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    Summary
    EudraCT Number:2016-003828-23
    Sponsor's Protocol Code Number:ML39310
    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:2017-01-31
    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 number2016-003828-23
    A.3Full title of the trial
    A pilot open-label study to assess the efficacy and safety of tocilizumab (TCZ) in patients with active Schnitzler’s syndrome (SchS)

    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A pilot open-label study to assess the efficacy and safety of tocilizumab (TCZ) in patients with active Schnitzler’s syndrome (SchS)

    A.4.1Sponsor's protocol code numberML39310
    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 SponsorCharité - Universitätsmedizin Berlin; Dpt. of Dermatology and Allergy
    B.1.3.4CountryGermany
    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 supportROCHE/CHUGAI Pharma AG
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCharité - Universitätsmedizin Berlin; Dpt. of Dermatology and Allergy
    B.5.2Functional name of contact pointKaroline Krause
    B.5.3 Address:
    B.5.3.1Street AddressCharitèplatz 1
    B.5.3.2Town/ cityBerlin
    B.5.3.3Post code10117
    B.5.3.4CountryGermany
    B.5.4Telephone number004930450518336
    B.5.5Fax number004930450518938
    B.5.6E-mailkaroline.krause@charite.de
    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 RoActemra
    D.2.1.1.2Name of the Marketing Authorisation holderRoche
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 nameRoActemra
    D.3.2Product code Ro 487-7533/F10
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    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
    Schnitzler’s syndrome (SchS)
    E.1.1.1Medical condition in easily understood language
    Schnitzler’s syndromes characterized by chronic urticarial skin lesions associated with fever attacks, bone and muscle pain, arthralgia or arthritis, fatigue and lymphadenopathy.
    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 19.1
    E.1.2Level PT
    E.1.2Classification code 10062908
    E.1.2Term Schnitzler's syndrome
    E.1.2System Organ Class 10040785 - Skin and subcutaneous tissue 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 assess the effect of TCZ on the clinical signs and symptoms of SchS
    E.2.2Secondary objectives of the trial
    To assess the effect of TCZ on inflammation markers (CRP, ESR, SAA, S100A12)
    To assess the effect of TCZ on the patient’s quality of life
    To assess the safety and tolerability following administration of TCZ to patients with SchS
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Adults (18 years or older)
    SchS diagnosis based on Strasbourg clinical criteria
    Active SchS, refractory to treatment with antihistamines, NSAIDS or colchicine, hydroxychloroquine or dapsone
    Patients who have a symptom score (PGA) of at least 8 (0-20) at baseline
    If necessary, concurrent/ongoing treatment with a stable dose of systemic corticosteroids not greater than 10mg/d for 14 days prior to screening
    If necessary, concurrent/ongoing treatment with a stable dose of antihistamines and NSAIDs for 7 days prior to screening
    Able to read, understand and willing to sign the informed consent form and abide with study procedures
    Willing, committed and able to return for all clinic visits and complete all study-related procedures, including willingness to have SC injections administered by a qualified person
    In females of childbearing potential: Negative pregnancy test within 28 days of randomization; males and females willing to use highly effective contraception (Pearl-Index < 1) during study treatment and for a minimum of 3 months after last dose of TCZ. Pregnancies occurring up to 90 days after the completion of the study medication must be reported to the investigator. A woman will be considered not of childbearing potential if she is post-menopausal for greater than two years or surgically sterile (bilateral tubal ligation, bilateral oophorectomy or hysterectomy)
    Subjects are considered eligible, if they meet the following tuberculosis (TB) screening criteria: no history of latent or active TB prior to screening, no signs or symptoms suggestive of active TB, no recent close contacts with a person with active TB, and negative QuantiFERON-TB test at screening (if QuantiFERON-TB test is positive, the patient can only be included if active TB is ruled out with appropriate measurements according to standard of care, e.g. the patient is pre-treated with isoniazide for 4 weeks).
