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    Summary
    EudraCT Number:2016-004996-33
    Sponsor's Protocol Code Number:VX15-809-114
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2017-03-10
    Trial 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 number2016-004996-33
    A.3Full title of the trial
    A Phase 4, Open-label Treatment, Randomized, Multicenter, 2-arm, Parallelgroup, Pilot Study of Adherence to Lumacaftor/Ivacaftor in CF Subjects Homozygous for the F508del-CFTR Mutation
    Estudio preliminar en fase IV, multicéntrico, aleatorizado, de tratamiento abierto y en dos grupos paralelos, del cumplimiento terapéutico del tratamiento con lumacaftor/ivacaftor en pacientes con fibrosis quística homocigóticos para la mutación F508del-CFTR
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del-CFTR Mutation
    Estudio preliminar para evaluar el uso de la tecnología inteligente de cumplimiento terapéutico para medir el cumplimiento de lumacaftor/ivacaftor en sujetos con FQ homocigotos para la mutación F508del-CFTR
    A.4.1Sponsor's protocol code numberVX15-809-114
    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 Incoporated
    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 Pharmaceutical 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.5.5Fax 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 Yes
    D.2.1.1.1Trade name Orkambi
    D.2.1.1.2Name of the Marketing Authorisation holderVERTEX PHARMACEUTICALS INCORPORATED
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameOrkambi
    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.3.9.2Current sponsor codeVX-809; VRT-826809; VRT-0826809
    D.3.9.3Other descriptive nameLUMACAFTOR
    D.3.9.4EV Substance CodeSUB130518
    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, VRT-813077
    D.3.9.3Other descriptive nameIVACAFTOR
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Cystic fibrosis subjects Homozygous for the F508del-CFTR Mutation
    Pacientes con fibrosis quística homocigóticos para la mutación F508del-CFTR
    E.1.1.1Medical condition in easily understood language
    Cystic fibrosis subjects Homozygous for the F508del-CFTR Mutation
    Pacientes con fibrosis quística homocigóticos para la mutación F508del-CFTR
    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 19.1
    E.1.2Level SOC
    E.1.2Classification code 10010331
    E.1.2Term Congenital, familial and genetic disorders
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the impact of smart adherence technology for monitoring LUM/IVA adherence rates among subjects 16 years of age and older with CF who are homozygous for the F508del-CFTR mutation.
    Evaluar la influencia de la tecnología inteligente de cumplimiento terapéutico para supervisar las tasas de cumplimiento terapéutico de LUM/IVA en sujetos de 16 años y mayores con FQ homocigotos para la mutación F508del-CFTR
    E.2.2Secondary objectives of the trial
    To collect subject and physician feedback on the use of smart adherence
    technology to monitor LUM/IVA adherence
    Reunir las opiniones de los sujetos y los médicos sobre el uso de la tecnología inteligente de cumplimiento terapéutico para supervisar el cumplimiento de LUM/IVA.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject and/or legally appointed and authorized representative (e.g., parents or legal guardian) will sign and date an informed consent form (ICF) and where appropriate, an assent form.
    2. Subject and/or legally appointed and authorized representative (e.g., parent or legal guardian) is willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
    3. Confirmed diagnosis of CF and homozygous for the F508del-CFTR mutation at Screening.
    4. Subjects (male and female) will be aged 16 years and older on the date of informed consent or, where appropriate, assent.
    5. FEV1 ≥40% of predicted normal for age, sex, and height at Screening.
    1. El sujeto y/o su representante legal nombrado y autorizado (p. ej., los padres o el tutor legal) firmarán y fecharán un formulario de consentimiento informado (FCI) y, cuando proceda, un formulario de asentimiento.
    2. El sujeto y/o su representante legal nombrado y autorizado (p. ej., los padres o el tutor legal) son capaces de cumplir con las visitas programadas, el plan de tratamiento, las restricciones del estudio, las pruebas analíticas, las normas sobre anticonceptivos y los demás procedimientos del estudio, y están dispuestos a hacerlo.
