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 |
Scar tissue in the lung that interferes with the ability to breathe |
|
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 | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the efficacy of VAY736 in patients with idiopathic pulmonary fibrosis |
|
E.2.2 | Secondary objectives of the trial |
Impact of VAY736 on survival and disease progression.
Other protocol-defined secondary objectives may apply |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Written informed consent must be obtained before any assessment is performed.
2.Male and female subjects 40 to 80 years of age inclusive
3.A diagnosis of definite or probable IPF within 5 years of the screening visit, as defined by the ATS/ERS/JRS/ALAT Diagnostic Guidelines
(Raghu et al 2011)
4.Presence of hilar/mediastinal adenopathy (>1cm in short-axis diameter), identified by screening HRCT scan of the chest
5.FVC 40-90% predicted (inclusive)
6.DLCO, corrected for hemoglobin, 25-79% predicted (inclusive)
7.FEV1/FVC >70%
8.Unlikely to die from cause other than IPF within the next 3 years, in the opinion of the investigator
9.Unlikely to undergo lung transplantation during this trial
10.Able to communicate well with the investigator, to understand and comply with the requirements of the study.
Other protocol-defined inclusion/exclusion criteria may apply |
|
E.4 | Principal exclusion criteria |
1. Emphysema > fibrosis on screening HRCT (must be confirmed by central reader)
2. Active viral, bacterial or other infections requiring systemic treatment at the time of screening or enrollment, or history of recurrent clinically significant infection or of bacterial infections with encapsulated organisms
3. History of major organ, hematopoietic stem cell or bone marrow transplant
4. History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes (e.g., mAb of IgG1 class) or to any of the constituents of the study drug (sucrose, L-Arginine hydrochloride, Lhistidine, polysorbate 80, hydrochloric acid)
5. Receipt of live/attenuated vaccine within a 2 month period before
baseline
6. History of primary or secondary immunodeficiency, including a positive Human Immunodeficiency Virus (HIV) (Enzyme-linked Immunosorbent Assay (ELISA) and Western blot) test result
7. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin, in situ cervical cancer), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases
8. Any one of the following screening values of complete blood count laboratory values: Hemoglobin levels below 8.0 g/dL; Total leukocyte count less than 2,000/μL; Platelets <100.0 x 109/L; Absolute neutrophil count (ANC) <1.5 x 109/L
9. Any surgical, medical (e.g., uncontrolled hypertension, heart failure or diabetes), psychiatric or additional physical condition that the Investigator feels may jeopardize the patient in case of participation in this study
10. Positive hepatitis B surface antigen (HBsAg); or positive total hepatitis B core antibody (anti-HBc); or positive hepatitis C antibody (anti-HCV) unless it can be documented that the patient has received highly-effective HCV-specific antiviral therapy, HCV RNA levels are measured, and HCV RNA is undetectable; i.e., any acute latent or chronic infection with hepatitis B or hepatitis C
11. Evidence of active or latent tuberculosis (TB) infection, as determined by Quantiferon test (after anti-TB treatment, patients with history of or latent TB may become eligible according to national guidelines)
Other protocol-defined inclusion/exclusion criteria may apply |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Change from baseline to end of treatment epoch (48 weeks of treatment) in Forced Vital Capacity (FVC). |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
end of treatment epoch (48 weeks of treatment) |
|
E.5.2 | Secondary end point(s) |
All-Cause mortality; Progression-free survival (PFS); Disease progression; Composite Endpoint; Change from baseline to end of treatment epoch (Week 48) in Diffusing Capacity of the Lungs (DLCO); Change from baseline to the end of treatment epoch (Week 48) in 6-minute walk test and in distance-saturation product; Change from baseline to the end of treatment epoch (Week 48) in resting oxygen saturation (on room air); Immunogenicity of VAY736; To assess the pharmacokinetics Cmin,ss of VAY736 after multiple s.c. doses; Idiopathic Pulmonary Fibrosis (IPF) -related Mortality;
Other protocol defined endpoints may apply |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
across the 48 weeks of treatment and at the end of study |
|
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 | 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 | 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 | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 25 |
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 |
France |
Germany |
Ireland |
Italy |
United Kingdom |
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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 2 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 6 |