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    Summary
    EudraCT Number:2012-003989-40
    Sponsor's Protocol Code Number:VX12-809-103
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-03-21
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2012-003989-40
    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
    Studio di fase 3, randomizzato, in doppio cieco, controllato con placebo, a gruppi paralleli, per la valutazione dell’efficacia e della sicurezza di lumacaftor in associazione con ivacaftor in soggetti di età pari e superiore a 12 anni, affetti da fibrosi cistica, omozigoti per la mutazione 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
    Uno studio condotto in pazienti con fibrosi cistica (una rara malattia ereditaria polmonare) per valutare l'efficacia e la sicurezza di una combinazione di due farmaci sperimentali
    A.4.1Sponsor's protocol code numberVX12-809-103
    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
    Fibrosi cistica in pazienti omozigoti per la mutazione F508del-CFTR
    E.1.1.1Medical condition in easily understood language
    Cystic fibrosis
    Fibrosi cistica
    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 14.1
    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
    Valutazione dell'efficacia di lumacaftor in associazione con ivacaftor alla settimana 24 in soggetti affetti da fibrosi cistica (FC), omozigoti per la mutazione 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)
    Valutazione della sicurezza di lumacaftor in associazione con ivacaftor alla settimana 24

    Valutazione della farmacocinetica di lumacaftor e del suo metabolita M28 (M28-lumafactor) e di ivacaftor e dei suoi metaboliti M1 e M6 (M1-ivacaftor e M6-ivacaftor)
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Optional part of the main study entitle: "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"


    A single optional blood sample (DNA Sample A) will be collected on Day 15 for potential exploratory evaluation of correlations between DNA markers with PKs, pharmacodynamics (PDs), treatment benefit, and AEs, for subjects who choose to participate in this assessment.
    A second optional blood sample (DNA Sample B) will be collected on Day 15 for potential exploratory evaluation of correlations between DNA markers with health and disease, especially CF, for subjects who choose to participate in this assessment.
    Optional blood samples for blood biomarker analysis will be collected on Day 1 and Week 24 for potential exploratory evaluation of correlations between blood biomarkers (e.g., proteins, peptides, lipids, vitamins, and endogenous metabolites) with PK, PD, treatment benefit, and AEs for subjects who choose to participate in this assessment.
    Optional sputum samples will be collected on Day 1 and Week 24 from subjects who are able to provide a sample and who choose to participate in this assessment, for potential exploratory evaluation of correlations between sputum biomarkers (e.g., qualitative and quantitative bacterial and viral assessments including genomic analyses, proteins, immune cells, inflammatory markers, peptides, lipids, endogenous metabolites, concentration of lumacaftor M28-lumacaftor and/or ivacaftor M1-ivacaftor and M6-ivacaftor with PK, PD, treatment benefit, and AEs. Optional sputum samples will not be collected from subjects randomized in Australia.
    Parte opzionale dello studio principale intitolato: "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"