    No participation in other clinical trials 4 weeks before and after participation in this study
    E.4Principal exclusion criteria
    Major surgery (including joint surgery) within 8 weeks prior to screening or planned major surgery within 6 months following randomization.
    Concurrent/ongoing treatment with biologics or recent treatment (less than 5 half lives)
    o With Anakinra within 7 days prior to screening, with canakinumab within 100 days prior to screening
    o with oral/parental corticosteriods greater than 10 mg/d within 2 weeks prior to screening
    o with Cyclosporin A Methotrexate, Dapsone, Chloroquine, Hydroxychloroquine, Azathioprine, Cyclophosphamide within 4 weeks prior to screening
    o other immunosuppressives within 4 weeks or 5 half lives prior to screening, whichever is longer
    o Previous treatment within six months of randomization with any cell-depleting therapies, including investigational agents or approved therapies, some examples include: CAMPATH, anti-CD4, anti-CD5, anti-CD3, anti-CD19 and anti-CD20.
    o Treatment with intravenous gamma globulin, plasmapheresis or Prosorba column within 6 months of baseline.
    Treatment with a live (attenuated) virus vaccine within 4 weeks prior to Baseline visit
    Significant medical condition rendering the patient immunocompromised or not suitable for a clinical trial
    Any previous treatment with alkylating agents such as chlorambucil, or with total lymphoid irradiation.
    History of severe allergic or anaphylactic reactions to human, humanized or murine monoclonal antibodies.
    An abnormal chest radiograph consistent with clinical signs of prior or present tuberculosis infection whether or not previously treated with anti-tuberculosis agents
    Significant concomitant illness such as, but not limited to, cardiac, renal, neurological, endocrinological, metabolic, or lymphatic disease that would adversely affect the subject’s participation or evaluation in this study
    Evidence of current HIV, Hepatitis B, or Hepatitis C infection by clinical or serological history
    Presence of any of the following laboratory abnormalities at enrollment visit: serum creatinine > 1.6 mg/dL (141 μmol/L) in female patients and > 1.9 mg/dL (168 μmol/L) in male patients, WBC <3,000/μl; platelet count <100000/μl ; ALT or AST >2 x ULN or total bilirubin >ULN, Hemoglobin <8.0 g/dL, neutrophil count <2,000 cells/μl or lymphocyte count <500/ μl
    Evidence of active, recurrent or latent systemic infection
    Active systemic inflammatory condition other than SchS including, but not limited to, rheumatoid arthritis
    History of malignancies within five years prior to screening other than a successfully treated non-metastatic cutaneous, basal, or squamous cell carcinoma and/or in situ cervical cancer
    Lactating females or pregnant females
    Enrollment in another investigational treatment or device study or use of an investigational agent, or less than 4 weeks or 5 half-lives, whichever is longer, since end of another investigational device or drug trial
    Subjects for whom there is concern about compliance with the protocol procedures
    Any medical condition which, in the opinion of the Investigator, would interfere with participation in the study or place the subject at risk
    History of substance abuse (drug or alcohol) or any other factor (e.g., serious psychiatric condition) that could limit the subject’s ability to comply with study procedures.
    Patients with known hypersensitivity to tocilizumab
    E.5 End points
    E.5.1Primary end point(s)
    Change in the investigator’s assessment of total disease activity (PGA) between baseline and TCZ treatment
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 20
    E.5.2Secondary end point(s)
    Proportion of patients with complete response (based on physician’s global assessment with no or minimal overall autoinflammatory disease activity and CRP </= 10 mg/l) at week 20
    Change in the patient based Schnitzler Activity Score (SchAS) during the treatment period (The SchAS combines the key symptoms of SchS).
    Change in inflammation markers (CRP, ESR, SAA, S100A12)
    Change in the patient’s quality of life (assessed by the Dermatology Life Quality Index and SF-36)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 20
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 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 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.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months6
    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 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: 8
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 No
    F.3.3.7Others Information not present in EudraCT
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    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)
    Patients will be treated in the autoinflammation reference center.
    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 Decision2017-04-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-02-28
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-04-10
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