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    Summary
    EudraCT Number:2018-000183-28
    Sponsor's Protocol Code Number:VX17-445-102
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-12-12
    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 number2018-000183-28
    A.3Full title of the trial
    A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
    Étude de phase 3, contrôlée, randomisée, en double aveugle, évaluant l’efficacité et l’innocuité du VX-445 en traitement combiné chez des sujets atteints de mucoviscidose, hétérozygotes pour la mutation F508del et porteurs d’une mutation à fonction minimale (F/MF)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
    Étude de phase 3 évaluant VX-445 en traitement combiné chez des sujets atteints de mucoviscidose, hétérozygotes pour la mutation F508del et porteurs d’une mutation à fonction minimale (F/MF)
    A.4.1Sponsor's protocol code numberVX17-445-102
    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
    B.5.3.3Post codeMA 02210-1862
    B.5.3.4CountryUnited States
    B.5.4Telephone number001877634 8789
    B.5.5Fax number001510595 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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product name100-mg VX-445/50-mg TEZ/75-mg IVA FDC
    D.3.2Product code VX-445/TEZ/IVA
    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 INNTezacaftor
    D.3.9.1CAS number 1152311-62-0
    D.3.9.2Current sponsor codeVX-661
    D.3.9.3Other descriptive nameVX-661
    D.3.9.4EV Substance CodeSUB33135
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVX-445
    D.3.9.1CAS number 2216712-66-0
    D.3.9.2Current sponsor codeVX-445
    D.3.9.3Other descriptive nameVX-445
    D.3.9.4EV Substance CodeSUB185183
    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 Yes
    D.2.1.1.1Trade name Kalydeco 150 mg film-coated tablets
    D.2.1.1.2Name of the Marketing Authorisation holder Vertex 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.3.1Product nameIvacaftor
    D.3.2Product code 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 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 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    E.1.1.1Medical condition in easily understood language
    Cystic Fibrosis
    Mucoviscidose
    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 efficacy of VX-445 in Triple Combination (TC) with TEZ and IVA in subjects with CF who are heterozygous for F508del and a minimal function mutation (F/MF subjects)
    Évaluer l’efficacité du VX-445 en traitement combiné triple (TCT) avec le tezacaftor (TEZ) et l’ivacaftor (IVA) chez des sujets atteints de mucoviscidose, hétérozygotes pour la mutation F508del et porteurs d’une mutation de fonction minimale (sujets F/MF)
    E.2.2Secondary objectives of the trial
    • To evaluate the safety of VX-445 in TC with TEZ and IVA
    • To evaluate the pharmacodynamics (PD) of VX-445 in TC with TEZ and IVA
    • To evaluate the pharmacokinetics (PK) of VX-445, TEZ, and IVA when administered in TC
    • Évaluer l’innocuité du VX-445 dans la mucoviscidose en association avec le TEZ et l’IVA
    • Évaluer la pharmacodynamie (PD) du VX-445 dans la mucoviscidose en association avec le TEZ et l’IVA
    • Évaluer la pharmacocinétique (PK) du VX-445, du TEZ et de l’IVA lorsqu’administrés en TCT
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form (ICF), and, when appropriate, an assent form.
    2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
    3. Age 12 years or older, on the date of informed consent.
    4. Confirmed diagnosis of CF as determined by the investigator.
    5. Heterozygous for F508del and an MF mutation (F/MF genotypes, see Appendix A for eligible MF mutations). If the screening CFTR genotype result is not received before randomization, a previous CFTR genotype laboratory report may be used to establish eligibility. Subjects who have been randomized and whose screening genotype does not confirm study eligibility must be discontinued from the study (Section 9.9).
    6. Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height (equations of the Global Lung Function Initiative [GLI]) at the Screening Visit. Spirometry measurements must meet American Thoracic Society/European Respiratory Society criteria for acceptability and repeatability.
    7. Stable CF disease as judged by the investigator.
    8. Willing to remain on a stable CF treatment regimen (as defined in Section 9.5) through completion of study participation.
