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    Summary
    EudraCT Number:2019-004823-20
    Sponsor's Protocol Code Number:TAK-743-3001
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-12-21
    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 ConcernedFrance - ANSM
    A.2EudraCT number2019-004823-20
    A.3Full title of the trial
    An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of
    Lanadelumab for Prevention Against Acute Attacks of Non-histaminergic
    Angioedema with Normal C1-Inhibitor (C1-INH)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Open-Label Study of Long-Term Safety and Efficacy of Lanadelumab for
    Prevention of Acute Attacks of Non-histaminergic Angioedema with
    Normal C1-Inhibitor
    A.4.1Sponsor's protocol code numberTAK-743-3001
    A.5.4Other Identifiers
    Name:INDNumber:116647
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/476/2020
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorTakeda Development Center Americas (TDCA)
    B.1.3.4CountryUnited States
    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 supportTakeda
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTakeda Development Center Americas (TDCA)
    B.5.2Functional name of contact pointJeremy Chadwick
    B.5.3 Address:
    B.5.3.1Street Address95 Hayden Avenue,
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number17814829378
    B.5.6E-mailJeremy.chadwick@takeda.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 Takhzyro
    D.2.1.1.2Name of the Marketing Authorisation holderShire Pharmaceuticals Ireland, 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 Yes
    D.2.5.1Orphan drug designation numberEU/3/15/1551
    D.3 Description of the IMP
    D.3.1Product namelanadelumab
    D.3.2Product code TAK-743, SHP643
    D.3.4Pharmaceutical form Solution for injection in pre-filled syringe
    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 INNLANADELUMAB
    D.3.9.1CAS number 1426055-14-2
    D.3.9.2Current sponsor codeTAK743, SHP643
    D.3.9.3Other descriptive nameDX2930
    D.3.9.4EV Substance CodeSUB189058
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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
    non-histaminergic angioedema with normal C1-INH
    E.1.1.1Medical condition in easily understood language
    Angioedema is a long-term and life-threatening disease. It manifests clinically as unpredictable, intermittent attacks of edema of the face, larynx, gastrointestinal tract, limbs and/or genitalia.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level HLT
    E.1.2Classification code 10002425
    E.1.2Term Angioedemas
    E.1.2System Organ Class 100000004858
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary: To evaluate the long-term safety of repeated subcutaneous (SC) administrations of lanadelumab in adolescents and adults with non-histaminergic angioedema with normal C1-INH
    E.2.2Secondary objectives of the trial
    Secondary:
    - To evaluate the long-term efficacy of lanadelumab in preventing angioedema attacks
    - To characterize pharmacokinetics (PK) and pharmacodynamics (PD) following long-term SC administration of lanadelumab
    - To assess the immunogenicity of chronically administered lanadelumab
    - To evaluate the effect of lanadelumab on health-related quality of life (HR-QoL)
    - To evaluate subject experience of injection
    - To evaluate the safety and efficacy of lanadelumab in subjects switched to the dosing regimen of 300 mg every 4 weeks (q4wks) lanadelumab
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males and females, 12 years of age and older diagnosed with non-histaminergic normal C1-INH angioedema at the time of enrollment into the antecedent Study SHP643-303.
    2. Subjects must have completed the treatment period (through Day 182) of Study SHP643-303 without reporting a clinically significant treatment-emergent adverse event (TEAE) that would preclude subsequent exposure to lanadelumab.
    3. Agree to adhere to the protocol-defined schedule of treatments, assessments, and procedures.
    4. Males, or non-pregnant, non-lactating females who are of child-bearing potential and who agree to be abstinent or agree to comply with the applicable contraceptive requirements of this protocol for the duration of the study;
    or 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.
    5. The subject (or the subject’s parent/legal guardian, if applicable) has provided written informed consent approved by the institutional review board/research ethics board/ethics committee (IRB/REB/EC) at any time prior to study start. If the subject is a minor (ie, <18 years of age), have a parent/legal guardian who is informed of the nature of the study provide written informed consent (ie, permission) for the minor to participate in the study before any study-specific procedures are performed. Assent will be obtained from minor subjects.
