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    Summary
    EudraCT Number:2009-016859-22
    Sponsor's Protocol Code Number:CACZ885D2307
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2012-03-09
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2009-016859-22
    A.3Full title of the trial
    A one-year open-label, multicenter trial to assess efficacy, safety and
    tolerability of canakinumab (ACZ885) and the efficacy and safety of
    childhood vaccinations in patients aged 4 years or younger with
    Cryopyrin Associated Periodic Syndromes (CAPS).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to check how safe, beneficial and tolerable the drug
    canakinumab, along with childhood vaccines, is for patients with
    Cryopyrin Associated Periodic Syndromes (CAPS).
    A.3.2Name or abbreviated title of the trial where available
    D2307
    A.4.1Sponsor's protocol code numberCACZ885D2307
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/208/2011
    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 AG
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressForum 1, Novartis Campus
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4056
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number41613241111
    B.5.5Fax number41613248001
    B.5.6E-mailclinicaltrial.enquiries@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 Yes
    D.2.1.1.1Trade name ILARIS
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Ltd
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 namecanakinumab
    D.3.2Product code ACZ885
    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 INNCANAKINUMAB
    D.3.9.1CAS number 914613-48-2
    D.3.9.2Current sponsor codeACZ885
    D.3.9.4EV Substance CodeSUB30137
    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) 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Cryopyrin Associated Periodic Syndromes (CAPS)
    E.1.1.1Medical condition in easily understood language
    CAPS is a group of inflammatory disorders (causing redness, swelling, pain and fever) caused by the body making too much of a protein called interleukin 1β (IL-1β).
    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 14.1
    E.1.2Level PT
    E.1.2Classification code 10068850
    E.1.2Term Cryopyrin associated periodic syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    This trial will assess the safety, efficacy and tolerability of ACZ885 in patients aged 4 years and younger with cryopyrin associated periodic syndromes (CAPS).
    E.2.2Secondary objectives of the trial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male and female patients that are 28 days to 60 months of age at the time of the screening visit.
    2. Body weight ≥ 2.5 kg.
    3. Parent or legal guardian's written informed consent is required before any assessment is performed for patients.
    4. At study entry, patients should have a clinical diagnosis of FCAS, MWS, or NOMID and symptoms requiring pharmacological intervention. Prior agreement between the Investigator and Novartis for study eligibility is required for patients who do not have a molecular diagnosis of NALP3 mutations available (either testing not performed, or testing performed but negative) upon study entry. For those patients who have not been molecularly tested for NALP3 mutations, molecular testing should be performed during the course of the study.
    5. For patients treated with an IL-1 blocking agent (i.e. anakinra,
    rilonacept), these treatments should be discontinued prior to the
    baseline visit and patients must demonstrate active disease prior to treatment.
    6. Patients who are scheduled to receive an immunization, according to their local vaccination guidelines, with an inactivated vaccine must be willing to participate in the assessment schedule for vaccinated patients.

    Other protocol-defined inclusion criteria may apply.
    E.4Principal exclusion criteria
    1. Preterm neonates for whom, in the Investigator's judgment,
    participation in the study is not deemed appropriate.
    2. History of recurrent and/or evidence of active bacterial, fungal, or viral infections (including HIV).
    3. Patients with immunodeficiency or treatment with
    immunosuppressive drugs.
    4. Live vaccinations within inferior or equal to 3 months prior to
    screening. No live vaccinations will be allowed throughout the course of this study and up to 3 months following the last dose.
    5. Patients with an increased risk of tuberculosis (TB) infection
    according to following risk factors:
    • Patients with recent close contact with persons known to have active pulmonary TB disease
    • Foreign-born patients from countries with a high prevalence of
    tuberculosis
    • Patients with recent tuberculosis infection (including children > 6
    months with a positive PPD test [defined as an induration of at least 10mm])
    • Patients with end-stage renal disease
    • Patients with diabetes mellitus
    • Patients receiving immunosuppressive therapy
    • Patients with hematologic cancers.
    6. Participation in another trial within the last 30 days or 5 half-lives of the investigational compound (whichever is longer).
    7. Familial and social conditions rendering regular medical assessment not possible.
    8. Pediatric patients with neutropenia (absolute neutrophil count [ANC] < 1.5 x 10^9/l)

    Other protocol-defined exclusion criteria may apply.
    E.5 End points
    E.5.1Primary end point(s)
    To assess the efficacy of canakinumab with respect to the treatment response in CAPS patients 4 years and younger
    E.5.1.1Timepoint(s) of evaluation of this end point
    A maximum of 56 weeks
    E.5.2Secondary end point(s)
    •To assess the efficacy of canakinumab with respect to the treatment response in CAPS patients 2 years and younger
    •Safety and tolerability as assessed by overall frequency of adverse events and number of patients completing the study in patients 2 years and younger and the overall population
    •To assess the presence of protective antibody levels following immunization with inactivated (killed) vaccines
    •To evaluate the safety of canakinumab treatment in pediatric patients receiving a concomitant vaccination
    •To evaluate the proportion of patients with vaccinated-associated reactions
    •To assess the reduction of inflammation marker (C-reactive protein (CRP) or serum amyloid A (SAA)) after treatment initiation
    E.5.2.1Timepoint(s) of evaluation of this end point
    A maximum of 56 weeks
    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 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
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 No
    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.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Canada
    Israel
    Switzerland
    E.8.7Trial has a data monitoring committee Yes
    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.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial 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: 15
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    paediatric patients
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 15
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: Canada
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