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    Summary
    EudraCT Number:2015-000543-16
    Sponsor's Protocol Code Number:VX14-809-109
    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:2015-06-08
    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 number2015-000543-16
    A.3Full title of the trial
    A Phase 3, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study in children aged 6 Through 11 Years With Cystic Fibrosis to assess the efficacy and safety of a combination of two experimental drugs
    A.4.1Sponsor's protocol code numberVX14-809-109
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/337/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorVertex Pharmaceuticals Incorporated
    B.1.3.4CountryUnited States
    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 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 organisationVertex Pharmaceuticals Incorporated
    B.5.2Functional name of contact pointClinical Trials and Medical Info
    B.5.3 Address:
    B.5.3.1Street Address50 Northern Avenue
    B.5.3.2Town/ cityBoston
    B.5.3.3Post code02210
    B.5.3.4CountryUnited States
    B.5.4Telephone number18776348789
    B.5.6E-mailmedicalinfo@vrtx.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 No
    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/3/10/761
    D.3 Description of the IMP
    D.3.1Product namelumacaftor/ivacaftor 100mg/125mg tablets
    D.3.2Product code VX-809 / VX-770
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation Yes
    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 number100
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    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
    Cystic Fibrosis
    E.1.1.1Medical condition in easily understood language
    Cystic Fibrosis
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene
    E.2.2Secondary objectives of the trial
    - To evaluate the safety of lumacaftor in combination with ivacaftor
    - To investigate the pharmacokinetics (PK) of lumacaftor and its metabolite (M28-lumacaftor) and ivacaftor and its metabolites (M1-ivacaftor and M6-ivacaftor)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
    - Subjects (males and females), will be between the ages of 6 and 11 years, inclusive, on the date of informed consent (and assent, if applicable).
    - Subjects who weigh ≥15 kg without shoes at the Screening Visit.
    - Subjects with confirmed diagnosis of CF at the Screening Visit. CF is defined as:
    • 2 CF causing mutations (all as documented in the subject's medical record) AND
    • chronic sinopulmonary disease OR gastrointestinal/nutritional abnormalities
    - Subjects who are homozygous for the F508del CFTR mutation (genotype to be confirmed at the Screening Visit).
    - Subjects with percent predicted FEV1 of ≥70 through ≤105 percentage points adjusted for age, sex, and height using the Wang equation at the Screening Visit.
    - Subjects with a screening LCI 2.5 result greater than or equal to 7.5
    - Subjects with stable CF disease as deemed by the investigator at the Screening Visit.
    - Subjects who are willing to remain on a stable CF medication regimen through Week 24 or, if applicable, through the Safety Follow up Visit.
    - Subjects who are able to swallow tablets.
    E.4Principal exclusion criteria
    - History of any comorbidity reviewed at the Screening Visit that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
    - Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (as deemed by the investigator).
    - Any of the following abnormal laboratory values at the Screening Visit:
    • Hemoglobin <10 g/dL
    • Abnormal liver function defined as any 3 or more of the following:
    • ≥3 × upper limit of normal (ULN) aspartate aminotransferase (AST)
    • ≥3 × ULN alanine aminotransferase (ALT)
    • ≥3 × ULN gamma glutamyl transpeptidase
    • ≥3 × ULN alkaline phosphatase
    • ALT or AST >5 × ULN
    • Total bilirubin >2 × ULN
    • Abnormal renal function defined as glomerular filtration rate ≤45 mL/min/1.73 m2 (calculated by the Counahan-Barratt equation)
    - An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug).
    - A standard 12 lead ECG demonstrating QTc >450 msec at the Screening Visit. If QTc exceeds 450 msec at the Screening Visit, the ECG should be repeated 2 more times during the Screening Period, and the average of the 3 QTc values should be used to determine the subject's eligibility.
    - History of solid organ or hematological transplantation at the Screening Visit.
    - Ongoing or prior participation in an investigational drug study (including studies investigating lumacaftor and/or ivacaftor) within 30 days of the Screening Visit.
    - Use of restricted medication or food within specified duration before the first dose of study drug
    - History of cataract/lens opacity or evidence of cataract/lens opacity, as determined by the ophthalmologic examination at the Screening Visit. The Screening Visit ophthalmologic examination does not need to be repeated if there is documentation of an examination meeting protocol criteria that was conducted within 3 months before the Screening Visit. All subjects will have an eye exam performed by an ophthalmologist at the Screening Visit and at the Week 24 Visit OR the Week 28 Safety Follow up Visit
    E.5 End points
    E.5.1Primary end point(s)
    Absolute change in lung clearance index 2.5 (LCI2.5)
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline through Week 24
    E.5.2Secondary end point(s)
    - Average absolute change in sweat chloride from baseline at Day 15 and at Week 4
    - Absolute change in body mass index (BMI) from baseline at Week 24
    - Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ R) respiratory domain score from baseline through Week 24
    - Absolute change in LCI5.0 from baseline through Week 24
    - Absolute change in sweat chloride from baseline at Week 24
    - Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) from baseline through Week 24
    - Relative change in ppFEV1 from baseline through Week 24
    - Absolute change in BMI for age z score from baseline at Week 24
    - Absolute change in weight from baseline at Week 24
    - Absolute change in weight for age z score from baseline at Week 24
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline through Week 24 or Baseline at week 24
    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 Yes
    E.6.6Pharmacokinetic Yes
    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) Yes
    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 Yes
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Denmark
    France
    Germany
    Sweden
    United Kingdom
    United States
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    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 years0
    E.8.9.2In all countries concerned by the trial months11
    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 Yes
    F.1.1Number of subjects for this age range: 200
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 200
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 66
    F.4.2.2In the whole clinical trial 200
    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)
    At the Week 24 Visit, subjects who complete study drug treatment and the visits in the Treatment Period will be offered the opportunity to enroll in the Treatment Cohort of an optional open label rollover study evaluating lumacaftor in combination with ivacaftor (enrollment will be based on the eligibility criteria specified within the rollover study).
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Cystic Fibrosis Foundation
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation European Cystic Fibrosis Society
    G.4.3.4Network Country Belgium
    G.4 Investigator Network to be involved in the Trial: 3
    G.4.1Name of Organisation Mukovszidose gGMBH
    G.4.3.4Network Country Germany
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-07-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-09-24
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-09-20
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