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    Summary
    EudraCT Number:2014-001393-34
    Sponsor's Protocol Code Number:MNILCAPS001
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2014-04-03
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2014-001393-34
    A.3Full title of the trial
    Ilaris® (canakinumab) efficacy and safety in CAPS patients without confirmed mutation on exon 3 of gene CIAS1 and young (<2yrs) CAPS patients with severe neurological (CINCA) phenotype.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Ilaris® (canakinumab) efficacy and safety in CAPS patients without confirmed mutation on exon 3 of gene CIAS1 and young (<2yrs) CAPS patients with severe neurological (CINCA) phenotype.
    A.4.1Sponsor's protocol code numberMNILCAPS001
    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 SponsorUZ Leuven
    B.1.3.4CountryBelgium
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportNovartis
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUZ Leuven
    B.5.2Functional name of contact pointProf Dr Carine Wouters
    B.5.3 Address:
    B.5.3.1Street Addressherestraat 49
    B.5.3.2Town/ cityLeuven
    B.5.3.3Post code3000
    B.5.3.4CountryBelgium
    B.5.4Telephone number3216343801
    B.5.6E-mailcarine.wouters@uzleuven.be
    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 Limited
    D.2.1.2Country which granted the Marketing AuthorisationBelgium
    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 nameIlaris
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPSubcutaneous use
    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
    E.1.1.1Medical condition in easily understood language
    CAPS
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    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
    • To evaluate if canakinumab at labeled dosing can induce and maintain clinical and biological remission in mutation-negative CAPS patients as well as in young (<2yrs) CAPS patients with CINCA phenotype
    E.2.2Secondary objectives of the trial
    • To evaluate the effect of canakinumab treatment on disease activity of CAPS
    • To assess the changes in CRP levels during the treatment period
    • To evaluate the safety of canakinumab in mutation-negative CAPS patients as well as in young (<2yrs) CAPS patients with CINCA phenotype.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female adults, adolescents and children aged 2 years and older with body weight of 7.5 kg or above. Younger (<2 yrs) patients with CINCA phenotype and central nervous system involvement will be included as soon as diagnosis is confirmed.
    2. Patients and/or their legally authorized representative(s) who have agreed to participate and have signed the informed consent
    3. Patients with the diagnosis of CAPS based upon the clinical and biological phenotype, and the opinion of the treating expert physician including: CRP > 10 mg/ l (confirmed by a lab protocol) and at least one of the following clinical manifestations:
    o General symptoms: fever, headache, fatigue, anemia
    o Skin: rash/non-pruritic urticaria
    o Osteo-articular system: arthralgia, arthritis, bone malformations
    o Ophtalmological problems: retinitis, conjunctivitis, uveitis, papilledema ; Progressive deafness (following cochlear nerve damage)
    o Neurological involvement: due to chronic inflammation: chronic aseptic meningitis, cerebral atrophy, intracranial hypertension, ventricle dilatation, calcification, papilledema
    o Renal dysfunction following AA amyloidosis
    4. For patients aged 2 years and older: no mutation confirmed on exon 3 of gene CIAS-1, following standard genetic mutation analysis.
    E.4Principal exclusion criteria
    1. Pregnant or breastfeeding women.
    2. Participation in any clinical trial investigation within 4 weeks prior to dosing or longer if
    required by local regulation.
    3. In case of previous treatment with biologic agents or DMARDs, an appropriate washout
    period (as according to the recognized duration of effect and half-lives)
    4. History of immunological compromisation.
    5. Positive test for or prior history of HIV (ELISA and Western blot), Hepatitis B (Hepatitis B surface antigen) or Hepatitis C.
    6. Presence of active infections or a history of pulmonary TB infection with or without
    documented adequate therapy. Subjects with current active TB, or recent close exposure to an individual with active TB are excluded from the study.
    7. Treatment with a live virus vaccine during 3 months prior to baseline visit. No live vaccines were allowed throughout the course of this study and up to 3 months following the last dose.
    8. History of malignancy except for treated basal cell carcinoma
    9. History of recurrent and/or evidence of active bacterial, fungal or viral infection(s).
    10. Presence of any of the following laboratory abnormalities: ALT or AST greater than 2 times the upper limit of normal (ULN), platelet count less than 100x109/L.
    11. Patients with neutropenia (absolute neutrophil count [ANC] < 1.5 x 109/l)
    12. History of significant medical conditions, which in the investigator’s opinion would exclude the patient from participating in this trial.
    13. Other Interleukin-1 therapy
    E.5 End points
    E.5.1Primary end point(s)
    Response will be defined as follows (all criteria to be fulfilled): Physician global assessment of auto-inflammatory disease activity ≤ minimal (using a 5-pointscale ranging from absent to severe) AND assessment of skin disease ≤ minimal (using a 5-point scale ranging from absent to severe) AND serological response: Serum C-reactive protein (CRP) < 10 mg/L.
    E.5.1.1Timepoint(s) of evaluation of this end point
    every 8 weeks from week 8 till week104
    E.5.2Secondary end point(s)
    * Physician global assessment of auto-inflammatory disease activity ≤ minimal (using a 5-pointscale ranging from absent to severe)
    * assessment of skin disease ≤ minimal (using a 5-point scale ranging from absent to severe)
    * serological response: Serum C-reactive protein (CRP) < 10 mg/L.
    E.5.2.1Timepoint(s) of evaluation of this end point
    every 8 weeks from week 8 till week104
    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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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.8.2.4Number of treatment arms in the trial1
    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 No
    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
    • Study duration: 104 weeks with possible extension depending on clinical response and evolution
    • Evaluations: patients will be evaluated before dosing, at baseline and every 8 weeks up to week 56. EOS visit will occur 8 weeks after the last canakinumab dose.
    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
    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: 3
    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: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    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 Yes
    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
    subjects under age incapable to give consent personally
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    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)
    depending on the clinical response and evolution an option is taken to extend the trial
    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 Decision2014-04-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-04-09
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2014-12-31
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