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    Summary
    EudraCT Number:2015-001644-11
    Sponsor's Protocol Code Number:VX15-809-110
    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:2016-03-31
    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 number2015-001644-11
    A.3Full title of the trial
    A Phase 3, Rollover Study to Evaluate the Safety and
    Efficacy of Long-term Treatment With Lumacaftor in
    Combination With Ivacaftor in Subjects Aged 6 Years
    and Older 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
    Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment with Lumacaftor in Combination with Ivacaftor.
    A.4.1Sponsor's protocol code numberVX15-809-110
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/005/2018
    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, Massachusetts
    B.5.3.3Post code02210
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1877 6348789
    B.5.5Fax number+15105958183
    B.5.6E-mailmedical_info@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 INNIvacaftor
    D.3.9.1CAS number 873054-44-5
    D.3.9.2Current sponsor codeVX-770
    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.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNlumacaftor
    D.3.9.1CAS number 936727-05-8
    D.3.9.2Current sponsor codeVX 809
    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.3.11 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.IMP: 2
    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 200mg/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 INNIvacaftor
    D.3.9.1CAS number 873054-44-5
    D.3.9.2Current sponsor codeVX-770
    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.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNLumacaftor
    D.3.9.1CAS number 936727-05-8
    D.3.9.2Current sponsor codeVX 809
    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.3.11 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
    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.1.2 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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the long term safety and tolerability of lumacaftor in combination with ivacaftor in subjects aged 6 years and older with cystic fibrosis (CF), homozygous for the F508del CFTR mutation, who are in the Treatment Cohort
    E.2.2Secondary objectives of the trial
    •To evaluate the long term efficacy and durability of lumacaftor in combination with ivacaftor for subjects in the Treatment Cohort
    •To evaluate the post treatment safety of lumacaftor in combination with ivacaftor for subjects in the Observational Cohort
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects who meet all of the following inclusion criteria will be eligible.
    1.Subject’s legally appointed and authorized representative (e.g., parent or legal guardian) will sign and date an ICF and the subject will sign and date an assent form (if applicable).
    2.Subjects entering the Treatment Cohort must meet both of the following criteria:
    •Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study drug treatment and the Week 26 Safety Follow up in Study 011B
    -Subjects who had study drug interruptions, but completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B are eligible (this is Day 1 of Study 110 for subjects at Study 110 active sites). Subjects who are not taking study drug at the end of the Treatment Period
    •Elect to enroll in the Treatment Cohort
    NOTE: Subjects who prematurely discontinued study drug treatment are not eligible for enrollment in the Treatment Cohort.
    Subjects entering the Observational Cohort must meet 1 of the following criteria:
    •Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study drug treatment and the Week 26 Safety Follow up in Study 011B
    •Subjects who received at least 4 weeks of study drug and completed visits up to Week 24 of Study 109 or Week 26 of Study 011B (this is Day 1 of Study 110 for subjects at Study 110 active sites) but are not taking study drug at the end of the Treatment Period.
    •Subjects who permanently discontinued study drug after receiving at least 4 weeks of study drug and remained in the study from the time of discontinuation of study drug treatment through the Week 24 Visit in Study 109 or the Week 26 Visit of Study 011B.
    3.Subjects who are willing to remain on a stable CF medication regimen through the Safety Follow up Visit (Treatment Cohort only).
    4.As deemed by the investigator, the subject’s legally appointed and authorized representative (e.g., parent or legal guardian) must be able to understand protocol requirements, restrictions, and instructions. The subject’s legally appointed and authorized representative should be able to ensure that the subject will comply with, and is likely to complete, the study as planned.
    E.4Principal exclusion criteria
    Subjects who meet any of the following exclusion criteria will not be eligible.
    1.History of any comorbidity or laboratory abnormality 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 (e.g., cirrhosis with portal hypertension).
    2.Pregnant and nursing females. Females of childbearing potential must have a negative urine pregnancy test at the Day 1 Visit before receiving the first dose of study drug.
