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    Summary
    EudraCT Number:2015-000097-35
    Sponsor's Protocol Code Number:CCFZ533X2204
    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:2015-06-05
    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 number2015-000097-35
    A.3Full title of the trial
    A multi-center, randomized, double-blind, placebo-controlled, parallel group study to preliminarily evaluate the safety, tolerability, pharmacokinetics and efficacy of CFZ533 in patients with moderate to severe myasthenia gravis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety, tolerability and efficacy of CFZ533 in the treatment of moderate to severe myasthenia gravis patients
    A.3.2Name or abbreviated title of the trial where available
    Safety,tolerability,pharmacokinetics and efficacy of CFZ533 in moderate to severe myasthenia gravis
    A.4.1Sponsor's protocol code numberCCFZ533X2204
    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 CFZ533
    D.3.4Pharmaceutical form Powder for solution for injection/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.3.9.3Other descriptive nameCFZ533
    D.3.9.4EV Substance CodeSUB130512
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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 placeboSolution for injection/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
    Acquired myasthenia gravis (MG) is an autoimmune disease that leads to fluctuating muscle weakness and fatigue. In the most common cases, muscle weakness is caused by circulating antibodies that bind to acetylcholine receptors at the postsynaptic neuromuscular junction, inhibiting the excitatory effects of the neurotransmitter acetylcholine.
    E.1.1.1Medical condition in easily understood language
    Myasthenia Gravis
    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 10028417
    E.1.2Term Myasthenia gravis
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • To evaluate the safety and tolerability of CFZ533 as an add-on therapy to standard of care in moderate to severe MG patients.
    • To evaluate the efficacy of IV CFZ533 as an add-on therapy to standard of care in patients with moderate to severe MG after 24 weeks of treatment.
    E.2.2Secondary objectives of the trial
    • To evaluate the efficacy of CFZ533 throughout the 24 weeks treatment period
    and the decay in efficacy throughout the 24 weeks follow-up period.
    • To evaluate changes in patient’s quality of life (QOL) throughout 24 weeks
    the treatment period.
    • To evaluate the pharmacokinetics of CFZ533
    • To evaluate the pharmacodynamics of CFZ533
    • To assess immunogenicity of CFZ533
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Diagnosis of MG class IIa to IVa inclusive (Myasthenia Gravis
    Foundation of America Clinical Classification).
    • Quantitative Myasthenia Gravis (QMG) score of 10 or greater. If the
    QMG score is < 15,
    no more than 4 points may be derived from items 1 or 2 (ocular motility
    disturbance and ptosis).
    • Documented history of acetylcholine receptor (AChR) or Muscle
    Specific Kinase (MuSK) antibody positive (assessed at screening if no
    medical history available).
    • Only one immunosuppressant or immunomodulatory drug at a stable
    dose is allowed during the study (i) azathioprine and mycophenolate
    mofetil must be stable for at least 4 months prior to randomization (ii)
    cyclosporine must be
    stable for at least 3 months prior to randomization.
    If the patient is on oral corticosteroids, methotrexate or tacrolimus at
    screening, the dose must be stable for at least 1 month prior to
    randomization.
    • If the patient is on cholinesterase inhibitors at screening, the dose
    must be stable for at least 2 weeks prior to randomization.
    • Women of child-bearing potential, defined as all women physiologically
    capable of becoming pregnant, may be included in the study if they are
    using highly effective methods of contraception during the study and for
    12 weeks after study treatment.
    E.4Principal exclusion criteria
    • MGFA grade I, IVb, or V disease.
    • Documented presence of unresected thymoma.
    • Patients having undergone thymectomy or thymo thymectomy
    (resection of thymoma) within 6 months of screening.
    • Patients having received any of the following treatments prior to
    randomization:
    IVIg or plasma exchange within 8 weeks oral or IV cyclosphosphamide
    treatment within 3 months; IV corticosteroid bolus (dose higher than 1
    mg/kg) within 3 months; belimumab within 6 months. For patients who
    received belimumab earlier, B cell count should be within normal range;
    rituximab within 12 months. For patients who received rituximab earlier,
    B cell count should be within normal range; any other biologic or an
    investigational drug within 1 month or five times the half-life, whichever
    is longer.
    • Live vaccines within 4 weeks prior to randomization.
    • Patients who are at significant risk for TE 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 prolonged partial thromboplastin time (PTT).
    E.5 End points
    E.5.1Primary end point(s)
    • Mean change from baseline in the QMG Score for Disease Severity after 24 weeks of treatment (primary endpoint).
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 24 weeks of treatment
    E.5.2Secondary end point(s)
    • Mean Change from baseline in QMG Score and MGC Score..
    • Proportion of patients with improvement by ≥ 3 points in QMG score.
    • Proportion of patients with worsening by ≥ 3 points in QMG score.
    • Proportion of patients intolerant to steroid taper.
    • Proportion of patients who discontinued due to inefficacy or worsening.
    • Mean change from baseline in the MG-ADL and MG QOL-15.
    • Free CFZ533 in plasma
    • Free CD40 on B cells, total CD40 on B cells and total soluble CD40 in plasma
    • Quantitative analysis of anti-CFZ533 antibodies in plasma
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the study
    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 No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    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 No
    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 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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
    Canada
    Denmark
    Germany
    Russian Federation
    Taiwan
    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
    LPLV
    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 months5
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months8
    E.8.9.2In all countries concerned by the trial days2
    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: 34
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 14
    F.4.2.2In the whole clinical trial 44
    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)
    After completion of the clinical trial, further treatment is left to the physician's discretion according to medical practice in this disease area.
    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 Decision2015-09-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-08-20
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-04-19
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