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    Summary
    EudraCT Number:2018-001573-24
    Sponsor's Protocol Code Number:1.0-22/3/2018
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-05-08
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2018-001573-24
    A.3Full title of the trial
    Functional Respiratory Imaging (FRI) to assess the short-term effect of the product ORKAMBI (lumacaftor/ ivacaftor) on lung function in ORKAMBI-naive patients with Cystic Fibrosis Homozygous for Phe508del.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of the short-term effect of ORKAMBI on lung function in patients with cystic fibrosis who have two copies of the F508del mutation using functional respiratory imaging
    A.4.1Sponsor's protocol code number1.0-22/3/2018
    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 SponsorAntwerp University Hospital
    B.1.3.4CountryBelgium
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportVertex Pharmaceuticals Incorporated
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAntwerp University Hospital
    B.5.2Functional name of contact pointKinderpneumologie UZA
    B.5.3 Address:
    B.5.3.1Street AddressWilrijkstraat 10
    B.5.3.2Town/ cityEdegem
    B.5.3.3Post code2650
    B.5.3.4CountryBelgium
    B.5.6E-mailkinderpneumologie@uza.be
    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 Orkambi
    D.2.1.1.2Name of the Marketing Authorisation holderVertex Pharmaceuticals (Europe) Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Film-coated tablet
    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 INNLUMACAFTOR
    D.3.9.3Other descriptive nameLUMACAFTOR
    D.3.9.4EV Substance CodeSUB130518
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIVACAFTOR
    D.3.9.1CAS number 873054-44-5
    D.3.9.4EV Substance CodeSUB33103
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number125
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Cystic Fibrosis Homozygous (homozygous for the F508del mutation)
    E.1.1.1Medical condition in easily understood language
    Patients with cystic fibrosis who have two copies of the F508del mutation
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10011762
    E.1.2Term Cystic fibrosis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to assess the efficacy of ORKAMBI on lung functionality across multiple FRI parameters.
    E.2.2Secondary objectives of the trial
    Secondary outcome variables are Patient Reported Outcome, lung function tests, digital lung auscultation, exercise tolerance and exacerbation frequencies.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Documented diagnosis of CF (homozygous for the F508del mutation must be present, this should be documented in the medical history).
    2. Age ≥ 12 years
    3. FEV1 ³ 50%
    4. Signed informed consent. If patient is a minor, parents/guardians must give written informed consent
    5. Patient must be on a stable regimen of CF medication for 4 weeks prior to Visit 1
    E.4Principal exclusion criteria
    1. FEV1 < 50%
    2. Anticipated requirement for hospitalization within the next three weeks
    3. History of pneumothorax within the past 6 months prior to Visit 1
    4. History of haemoptysis requiring embolization within the past 12 months prior to Visit 1
    5. Unable or unwilling to complete study visits or provide follow-up data as required per the study protocol
    6. Has taken Intravenous (IV) antibiotics within the past 4 weeks prior to Visit 1
    7. Has ongoing exacerbation or Allergic bronchopulmonary aspergillosis (ABPA)
    8. Pregnant or lactating female
    9. Posttransplant patients
    10. Patients with severe hepatic impairment
    E.5 End points
    E.5.1Primary end point(s)
    FRI parameters:
    • Specific airway resistance (siRaw)
    • Specific Airway volumes (siVaw)
    E.5.1.1Timepoint(s) of evaluation of this end point
    HRCT scans will be taken at baseline (visit 1) and after 3 months of treatment (Visit 4).
    E.5.2Secondary end point(s)
    FRI parameters:
    • Lung and lobe volumes
    • Internal airflow distribution
    • Airway wall thickness
    • Blood vessel volume
    • Air trapping
    • Deposition of inhaled medications

    Spirometry measurements:
    • FEV1
    • FVC
    • FEV1/FVC

    Lung Clearance Index (LCI)

    Exercise Tolerance:
    • 6 Minute Walk Test

    Patient Reported Outcome (PRO):
    • Borg Category Ratio 10 Scale: measure of the present dyspnea and leg fatigue before and after exercise
    • Cystic Fibrosis Questionnaire- Revised (CFQ-R) respiratory domain score: measure Health-Related Quality of Life

    Exacerbation frequency:
    • Exacerbations requiring oral antibiotics
    • Exacerbations requiring intravenous antibiotics
    E.5.2.1Timepoint(s) of evaluation of this end point
    HRCT scans will be taken at baseline (visit 1) and after 3 months of treatment (visit 4).

    Spirometry will taken at baseline (visit 1), after 1 month of tratement (visit 2), after 2 months of treatment (visit 3) and after 3 months of teratment (visit 4).

    LCI will be done at baseline (visit 1) and after 3 months of treatment (visit 4).

    6 MWT and PRO will be done at baseline (visit 1) and after 3 months of treatment (visit 4).

    Exacerbation frequency will be checked at every visit (1,2,3, and 4).
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    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
    LPLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 10
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) No
    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 state20
    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)
    Normal treatment of CF patients.
    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 Decision2018-06-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-11
    P. End of Trial
    P.End of Trial StatusCompleted
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