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    Summary
    EudraCT Number:2013-004808-19
    Sponsor's Protocol Code Number:CCFZ533X2203
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-05-06
    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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2013-004808-19
    A.3Full title of the trial
    A multi-center, randomized, double-blind, placebo-controlled, parallel group study to assess the safety, tolerability, pharmacokinetics and preliminary efficacy of CFZ533 in patients with primary Sjögren’s syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study in patients with primary Sjögren’s syndrome with the aim to assess safety, tolerability, pharmacokinetics (way the body absorbs, distributes, and gets rid of the drug) and preliminary efficacy of CFZ533
    A.3.2Name or abbreviated title of the trial where available
    Safety, pharmacokinetics and preliminary efficacy study of CFZ533 in patients with primary Sjögren's
    A.4.1Sponsor's protocol code numberCCFZ533X2203
    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 SponsorNovartis Pharma Services AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma Services AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Hungary Kft. Pharma
    B.5.2Functional name of contact pointPublic Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressBartók Béla út 43-47.
    B.5.3.2Town/ cityBudapest
    B.5.3.3Post code1114
    B.5.3.4CountryHungary
    B.5.4Telephone number+361457-6500
    B.5.5Fax number+361457-6600
    B.5.6E-mailinfoph.hungary@novartis.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.3.2Product code CFZ533
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot yet established
    D.3.9.2Current sponsor codeCFZ533
    D.3.9.4EV Substance CodeSUB130512
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 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.3.2Product code CFZ533
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot yet established
    D.3.9.2Current sponsor codeCFZ533
    D.3.9.4EV Substance CodeSUB130512
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPowder for solution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPowder for solution for injection
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    primary Sjögren’s syndrome
    E.1.1.1Medical condition in easily understood language
    autoimmune condition of unknown cause where the body's own self-defenses attack glands that secrete fluids resulting in inflammation of the glands. The inflammatory process can involve other organs.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level PT
    E.1.2Classification code 10040767
    E.1.2Term Sjogren's syndrome
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue 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 assess the safety and tolerability of multiple intravenous infusion of CFZ533 in patients with primary Sjögren’s syndrome as measured by adverse events (AEs).
    • To compare the effect of multiple intravenous infusion of CFZ533 versus placebo on the clinical disease activity of primary Sjögren’s syndrome patients as measured by the change of an EULAR Sjögren’s Syndrome Disease Activity Index (ESSDAI) after 12 weeks treatment.
    E.2.2Secondary objectives of the trial
    • To assess the safety and tolerability of multiple subcutaneous doses of CFZ533 in patients with primary Sjögren’s syndrome as measured by adverse events (AEs).
    • To compare the effect of multiple subcutaneous doses of CFZ533 versus placebo on the clinical disease activity of primary Sjögren’s syndrome patients as measured by the change of an EULAR Sjögren’s Syndrome Disease Activity Index (ESSDAI) after 12 weeks treatment.
    • To assess the pharmacokinetics of multiple subcutaneous doses and multiple intravenous infusion of CFZ533 in primary Sjögren’s syndrome patients.

