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    Summary
    EudraCT Number:2019-001235-31
    Sponsor's Protocol Code Number:DEALSZ-2019-001
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-11-11
    Trial 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 number2019-001235-31
    A.3Full title of the trial
    Factor XII-associated cold autoinflammatory syndrome (FACAS) linked to
    kallikrein-kinin pathology: Proof of concept treatment with Lanadelumab
    (DX-2930)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Lanadelumab in FACAS
    A.3.2Name or abbreviated title of the trial where available
    LANA-FXII
    A.4.1Sponsor's protocol code numberDEALSZ-2019-001
    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
    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 support Takeda Pharma Vertrieb GmbH & Co. KG
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationInstitute of Allergology
    B.5.2Functional name of contact pointProf. Marcus Maurer
    B.5.3 Address:
    B.5.3.1Street AddressHindenburgdamm 30
    B.5.3.2Town/ cityBerlin
    B.5.3.3Post code12203
    B.5.3.4CountryGermany
    B.5.4Telephone number004930450518043
    B.5.5Fax number0049304507518972
    B.5.6E-mailmarcus.maurer@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 Takhzyro® 300 mg Injektionslösung
    D.2.1.1.2Name of the Marketing Authorisation holderTakeda Pharma Vertrieb GmbH & Co. KG
    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 nameLanadelumab
    D.3.2Product code na
    D.3.4Pharmaceutical form Solution for injection/infusion in pre-filled syringe
    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 No
    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
    Lanadelumab in patients with Factor XII-associated cold autoinflammatory syndrome (FACAS)
    E.1.1.1Medical condition in easily understood language
    FACAS
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10072220
    E.1.2Term Autoinflammatory disease
    E.1.2System Organ Class 10021428 - Immune system 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 Lanadelumab on the clinical signs and symptoms of FXII-associated cold autoinflammatory syndrome (reduction in symptomatic days with urticarial rash, arthralgia, headache, chills)
    E.2.2Secondary objectives of the trial
    • To assess the effect of Lanadelumab on inflammation markers (CRP, ESR, SAA, S100A8/9)
    • To assess the effect of Lanadelumab on the patients' quality of life
    • To assess the safety and tolerability following administration of Lanadelumab
    • To assess the effect of Lanadelumab on the kallikrein-kinin pathway and cytokine release (prekallikrein levels, cHMWK levels, IL-1ß release)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Adolescents (12 - 17 years old) and adults (18 years or older)
    • Documented FXII-associated autoinflammatory disorder (FACAS) by positive genetic analysis result
    • Clinical symptoms of cold-associated wheals, arthralgia, headache, fatigue
    • Able to read, understand and willing to sign the informed consent form and abide with study procedures
    Males and females who are fertile and sexually active must adhere to contraception requirements for the duration of the study as follows:
    • Females of childbearing potential must agree to be abstinent or else use one of the following medically acceptable forms of contraception from the screening period through 30 days after the final study visit: progestin-only oral contraceptive, intrauterine device, progestin depot injection or progestin implant. A female whose male partner has had a vasectomy must agree to use one additional form of highly effective contraception (Pearl-Index <1).
    • Females of non-childbearing potential, defined as surgically sterile (status post hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) or post-menopausal for at least 12 months do not require contraception during the study.
    • Males, including males who are surgically sterile (post vasectomy), with female partner(s) of childbearing potential must agree to be abstinent or else the female partner(s) have to use a highly effective form of contraception (Pearl-Index <1) from the screening period through 60 days after the final study visit.
    E.4Principal exclusion criteria
    • Any other forms of urticaria or angioedema not related to genetic mutations within the FXII gene
    • Concurrent/ongoing treatment with biologics or recent treatment (less than 5 half-lives)
    • With anakinra within 7 days prior to screening, with canakinumab within 100 days prior to screening
    • with oral/parental corticosteriods greater than 10 mg/d within 2 weeks prior to screening
    o other immunosuppressives within 4 weeks or 5 half-lives prior to screening, whichever is longer
    • Treatment with a live (attenuated) virus vaccine within 4 weeks prior to Baseline visit
    • Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral
    contraceptives or hormonal replacement therapy) within 4 weeks prior to screening
    • Use of prophylactic therapy with C1-INH, attenuated androgens, or antifibrinolytics within 2 weeks prior to the start of the treatment period
    (Day 0)
    • Any of the following liver function test abnormalities:
    o alanine aminotransferase (ALT) > 3x upper limit of normal, or
    o aspartate aminotransferase (AST) > 3x upper limit of normal, or
    o total bilirubin > 2x upper limit of normal (unless the bilirubin
    elevation is a result of Gilbert's Syndrome).
