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    Summary
    EudraCT Number:2012-003990-24
    Sponsor's Protocol Code Number:VX12-809-104
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-05-16
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2012-003990-24
    A.3Full title of the trial
    A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
    Estudio de fase 3, aleatorizado, doble ciego, controlado con placebo, de grupos paralelos para evaluar la eficacia y la seguridad de lumacaftor en combinación con ivacaftor en pacientes de 12 o más años de edad con fibrosis quística y homocigóticos para la mutación F508del-CFTR
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study in people with Cystic Fibrosis ( a rare hereditary pulmonary disease) to assess the efficacy and safety of a combination of two experimental drugs
    Estudio en personas con Fibrosis Quística (una rara enfermedad pulmonar hereditaria) para evaluar la eficacia y seguridad de la combinación de dos fármacos experimentales.
    A.4.1Sponsor's protocol code numberVX12-809-104
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/121/2012
    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 Address130 Waverly Street
    B.5.3.2Town/ cityCambridge, MA
    B.5.3.3Post code02139-4242
    B.5.3.4CountryUnited States
    B.5.4Telephone number+ 18776348789
    B.5.5Fax number+ 15105958183
    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 200mg/125mg tablets
    D.3.2Product code VX-809 / VX-770
    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.3.9.1CAS number 936727-05-8
    D.3.9.2Current sponsor codeVX-809
    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.3.9.2Current 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.3.11 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/83mg tablet
    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.3.9.1CAS number 936727-05-8
    D.3.9.2Current sponsor codeVX-809
    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.3.9.2Current 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 number83
    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: 3
    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/3/8/556
    D.3 Description of the IMP
    D.3.1Product nameivacaftor 125mg tablet
    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 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.3.11 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
    D.8 Placebo: 2
    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
    D.8 Placebo: 3
    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 in patients homozygous for the F508del-CFTR Mutation
    Fibrosis Quística en pacientes homocigóticos para la mutación del gen F508del-CFTR
    E.1.1.1Medical condition in easily understood language
    Cystic fibrosis
    Fibrosis Quística
    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 16.0
    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 efficacy of lumacaftor in combination with ivacaftor through Week 24 in subjects with Cystic Fibrosis who are homozygous for the F508del-CFTR mutation
    Evaluar la eficacia de lumacaftor en combinación con ivacaftor hasta la semana 24 en pacientes con Fibrosis Quística que son homocigóticos para la mutación F508del-CFTR
    E.2.2Secondary objectives of the trial
    To evaluate the safety of lumacaftor in combination with ivacaftor through Week 24

    To investigate the pharmacokinetics (PK) of lumacaftor and its metabolite, M28 (M28 lumacaftor) and ivacaftor and its metabolites M1 and M6 (M1 ivacaftor and M6 ivacaftor)
    Evaluar la seguridad de lumacaftor en combinación con ivacaftor hasta la semana 24.

    Para investigar la farmacocinética (PK) de lumacaftor y su metabolito, M28 (M28 lumacaftor) e ivacaftor y sus metabolitos M1 y M6 (M1 ivacaftor y M6 ivacaftor)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Males and females, aged 12 years or older on the date of informed consent or, where appropriate, date of assent
    Confirmed diagnosis of CF
    Homozygous for the F508del CFTR mutation
    FEV1 ?40% and ?90% of predicted normal for age, sex, and height
    Willing to remain on a stable CF medication regimen through Week 24 or, if applicable, the Safety Follow up Visit
    Varones y mujeres, que tengan 12 años o más de edad en el momento de firmar el consentimiento informado o, cuando proceda, el documento de asentimiento.
    Diagnóstico confirmado de FQ
    Homocigosis para la mutación F508del-CFTR
    FEV1 ?40% y ?90% del valor normal esperado para la edad, sexo y la talla del paciente.
    Disposición a recibir un tratamiento estable con medicación para FQ hasta la semana 24 o, en su caso, hasta la visita de seguimiento.
    E.4Principal exclusion criteria
    An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before first dose of study drug
    History of solid organ or hematological transplantation
    History of alcohol or drug abuse in the past year
    Ongoing or prior participation in an investigational drug study (including studies investigating lumacaftor and/or ivacaftor) within 30 days of screening
    Use of moderate to strong inhibitors or inducers of CYP3A, including consumption of certain herbal medications (e.g., St. John's Wort) and certain fruit and fruit juices within 14 days before Day 1 of dosing
    Exacerbación aguda de una infección de las vías respiratorias altas o bajas, o cambios en el tratamiento (incluidos antibióticos) administrado para la enfermedad pulmonar en las 4 semanas anteriores al día 1 (primera dosis de la medicación del estudio).
    Antecedente de transplante de órgano o hematológico
    Antecedentes de alcoholismo o drogadicción en el último año
    Participación actual o previa en un estudio con un fármaco en investigación (incluidos los estudios que han investigado lumacaftor o ivacaftor) en los 30 días previos a la selección.
    Uso de inhibidores o inductores moderados o potentes de la CYP3A, como el consumo de ciertos medicamentos de origen vegetal (por ejemplo, hipérico) y ciertas frutas y zumos de fruta desde 14 días antes del día 1
    E.5 End points
    E.5.1Primary end point(s)
    Relative change in percent predicted forced expiratory volume in 1 second (FEV1) from baseline through Week 24
    Variación relativa del porcentaje del valor esperado del volumen espiratorio forzado en 1 segundo (FEV1) entre el momento basal y la semana 24.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 24
    Semana 24
    E.5.2Secondary end point(s)
    Absolute change in body mass index (BMI) from baseline at Week 24

    Number of pulmonary exacerbations from baseline through Week 24

    Absolute change in Cystic Fibrosis Questionnaire ? Revised (CFQ-R) respiratory domain score from baseline through Week 24

    Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values (hematology, serum chemistry, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), pulse oximetry and vital signs
    Variación absoluta del índice de masa corporal (IMC) entre el momento basal y la semana 24.

    Número de exacerbaciones pulmonares hasta la semana 24

    Variación absoluta de la puntuación obtenida en el dominio respiratorio del cuestionario revisado de la fibrosis quística (CFQ-R) entre el momento basal y la semana 24.

    Evaluaciones de la seguridad y la tolerabilidad basadas en los acontecimientos adversos (AA), los valores analíticos (hematología, bioquímica sérica, pruebas de coagulación y análisis de orina), los electrocardiogramas (ECG) digitales convencionales, y las constantes vitales y la pulsioximetría.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Weeks 24 and 28
    Semanas 24 y 28
    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 Yes
    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 trial3
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA45
    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
    Austria
    Belgium
    Canada
    Denmark
    France
    Germany
    Spain
    United Kingdom
    United States
    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
    LVLS Last Visit Last Subject
    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 months10
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    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: 273
    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: 273
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 319
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children, age 12-17 years old, are included in the study.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state49
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 322
    F.4.2.2In the whole clinical trial 501
    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 the visits in the Treatment Period, regardless of whether they have prematurely discontinued study drug treatment, will be offered the opportunity to enroll in a Treatment Cohort or Observational Cohort in a rollover study of lumacaftor in combination with ivacaftor (VX12 809 105 [Study 105]).
    En la visita de la semana 24, los pacientes que completen las visitas del período de tratamiento, independientemente de que hayan suspendido prematuramente el tratamiento con el fármaco del estudio, tendrán la posibilidad de participar en launa cohorte de tratamiento deo en la cohorte de observación en un estudio de extensión de seguridad de lumacaftor en combinación con ivacaftor (VX12 809 105 [estudio 105]).
    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 Decision2013-08-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-06-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-04-25
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