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    Summary
    EudraCT Number:2019-001693-28
    Sponsor's Protocol Code Number:CONE-01
    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:2019-09-02
    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 number2019-001693-28
    A.3Full title of the trial
    Prospective, open-label, single arm, multicenter, pharmacokinetic, and safety study of a single dose intravenous human plasma-derived C1 Esterase Inhibitor (C1-INH) concentrate in patients with congenital C1-INH deficiency and hereditary angioedema
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Single-arm pharmacokinetic phase 2a study of a single dose intravenous human plasma-derived C1-INH involving 20 HAE type I or type II patients, aged 18 years or older
    A.3.2Name or abbreviated title of the trial where available
    CONE-01
    A.4.1Sponsor's protocol code numberCONE-01
    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 SponsorOctapharma Pharmazeutika Produktionsges.m.b.H.
    B.1.3.4CountryAustria
    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 supportOctapharma Pharmazeutika Produktionsges.m.b.H.
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOctapharma Pharmazeutika Produktionsges.m.b.H.
    B.5.2Functional name of contact pointGlobal Clinical Project Manager
    B.5.3 Address:
    B.5.3.1Street AddressOberlaaer Straße 235
    B.5.3.2Town/ cityVienna
    B.5.3.3Post code1100
    B.5.3.4CountryAustria
    B.5.4Telephone number+43(0)1 610 321315
    B.5.5Fax number+43(0)1 610 329249
    B.5.6E-mailvera.buerger@octapharma.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 nameC1 Esterase Inhibitor Human
    D.3.2Product code OCTA-C1-INH
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeOCTA-C1-INH
    D.3.9.3Other descriptive nameC1 ESTERASE INHIBITOR (HUMAN)
    D.3.9.4EV Substance CodeSUB39564
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200 IU/mL
    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) 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 Yes
    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
    Hereditary angioedema type I and type II
    E.1.1.1Medical condition in easily understood language
    Hereditary angioedema (HAE)
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 23.1
    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.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10080956
    E.1.2Term Hereditary angioedema type I
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10080960
    E.1.2Term Hereditary angioedema type II
    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
    The primary objective of this study is to assess the pharmacokinetic (PK) characteristics of OCTA-C1-INH after a single intravenous (IV) administration in HAE patients who are not experiencing an HAE attack.
    E.2.2Secondary objectives of the trial
    • To assess blood level changes of C1-INH antigen and C4 level after a single IV administration of OCTA-C1-INH.
    • To assess the safety of OCTA-C1-INH IV administration.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Documented congenital C1-INH deficiency with C1-INH functional activity less than 50% and C4 level below the laboratory reference range.
    2. Age ≥18 years at informed consent date.
    3. Signed informed consent.
    4. Patient must be capable to understand and comply with the relevant aspects of the study protocol.
    5. Women of childbearing potential must have a negative pregnancy test at screening as well as pre-infusion and must agree to use acceptable methods of contraception from screening until final visit.
    6. Fertile male patients must agree to use acceptable methods of contraception from screening until final visit.
    E.4Principal exclusion criteria
    1. Any signs of an HAE attack OR HAE attack within 7 days prior to dosing with the IMP (OCTA-C1-INH) OR more than a total of 9 HAE attacks over the previous 3 months prior to dosing with the IMP.
    2. Patients who have received prophylactic or acute treatment with C1-INH (Berinert®, Cinryze®, HAEgarda®, Ruconest®, etc.), non-biological bradykinin pathway inhibitors (e.g., ecallantide, icatibant), or treatment with tranexamic acid within 2 weeks prior to dosing with the IMP.
    3. Patients who have received treatment with lanadelumab within 11 weeks prior to dosing with the IMP.
    4. Patients with planned dental, medical, or surgical procedures who will need pre-procedural HAE prophylaxis during the study period.
    5. Female patients taking estrogen-containing contraceptive regimen, hormone replacement therapy (excepting progesterone-only contraceptives, which are permitted), or selective estrogen receptor modulators (e.g., tamoxifen). Any patients on specific androgen therapy (e.g., testosterone, danazol, dehydroepiandrosterone/androstenedione).
    6. Any change (start, stop, or change in dose) in androgen therapy (e.g., oxandrolone, stanozolol) in the last 14 days prior to dosing with the IMP.
    7. Participated in any other investigational drug evaluation or received blood or a blood product, except for C1-INH, within 30 days prior to dosing with the IMP.
    8. Live viral vaccination within 30 days prior to screening.
    9. Acute infectious illness characterized by rapid onset of disease, a relatively brief period of symptoms, and resolution within a short period of time.
    10. Risk factors for thromboembolic events, including presence of indwelling venous catheter or access device, history of thrombosis, underlying atherosclerosis, morbid obesity (defined as BMI of ≥35 kg/m2 and experiencing obesity-related health conditions or ≥40 to 44.9 kg/m2), immobility, or medications known to increase thromboembolic risk.
    11. History of allergic reaction to C1-INH products or other blood products.
    12. History of clinically relevant antibody development against C1-INH.
    13. Any history of B-cell malignancy that was unresolved in the past 5 years.
    14. Pregnancy or lactation.
    15. Any clinically significant medical or psychiatric condition that, in the opinion of the Investigator, would interfere with the patient’s ability to participate in the study.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the PK parameters of OCTA-C1-INH, measured as C1-INH activity.
    The PK parameters that will be assessed for OCTA-C1-INH include:
    • Blood concentrations at each sampling time
    • Maximum blood concentration (Cmax)
    • Time to maximum concentration (Tmax)
    • Clearance (CL)
    • Area under the concentration-time curve (AUC)
    • AUC normalized by the dose (AUCnorm)
    • Mean residence time (MRT)
    • Incremental recovery (IR)
    • Volume of distribution (Vd)
    • Elimination half-life (T1/2)
    E.5.1.1Timepoint(s) of evaluation of this end point
    PK sampling (pre-injection, post-injection: at 0 and 15 minutes, 1 hour, 2, 6, 12, 24, 48, 72, 120, 144, and 168 hours)
    E.5.2Secondary end point(s)
    • PK parameters of the following analytes:
    - C1-INH antigen
    - C4 level
    All PK parameters determined for the primary PK endpoint will also be determined for the secondary PK endpoints.
    • Safety Parameters:
    - Number and severity of adverse events of special interest (AESIs) of thromboembolic event (TEE) type
    - Change in vital signs from pre- to post-injection
    - Change in laboratory parameters from pre- to post-injection
    - Serology testing, blood nuclear antigen tests for hepatitis A virus [HAV], hepatitis B virus [HBV], hepatitis C virus [HCV], human immunodeficiency virus [HIV]-1/2, and parvovirus B19
    - Anti-C1-INH antibodies
    E.5.2.1Timepoint(s) of evaluation of this end point
    PK sampling (pre-injection, post-injection: at 0 and 15 minutes, 1 hour, 2, 6, 12, 24, 48, 72, 120, 144, and 168 hours)
    safety (at all visits from the timepoint of injection until end of study)
    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 No
    E.6.6Pharmacokinetic Yes
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA1
    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
    Belarus
    Russian Federation
    Ukraine
    Germany
    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: 17
    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 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 No
    F.4 Planned number of subjects to be included
    F.4.1In the member state0
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 20
    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)
    Standard care
    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 Decision2020-02-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-11-28
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-02-17
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