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    Summary
    EudraCT Number:2007-004367-22
    Sponsor's Protocol Code Number:CACZ885D2306
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2008-03-07
    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 ConcernedUK - MHRA
    A.2EudraCT number2007-004367-22
    A.3Full title of the trial
    An open-label, long-term safety and efficacy study of ACZ885 (anti-interleukin-1β monoclonal antibody) administered for at least 6 months in patients with the
    following cryopyrin-associated periodic syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
    A.3.2Name or abbreviated title of the trial where available
    D2306
    A.4.1Sponsor's protocol code numberCACZ885D2306
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberNot available
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/07/439
    D.3 Description of the IMP
    D.3.1Product nameACZ885
    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 (WHO approval pending)
    D.3.9.1CAS number -
    D.3.9.2Current sponsor codeACZ885
    D.3.9.3Other descriptive nameRecombinant human monoclonal antibody to human IL-1beta of the IgG1/K class
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150 mg
    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 typeHuman Monoclonal Antibody
    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
    The following cryopyrin-associated periodic syndromes:

    Familial Cold Autoinflammatory Syndrome,

    Muckle-Wells Syndrome,

    or Neonatal Onset Multisystem Inflammatory 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
    Long-term safety and tolerability of ACZ885 in patients who participated in the A2102, D2201 or D2304 studies or in newly identified patients with the following cryopyrin-associated periodic syndromes (CAPS): Familial Cold Autoinflammatory Syndrome FCAS), Muckle-Wells Syndrome (MWS) or Neonatal Onset Multisystem Inflammatory Disease (NOMID).
    E.2.2Secondary objectives of the trial
    1. Maintenance of response over time defined by the number of patients who do not relapse as determined by the Physician’s global assessment of autoinflammatory disease activity, assessment of skin disease and inflammation markers.
    2. to Assess number of patients who require a dose adjustment or an administration frequency adjustment, with subsequent maintenance of response over time as determined by the Physician’s global assessment of autoinflammatory disease activity, assessment of skin disease and CRP and/or SAA.
    3. Immunogenicity of ACZ885. PK of ACZ885, in particular, comparison of the new HSA- (human serum albumin) drug formulation with previously used HSA+ formulation.
    4. Long-term effects of ACZ885 on disease progression with regards to deafness, kidney function, neurological and ophthalmological symptoms.
    5. Long term maintenance of Health-Related Quality of Life
    (HRQoL) of ACZ885 by using the HAQ©, SF-36®, FACIT-Fatigue©, and CHQ-PF28©.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male and female patients at least 4 years of age at the time of the screening visit.
    2. Patient’s informed consent for ≥ 18 years of age before any assessment is
    performed.
    3. Parent or legal guardian’s written informed consent and child’s assent, if
    appropriate, are required before any assessment is performed for patients
    <18 years of age.
    4. Patients with a diagnosis of FCAS, MWS, or
    NOMID.
    At study entry, new ACZ885 NAIVE patients should have symptoms requiring pharmacological intervention.
    At the time of screening, patients can be either untreated or treated (i.e. under
    ACZ885, anakinra, or any other investigational IL-1 blocking therapy). 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 MOLECULLARLY TESTED FOR NALP3 MUTATIONS, MOLECULAR TESTING SHOULD BE PERFORMED DURING THE COURSE OF THE STUDY.)
    5. For patients under anakinra therapy or any other investigational IL-1 blocking
    therapy, these treatments should be discontinued prior to the baseline visit (see
    Section 6.5.8 for specific time points for discontinuing treatments prior to the
    baseline visit).
    6. Patients from the A2102 study may enter this study upon signing informed
    consent irrespective of whether they are in remission or flaring. However, dosing
    at Visit 2 (Baseline Visit) can only occur if either 1) the patient is experiencing
    disease flare or 2) at least two months have elapsed from their last injection even
    in the absence of flare, whichever is earlier.
    7. Patients who completed the D2304 study may enter this study upon signing
    informed consent. A patient is defined as completing the study if he/she
    completed the D2304 study up to and including Visit 15 (End of Study Visit).
    8. PATIENTS WHO COMPLETED THE DD201 STUDY MAY ENTER THIS STUDY UPON SIGNING INFORMED CONSENT. A PATIENT IS DEFINED AS COMPLETING THE STUDY IF HE/SHE COMPLETED THE D2201 STUDY UP TO AN INCLUDING THE END OF STUDY VISIT.
    9. Patients who discontinued from the A2102, D2201 or D2304 studies and for whom in the
    Investigator’s judgment (with prior agreement from Novartis) treatment with
    ACZ885 in this study is considered appropriate.
    10. Body weight ≥ 15 kg.
    11. Able to communicate with the investigator and comply with the requirements of
    the study (for children the parent can assist when necessary).
    E.4Principal exclusion criteria
    1. Pregnant or nursing (lactating) women.