    E.4Principal exclusion criteria
    1. Discontinued from Study SHP643-303 after enrollment but before Visit 26 for any reason.
    2. Presence of important safety concerns identified in Study SHP643-303 that would preclude participation in this study.
    3. Dosing with an investigational product (IP, not including IP defined in antecedent Study SHP643-303) or exposure to an investigational device within 4 weeks prior to Day 0.
    4. Subject has a known hypersensitivity to the investigational product or its components.
    5. Have any condition (surgical or medical) that, in the opinion of the investigator or sponsor, may compromise their safety or compliance, preclude the successful conduct of the study, or interfere with interpretation of the results (eg, significant pre-existing illness or other major comorbidities that the investigator considers may confound the interpretation of study results).
    E.5 End points
    E.5.1Primary end point(s)
    Safety measures, including:
    - Adverse events (AEs), including serious adverse events (SAEs) and adverse events of special interest (AESI)
    - Clinical laboratory testing (hematology, clinical chemistry, coagulation, and urinalysis)
    - Vitals signs including blood pressure, heart rate (HR), body temperature
    - Weight and height (height for subjects <18 years old)
    - 12-lead ECGs
    E.5.1.1Timepoint(s) of evaluation of this end point
    multiple timepoints as per Protocol's Schedule of Activities
    E.5.2Secondary end point(s)
    - Number of investigator-confirmed angioedema attacks during the treatment period
    - Number of moderate or severe angioedema attacks during the treatment period
    - Number of high-morbidity angioedema attacks during the treatment period; a high-morbidity angioedema attack is defined as any attack that has at least one of the following characteristics: severe, results in hospitalization (except hospitalization for observation <24 hours), hemodynamically significant (systolic blood pressure <90 mmHg, requires intravenous (IV) hydration, or associated with syncope or near-syncope) or laryngeal.
    - Analysis of pharmacokinetics (PK) effects through measurement of plasma concentrations of lanadelumab.
    - Evaluation of the pharmacodynamic (PD) effects of lanadelumab through cHMWK and fluorogenic plasma kallikrein (pKal) assay with FXIIa activation.
    - Presence of anti-drug antibodies (ADAs), including evaluation of neutralizing antibodies (if any confirmed positive anti-drug antibodies are detected)
    - Health-related quality of life assessments will be assessed using the AE-QoL questionnaire.
    - Lanadelumab Injection Report

    Safety measures, including:
    - AEs, including SAEs and AESIs
    - Clinical laboratory testing (hematology, clinical chemistry, coagulation, and urinalysis)
    - Vitals signs including blood pressure (BP), HR, body temperature
    - Weight and height (height for subjects <18 years old)
    - 12-lead ECGs

    Efficacy measures, including:
    - Number of investigator-confirmed angioedema attacks during the treatment period
    - Number of moderate or severe angioedema attacks during the treatment period
    - Number of high-morbidity angioedema attacks during the treatment period; a high-morbidity angioedema attack is defined as any attack that has at least one of the following characteristics: severe, results in hospitalization (except hospitalization for observation <24 hours), hemodynamically significant (systolic blood pressure <90 mmHg, requires IV hydration, or associated with syncope or near-syncope) or laryngeal.
    E.5.2.1Timepoint(s) of evaluation of this end point
    multiple timepoints as per Protocol's Schedule of Activities
    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 Yes
    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) 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 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 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA32
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    Germany
    Hungary
    Italy
    Japan
    Netherlands
    Poland
    Spain
    United States
    France
    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 years1
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months4
    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: 2
    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: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 70
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 38
    F.4.2.2In the whole clinical trial 75
    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)
    No aftercare is planned for this study.
    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 Decision2022-01-25
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-05-05
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