    1. Le sujet (ou son représentant légalement désigné et autorisé) signera et datera un formulaire de consentement éclairé (ICF) et lorsque cela est approprié, un formulaire d’assentiment.
    2. Être prêt(e) et en mesure de se conformer aux visites planifiées, au schéma thérapeutique, aux restrictions de l’étude, aux analyses de laboratoire, aux méthodes contraceptives et à d’autres procédures de l’étude.
    3. Être âgé(e) de 12 ans ou plus à la date du consentement éclairé.
    4. Présenter un diagnostic confirmé de mucoviscidose (MV) selon la détermination de l’investigateur.
    5. Être hétérozygote pour la mutation F508del et une mutation MF (génotypes F/MF, voir l’Annexe A pour les mutations MF éligibles). Pour établir l’éligibilité, on pourra utiliser un rapport de laboratoire antérieur du génotype CFTR, si le résultat du génotype CFTR à la sélection n’est pas reçu avant la randomisation. Les sujets qui ont été randomisés et ceux dont le génotype de sélection ne confirme pas l’éligibilité à l’étude doivent être suspendus de l’étude (Section 9.9).
    6. Une valeur du volume expiratoire maximal par seconde (VEMS) comprise entre ≥ 40 % et ≤90 % de moyenne prédite pour l’âge, le sexe et la taille (équations de la Global Lung Function Initiative [GLI]) à la visite de sélection. Les mesures de spirométrie doivent répondre aux critères de l’ATS/ERS (American Thoracic Society/European Respiratory Society) en terme d’acceptabilité et de reproductibilité.
    7. Mucoviscidose stable selon l’investigateur.
    8. Être prêt(e) à respecter le schéma thérapeutique d’une mucoviscidose stable (défini dans la section 9.5) jusqu’à la fin de la participation à l’étude.
    E.4Principal exclusion criteria
    1. History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug(s) to the subject. This includes, but is not limited to, the following:
    • Clinically significant cirrhosis with or without portal hypertension
    • Solid organ or hematological transplantation.
    • Alcohol or drug abuse in the past year, including, but not limited to, cannabis, cocaine, and opiates, as deemed by the investigator.
    • Cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage 0 cervical carcinoma in situ (all 3 with no recurrence for the last 5 years)
    2. History of hemolysis.
    3. Glucose-6-phosphate dehydrogenase (G6PD) deficiency, defined as G6PD activity less than the lower limit of normal (LLN) or 70% of the mean of the LLN and the upper limit of normal (ULN), whichever is greater. A documented historical quantitative G6PD activity value measured in an established laboratory that runs the assay routinely and obtained during a prior Vertex study or as part of routine clinical care may be used to establish eligibility.
    4. Any of the following abnormal laboratory values at screening:
    • Hemoglobin <10 g/dL
    • Total bilirubin ≥2 × ULN
    • Aspartate transaminase (AST), alanine transaminase (ALT), gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) ≥3 × ULN
    • Abnormal renal function defined as glomerular filtration rate ≤50 mL/min/1.73 m2 (calculated by the Modification of Diet in Renal Disease Study Equation) for subjects ≥18 years of age and ≤45 mL/min/1.73 m2 (calculated by the Counahan-Barratt equation) for subjects aged 12 to 17 years (inclusive)
    5. An acute upper or lower respiratory infection, PEx, or changes in therapy (including antibiotics) for sinopulmonary disease within 28 days before the first dose of study drug (Day 1).
    6. Lung infection with organisms associated with a more rapid decline in pulmonary status (including, but not limited to, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus). For subjects who have had a history of a positive culture, the investigator will apply the following criteria to establish whether the subject is free of infection with such organisms:
    • The subject has not had a respiratory tract culture positive for these organisms within the 12 months before the date of informed consent.
    • The subject has had at least 2 respiratory tract cultures negative for such organisms within the 12 months before the date of informed consent, with the first and last of these separated by at least 3 months, and the most recent one within the 6 months before the date of informed consent.
    7. An acute illness not related to CF (e.g., gastroenteritis) within 14 days before the first dose of study drug (Day 1).
