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    Summary
    EudraCT Number:2015-001267-39
    Sponsor's Protocol Code Number:VX15-770-123
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2015-12-04
    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 ConcernedFrance - ANSM
    A.2EudraCT number2015-001267-39
    A.3Full title of the trial
    A Phase 3b, 2-part, Randomized, Double-blind, Placebo-controlled Crossover Study With a Long term Open-label Period to Investigate Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
    Étude croisée de phase IIIb, en 2 parties, randomisée, en double aveugle, contrôlée contre placebo avec une période en ouvert à long terme visant à évaluer l’ivacaftor chez des sujets atteints de mucoviscidose âgés de 3 à 5 ans et présentant une mutation de défaut de régulation (classe III) du gène CFTR spécifique
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the efficacy and safety of ivacaftor in children aged 3 to 5 with Cystic Fibrosis (a rare hereditary disease that affects the lungs, digestive system and other organs).
    Étude visant à évaluer l’efficacité et la sécurité d’emploi de l’ivacaftor chez des sujets atteints de mucoviscidose âgés de 3 à 5 ans ( une maladie héréditaire rare qui affecte les poumons, le système digestif et d'autres organes).
    A.4.1Sponsor's protocol code numberVX15-770-123
    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 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, MA
    B.5.3.3Post code02210
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1877634 8789
    B.5.5Fax number+1510595 8183
    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/3/08/556
    D.3 Description of the IMP
    D.3.1Product nameIvacaftor
    D.3.2Product code VX-770
    D.3.4Pharmaceutical form Granules
    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 number50
    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/3/08/556
    D.3 Description of the IMP
    D.3.1Product nameIvacaftor
    D.3.2Product code VX-770
    D.3.4Pharmaceutical form Granules
    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 number75
    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/3/08/556
    D.3 Description of the IMP
    D.3.1Product nameIvacaftor
    D.3.2Product code VX-770
    D.3.4Pharmaceutical form Granules in sachet
    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 number50
    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: 4
    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/3/08/556
    D.3 Description of the IMP
    D.3.1Product nameIvacaftor
    D.3.2Product code VX-770
    D.3.4Pharmaceutical form Granules in sachet
    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 number75
    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: 5
    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 Kalydeco
    D.2.1.1.2Name of the Marketing Authorisation holderVertex Pharmaceuticals (Europe) Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/08/556
    D.3 Description of the IMP
    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 number150
    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 placeboGranules
    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 placeboGranules
    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
    Mucoviscidose
    E.1.1.1Medical condition in easily understood language
    Cystic Fibrosis
    Mucoviscidose
    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 18.1
    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 ivacaftor treatment in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation, and who are 3 through 5 years of age at the start of the study, as measured by lung clearance index (LCI)
    Évaluer l’efficacité du traitement par ivacaftor, mesurée par l’indice de clairance pulmonaire (ICP) chez des sujets atteints de mucoviscidose présentant une mutation de défaut de régulation (classe III) du gène CFTR (de l’anglais CF transmembrane conductance regulator) spécifique et qui sont âgés de 3 à 5 ans au début de l’étude.
    E.2.2Secondary objectives of the trial
    • To evaluate disease progression as measured by changes in computed tomography (CT) scan
    • To evaluate disease progression as measured by changes in pancreatic function
    • To evaluate the safety of ivacaftor treatment, as measured by clinical laboratory measures (including liver function tests [LFTs]), ophthalmologic examinations, and adverse events (AEs)
    • Progression de la maladie, mesurée par les variations des résultats de la tomodensitométrie (TDM)
    •Progression de la maladie, mesurée par les variations des résultats de la fonction pancréatique
    • Sécurité d’emploi du traitement par ivacaftor
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female with confirmed diagnosis of CF, defined as:
    - a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis
    OR
    2 CF causing mutations (all as documented in the subject's medical record)
    AND
    - Clinical manifestations of CF
    2. Must have 1 of the following CFTR gating mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.
    3. Subjects will be between the ages of 3 and 5 years, inclusive, at Screening and Day 1
    4. Weight ≥8 kg to <25 kg at Screening and Day 1
    5. Hematology, serum chemistry, and coagulation at Screening with no clinically significant abnormalities or concomitant diagnosis that would interfere with the LCI and CT scan study assessments, as judged by the investigator
    1. Patients de sexe masculin ou féminin présentant un diagnostic confirmé de mucoviscidose défini comme suit:
    - concentration de chlorure dans la sueur ≥ 60 mmol/l mesurée au moyen d’un test quantitatif par iontophorèse à la pilocarpine ;
    OU
    2 mutations causant la mucoviscidose (les deux doivent être documentées dans le dossier médical du sujet) ;
    ET
    - manifestations cliniques de la mucoviscidose.