    Nel Giorno 15 sarà prelevato un singolo campione di sangue facoltativo (campione di DNA A) dai soggetti che scelgono di partecipare alla valutazione esplorativa potenziale delle correlazioni tra i marcatori di DNA e la farmacocinetica, la farmacodinamica, il beneficio del trattamento e gli EA.
    Nel Giorno 15 sarà prelevato un secondo campione di sangue facoltativo (campione di DNA B) dai soggetti che scelgono di partecipare alla valutazione esplorativa potenziale delle correlazioni tra i marcatori di DNA e lo stato di salute e la malattia, in particolare la FC.
    Nel Giorno 1 e alla Settimana 24 saranno prelevati campioni di sangue facoltativi dai soggetti che scelgono di partecipare all'analisi dei biomarcatori ematici al fine di condurre una valutazione esplorativa potenziale delle correlazioni tra detti biomarcatori ematici (ad es. proteine, peptidi, lipidi, vitamine, e metaboliti endogeni) e la farmacocinetica, la farmacodinamica, il beneficio del trattamento e gli EA.
    Nel Giorno 1 e alla Settimana 24 saranno prelevati campioni facoltativi di espettorato dai soggetti in grado di fornire un campione e che scelgono di partecipare a questa valutazione esplorativa potenziale delle correlazioni tra i biomarcatori nell'espettorato (ad es. valutazioni virali e batteriche qualitative e quantitative, comprese analisi genomiche, proteine, cellule immunitarie, marcatori di infiammazione, peptidi, lipidi, metaboliti endogeni, concentrazione di lumacaftor , M28-lumacaftor e/o di ivacaftor , M1-ivacaftor e M6-ivacaftor ) e la farmacocinetica, la farmacodinamica, il beneficio di trattamento e gli EA. I campioni opzionali di espettorato non verranno raccolti dai soggetti randomizzati in Australia.
    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
    • Maschi e femmine, di età di 12 anni o maggiore alla data del consenso informato o, se applicabile, la data di assenso
    • Diagnosi confermata di fibrosi cistica
    • Mutazione omozigote per F508del CFTR
    • FEV1 ≥40% e ≤90% del previsto normale per età, sesso e altezza
    • Capacità di rimanere con un regime terapeutico stabile per la fibrosi cistica fino alla settimana 24 o, se applicabile, fino alla visita di Follow up di sicurezza
    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
    • Un'infezione acuta delle vie respiratorie superiori o inferiori, esacerbazione polmonare o modifiche della terapia (compresi gli antibiotici) per la malattia polmonare entro 4 settimane prima della prima dose del farmaco in studio
    • Storia di trapianto di organi solidi o di trapianti ematologici
    • Storia di abuso di alcool o di droga nell'ultimo anno
    • Partecipazione in corso o precedente in uno studio con farmaco sperimentale (tra cui studi che sperimentano lumacaftor e / o ivacaftor) entro i 30 giorni dello screening
    • Utilizzo inibitori o induttori di CYP3A di grado da moderato a potente, tra cui il consumo di alcuni farmaci a base di erbe (ad esempio, l'iperico) e alcuni tipi di frutta e succhi di frutta nei 14 giorni precedenti il giorno 1 del trattamento
    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
    Variazione relativa del valore percentuale del volume espiratorio forzato in 1 secondo (FEV1) predetto, osservata alla settimana 24 rispetto al basale
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 24
    Settimana 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
    Variazione assoluta dell'indice di massa corporea (IMC), osservata alla settimana 24 rispetto al basale

    Numero di esacerbazioni polmonari fino alla settimana 24

    Variazione assoluta del punteggio del dominio relativo alla funzionalità respiratoria nel questionario sulla fibrosi cistica rivisto (CFQ R), osservata alla settimana 24 rispetto al basale

    Valutazioni della sicurezza e della tollerabilità, basate su eventi avversi (EA), valori delle analisi cliniche di laboratorio (ematologia, chimica sierica, studi di coagulazione ed esame delle urine), elettrocardiogrammi (ECG) digitali standard, pulsossimetria e segni vitali
    E.5.2.1Timepoint(s) of evaluation of this end point
    Weeks 24 and 28
    Settimana 24 e 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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA48
    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
    Canada
    Czech Republic
    France
    Germany
    Ireland
    Italy
    Netherlands
    Sweden
    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
    LVLS Ultima visita dell'ultimo soggetto
    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 months3
    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: 264
    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: 264
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 349
    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 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]).
    Alla Visita settimana 24, ai soggetti che completano le visite nel periodo di trattamento, indipendentemente dal fatto che hanno prematuramente interrotto il trattamento farmacologico di studio, verrà offerta la possibilità di partecipare a un trattamento di coorte o ad una coorte osservazionale in studio di rollover su lumacaftor in combinazione con ivacaftor (VX12 809 105 [Studio 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 Decision2013-05-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-04-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-04-29
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