    8. Ongoing or prior participation in a study of an investigational treatment within 28 days or 5 terminal half-lives (whichever is longer) before screening. The duration of the elapsed time may be longer if required by local regulations.
    9. Use of prohibited medications as defined in Table 9-2, within the specified window before the first dose of study drug (Day 1).
    10. Pregnant or nursing females. Females of childbearing potential must have a negative pregnancy test at screening (serum test) and Day 1 (urine test).
    11. The subject or a close relative of the subject is the investigator or a subinvestigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site. However, an adult (aged 18 years or older) who is a relative of a study staff member may be randomized in the study provided that
    • the adult lives independently of and does not reside with the study staff member, and
    • the adult participates in the study at a site other than the site at which the family member is employed.
    1. Antécédents de pathologie ou d’affection clinique quelconque qui, d’après l’investigateur, pourrait fausser les résultats de l’étude ou représenter un risque supplémentaire dans le cadre de l’administration du ou des médicament(s) de l’étude au patient. Cela inclut, mais sans s’y limiter, les critères suivants :
    • Une cirrhose cliniquement significative avec ou sans hypertension portale
    • Transplantation d’organe solide ou hématologique.
    • Consommation abusive de drogues et d’alcool au cours de l’année écoulée, y compris mais sans s’y limiter, cannabis, cocaïne et opiacés, selon la détermination de l’investigateur.
    • Cancer, sauf carcinomes cutanés des cellules squameuses ou des cellules basales de la peau et carcinome in situ de stade 0 du col de l’utérus (dans le cas des trois cancers, sans récidive pendant les 5 dernières années)
    2. Antécédents d’hémolyse.
    3. Déficit en glucose-6-phosphate déshydrogénase (G6PD), défini par une activité G6PD inférieure à la limite inférieure de la normale (LIN) ou à 70 % de la moyenne de la LIN et de la limite supérieure de la normale (LSN), la limite supérieure prévalant. Pour établir l’éligibilité, on pourra utiliser une valeur d’activité quantitative historique de la G6PDdocumentée mesurée dans un laboratoire établi qui analyse périodiquement le dosage, et obtenue pendant une étude Vertex antérieure ou dans le cadre des soins cliniques périodiques.
    4. L’une des valeurs cliniques anormales suivantes à la sélection :
    • Hémoglobine <10 g/dl
    • Bilirubine totale ≥2 × LSN
    • Aspartate transaminase (ASAT), alanine transaminase (ALAT), gamma glutamyl transférase (GGT) ou phosphatase alcaline (ALP) ≥3 × LSN
    • Fonction rénale définie par une filtration glomérulaire de ≤50 ml/min/1,73 m2 (calculée par l’équation MDRD de la modification du régime alimentaire au sein du groupe de patients atteints d’insuffisance rénale) pour les sujets âgés de ≥18 ans, et de ≤45 ml/min/1,73 m2 (calculée par l’équation de Counahan-Barratt) pour les sujets âgés de 12 à 17 ans (inclus)
    5. Infection aiguë des voies respiratoires inférieures ou supérieures, ExP ou modifications de traitement (dont les antibiotiques) de la maladie pulmonaire dans les 28 jours précédant la première dose de médicament de l’étude (Jour 1).
    6. Infection pulmonaire par des organismes associés à un déclin accéléré de la fonction pulmonaire (y compris mais sans s’y limiter Burkholderia cenocepacia, Burkholderia dolosa et Mycobacterium abscessus). L’investigateur appliquera les critères suivants pour les sujets ayant des antécédents de culture positive, afin d’établir si le sujet est exempt d’infection par ces organismes :
    • Le sujet n’a pas présenté de culture positive des voies respiratoires à ces organismes dans les 12 mois précédant la date de consentement éclairé.
    • Le sujet a présenté au moins 2 cultures négatives des voies respiratoires à ces organismes dans les 12 mois précédant la date du consentement éclairé, la première et la dernière de ces trois cultures respectant un intervalle d’au moins 3 mois, et la plus récente intervenant dans les 6 mois précédant la date du consentement éclairé.
    7. Une pathologie aiguë non liée à la mucoviscidose (ex. gastro-entérite) dans les 14 jours précédant la première dose de médicament de l’étude (Jour 1).
