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    Summary
    EudraCT Number:2006-005455-15
    Sponsor's Protocol Code Number:CACZ885D2304
    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:2007-02-22
    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 number2006-005455-15
    A.3Full title of the trial
    A three-part multicenter study, with a randomized, doubleblind, placebo controlled, withdrawal design in Part II to assess efficacy, safety, and tolerability of ACZ885 (antiinterleukin-1β monoclonal antibody) in patients with Muckle-Wells Syndrome
    A.4.1Sponsor's protocol code numberCACZ885D2304
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 ACZ885
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNCanakinumab - proposed
    D.3.9.2Current sponsor codeACZ885
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 placeboPowder and solvent for solution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Muckle-Wells Syndrome (Autoinflammatory Disease)
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10064569
    E.1.2Term Muckle-Wells syndrome
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess efficacy (% of patients who relapse) of ACZ885 compared with placebo in Part II as determined by the Physician’s global assessment of autoinflammatory disease activity, assessment of skin disease and inflammation markers (C-reactive protein (CRP) and/or serum amyloid A (SAA).
    E.2.2Secondary objectives of the trial
    To assess the safety, tolerability and immunogenicity of ACZ885. To assess overall efficacy (response rate) of ACZ885 in Part I and Part III as determined by the
    Physician’s global assessment of autoinflammatory disease activity, assessment of skin disease and inflammation markers. To evaluate the PK and PD of ACZ885. To assess the effect of ACZ885 on disease progression with regards to deafness, kidney function, neurological and ophthalmological symptoms.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    From the complete list of the protocol:

    Molecular diagnosis of NALP3 mutations and clinical picture resembling MWS.

    Male and female patients aged 4 to 75 years at the time of the screening visit.

    For patients under anakinra therapy or any other investigational IL-1 blocking therapy, willingness to discontinue the treatment.
    E.4Principal exclusion criteria
    From the complete list of the protocol:

    Use of the following therapies:

    • Etanercept in the 4 weeks prior to the baseline visit (Day 1)

    • Adalimumab in the 8 weeks prior to the baseline visit (Day 1)

    • Infliximab in the 12 weeks prior to the baseline visit (Day 1)
    • Any other investigational biologics in the 8 weeks prior to the baseline visit (Day 1) (with the exception of anakinra therapy and IL-1 blocking therapies –see below)

    • Patients who are on IL-1 blocking biological therapies (including anakinra therapy) need to discontinue this treatment. As soon as the criteria for relapse are fulfilled, patients can enter the study and receive ACZ885 treatment. This run-in phase reduces the classical wash-out phase to a medically meaningful time and avoids unnecessary suffering for the patient in case a predefined wash-out period per protocol is too long for an individual subject.

    • Leflunomide in the 4 weeks prior to the baseline visit (Day 1)

    • Thalidomide in the 4 weeks prior to the baseline visit (Day 1)

    • Cyclosporine in the 4 weeks prior to the baseline visit (Day 1)

    • i.v. immunoglobulin (i.v. Ig) in the 8 weeks prior to the baseline visit (Day 1)

    • 6-Mercaptopurine, azathioprine, cyclophosphamide, or chlorambucil in the 12 weeks prior to the baseline visit (Day 1)

    • Colchicine, dapsone, mycophenolate mofetil in the 3 weeks prior to the baseline visit (Day 1)

    • Corticosteroids ≥20mg/day or >0.4 mg/kg, whichever applies, in the 1 week prior to the baseline visit (Day 1)
    E.5 End points
    E.5.1Primary end point(s)
    The primary variable is the proportion of patients with disease flare in the withdrawal
    period (Part II). Patients who prematurely discontinue study in the withdrawal phase due to any reason will be considered as having disease flare.

    The primary objective of the study is to show superiority of ACZ885 compared to placebo regarding the primary variable in Part II. The two treatment groups will be compared using an exact test (based on hypergeometric probabilities) about the common odds ratio, adjusting for cohort. The null hypothesis to be tested is that the common odds ratio is equal to 1, i.e. the probability of having disease flare is the same for both groups versus the common odds ratio is not equal to 1, i.e. the
    probability of having disease flare is different for both groups. Exact two-sided p-value, the common odds ratio and exact 95% confidence interval for the common odds ratio will be computed. The primary analysis will be based on the ITT population of Part II.
    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 Yes
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity assessment
    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 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 No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Randomised withdrawal design. Double blind in Part II.
    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.8.3 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 EEA11
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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 at the end of part III. At the end of part III an extension study is planned to allow patients to continue treatment.
    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 months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2007-02-22. Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Parents consent or guardians consent mandatory for children and adolescents.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 17
    F.4.2.2In the whole clinical trial 20
    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)
    Extension study planned.
    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 Decision2007-05-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2007-06-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2008-10-29
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