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    Summary
    EudraCT Number:2013-000250-22
    Sponsor's Protocol Code Number:CVAY736X2201
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-10-16
    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 ConcernedGermany - PEI
    A.2EudraCT number2013-000250-22
    A.3Full title of the trial
    A single dose, double-blind, placebo-controlled, parallel study to assess the pharmacodynamics, pharmacokinetics and safety and tolerability of VAY736 in patients with primary Sjögren’s syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A single dose study to assess the effect of VAY736 in patients with primary Sjögren’s syndrome
    A.3.2Name or abbreviated title of the trial where available
    CVAY736X2201
    A.4.1Sponsor's protocol code numberCVAY736X2201
    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 Pharma GmbH
    B.5.2Functional name of contact pointMedizinischer Infoservice (MCC)
    B.5.3 Address:
    B.5.3.1Street AddressRoonstrasse 25
    B.5.3.2Town/ cityNürnberg
    B.5.3.3Post code90429
    B.5.3.4CountryGermany
    B.5.4Telephone number+491802232300
    B.5.5Fax number+4991127312160
    B.5.6E-mailinfoservice.novartis@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 VAY736
    D.3.4Pharmaceutical form Powder for concentrate for solution for infusion
    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 INNVAY736
    D.3.9.2Current sponsor codeVAY736
    D.3.9.3Other descriptive nameVAY736
    D.3.9.4EV Substance CodeSUB31641
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number3 to 10
    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 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 Yes
    D.3.11.13.1Other medicinal product typemonoclonal antibody
    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 placeboSolution for infusion
    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
    Seropositive primary Sjögren’s syndrome
    E.1.1.1Medical condition in easily understood language
    Sjögren's syndrome
    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 19.0
    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 compare the effect of a single i.v. dose VAY736 versus placebo on the clinical disease activity of primary Sjögren’s syndrome patients as measured by the change of a modified EULAR Sjögren’s Syndrome Disease Activity Index (ESSDAI) between base line and week 12
    • To assess the safety and tolerability of a single i.v. dose VAY736 in patients with primary Sjögren’s syndrome as measured by adverse events (AEs)
    E.2.2Secondary objectives of the trial
    • To evaluate the effect of a single i.v. dose VAY736 versus placebo on self-reported outcomes in pSS patients at 12 weeks compared to baseline 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.
    • To determine the changes in the physician global assessment of the patient’s overall disease activity between baseline and week 12 as recorded by a visual analog scale (VAS)
    • To determine the changes in the patients’ global assessment of their disease activity between baseline and week 12 as recorded by a VAS
    • To determine the pharmacokinetics following a single dose i.v. VAY736 in pSS patients
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent must be obtained before any assessment is performed
    2. Male and female subjects age 18 to 75 years of age included
    3. Fulfilled revised European US consensus criteria for pSS (Vitali et al 2002); if key inclusion criteria are missing from medical history, information will be obtained at screening and may be used as a baseline value if required
    4. ESSDAI value ≥ 6
    5. Elevated serum titers at screening of ANA (≥ 1:160) and of RF
    OR
    Seropositive at screening for anti-SSA (± anti-SSB) antibodies
    6. Stimulated whole salivary flow rate at screening of > 0 mL/min
    7. Subjects must weigh at least 50 kg and no more than 150 kg to
    participate in the study, and must have a body mass index (BMI) within
    the range of 18 - 35 kg/m2
    8. Able to communicate well with the investigator, to understand and
    comply with the requirements of the study.
    E.4Principal exclusion criteria
    - Prior or previous use of (specific dosages and intervals prior to study start may apply): B-cell depleting therapy (e.g., rituximab), Prednisone, anti-BAFF mAb, CTLA4-Fc Ig (abatacept), anti-TNF-α mAb, cyclophosphamide, methotrexate, azathioprine and medications known to cause dry mouth. Hydroxychloroquine or methotrexate in a consistent dose for ≥ 3 months prior to randomization is allowed.
    - Active or recent history of clinically significant infection
    - Vaccination within 2 month prior to study
    - History of primary or secondary immunodeficiency
    - Other protocol-defined inclusion/exclusion criteria may apply.
    E.5 End points
    E.5.1Primary end point(s)
    The primary aim of this study is to evaluate the effect of a single i.v. dose VAY736 on the clinical disease activity of primary Sjögren’s syndrome patients as measured by the ESSDAI at week 12 compared to baseline. The statistical analysis model will include data on the ESSDAI from all timepoints at which it was recorded (baseline, weeks 6, 12 and 24) but the primary comparison is made for week 12.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary efficacy variable is the ESSDAI recorded at baseline and week 12.
    E.5.2Secondary end point(s)
    Secondary efficacy variables supporting the exploratory objectives are:
    • EULAR Sjögren’s Syndrome Patient Reported Intensity (ESSPRI) change from baseline.
    • Change in physician global assessment of the patient’s overall disease activity between baseline and week 12 as recorded by a visual analog scale (VAS)
    • Change in the patient’s global assessment of their disease activity between baseline and week 12 as recorded by a VAS
    E.5.2.1Timepoint(s) of evaluation of this end point
    The secondary efficacy variables will be analyzed following the same approach used for the primary efficacy variable.
    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 Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    tolerability, immunogenicity
    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
    non-confirmatory
    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 EEA1
    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
    The study will complete when the last subject completes their Study Completion visit, and any repeat assessments associated with this visit have been documented and followed-up appropriately by the Investigator.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months3
    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 months8
    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: 24
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Patients with active primary Sjögren’s syndrome
    F.4 Planned number of subjects to be included
    F.4.1In the member state24
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 28
    F.4.2.2In the whole clinical trial 28
    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)
    Each subject will be required to complete the study in its entirety and thereafter no further study treatment will be made available to them.

    The investigator must provide follow-up medical care for all patients who are prematurely withdrawn from the study, or must refer them for appropriate ongoing care.
    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 Decision2014-04-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-11-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-02-07
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