    8. Une participation antérieure ou en cours à l’étude d’un traitement expérimental dans les 28 jours ou 5 demi-vies terminales (selon l’éventualité la plus longue) précédant la sélection. La durée du temps écoulé peut être plus longue si les réglementations en vigueur localement l’exigent.
    9. L’utilisation de médicaments prohibés tels que définis dans le tableau 9-2, dans la fenêtre temporelle spécifiée, avant la première dose de médicament de l’étude (Jour 1).
    10. Femmes enceintes ou allaitantes. Les femmes en âge de procréer doivent présenter un test de grossesse négatif à la sélection (test sérique) et au jour 1 (test urinaire).
    11. Le sujet ou un parent proche du sujet est investigateur ou sous-investigateur, assistant de recherche, pharmacien, coordonnateur de l’étude ou membre d’un personnel directement impliqué dans la conduite de l’étude au centre. Les sujets adultes (âgés de 18 ans ou plus) apparentés à un membre du personnel de l’étude peuvent toutefois être randomisés dans l’étude à condition que
    • l’adulte vive de façon autonome et ne réside pas avec le membre du personnel de l’étude, et que
    • l’adulte participe à l’étude dans un centre différent de celui où le membre de sa famille est employé.
    E.5 End points
    E.5.1Primary end point(s)
    Absolute change in ppFEV1 from baseline at Week 4
    Variation absolue en pourcentage du volume expiratoire maximal par seconde (VEMS) prédit entre la visite de référence et la Semaine 4
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 4
    Semaine 4
    E.5.2Secondary end point(s)
    Key Secondary Endpoints
     Absolute change in ppFEV1 from baseline through Week 24
     Number of pulmonary exacerbations (PEx) through Week 24
     Absolute change in SwCl from baseline through Week 24
     Absolute change in CFQ-R respiratory domain score from baseline through Week 24
     Absolute change in body mass index (BMI) from baseline at Week 24
     Absolute change in SwCl from baseline at Week 4
     Absolute change in CFQ-R respiratory domain score from baseline at Week 4
    Other Secondary Endpoints
     Time-to-first PEx through Week 24
     Absolute change in BMI z-score from baseline at Week 24
    Absolute change in body weight from baseline at Week 24
     Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry
     PK parameters of VX-445, TEZ, M1-TEZ, and IVA
    • Variation absolue du VEMS entre la visite de référence et la Semaine 24
    • Nombre d’exacerbations pulmonaires (ExP) jusqu’à la Semaine 24
    • Variation absolue du taux de chlore sudoral (Cl sudoral) entre la visite de référence et la Semaine 24
    • Variation absolue du score du domaine « respiratoire » du questionnaire CFQ-R (CF Questionnaire-Revised) entre la visite de référence et la Semaine 24
    • Variation absolue de l’indice de masse corporelle (IMC) entre la visite de référence et la Semaine 24
    • Variation absolue du Cl sudoral entre la visite de référence et la Semaine 4
    • Variation absolue du score du domaine « respiratoire » du questionnaire CFQ-R entre la visite de référence et la Semaine 4
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 4;
    Baseline through Week 24;
    At Week 24
    Semaine 4;
    Entre la visite de référence et la Semaine 24;
    A la semaine 24
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA53
    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
    Czech Republic
    France
    Germany
    Greece
    Italy
    Netherlands
    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
    LSLV
    Denière visite du dernier patient
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days11
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months7
    E.8.9.2In all countries concerned by the trial days11
    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: 70
    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: 70
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 290
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    adolescents 12-17 years
    F.4 Planned number of subjects to be included
    F.4.1In the member state21
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 134
    F.4.2.2In the whole clinical trial 360
    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)
    An open-label study will be available for subjects who complete the Week 24 Visit and are eligible. The Safety Follow-up Visit is not required for subjects who complete the Week 24 Visit and enroll in an open-label study within 28 days after the last dose of study drug.
    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 Decision2018-09-11
    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 StatusCompleted
    P.Date of the global end of the trial2019-04-24
    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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