    3. Tiene un diagnóstico confirmado de FQ y es homocigótico para la mutación F508del-CFTR en la selección.
    4. Los sujetos (de ambos sexos) tendrán 16 años de edad o más en la fecha de la firma del consentimiento o asentimiento informado, según proceda.
    5. VEF1 ≥ 40 % del valor normal esperado para la edad, el sexo y la estatura en la selección.
    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 LUM/IVA to the subject, e.g., history of advanced liver disease.
    2. Presence of moderate or severe hepatic impairment (Child-Pugh Class B or C).
    3. Subjects currently receiving invasive mechanical ventilation.
    4. Known history of alcohol or drug abuse in the past year, including but not limited to cannabis, cocaine, and opiates, as deemed by the investigator.
    5. Any of the following abnormal laboratory values during screening:
    o Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >5 × ULN o ALT or AST >3 × ULN with total bilirubin >2 × ULN o Glomerular filtration rate ≤30 mL/min/1.73 m2. This will be calculated by the Modification of Diet in Renal Disease study equation for subjects ≥18 years of age and calculated by the Counahan-Barratt equation for subjects aged 12 to 17 years.
    6. Pregnant or nursing females (females of childbearing potential must have a negative pregnancy test at screening and Day 1).
    7. Female subjects and female partners of male subjects who plan to become pregnant during Treatment Period or within 90 days following the last dose of LUM/IVA.
    8. Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements outlined in Section 11.5.7.1.
    9. History of solid organ or hematological transplantation.
    10. Ongoing or prior participation in an investigational drug study (including studies
    investigating LUM and/or IVA) within 30 days of screening
    - A washout period of 5 terminal half-lives of the previous investigational LUM/IVA or 30 days, whichever is longer
    - The duration of the elapsed time may be longer if required by local regulations.
    11. Current use of commercial LUM/IVA combination therapy.
    12. Subject, or 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.
    1. Antecedentes de cualquier enfermedad o cualquier dolencia médica que, en opinión del investigador, pudiera confundir los resultados del estudio o suponer un riesgo añadido para la administración de L(s) al sujeto, p. ej., antecedentes de hepatopatía avanzada.
    2. Presencia de insuficiencia hepática moderada o grave (clase B o C de Child-Pugh).
    3. Sujetos que actualmente reciben ventilación mecánica invasiva.
    4. Antecedentes conocidos de alcoholismo o toxicomanía en el último año, incluidos, entre otros, cannabis, cocaína y opiáceos, en opinión del investigador.
    5. Alguna de las siguientes anomalías analíticas durante la selección:
    - Valores de alanina aminotransferasa (ALT) o aspartato aminotransferasa (AST) > 5 veces el LSN de ALT o > 3 veces el LSN de AST con bilirrubina total > 2 veces el LSN.
    - Filtración glomerular ≤ 30 ml/min/1,73 m2. Se calculará según la ecuación del estudio Modificación de la alimentación en la nefropatía (Modification of Diet in Renal Disease) para sujetos ≥ 18 años de edad y según la ecuación de Counahan-Barratt para sujetos de 12 a 17 años.
    6. Mujeres embarazadas o lactando (las mujeres con capacidad de procrear deben obtener un resultado negativo en la prueba de embarazo en la selección y en el día 1).
    7. Sujetos mujeres y parejas femeninas de los sujetos varones que tengan previsto quedarse embarazadas durante el período de tratamiento o en los 90 días siguientes a la última dosis de LUM/IVA.
    8. Sujetos con capacidad de procrear sexualmente activos que no estén dispuestos a cumplir los requisitos sobre anticoncepción que se indican en la sección 11.5.7.1.