    2. Le sujet doit présenter 1 des mutations de défaut de régulation (classe III) du gène CFTR suivantes sur au moins 1 allèle : G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P ou G1349D.
    3. Les sujets seront âgés de 3 à 5 ans inclus, au moment de la sélection et au jour 1.
    4. Poids ≥ 8 kg à <25 kg au moment de la sélection et au jour 1.
    5. Hématologie, chimie sérique et coagulation au moment de la sélection sans anomalies cliniquement significatives ou diagnostics concomitants susceptibles de gêner les évaluations de l’étude concernant l’ICP et les TDM, selon l’avis de l’investigateur.
    E.4Principal exclusion criteria
    1. History of any illness or condition that, in the opinion of the investigator, might confound the results of the study, impact use of the LCI or CT scan as assessments or pose an additional risk in administering study drug to the subject
    2. An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
    3. Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (in the opinion of the investigator)
    4. Abnormal liver function, at Screening, defined as 3 × upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase (ALP), and total bilirubin
    5. History of solid organ or hematological transplantation
    6. Any clinically significant "non-CF-related" illness within 2 weeks before Day 1. "Illness" is defined as an acute (serious or nonserious) condition (e.g., gastroenteritis)
    7. Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1
    8. Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives (whichever is longer or as determined by the local requirements) before Screening
    1. Antécédents de toute pathologie ou affection qui, selon l’avis de l’investigateur, pourraient fausser les résultats de l’étude, avoir une incidence sur les évaluations liées à l'ICP ou aux TDM, ou présenter un risque supplémentaire lors de l’administration du médicament à l’étude au sujet.
    2. Infection aiguë des voies respiratoires supérieures ou inférieures, exacerbation pulmonaire ou modifications du traitement (dont antibiotiques) pour la pathologie pulmonaire dans les 4 semaines précédant le jour 1.
    3. Toute anomalie biologique cliniquement significative lors de la visite de sélection qui pourrait gêner les évaluations de l’étude ou présenter un risque inutile pour le sujet (selon l’avis de l’investigateur).
    4. Fonction hépatique anormale lors de la sélection, définie comme des valeurs 3 × la limite supérieure de la normale (LSN), pour 3 ou plus des paramètres suivants : aspartate aminotransférase (ASAT) sérique, alanine aminotransférase (ALAT) sérique, gamma-glutamyl transpeptidase (GGT), phosphatase alcaline (PAL) sérique et bilirubine totale.
    5. Antécédents de greffe d’un organe solide ou de greffe hématologique.
    6. Toute pathologie cliniquement significative « non associée à la mucoviscidose » au cours des 2 semaines précédant le jour 1. Le terme « pathologie » signifie une affection aiguë (grave ou non grave) (par exemple, gastro-entérite).
    7. Utilisation de tout inducteur ou inhibiteur modéré ou puissant du cytochrome P450 (CYP) 3A au cours des 2 semaines précédant le jour 1.
    8. Participation à une étude clinique impliquant l’administration d’un médicament expérimental ou commercialisé au cours des 30 jours ou 5 demi-vies terminales (l’éventualité la plus longue étant retenue ou selon la réglementation locale) précédant la sélection.