    • Pregnancy or breastfeeding.
    • Subject has any condition that, in the opinion of the Investigator or Sponsor, may compromise their safety or compliance, preclude
    successful conduct of the study, or interfere with interpretation of the results (e.g., history of substance abuse or dependence, a significant
    pre-existing illness or other major comorbidity that the Investigator considers may confound the interpretation of study results).
    • Significant medical condition rendering the patient immuno-compromised or not suitable for a clinical trial
    • 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
    • Patients with known hypersensitivity to any constituent of the products of lanadelumab
    • Dementia, altered mental status, or any psychiatric condition, or stay in an institution further to an official or court order that would prohibit
    the understanding or rendering of informed consent or participation in the study.
    • Subjects who are study site employees, or immediate family members of a study site or sponsor employee.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of the study is the change in patient-reported disease activity total score (mean of weeks 9 to 12 compared to mean of weeks -4 to -1 (baseline)).
    Patient-reported disease activity is assessed by a daily health assessment form (FACAS-DHAF) evaluating the severity of 5 key symptoms of FACAS: urticarial rash, fatigue, chills/fever, arthralgia and headache. Patients are asked to grade the severity of each symptom daily on a numeric rating scale (NRS) ranging from 0 (very good) to 10 (very bad). The daily total score is defined as the mean of the 5 individual symptom scores. The total score at baseline is defined as the mean of daily total scores from week -4 to -1.
    The total score at weeks 9 to 12 is defined as the mean of daily total scores from week 9 to 12. The primary endpoint is the difference of these two scores (total score at weeks 9 to 12 minus total score at baseline).
    The mean score over 4 weeks will be calculated if at least 70% of the daily patient-reported assessments are available (e.g. if at least 20 out of 28 diary values are available). Otherwise, the mean score over 4 weeks will be regarded as missing.
    E.5.1.1Timepoint(s) of evaluation of this end point
    weeks 9 to 12 compared to weeks -4 to -1 (baseline)
    E.5.2Secondary end point(s)
    • Change in PR-DA urticarial rash score at weeks 9 to 12 and weeks 24 to 28, each compared to weeks -4 to -1 (baseline).
    • Change in PR-DA fatigue score at weeks 9 to 12 and weeks 24 to 28, each compared to weeks -4 to -1 (baseline).
    • Change in PR-DA chills/ fever score at weeks 9 to 12 and weeks 24 to 28, each compared to weeks -4 to -1 (baseline).
    • Change in PR-DA arthralgia score at weeks 9 to 12 and weeks 24 to 28, each compared to weeks -4 to -1 (baseline).
    • Change in PR-DA headache score at weeks 9 to 12 and weeks 24 to 28, each compared to weeks -4 to -1 (baseline).
    • Change in PR-DA total score at weeks 24 to 28 compared to weeks -4 to -1 (baseline).
    • Change in inflammation markers CRP, ESR, SAA, S100 A8/9 (from Baseline to week 12 and week 28).
    • Change in disease-specific quality-of-life assessed by Dermatology Life Quality Index (DLQI) over the study (from Baseline to week 12 and week 28).
    • Changes in generic quality-of-life assessed by 36-Item Short Form Health Survey (SF-36) (from Baseline to week 12 and week 28).
    • Safety: physical examination, routine safety laboratory assessments, vital signs, and adverse events.
    • Change in physician global assessment as assessed by verbal rating scale from 0-10 (0=no symptoms; 10=very severe symptoms) over the study (from Baseline to week 12 and week 28).
    • Changes from Baseline to week 28 of plasma levels of potential biomarkers in Lanadelumab-treated patients (e.g. FXII, prekallikrein and cHMWK levels, IL-1ß release from donor PBMCs).
    E.5.2.1Timepoint(s) of evaluation of this end point
    from Baseline to week 12 and week 28
    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 No
    E.6.5Efficacy No
    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 Yes
    E.8.1.7.1Other trial design description
    exploratory, proof-of-concept study
    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 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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    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: 1
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 3
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state4
    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)
    none
    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 Decision2019-11-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-11-25
    P. End of Trial
    P.End of Trial StatusOngoing
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