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    Summary
    EudraCT Number:2019-001314-41
    Sponsor's Protocol Code Number:69501
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-05-06
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2019-001314-41
    A.3Full title of the trial
    Kinetics of ivacaftor at Switch Orkambi Symkevi study
    Kinetiek van ivacaftor op het moment van Switch Orkambi Symkevi onderzoek
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This study investigates the effect of switching from Orkambi treatment to Symkevi. In particuar we want to investigate the effect of the switch on the uptake, concentration in the blood an degradation of ivacaftor.
    Dit onderzoek brengt de effecten in kaart van het overstappen van Orkambi (bevat de werkzame stoffen lumacaftor en ivacaftor) naar Symkevi (bevat de werkzame stoffen tezacaftor en ivacaftor). In het bijzonder willen we onderzoeken wat de invloed is van het wijzigen van lumacaftor in tezacaftor op de opname, beschikbaarheid en afbraak van ivacaftor.
    A.3.2Name or abbreviated title of the trial where available
    SOS
    SOS
    A.4.1Sponsor's protocol code number69501
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    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: contains lumacaftor and ivacaftor
    D.2.1.1.2Name of the Marketing Authorisation holderVertex
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 Coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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 Symkevi
    D.2.1.1.2Name of the Marketing Authorisation holderVertex
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 nameSymkevi
    D.3.4Pharmaceutical form Coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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 Yes
    D.2.1.1.1Trade name Kalydeco
    D.2.1.1.2Name of the Marketing Authorisation holderVertex Pharmaceuticals (Ireland) Limited
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 nameKalydeco
    D.3.4Pharmaceutical form Coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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
    Cystic fibrose
    E.1.1.1Medical condition in easily understood language
    mucoviscoidosis
    taaislijmziekte
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary study endpoint is the ivacaftor through concentration.
    Het primaire eindpunt is de dalspiegel van ivacaftor
    E.2.2Secondary objectives of the trial
    - tezacaftor and lumacaftor through concentrations
    - hydroxymethyl-ivacaftor and ivacaftor-carboxylate concentrations
    - ivacaftor, tezacaftor and lumacaftor concentration at T-max
    - dalspiegels van tezacaftor en lumacaftor
    - concentraties van hydroxymethyl-ivacaftor en ivacaftor-carboxylaat
    - concentraties op T-max (optional part of the study) ivacaftor, tezacaftor en lumacaftor (topspiegels)
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Kinetics of ivacaftor at Switch Orkambi Symkevi (SOS)
    Optional study: ivacaftor, tezacaftor and lumacaftor concentration at T-max
    Kinetiek van ivacaftor tijdens Switch Orkambi Symkevi (SOS)
    Optioneel onderzoek: ivacaftor, tezacaftor en lumacaftor concentratie op T-max (topspiegel)
    E.3Principal inclusion criteria
    • The subject is homozygous for the F508del mutation in the CFTR gene.
    • The subject is aged 18 years or older.
    • The subject used Orkambi continuous for at least a month before enrolling in the study.
    • The subject used Orkambi in a dose of twice-daily 2 tablets of 200/125 mg lum/iva.
    • The subject will switch from Orkambi therapy to Symkevi.
    • The subject will initiate Symkevi therapy with a dosage of once-daily 1 tablet of 100/150 mg TEZ/IVA and once-daily 1 tablet of 150 mg IVA
    • The subject has signed and dated a written informed consent.
    • de proefpersoon heeft een homozygote F508del mutatie in het CFTR gen
    • de proefpersoon is 18 jaar of ouder
    • de proefpersoon heeft voor inclusie in het onderzoek gedurende ten minste 1 maand continue Orkambi gebruikt
    • de proefpersoon heeft een dosering van twee maal per dag 2 tabletten van 200/125 mg LUM/IVA gebruikt
    • de proefpersoon zal overstappen van Orkambi (LUM/IVA) naar Symkevi (TEZ/IVA)
    • de proefpersoon zal de Symkevi medicatie aanvangen met een dosering van 1 maal per dag 100/150 mg TEZ/IVA en 1 maal per dag 150 mg IVA
    E.4Principal exclusion criteria
    • The subject is deemed unfit to participate in this study by the treating physician.
    • The subject is experiencing an exacerbation needing treatment with IV antibiotics
    • The subject is door de behandelaar als 'onfit voor deelname aan het onderzoek' aangemerkt
    • The subject heeft een pulmonale exacerbatie waarvoor behandeling met intraveneuze antibiotica noodzakelijk is,
    E.5 End points
    E.5.1Primary end point(s)
    - The primary study endpoint is the ivacaftor through concentration.
    Het primaire eindpunt is de dalspiegel van ivacaftor
    E.5.1.1Timepoint(s) of evaluation of this end point
    After at least one month of treatment with Orkambi, just before subject is supposed to take the next morning dose Orkambi
    After at least one month of treatment with Symkevi, just before subject is supposed to take the next morning dose Symkevi
    Na ten minste 1 maand gebruik van Orkambi, op het moment dat patiënt de volgende ochtenddosis Orkambi moet innemen.
    Na ten minste 1 maand gebruik van Symkevi, op het moment dat patiënt de volgende ochtenddosis Symkevi moet innemen
    E.5.2Secondary end point(s)
    - tezacaftor and lumacaftor through concentrations
    - hydroxymethyl-ivacaftor and ivacaftor-carboxylate concentrations
    - ivacaftor, tezacaftor and lumacaftor concentration at T-max
    - dalspiegels van tezacaftor en lumacaftor
    - concentraties van hydroxymethyl-ivacaftor en ivacaftor-carboxylaat
    - concentraties op T-max (optional part of the study) ivacaftor, tezacaftor en lumacaftor (topspiegels)
    E.5.2.1Timepoint(s) of evaluation of this end point
    After at least one month of treatment with Orkambi, 3-5 hours after subject has taken morning dose Orkambi
    After at least one month of treatment with Symkevi, 3-5 hours after subject has taken morning dose Symkevi
    Na ten minste 1 maand gebruik van Orkambi, 3-5 uur nadat de proefpersoon een ochtenddosis Orkambiheeft ingenomen.
    Na ten minste 1 maand gebruik van Symkevi, 3-5 uur nadat de proefpersoon een ochtenddosis Symkevi heeft ingenomen.
    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 Yes
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 Yes
    E.8.1.7.1Other trial design description
    observationeel
    observational
    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.8.3 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 concerned2
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 100
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 state100
    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)
    Not different from expected nromal treatment
    Niet anders dan reguliere zorg
    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 Decision2019-05-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-05-30
    P. End of Trial
    P.End of Trial StatusCompleted
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