    9. Antecedentes de trasplante de un órgano sólido o hematológico.
    10. Participación actual o previa en un estudio con un fármaco en investigación (incluidos los estudios con LUM y/o IVA en investigación) en los 30 días anteriores a la selección
    - Un período de reposo farmacológico de 5 semividas terminales de la combinación LUM/IVA en investigación previo o 30 días, el período que sea más largo.
    - La duración del tiempo transcurrido puede ser mayor si lo exige la normativa local.
    11. Uso actual del tratamiento combinado con LUM/IVA comercializado.
    12. El sujeto o un familiar cercano suyo es el investigador o un investigador asociado, un ayudante de la investigación, un farmacéutico, un coordinador del estudio u otro miembro del personal que participa directamente en la realización del estudio en ese centro.
    E.5 End points
    E.5.1Primary end point(s)
    Mean percentage adherence to LUM/IVA treatment over 48 weeks
    Media del porcentaje de cumplimiento terapéutico del tratamiento con LUM/IVA durante 48 semanas
    E.5.1.1Timepoint(s) of evaluation of this end point
    48 weeks
    48 semanas
    E.5.2Secondary end point(s)
    1. Mean percentage adherence to LUM/IVA treatment over 24 weeks
    2. Mean percentage adherence to LUM/IVA treatment from Week 25 through Week 48
    3. Proportion of subjects with ≥80% LUM/IVA adherence over 24 weeks
    4. Proportion of subjects with ≥80% LUM/IVA adherence over 48 weeks
    5. Proportion of subjects with ≥80% LUM/IVA adherence from Week 25 through Week 48
    6. Proportion of subjects with a non-physician-directed LUM/IVA interruption ≥72 hours over 48 weeks
    7. Number of non-physician-directed LUM/IVA interruptions ≥72 hours over 48 weeks
    8. Time to the first non-physician-directed LUM/IVA interruption ≥72 hours over 48 weeks
    1. Media del porcentaje de cumplimiento terapéutico del tratamiento con LUM/IVA durante 24 semanas
    2. Media del porcentaje de cumplimiento terapéutico del tratamiento con LUM/IVA desde la semana 25 hasta la semana 48 inclusive
    3. Porcentaje de sujetos con cumplimiento terapéutico de LUM/IVA ≥ 80 % durante 24 semanas
    4. Porcentaje de sujetos con cumplimiento terapéutico de LUM/IVA ≥ 80 % durante 48 semanas
    5. Porcentaje de sujetos con cumplimiento terapéutico de LUM/IVA ≥ 80 % desde la semana 25 hasta la semana 48 inclusive
    6. Porcentaje de sujetos con una interrupción de LUM/IVA ≥ 72 horas no indicada por el médico a lo largo de las 48 semanas
    7. Número de interrupciones de LUM/IVA ≥ 72 horas no indicadas por el médico a lo largo de las 48 semanas
    8. Tiempo hasta la primera interrupción de LUM/IVA ≥ 72 horas no indicada por el médico a lo largo de las 48 semanas
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Over 24 weeks
    2. From Week 25 through Week 48
    3. Over 24 weeks
    4. Over 48 weeks
    5. From Week 25 through Week 48
    6. Over 48 weeks
    7. Over 48 weeks
    8. Over 48 weeks
    1. Durante 24 semanas
    2. Desde la semana 25 hasta la semana 48 inclusive
    3. Durante 24 semanas
    4. Durante 48 semanas
    5. Desde la semana 25 hasta la semana 48 inclusive
    6. Durante 48 semanas
    7. Durante 48 semanas
    8. Durante 48 semanas
    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 No
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    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 Yes
    E.6.13.1Other scope of the trial description
    Adherence
    Adherencia
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Cada brazo de estudio incluirá una tecnología inteligente con alerta de dosificación activa o inacti
    Each study arm will include a smart technology with either active or inactive dosing alert features.
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LPLV
    UVUP
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    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: 15
    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: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 60
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 42
    F.4.2.2In the whole clinical trial 75
    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 Decision2017-05-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-04-26
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    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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