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    Summary
    EudraCT Number:2017-004134-29
    Sponsor's Protocol Code Number:VX17-659-105
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-03-21
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2017-004134-29
    A.3Full title of the trial
    A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
    Estudio abierto en fase III para evaluar la seguridad a largo plazo y la eficacia de la politerapia con VX-659 en pacientes con fibrosis quística, homocigotos o heterocigotos para la mutación F508del.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis
    Estudio para evaluar la eficacia y la seguridad a largo plazo de la politerapia con VX-659
    A.4.1Sponsor's protocol code numberVX17-659-105
    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
    B.5.3.4CountryUnited States
    B.5.5Fax number+1 510 595 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 nameVX-659/Tezacaftor/Ivacaftor
    D.3.2Product code VX-659/ 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 INNVX-659
    D.3.9.3Other descriptive nameVX-659
    D.3.9.4EV Substance CodeSUB184589
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number120
    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.3.9.4EV Substance CodeSUB188271
    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.3.11 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 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/3/008/556
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Cystic fibrosis
    Fibrosis quística
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10011762
    E.1.2Term Cystic fibrosis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation.
    Evaluar la tolerabilidad y la seguridad a largo plazo de VX-659 en combinación triple (CT) con tezacaftor (TEZ) e ivacaftor (IVA) en sujetos con fibrosis quística (FQ) que son homocigotos o heterocigotos para la mutación F508del.
    E.2.2Secondary objectives of the trial
    1) To evaluate the long-term efficacy of VX-659 in TC with TEZ and IVA
    2) To evaluate the pharmacodynamics (PD) of VX-659 in TC with TEZ and IVA
    1) Evaluar la eficacia a largo plazo de VX-659 en CT con TEZ e IVA.
    2) Evaluar la farmacodinámica (FD) de VX-659 en CT con TEZ e IVA.
    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. Did not withdraw consent from a parent study.

    4. Meets at least 1 of the following criteria:

    • Completed study drug treatment in a parent study.

    • Had study drug interruption(s) in a parent study, but completed study visits up to the last scheduled visit of the Treatment Period of a parent study.

    5. Willing to remain on a stable CF treatment regimen through completion of study participation.
    1. El paciente (o su representante legal autorizado) firmará y fechará un formulario de consentimiento informado (FCI) y, cuando proceda, un formulario de asentimiento.

    2. El paciente debe estar dispuesto a, y ser capaz de cumplir con, las visitas programadas, el plan de tratamiento, las restricciones del estudio, los análisis clínicos, las directrices sobre anticonceptivos y otros procedimientos del estudio.

    3. No retiró el consentimiento de un estudio original.

    4. Cumple como mínimo 1 de los 3 siguientes requisitos:
    • Completó el tratamiento con el fármaco de estudio en el estudio original.
    • Sufrió una o varias interrupciones en el estudio original, pero completó las visitas del estudio hasta la última visita programada del periodo de tratamiento del estudio original.

    5. El paciente debe estar dispuesto a permanecer en un régimen estable de tratamiento de FQ (según se define en la Sección 9.5) hasta la finalización de su participación en el estudio.
    E.4Principal exclusion criteria
    1. History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.

    2. Pregnant and nursing females. Females of childbearing potential must have a negative pregnancy test at the Day 1 Visit before receiving the first dose of study drug.

    3. History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator. (e.g., subjects with a history of allergy or hypersensitivity to the study drug.)

    4. Current participation in an investigational drug trial (other than a parent study). Participation in a noninterventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) and screening for another Vertex study is permitted.
    1. Antecedentes de una enfermedad asociada que, en opinión del investigador, podría confundir los resultados del estudio o suponer un riesgo adicional al administrar el fármaco del estudio al paciente.

    2. Mujeres embarazadas y en periodo de lactancia. Las mujeres con capacidad de procrear tienen que tener un resultado negativo en la prueba de embarazo en la visita del día 1 antes de recibir la primera dosis del fármaco del estudio.

