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    Summary
    EudraCT Number:2014-002655-26
    Sponsor's Protocol Code Number:BCX4161-301
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-12-03
    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 ConcernedGermany - BfArM
    A.2EudraCT number2014-002655-26
    A.3Full title of the trial
    OPuS-2 A multicentre, randomised, double blind, placebo controlled, parallel group study to evaluate the efficacy and safety of two dose levels of BCX4161 for 12 weeks as an oral prophylaxis treatment for attacks of hereditary angioedema.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    OPuS2 - A study to assess two doses of BCX4161 in the prevention of HAE attacks in patients over a 12 week period
    A.3.2Name or abbreviated title of the trial where available
    OPuS 2
    A.4.1Sponsor's protocol code numberBCX4161-301
    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 Ltd
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address1 Lyric Square
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeW6 0NB
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+440203751 1350
    B.5.6E-mailregulatoryaffairs@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, soft
    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, soft
    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] - 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 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 determine the efficacy of prophylactic BCX4161 300 mg and 500 mg administered three times daily for 12 weeks compared to placebo
    E.2.2Secondary objectives of the trial
    To assess the safety and tolerability of BCX4161 at 300 mg and 500 mg administered three times daily for 12 weeks
    To assess effects of BCX4161 on HAE disease activity and HAE attack characteristics
    To evaluate the effects of BCX4161 on quality of life
    To describe the pharmacokinetics of BCX4161
    To characterize the pharmacodynamic effects of BCX4161
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Opus 2 - PK, Amylase and Lipase Sub Study
    Objective - To obtain concentrations enabling non-compartmental and population pharmacokinetic analyses and to characterize the time course of amylase and lipase levels following study drug dosing after single and multiple doses.
    E.3Principal inclusion criteria
    1. Males and non-pregnant, non-lactating females age ≥18 years.
    2. Body mass index (BMI) of 19-36 kg/m2.
    3. A clinical diagnosis of hereditary angioedema Type 1 or Type 2 as documented by:
    a. A low C1 INH antigenic level OR
    b. A normal C1 INH antigenic level and a low C1 INH functional level
    4. Female participants must meet one of the following requirements:
    • A woman of childbearing potential (defined as a non-menopausal female who has not had a hysterectomy, bilateral oophorectomy, or documented ovarian failure) who agrees to use a highly effective method of contraception for the study duration and for 30 days after study drug dosing.
    Female subjects who report being postmenopausal for ≤ 2 years and have a screening follicle stimulating hormone (FSH) ≤ 40 mIU/mL must agree to use a highly effective contraceptive method
    • A woman of non-childbearing potential (defined as being postmenopausal for > 2 years, having a screening FSH > 40 mIU/mL if postmenopausal for ≤ 2 years, or a woman who has had a hysterectomy, bilateral oophorectomy, or documented ovarian failure).
    • A woman declaring herself as either sexually abstinent or exclusively having female sexual partners.
    5. Male participants must comply with the following requirements:
    • Must agree to utilize one 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) through Week 12 and for 90 days after the last dose of study drug.
    • Must abstain from sperm donation through Week 12 and for 90 days after the last dose of study drug.
    6. Any concomitant medication at screening must be anticipated to be continued through the entire study and be of a stable dose and regimen for the duration of the entire study, including oral androgens prescribed for the prevention of HAE attacks.
    7. Access to and ability to use one or more acute medications approved by the relevant competent authority for the treatment of acute attacks of HAE. Cinryze used for acute treatment of HAE attacks is an acceptable medication for this purpose.
    8. Written informed consent.
    9. A documented HAE attack rate established by one of the following criteria:
    a. Previous documentation of pre-determined HAE attacks per month for 3 consecutive months (93 days) within the 6 months prior to screening.
    For subjects who have received effective prophylaxis, there must be documentation of a pre-determined attack rate per month for 3 consecutive months (93 days) within the 12 months prior to screening.
    A documented attack rate cannot be used for subjects who have received effective prophylaxis for more than 9 months immediately prior to screening.
    b. A subject diary record of unique HAE attacks collected in a run-in period of a maximum of 2 months (62 days).
    10. In the opinion of the Investigator, the subject is expected to adequately comply with all required study procedures for the duration of the study.
    E.4Principal exclusion criteria
    1. Females who are pregnant (positive urine or serum pregnancy test) or breast feeding at the screening visit or who are planning to become pregnant during the study.
    2. 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. For example, subjects with chronic renal failure, decompensated liver disease, or chronic heart failure would not be eligible under this criterion.
    3. 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.
    4. Use of an estrogen- and/or progestin-containing hormonal contraceptive within 60 days prior of the screening visit; or expected initiation at any time through the end of the study (follow up). IUDs containing progestin can be placed at any time prior to the screening visit.
    5. Use within the 7 days prior to the screening visit or expected use at any time through the end of the study of C1 INH or tranexamic acid for prophylaxis of HAE attacks. Use of a C1INH therapy for treatment of acute attacks is not excluded.
    6. Use within the 30 days prior to the screening visit or expected use at any time through the end of the study of androgens for prophylaxis of HAE attacks, except for subjects who meet the required attack frequency inclusion criterion while currently being treated with androgens for prophylaxis. Such subjects must have been on a stable dose of androgens for the 90 days prior to the screening visit and agree to remain on their current dose of androgens for the duration of the study.
    7. Prior enrollment in OPuS-1.
    8. INR > 1.3 x ULN.
    9. Creatinine clearance (CLcr) less than 60 mL/min.
    10. Concurrent use of angiotensin converting enzyme inhibitors from the screening visit or expected use at any time through the end of the study.
    11. Current or past medical history of acute pancreatitis, chronic pancreatitis, or pancreatic insufficiency.
    12. Clinically significant abnormal ECG as assessed by the Investigator.
    13. Any abnormal laboratory or urinalysis finding at the screening visit that, in the opinion of the Investigator or Sponsor, is clinically significant and relevant for this study.
    14. Current participation in any other investigational drug study or within 30 days prior to the screening visit.
    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 drugs of abuse screen (unless as used as medical treatment, e.g., with a prescription).
    17. Positive for hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV).
    E.5 End points
    E.5.1Primary end point(s)
    The mean acute angioedema attack rate in each active treatment group and placebo
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of Treatment - 12 weeks
    E.5.2Secondary end point(s)
    • Number of attack-free days
    • Number of subjects who are attack-free
    • Disease activity, as measured by the AAS84 (AAS over 84 days)
    • Quality of Life, as measured by the AE-QoL and EQ-5D-5L
    • Acute HAE attack medication administrations
    • Discontinuations due to lack of efficacy
    E.5.2.1Timepoint(s) of evaluation of this end point
    End of Treatment - 12 weeks
    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 Yes
    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) Yes
    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 Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    Switzerland
    United States
    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 years1
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    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: 84
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 12
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 35
    F.4.2.2In the whole clinical trial 96
    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)
    Subjects will return to their normal standard of care under their physician. All subjects who tolerated active treatment and completed the study will be eligible for a planned open label treatment protocol to be initiated early 2016.
    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 Decision2015-02-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-02-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-01-08
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