    2. Women of child-bearing potential unless they meet the following definition of postmenopausal state: 12 months of natural (spontaneous) amenorrhea or 6 months of spontaneous amenorrhea with serum FSH levels >40 mIU/mL or are using one or more of the acceptable methods of contraception detailed in the protocol are used.
    3. Participation in any clinical investigation within 4 weeks prior to dosing or longer if
    required by local regulation with the exception of trials with anakinra, other
    investigational IL-1 blocking therapies, and/or ACZ885.
    4. History of being immunocompromised, including a positive HIV at screening
    (ELISA and Western blot) test result.
    5. A positive HBsAg or Hepatitis C antibody test result.
    6. Live vaccinations within 3 months prior to the start of the trial, during the trial, and
    up to 3 months following the last dose.
    7. History of drug or alcohol abuse within the 12 months prior to dosing.
    8. Donation or loss of 400 mL or more of blood within 8 weeks prior to dosing for
    adults (For children the cut-off amount of donation or loss of blood should be
    based on the Investigator's judgment & according to the local regulations. Please
    also refer to Appendix 4 for the recommended sample volume per blood draw for
    children).
    9. History of significant medical conditions, which in the Investigator’s opinion would
    exclude the patient from participating in this trial (this can be discussed with
    Novartis on a case by case basis in case of uncertainty).
    10. History of recurrent and/or evidence of active bacterial, fungal, or viral infections.
    11. Positive tuberculin skin test reaction (PPD 5 tuberculin units or as according to
    local standard practice) (≥ 5 mm induration) at 48 to 72 hours after administration at the screening visit or within 2 months prior to the screening visit, according to
    national guidelines. Patients who have a positive PPD skin test with a
    documentation of BCG vaccination, who are at low environmental risk for
    tuberculosis (TB) infection or reactivation, and have a negative chest X-ray can be
    included. A positive PPD test has been defined using the [MMWR 2000
    guidance], summarized as criteria for tuberculin positivity by risk group:
    • equal or greater than 15 mm of induration for persons with no risk factors for
    TB
    • equal or greater than 10 mm of induration for persons with an increased
    probability of recent infection or with other clinical conditions that increased
    the risk for TB
    • equal or greater than 5 mm of induration for very high risk population (HIV),
    contact TB cases, immunosuppression (organ transplantation, steroids
    > 15 mg/day of prednisone for 1 month or more).
    12.Precaution against tuberculosis should be handled according to the best medical
    practice consistent to the local standards in each country with prior consultation
    with Novartis. Patients requiring administration of antibiotics against latent
    tuberculosis should complete their treatment and should be considered cured
    prior to being re-considered for entry into this study.
    13. Use of:
    • Etanercept in the 4 weeks prior to the baseline visit (Day 1) and thereafter
    • Adalimumab in the 8 weeks prior to the baseline visit (Day 1) and thereafter
    • Infliximab in the 12 weeks prior to the baseline visit (Day 1) and thereafter
    • Rituximab in the 26 weeks prior to the baseline visit (Day 1) and thereafter
    • Any other investigational biologics in the 8 weeks prior to the baseline visit
    (Day 1) and thereafter (with the exception of anakinra therapy –see below)
    • Kineret (anakinra therapy) 1 day prior to the baseline visit (Day 1) and
    thereafter
    • Leflunomide in the 4 weeks prior to the baseline visit (Day 1) and thereafter.
    After the completion of leflunomide treatment a cholestiramine in dose 8 g 3
    times per day for 14 days is recommended.
    • Thalidomide in the 4 weeks prior to the baseline visit (Day 1) and thereafter
    • Cyclosporine in the 4 weeks prior to the baseline visit (Day 1) and thereafter
    • i.v. immunoglobulin (i.v. Ig) in the 8 weeks prior to the baseline visit (Day 1)
    and thereafter
    • 6-Mercaptopurine, azathioprine, cyclophosphamide, or chlorambucil in the
    12 weeks prior to the baseline visit (Day 1) and thereafter
    • Dapsone, mycophenolate mofetil in the 3 weeks prior to the baseline visit
    (Day 1) and thereafter
    ≥20mg/day or >0.4 mg/kg, whichever applies, in the 1 week
    prior to the baseline visit (Day 1) and thereafter
    • Use of other investigational non-biological drugs at the time of enrollment,
    within 30 days or 5 half-lives of enrollment, whichever is longer.
    14. History of hypersensitivity to any of the study drugs or to drugs of similar chemical
    classes
    E.5 End points
    E.5.1Primary end point(s)
    Assess Long term safety and tolerability
    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 Yes
    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) 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 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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    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 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 68
    F.4.2.2In the whole clinical trial 80
    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)
    Expected that product has a MA at that time.
    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 Decision2008-04-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2008-05-01
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2010-04-29
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