    3.Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
    4.History of drug intolerance in the prior study that would pose an additional risk to the subject in the opinion of investigator, and which should be discussed with the Vertex medical monitor. Examples of subjects who may not be eligible for the treatment cohort include (but are not limited to) the following:
    •Subjects with a history of allergy or hypersensitivity to the study drug
    •Liver function test (LFT) abnormality during study drug treatment in the previous study (Study 109 or Study 011B) for which a clear cause was not identified:
    oAbnormal liver function defined as any 2 or more of the following:
    a.≥3 × upper limit of normal (ULN) aspartate aminotransferase (AST)
    b.≥3 × ULN alanine aminotransferase (ALT)
    c.≥3 × ULN gamma glutamyl transpeptidase
    d.≥3 × ULN alkaline phosphatase
    oALT or AST >5 × ULN
    oTotal bilirubin >2 × ULN
    oOther LFT abnormalities that would pose an additional risk to the subject in the opinion of investigator or Vertex
    •Other severe or life-threatening reactions to the study drug in the previous study
    5.History of poor compliance with study drug and/or procedures in the previous study as deemed by the investigator.
    6.Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor). NOTE: participation in a noninterventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) is permitted.
    E.5 End points
    E.5.1Primary end point(s)
    Treatment Cohort:
    Safety and tolerability assessments of long term treatment of lumacaftor in combination with ivacaftor based on adverse events (AEs), clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard 12 lead electrocardiograms (ECGs), vital signs, pulse oximetry, ophthalmological examinations, and spirometry
    Observational Cohort:
    Not applicable
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline through week 96 with a Safety Follow up Visit 4 weeks [± 7 days] after the last dose).
    E.5.2Secondary end point(s)
    Treatment Cohort:
    The following efficacy endpoints will be analyzed using baseline values in the previous study:
    •Absolute change from baseline in LCI2.5 (subjects from Study 109 and the Study 011B LCI Substudy only)
    •Absolute change from baseline in sweat chloride
    •Absolute change from baseline in body mass index (BMI)
    •Absolute change from baseline in Cystic Fibrosis Questionnaire Revised (CFQ R) respiratory domain score
    The following efficacy endpoints will be analyzed using baseline values in the current study:
    •Absolute change from baseline in LCI2.5 (subjects from Study 109 and the Study 011B LCI Substudy only)
    •Absolute change from baseline in sweat chloride
    •Absolute change from baseline in BMI
    •Absolute change from baseline in CFQ R respiratory domain score
    Observational Cohort:
    Safety, as determined by serious adverse events (SAEs)
    E.5.2.1Timepoint(s) of evaluation of this end point
    From baseline through week 96 with a Safety Follow up Visit 4 weeks [± 7 days] after the last dose).
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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.8.2.4Number of treatment arms in the trial1
    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 concerned4
    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
    Treatment Cohort
    LVLS (or until commercial availability for eligible subjects who are not participating in the Imaging Substudy) with a Safety Follow up Visit 4 weeks [± 7 days] after the last dose).
    Observational Cohort
    For the Observational Cohort, maximum subject participation will be approximately 2 years.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months24
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months24
    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: 256
    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: 251
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 66
    F.4.2.2In the whole clinical trial 256
    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)
    Patients could switch to commercially available treatment.
    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 ECFS Clinical Trials Network, University Hospital Leuven
    G.4.3.4Network Country Belgium
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation Cystic Fibrosis Foundation Therapeutics, Inc
    G.4.3.4Network Country United States
    G.4 Investigator Network to be involved in the Trial: 3
    G.4.1Name of Organisation Mukoviszidose Institut gGmbH (German Cystic Fibrosis Association)
    G.4.3.4Network Country Germany
    G.4 Investigator Network to be involved in the Trial: 4
    G.4.1Name of Organisation The Sydney Children's Hospitals Network (Randwick and Westmead)
    G.4.3.4Network Country Australia
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-05-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-05-25
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-04-23
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