    Other protocol defined inclusion criteria may apply;
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Written informed consent must be obtained before any assessment is performed;
    • Male and female patients 18 to 75 years of age included;
    • Subjects must have a body weight of 50 – 150 kg (inclusive);
    • Diagnosis of primary Sjögren’s syndrome according to revised EU/US consensus criteria (Vitali et al 2002);
    • Moderate to severe disease activity as determined by ESSDAI score ≥ 6;
    • Presence of autoantibodies at screening as determined by any of the following:
     Elevated serum titers of ANA (≥ 1:160) and positive rheumatoid factor (RF); or,
     Positive anti-SSA
    • Stimulated whole salivary flow rate > 0 mL/min;
    • If the patient is on oral glucocorticoid treatment at screening, the dose must NOT exceed 10 mg prednisone or equivalent per day, and must be stable for at least 2 weeks prior to randomization and for the duration of the study;
    • If the patient is on chloroquine or hydroxychloroquine at screening, the dose must be stable for at least 4 weeks prior to randomization and for the duration of the study;
    • If the patient is treated with oral or parenteral methotrexate at screening, the dose must NOT exceed 25 mg per week for at least 3 months prior to randomization and must be stable for the duration of the study;
    • If the patient is treated with oral azathioprine at screening, the dose must NOT exceed 100 mg per day for at least 3 months prior to randomization and must be stable for the duration of the study;
    • Subject must be able to communicate well
    E.4Principal exclusion criteria
    • Secondary Sjögren’s syndrome. Patients with laboratory or clinical signs of another connective tissue disease (e.g., systemic lupus erythematous) may be eligible at the investigators discretion;
    • Use other investigational drugs at the time of enrollment, or is within five half-lives of using other investigational drugs or longer if required by local regulations, at the time of enrollment;
    • History of hypersensitivity to study drug or to drugs of similar chemical classes;
    • Patients having received the following treatments (within given timeframe before randomization):
     Oral or i.v. cyclosphosphamide treatment within 6 months;
     i.v. corticosteroid bolus with dose > 1 mg/kg within 3 months;
     Rituximab within 12 months. For patient who received rituximab earlier, B cell count should be within normal range;
     Belimumab within 6 month;
     Any other biologic within 1 month or five times the half-life, whichever is longer;
     Any other immunosuppressives (despite methotrexate, glucocorticoids, and
    hydroxychloroquine on stable doses as described in the inclusion criteria 8, 9, 10, 11) such as cyclosporine A or mycophenolate within 3 months;
    • Patients where the primary cause of sicca symptoms (e.g. dry mouth, dry eyes) as judged by the investigator, is attributable to a medication used regularly or intermittently (rather than to primary Sjogren`s syndrome)
    • Patients who are at significant risk for thromboembolic events as judged by the investigator or have any one of the following:
     History of either thrombosis or 3 or more spontaneous abortions with or without the presence of anti-cardiolipin autoantibodies;
     Presence of lupus anticoagulant or prolonged partial thromboplastin time (PTT);
    • Pancreatic injury or pancreatitis as indicated by abnormal signs or symptoms of pancreatitis or clinically significant elevations in amylase or lipase;
    • History or presence of any medically significant cardiac condition which according to the investigator may jeopardize the patient in case of participation in the study, including ischemic heart disease, heart failure, cardiomyopathy, myocardial infarction or stroke;
    • Sitting vital signs outside of the following ranges at screening or baseline: body temperature: 35.0 - 37.5°C, systolic blood pressure: 90 - 145 mmHg, diastolic blood pressure: 50 - 90 mmHg, pulse rate 50 - 100 bpm.
    • History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases;
    • Signs or symptoms of a clinically significant systemic viral, bacterial or fungal infection within 30 days of randomization;

    Other protocol defined exclusion criteria may apply.
    E.5 End points
    E.5.1Primary end point(s)
    a. adverse event (AE) monitoring
    b. change of an EULAR Sjögren’s Syndrome Disease Activity Index (ESSDAI) after 12 weeks treatment
    E.5.1.1Timepoint(s) of evaluation of this end point
    a. from screening to End Of Study (EOS) for all AEs/ until 30 days after
    EOS for SAEs
    b. at screening, baseline and at week-3, 5, 9, 13, 15, 17, 21, 25, 29, 33

    For further information, please refer to the Assessment schedule in the protocol
    E.5.2Secondary end point(s)
    a. Pharmacokinetics parameters
    b. Effect on self-reported outcomes as measured by the EULAR Sjögren’s Syndrome Patient Reported Intensity (ESSPRI), the Short Form (36) Health Survey (SF-36) and the Multidimensional Fatigue Inventory (MFI) Questionnaire after 12 weeks treatment.
    c. Changes in the physician global assessment of the patient’s overall disease activity as recorded by a visual analog scale (VAS) after 12 weeks treatment.
    d. Changes in the patients global assessment of their disease activity as recorded by a VAS after 12 weeks treatment.
    E.5.2.1Timepoint(s) of evaluation of this end point
    a. At week-1, 3, 5, 9, 11, 13, 15, 17, 19, 21, 25, 29, 33
    b. At baseline and at week-9, 13, 17, 21, 25, 29, 33
    c. At baseline and at week-3, 5, 9, 13, 15, 17, 21, 25, 29, 33
    d. At baseline and at week-3, 5, 9, 13, 15, 17, 21, 25, 29, 33

    For further information, please refer to the Assessment schedule in the protocol
    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 Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    open-label in part 2
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    Switzerland
    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.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days6
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months5
    E.8.9.2In all countries concerned by the trial days6
    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: 37
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 42
    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
    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 Decision2014-07-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-06-24
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-06-29
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