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    Summary
    EudraCT Number:2013-002319-82
    Sponsor's Protocol Code Number:BCX4161-203
    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:2013-11-20
    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 number2013-002319-82
    A.3Full title of the trial
    A Phase 2a double-blind placebo-controlled 2-period crossover study to evaluate the safety and efficacy of BCX4161 as a prophylactic treatment to reduce the frequency of attacks in subjects with hereditary angioedema
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An early clinical trial of BCX4161 in patients with hereditary angioedema compared to treatment with placebo.
    A.4.1Sponsor's protocol code numberBCX4161-203
    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 SponsorBioCryst Pharmaceuticals Inc.
    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 supportBioCryst Pharmaceuticals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAMS Advanced Medical Services
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address500 Chiswick High Road
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeW4 5RG
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number440208956 2606
    B.5.5Fax number440208956 2358
    B.5.6E-maillisa.frenz@ams-europe.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 No
    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 nameBCX4161
    D.3.2Product code BCX4161
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBCX4161
    D.3.9.2Current sponsor codeBCX4161
    D.3.9.3Other descriptive nameBCX4161
    D.3.9.4EV Substance CodeSUB122648
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Hereditary Angioedema
    E.1.1.1Medical condition in easily understood language
    Hereditary Angioedema
    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 16.1
    E.1.2Level PT
    E.1.2Classification code 10019860
    E.1.2Term Hereditary angioedema
    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 the effect of prophylactic BCX4161 as measured by the frequency of attacks in subjects with hereditary angioedema (HAE)
    E.2.2Secondary objectives of the trial
    To evaluate the safety and tolerability of prophylactic BCX4161 in subjects with HAE type 1 or 2
    To describe the pharmacokinetics of BCX4161 in subjects with HAE type 1 or 2
    To characterize the pharmacodynamic effects of BCX4161in subjects with HAE
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males and non-pregnant, non-lactating females of age 18-65 years
    2. A clinical diagnosis of hereditary angioedema as documented at any time in the medical records or at screening by a low C4 level AND:
    a. A low C1INH antigenic level OR
    b. A normal or increased C1INH antigenic level and a low C1INH functional level
    3. Documentation of an average of 1 attack per week over at least a 3 month period within the past year
    4. Body mass index (BMI) of 19-36 kg/m2
    5. Female participants must meet one of the following requirements:
    a. A woman of childbearing potential (defined as a nonmenopausal female who has not had a hysterectomy, bilateral oophorectomy, or documented ovarian failure) who agrees to use 2 highly effective contraceptive methods for the study duration and for 30 days after study drug dosing. Two or more of the following methods are acceptable and must include at least one barrier method: Surgical sterilization (i.e. bilateral tubal ligation), placement of an intrauterine device or intrauterine system, hormonal contraception (implantable, patch, oral, vaginal ring), barrier methods (either their partners use of a condom or the subjects use of an occlusive cap [diaphragm, or cervical/vault caps] with spermicidal foam/gel/film/cream/suppository). Estrogen-and progestin containing hormonal contraception is only permitted if it has been used for at least 60 days prior to screening (IUDs with progestin are permitted to be placed at any time prior to or during screening). Female subjects who report being postmenopausal for ≤ 2 years and have a screening follicle stimulating hormone (FSH) ≤ 40 mIU/mL must agree to use 2 highly effective contraceptive methods for the study period and for 30 days after the last dose of study drug (as defined above).
    b. A woman of nonchildbearing potential (defined as postmenopausal for > 2 years or a screening FSH > 40 mIU/mL if postmenopausal for ≤ 2 years or have had a hysterectomy, bilateral oophorectomy, or documented ovarian failure)
    6. Male participants must comply with the following requirements:
    a. Must agree to utilize a highly effective contraceptive method with female partners of childbearing potential (defined as postmenopausal ≤ 2 years or a nonmenopausal female who has not had a hysterectomy, bilateral oophorectomy, or documented ovarian failure), where “highly effective contraceptive method” is defined as having had a vasectomy, or utilizing 2 forms of contraception, 1 of which must be a condom, during intercourse, for the study duration and for 90 days after the last dose of the study drug.
    b. Must abstain from sperm donation for the study period and for 90 days after the last dose of study drug
    7. Any concomitant medication at screening must be anticipated to be continued through the study and be of a stable dose and regimen for the duration of the study
    8. Written informed consent
    E.4Principal exclusion criteria
    1. Age < 18 or > 65 years
    2. Females who are pregnant (positive urine or serum pregnancy test) or breast feeding at screening or who are planning to become pregnant during the study period
    3. Any clinically significant medical condition or medical history that, in the opinion of the Investigator or Sponsor, would interfere with the subject’s safety or ability to participate in the study
    4. 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
    5. Use of an estrogen-or porgestin containing hormonal contraceptive that was initiated less than 60 days prior to screening. IUDs containing progestin can be placed at any time prior to or during the screening period.
    6. Use within the 7 days prior to screening or planned use through the study of C1 inhibitor (C1 INH) or tranexamic acid for prophylaxis of HAE attacks. Use of a C1INH therapy for treatment of acute attacks is not excluded
    7. Use within the 30 days prior to screening or planned use through the study of anabolic steroids for prophylaxis of HAE attacks
    8. Concurrent daily use of anticoagulants, antiplatelet drugs (with the exception of low-dose aspirin for cardioprotection) or NSAIDS from screening through the end of the study
    9. Abnormal ECG (defined as any screening or baseline QTcF > 470 msec, PR > 220msec, or ventricular and/or atrial premature contractions that are more frequent than occasional, and/or as couplets or higher in grouping)
    10. Family history of sudden death to causes other than HAE
    11. History of QT prolongation
    12. Any abnormal laboratory or urinalysis finding at screening that, in the opinion of the Investigator or Sponsor, is clinically significant and relevant for this study
    13. Prolonged activated partial thromboplastin time (aPTT) or prothrombin time (PT) at screening (defined as any screening or baseline Grade 1 or greater toxicity)
    14. Current participation in any other investigational drug study or within the past 30 days of screening
    15. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse (self-reported alcoholic intake > 3 units alcohol/day)
    16. Positive for hepatitis B surface antigen (HBV), hepatitis C antibody (HCV), or human immunodeficiency virus (HIV) type 1
    E.5 End points
    E.5.1Primary end point(s)
    Number of acute attacks (independently adjudicated)
    Symptoms and location of any attack
    Severity of any attack (using the Angioedema Activity Score- AAS)
    Treatment used to manage any attack
    E.5.1.1Timepoint(s) of evaluation of this end point
    At the end of each 28 day treatment period - ie Day 29 and Day 64
    E.5.2Secondary end point(s)
    AEs, laboratory analyses (clinical chemistry, hematology and urinalysis), vital signs, ECGs and physical examinations
    Pharmacokinetic endpoints
    kallikrein inhibition by BCX4161
    Prothrombin time, activated partial prothrombin time
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the treatment periods, and seven days post final dose
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    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 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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA10
    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 years
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months8
    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: 25
    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 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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 25
    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 remain under the care of their physician and receive the standard care treatment regime for that institution.
    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 Decision2013-12-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-02-03
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-05-13
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