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    The EU Clinical Trials Register currently displays   43845   clinical trials with a EudraCT protocol, of which   7282   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2016-002744-17
    Sponsor's Protocol Code Number:MA39189
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-04-05
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2016-002744-17
    A.3Full title of the trial
    MULTICENTER, INTERNATIONAL, DOUBLEBLIND, TWO-ARM, RANDOMIZED, PLACEBO CONTROLLED PHASE II TRIAL OF PIRFENIDONE IN PATIENTS WITH UNCLASSIFIABLE PROGRESSIVE FIBROSING ILD
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of Pirfenidone in Patients with Unclassifiable Progressive Fibrosing Interstitial Lung Disease
    A.4.1Sponsor's protocol code numberMA39189
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorF. Hoffmann-La Roche Ltd
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportF. Hoffmann-La Roche Ltd.
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationF. Hoffmann-La Roche Ltd.
    B.5.2Functional name of contact pointTrial Information Support Line-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4070
    B.5.3.4CountrySwitzerland
    B.5.6E-mailglobal.rochegenentechtrials@roche.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Esbriet
    D.2.1.1.2Name of the Marketing Authorisation holderRoche Registration GmbH
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/04/241
    D.3 Description of the IMP
    D.3.1Product nameEsbriet
    D.3.2Product code RO0220912/F01
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPIRFENIDONE
    D.3.9.1CAS number 53179-13-8
    D.3.9.2Current sponsor codeRO0220912/F01
    D.3.9.4EV Substance CodeSUB09907MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number267
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Fibrosing interstitial lung disease (ILD) of unknown origin
    E.1.1.1Medical condition in easily understood language
    Interstitial lung disease is a group of lung diseases affecting the interstitium (the tissue and space around the air sacs of the lungs).
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10022611
    E.1.2Term Interstitial lung disease
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    •To evaluate the effect of pirfenidone versus (vs.) placebo on lung function parameters
    E.2.2Secondary objectives of the trial
    •To evaluate the efficacy of pirfenidone vs. placebo from baseline (Day 1) until Week 24 on other functional parameters, outcomes, and patient-reported outcomes
    •To evaluate the safety of pirfenidone vs. placebo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Age >= 18-85 years
    - Confirmed fibrosing ILD which, following multidisciplinary team review, cannot be classified with either high or moderate confidence as a specific idiopathic interstitial pneumonia or other defined ILD
    - Progressive disease as considered by the investigator as patient deterioration within the last 6 months, which is defined as a rate of decline in forced vital capacity (FVC) >5% or a significant symptomatic worsening not due to cardiac, pulmonary vascular or other causes
    - Extent of fibrosis >10% on high-resolution computed tomography within the last 12 months
    -Forced vital capacity >= 45% of predicted value
    -Diffusing capacity of the lung for carbon monoxide (DLco) >= 30% of predicted value
    -Forced expiratory volume in 1 second/FVC ratio >= 0.7
    -Able to do 6-minute walk distance (6MWD) >= 150 meters
    -For women of childbearing potential: agreement to remain abstinent or use a contraceptive method with a failure rate of < 1% per year during the treatment period and for at least 58 days after the last dose of trial treatment
    -For men, agreement to remain abstinent or use contraceptive measures, and agreement to refrain from donating sperm
    E.4Principal exclusion criteria
    -Diagnosis with moderate or high confidence of nonspecific interstitial pneumonia and any ILD with an identifiable cause such as connective tissue disease-ILD, chronic hypersensitivity pneumonitis, or others
    -Diagnosis of idiopathic pulmonary fibrosis independent of the confidence level
    -History of unstable angina or myocardial infarction during the previous 6 months
    -Treatment with high dose systemic corticosteroids, or any immunosuppressant other than mycophenolate mofetil/acid (MMF), at any time at least 4 weeks prior to the screening period. Patients being treated with MMF should be on a stable dose that is expected to remain stable throughout the trial and was started at least 3 months prior to screening
    -Patients previously treated with pirfenidone or nintedanib
    -Patients treated with N-acetyl-cysteine for fibrotic lung disease, at any time within the 4 weeks of the screening period
    -Drug treatment for any type of pulmonary hypertension
    -Participation in a trial of an investigational medicinal product within the last 4 weeks
    -Significant co-existent emphysema (extent greater than extent of fibrosis on high-resolution computed tomography within the last 12 months)
    -Significant other organ co-morbidity including hepatic or renal impairment
    -Predicted life expectancy < 12 months or on an active transplant waiting list
