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    Summary
    EudraCT Number:2017-001078-41
    Sponsor's Protocol Code Number:VX13-809-011
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2017-03-27
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2017-001078-41
    A.3Full title of the trial
    A Phase 3, Open-label Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study in people with cystic fibrosis (a rare pulmonary disease) to assess the pharmacokinetics and safety of a combination of two experimental drugs"
    A.4.1Sponsor's protocol code numberVX13-809-011
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01897233
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/220/2016
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of Sponsor Vertex 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 support Vertex Pharmaceuticals Incorporated
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation Vertex 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, Massachusetts
    B.5.3.3Post code02210
    B.5.3.4CountryUnited States
    B.5.4Telephone number1877634-8789
    B.5.5Fax number1510595-8183
    B.5.6E-mailmedical_info@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 lumacaftor/ivacaftor
    D.2.1.1.2Name of the Marketing Authorisation holderVertex Pharmaceuticals (Europe) Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 namelumacaftor/ivacaftor
    D.3.2Product code VX-809/VX-770
    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 INNLUMACAFTOR
    D.3.9.1CAS number 936727-05-8
    D.3.9.2Current sponsor codeVX-809
    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 number100
    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 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
    E.1.1.1Medical condition in easily understood language
    Cystic Fibrosis
    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 20.0
    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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A: To evaluate the PK of multiple doses of lumacaftor in combination with ivacaftor
    Part B - To evaluate the safety and tolerability of lumacaftor in combination with ivacaftor through
    Week 24
    E.2.2Secondary objectives of the trial
    Part A:
    - To investigate the PK of a lumacaftor metabolite, M28 (M28-lumacaftor), and ivacaftor
    metabolites, M1 and M6 (M1-ivacaftor and M6-ivacaftor)
    - To evaluate the safety and tolerability of multiple doses of lumacaftor in combination
    with ivacaftor

    Part B:
    - To evaluate the PD of lumacaftor in combination with ivacaftor through Week 24
    - To evaluate the off-drug response after the Washout Period (Week 24 to Week 26)
    - To evaluate the PK of lumacaftor, M28-lumacaftor, ivacaftor, M1-ivacaftor, and
    M6-ivacaftor for lumacaftor in combination with ivacaftor
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Subjects (males and/or females), 6 through 11 years of age, inclusive, on the date of
    informed consent (and assent, if applicable) (Part A and Part B).
    •Confirmed diagnosis of CF defined as: with 2 CF-causing mutations, chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities
    •Subjects who weigh ≥15 kg without shoes at Screening Visit
    •Subjects who are homozygous for the F508del-CFTR mutation
    •Subjects with percent predicted forced expiratory volume in 1 second (FEV1) of 70% to 105% (inclusive) (Part A) or ≥40% (Part B) at Screening Visit
    •Subjects with stable CF disease and who are willing to remain on stable CF medication regimen
    •Able to swallow tablets
    E.4Principal exclusion criteria
    •History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
    •Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1 of the study
    •Abnormal liver function as defined in the protocol at Screening Visit
    •Abnormal renal function as defined in the protocol at Screening Visit
    •History of solid organ or hematological transplantation
    •Ongoing participation in an investigational drug study or prior participation in an investigational drug study within 30 days prior of Screening Visit
    •History or evidence of lens opacity or cataract at Screening Visit
    •Colonization with organisms associated with a more rapid decline in pulmonary status at Screening Visit (Part A only)
    •A standard 12-lead ECG demonstrating QTcF >450 msec at Screening Visit
    E.5 End points
    E.5.1Primary end point(s)
    Part A:
    Lumacaftor and ivacaftor PK parameters, including maximum observed concentration (Cmax)
    and area under the concentration versus time curve from time of dosing to time tau (AUC0-τ)

    Part B:
    Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values
    (serum chemistry, hematology, coagulation studies, and urinalysis), standard 12-lead
    electrocardiograms (ECGs), vital signs, pulse oximetry, ophthalmological examinations, and
    spirometry
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part A:
    Day1, Day 14

    Part B:
    Day 1 up to week 26
    E.5.2Secondary end point(s)
    Part A:
    - M28-lumacaftor, M1-ivacaftor, and M6-ivacaftor PK parameters, including Cmax and AUC0-τ
    - Safety and tolerability of lumacaftor in combination with ivacaftor as determined by AEs,
    clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard 12-lead ECGs, vital signs, pulse oximetry, and spirometry

    Part B:
    - Average absolute change from baseline in sweat chloride at Day 15 and at Week 4
    - Absolute change from baseline in body mass index (BMI) and BMI-for-age z-score at Week 24
    - PK parameters of lumacaftor, M28-lumacaftor, ivacaftor, M1-ivacaftor, and M6-ivacaftor
    - Absolute change from baseline in weight and weight-for-age z-score at Week 24
    - Absolute change from baseline in height and height-for-age z-score at Week 24
    - Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score at Week 24
    - Absolute change from baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) domains at Week 24
    - Absolute change in sweat chloride from Week 24 at Week 26
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part A:
    Day 1, Day 1 up to Day 28

    Part B:
    through week 26
    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 No
    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 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 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 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Canada
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days16
    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: 56
    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: 56
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 58
    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)
    No specific plans for additional treatment after the subject has ended the participation in the trial
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
    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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