related causes and/or caused by IPF) .
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
•Week 52 •Time to first hospitalization •Time to death |
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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 | No |
E.6.7 | Pharmacodynamic | No |
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 | 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) | 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 | 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 | 7 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 70 |
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 |
Belgium |
Canada |
France |
Germany |
Israel |
Italy |
Poland |
Spain |
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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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 | 1 |
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 | 1 |
E.8.9.2 | In all countries concerned by the trial days | 0 |