    E.5 End points
    E.5.1Primary end point(s)
    Part 1
    Absolute change from baseline in LCI2.5 through 8 weeks of treatment
    Partie 1:
    Variation absolue de l’ICP2,5 après 8 semaines de traitement par rapport à la référence
    E.5.1.1Timepoint(s) of evaluation of this end point
    8 weeks
    8 semaines
    E.5.2Secondary end point(s)
    Part 1
    • Absolute change from baseline in serum levels of immunoreactive trypsinogen at 8 weeks of treatment
    • Absolute change from baseline in fecal elastase-1 at 8 weeks of treatment
    • Absolute change from baseline in weight at 8 weeks of treatment
    • Absolute change from baseline in body mass index (BMI) at 8 weeks of treatment
    • Safety, as determined by AEs, clinical laboratory values (including LFTs), and other relevant assessments

    Part 2:
    • Absolute change from baseline in LCI2.5 at Weeks 48, 96, and 144
    • Absolute change from baseline in CT scan at Week 96
    • Absolute change from baseline in serum levels of immunoreactive trypsinogen at Weeks 48, 96, and 144
    • Absolute change from baseline in fecal elastase-1 at Weeks 48, 96, and 144
    • Absolute change from baseline in weight-for-age z-score at Weeks 48, 96, and 144
    • Absolute change from baseline in height-for-age z-score at Weeks 48, 96, and 144
    • Absolute change from baseline in BMI-for-age z-score at Weeks 48, 96, and 144
    • Safety, as determined by AEs, clinical laboratory values (including LFTs), and ophthalmologic examinations
    Partie 1:
    •Variation absolue des taux sériques de trypsinogène immunoréactif après 8 semaines de traitement par rapport à la référence
    •Variation absolue du taux d’élastase-1 fécale après 8 semaines de traitement par rapport à la référence
    •Variation absolue du poids après 8 semaines de traitement par rapport à la référence
    •Variation absolue de l’indice de masse corporelle (IMC) après 8 semaines de traitement par rapport à la référence
    •Sécurité d’emploi, déterminée par les EI, les valeurs biologiques cliniques (y compris les TFH), les examens ophtalmologiques, les examens cliniques et les signes vitaux

    Partie 2:
    • Variation absolue de l’ICP2,5 entre la référence et les semaines 48, 96 et 144
    • Variation absolue du résultat de TDM entre la référence et la semaine 96
    • Variation absolue des taux sériques de trypsinogène immunoréactif entre la référence et les semaines 48, 96 et 144
    •Variation absolue du taux d’élastase-1 fécale entre la référence et les semaines 48, 96 et 144
    •Variation absolue du z-score du poids pour l’âge entre la référence et les semaines 48, 96 et 144
    • Variation absolue du z-score de la taille pour l’âge entre la référence et les semaines 48, 96 et 144
    • Variation absolue du z-score de l’IMC pour l’âge entre la référence et les semaines 48, 96 et 144
    • Sécurité d’emploi, déterminée par les EI, les valeurs biologiques cliniques (y compris les TFH), les examens ophtalmologiques, les examens cliniques et les signes vitaux
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part 1: 8 weeks
    Part 2:
    •Absolute change from baseline in LCI2.5 at Weeks 48, 96, and 144
    • Absolute change from baseline in CT scan at Week 96
    • Absolute change from baseline in serum levels of immunoreactive trypsinogen at Weeks 48, 96, and 144
    • Absolute change from baseline in fecal elastase-1 at Weeks 48, 96, and 144
    • Absolute change from baseline in weight-for-age z-score at Weeks 48, 96, and 144
    • Absolute change from baseline in height-for-age z-score at Weeks 48, 96, and 144
    • Absolute change from baseline in BMI-for-age z-score at Weeks 48, 96, and 144
    • Safety: throughout the study
    Partie 1: 8 semaines
    Partie 2:
    • Variation absolue de l’ICP2,5 entre la référence et les semaines 48, 96 et 144
    • Variation absolue du résultat de TDM entre la référence et la semaine 96
    • Variation absolue des taux sériques de trypsinogène immunoréactif entre la référence et les semaines 48, 96 et 144
    •Variation absolue du taux d’élastase-1 fécale entre la référence et les semaines 48, 96 et 144
    •Variation absolue du z-score du poids pour l’âge entre la référence et les semaines 48, 96 et 144
    • Variation absolue du z-score de la taille pour l’âge entre la référence et les semaines 48, 96 et 144
    • Variation absolue du z-score de l’IMC pour l’âge entre la référence et les semaines 48, 96 et 144
    • Sécurité d’emploi: tout au long de l'étude
    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 No
    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 No
    E.8.1.6Cross over Yes
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    La partie 2 de l'étude est une période en ouvert à long terme
    Part 2 of the study is a long-term open-label period
    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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    France
    United Kingdom
    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
    Dernière visite dernier sujet
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months5
    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: 50
    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: 50
    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 No
    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 aged 3 to 5 years
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 26
    F.4.2.2In the whole clinical trial 50
    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)
    None
    Aucun
    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 Decision2015-11-05
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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