    -Use of any tobacco product in the 12 weeks prior to the start of screening, or any unwillingness to abstain from their use through to the Follow-up Visit
    -Illicit drug or alcohol abuse within 12 months prior to screening
    -Planned major surgery during the trial
    -Hypersensitivity to the active substance or to any of the excipients of pirfenidone
    -History of angioedema
    -Concomitant use of fluvoxamine
    -Clinical evidence of any active infection
    -Any history of hepatic impairment, elevation of transaminase enzymes, or liver function test results as: Total bilirubin above the upper limit of normal (ULN), Aspartate aminotransferase or alanine aminotransferase >1.5 × ULN, and Alkaline phosphatase >2.0 × ULN
    -Creatinine clearance < 30 millilitre (mL) per minute, calculated using the Cockcroft-Gault formula
    -Any serious medical condition, clinically significant abnormality on an Electrocardiogram (ECG) at screening, or laboratory test results
    -An ECG with a heart rate corrected QT interval using Fridericia’s formula as >= 500 milliseconds at screening, or a family or personal history of long QT syndrome
    E.5 End points
    E.5.1Primary end point(s)
    Rate of decline in FVC measured in mL by daily handheld spirometer over the 24-week double-blind treatment period
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to Week 24
    E.5.2Secondary end point(s)
    1.Change in percent predicted FVC and in mL measured by spirometry during clinic visits
    2.Categorical change in FVC of > 5% , measured both by daily spirometry as well as by spirometry during clinic visits
    3.Categorical change in FVC of > 10%, measured both by daily spirometry as well as by spirometry during clinic visits
    4.Change in percent predicted DLco
    5.Change in 6MWD in meters
    6.Change in University of California, San Diego-Shortness of Breath Questionnaire score
    7.Change in score in Leicester Cough Questionnaire
    8.Change in cough visual analog scale
    9.Change in total and sub-scores of the St. George's Respiratory Questionnaire
    10.Number of participants with non-elective hospitalization, both respiratory and all cause
    11.Incidence of, and time to first, investigator-reported acute exacerbations
    12.Progression-free survival (PFS), defined as the time to the first occurrence of a >10% absolute decline in percent predicted FVC, a >50 m decline of 6MWD, or death
    13.PFS, alternatively defined as the time to the first occurrence of a >10% absolute decline in percent predicted FVC, non-elective respiratory hospitalization, or death
    14.Time to death from any cause
    15.Time to death from respiratory diseases
    16.Nature, frequency, severity, and timing of treatment-emergent adverse events
    17.Dose reductions and treatment interruptions
    18.Abnormalities in clinical laboratory test results
    19.Abnormalities in 12-lead ECG
    20.Number of participants withdrawn from trial treatment or trial discontinuations
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-5. Screening (Days -21 to -1) to Week 24
    6-9. Week 1 (Day 1) to Week 24
    10. Up to Week 91
    11-13. Week 1 (Day 1) to Week 24
    14-16. Up to Week 91
    17. Week 1 to Week 24
    18. Screening to Week 24
    19. Screening to Week 28
    20. Up to Week 91
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Assessments of the exploratory biomarkers and their relationship with drug responses
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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.8.4.1Number of sites anticipated in Member State concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA50
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Belgium
    Canada
    Czech Republic
    Denmark
    Germany
    Ireland
    Israel
    Italy
    Poland
    Portugal
    Spain
    United Kingdom
    E.8.7Trial 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
    The end of the clinical trial is defined as the date when the last patient, last visit (LPLV) occurs or the date at which the last data point which is required for the statistical analysis is received, whichever is the later date.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months9
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 100
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 150
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 148
    F.4.2.2In the whole clinical trial 250
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    After completion of the double-blind treatment period, the Sponsor will offer the possibility to the patients to receive continued access to pirfenidone in a safety follow-up period of up to 12 months, within this clinical trial protocol. Following the end of the 12-month safety follow-up period, the Sponsor will offer post-trial access to the trial treatment (pirfenidone) free of charge to eligible patients in accordance with the Roche Global Policy on Continued Access to IMP.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-05-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-04-25
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-01-10
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