    3. Antecedentes de intolerancia al fármaco en el estudio original que supondrían un riesgo adicional para el paciente en la opinión del investigador (por ej. pacientes con antecedentes de alergia o hipersensibilidad al fármaco del estudio).

    4. Participación actual en un estudio de fármaco en investigación (que no sea un estudio original). Se permite la participación en un estudio no intervencionista (incluidos los estudios de observación, estudios de archivos y estudios que requieran extracciones de sangre sin administración de los fármacos del estudio) y participar en procesos de selección para otro estudio Vertex.
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability of long-term treatment with VX-659 in TC with TEZ and IVA based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry.
    Seguridad y tolerabilidad del tratamiento a largo plazo con VX-659 en CT con TEZ e IVA basadas en los acontecimientos adversos (AA), valores de los análisis clínicos, ECG, constantes vitales y pulsioximetría.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Treatment period, ETT visit, Safety Follow-up after last dose.
    Periodo de tratamiento, visita ETT, seguridad de seguimiento después de la última dosis
    E.5.2Secondary end point(s)
    1. Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1)
    2. Absolute change in sweat chloride (SwCl)
    3. Number of pulmonary exacerbations (PEx)
    4. Time-to-first PEx
    5. Absolute change in body mass index (BMI)
    6. Absolute change in BMI z-score
    7. Absolute change in body weight
    8. Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score
    1. Cambio absoluto en el volumen espiratorio forzado porcentual previsto en 1 segundo (VEFpp1) con respecto al inicio.
    2. Cambio absoluto de cloruro en sudor (ClSu).
    3. Número de reagudizaciones pulmonares (ReP).
    4. Período hasta primera ReP.
    5. Variación absoluta en el índice de masa corporal (IMC).
    6. Variación absoluta de la puntuación z del IMC.
    7. Variación absoluta en el peso corporal.
    8. Variación absoluta en el cuestionario sobre la FQ revisado (CFQ-R) respecto al inicio.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Treatment period, ETT Visit, Safety Follow-Up After Last Dose.
    2. Day 1,15, Week 4,8,16,24, 96.
    3. Treatment period, ETT Visit, Safety Follow-Up After Last Dose.
    4. Treatment period, ETT Visit, Safety Follow-Up After Last Dose.
    5. Treatment period, ETT Visit, Safety Follow-Up After Last Dose.
    6. Treatment period, ETT Visit, Safety Follow-Up After Last Dose.
    7. Treatment period, ETT Visit, Safety Follow-Up After Last Dose.
    8. Day1, Weeks 4,8,24, 48, 72, 96, ETT Visit, Safety Follow-Up After Last Dose.
    1. Periodo de tratamiento, visita ETT, seguridad de seguimiento después de la última dosis
    2. Día 1, 15, Semana 4, 8, 16, 24, 96
    3. Periodo de tratamiento, visita ETT, seguridad de seguimiento después de la última dosis
    4. Periodo de tratamiento, visita ETT, seguridad de seguimiento después de la última dosis
    5. Periodo de tratamiento, visita ETT, seguridad de seguimiento después de la última dosis
    6. Periodo de tratamiento, visita ETT, seguridad de seguimiento después de la última dosis
    7. Periodo de tratamiento, visita ETT, seguridad de seguimiento después de la última dosis
    8. día 1, Semanas 4, 8, 24, 48, 72, 96, Vista ETT,seguridad de seguimiento después de la última dosis
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    Tolerabilidad
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA36
    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
    Denmark
    Germany
    Ireland
    Israel
    Norway
    Poland
    Spain
    Switzerland
    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
    The end of study is defined as the last scheduled visit or, for subjects who have been lost to follow-up, the last contact, whichever occurs later, for the latest completing subject in the study.
    El final del estudio se define como la última visita programada o, para los sujetos que se perdieron durante el seguimiento, el último contacto, lo que ocurra más tarde, para el último sujeto que complete el estudio.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    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: 153
    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: 153
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 307
    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 state36
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 190
    F.4.2.2In the whole clinical trial 460
    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.
    Ninguno
    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 Decision2018-05-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-05-04
    P. End of Trial
    P.End of Trial StatusOngoing
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