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    Summary
    EudraCT Number:2018-002624-16
    Sponsor's Protocol Code Number:P170907J
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2018-11-21
    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 ConcernedFrance - ANSM
    A.2EudraCT number2018-002624-16
    A.3Full title of the trial
    Validation of respiratory epithelial functional assessment
    to predict clinical efficacy of Orkambi®.
    Pathway to personalized therapy in Cystic Fibrosis
    PREDICT-CF
    Validation de l'exploration fonctionnelle épithéliale pour prédire la réponse clinique de Orkambi® un modulateur de la protéine CFTR. Vers une thérapie personnalisée de la mucoviscidose. PREDICT-CF
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    NA
    NA
    A.3.2Name or abbreviated title of the trial where available
    PREDICT-CF
    A.4.1Sponsor's protocol code numberP170907J
    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 SponsorASSISTANC-PUBLIQUE-HOPITAUX DE PARIS (AP-HP)
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportDGOS
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationASSISTANCE PUBLIQUE-HOPITAUX DE PARIS (AP-HP)
    B.5.2Functional name of contact pointDRCI Hôpital Saint-Louis
    B.5.3 Address:
    B.5.3.1Street Address1, Avenue Claude Vellefaux
    B.5.3.2Town/ cityPARIS
    B.5.3.3Post code75010
    B.5.3.4CountryFrance
    B.5.6E-mailsylvie.prieur@aphp.fr
    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® Lumacaflor/Ivacaflor. CAS lumacaflor : 936727-05-8 CAS ivacaflor : 873054-44-5
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 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
    Homozygous F508del patient aged 12 years or older
    NA
    E.1.1.1Medical condition in easily understood language
    Homozygous F508del patient aged 12 years or older
    Patient with an indication for Orkambi®
    NA
    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 No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To investigate the predictive value for improvement of percentage of predicted FEV1 at 24 weeks of treatment with Orkambi® of a test quantifying in vitro the correction of CFTR activity, as assessed by the change in cyclic AMP (cAMP) dependant Chloride (Cl-) secretion in patient derived HNE culture after Lumacaftor/Ivacaftor 48 hrs incubation.
    NA
    E.2.2Secondary objectives of the trial
    1) to assess the predictive value of the test for improvement of the absolute change in the Z-score of the predicted FEV1 from baseline to week 24 of Orkambi®
    2) to assess the predictive value of the test for improvement of the absolute change in the percentage / Z-score of predicted FEV1 from baseline to week 48 of Orkambi®
    3) to assess the predictive value of the test for the other parameters of the lung function response (Forced Vital Capacity, Residual Functional Capacity) to Orkambi® at week 24 and 48
    4) to assess the predictive value of the test for the Lung Clearance Index response to Orkambi® at week 48
    5) to assess the predictive value of the test for the nutritional response to Orkambi® at week 24 and 48
    6)7)8)9)10)11) cf protocol
    NA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Homozygous F508del patient aged 12 years or older
    - Patient with an indication for Orkambi® treatment according to the marketing authorization application
    - Patient never received Orkambi® in the past
    - Signed Informed consent form by the patient (if aged ≥ 18 years), or by parents / legal guardian and patient’s agreement (if aged < 18 years)
    - Patient affiliated to the health insurance system
    NA
    E.4Principal exclusion criteria
    - Homozygous F508del patients who do not meet the treatment indications according to the marketing authorization application
    - Patients refusing Orkambi®
    - CF patients not homozygous for the p.Phe508del mutation
    - Active smoker
    - Severe nasal mucosa disrepair
    - Contraindications to xylocaine anesthesia,
    - Participation with another interventional study with drug

    NA
    E.5 End points
    E.5.1Primary end point(s)
    Absolute change in the percentage of predicted forced expiratory volume in 1 second (%FEV1) from baseline to week 24 of Orkambi®.
    The response to Orkambi® is defined by an absolute change ≥ 10 % after 24 weeks of treatment.
    NA
    E.5.1.1Timepoint(s) of evaluation of this end point
    after 24 weeks of treatment.
    NA
    E.5.2Secondary end point(s)
    1) Absolute change in the Z-score of forced expiratory volume in 1 second (FEV1) from baseline to week 24
    2) Absolute change in percent predicted/Z-score of forced expiratory volume in 1 second (FEV1) from baseline through week 48
    3) Absolute change in percent predicted of forced vital capacity (%FVC) and Functional Residual Capacity (%RFC) from baseline through week 24 and 48
    4) Absolute change in lung clearance index 2.5 (LCI2.5) from baseline through Week 48
    5) Absolute change in height and weight-for-age-z-score from baseline to week 24 and 48
    6) Absolute change in CFU of sputum microorganisms from baseline to week 24 and 48
    7) Number of exacerbations to week 48 in comparison to the year previous treatment with Orkambi®
    8)9)10)11) cf protocol
    The response to Orkambi® will also be studied in secondary endpoint as an absolute change in the percentage of FEV1 ≥ 5 % after 24 and 48 weeks of treatment.
    NA
    E.5.2.1Timepoint(s) of evaluation of this end point
    to week 24 or to week 48 or to week24 and week 48
    NA
    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 Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    The predictive value for improvement of percentage of predicted FEV1 at 24 weeks of treatment with Orkambi®
    NA
    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
    NA
    Prospective cohort study
    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 concerned17
    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
    NA
    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 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: 52
    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: 52
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 50
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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.4.1In the member state104
    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)
    Therapeutic care of patients stays identical during research and post research as usual care.
    NA
    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 Decision2018-12-13
    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 Status
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