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    Summary
    EudraCT Number:2011-000987-92
    Sponsor's Protocol Code Number:C11209
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-04-17
    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 ConcernedCzechia - SUKL
    A.2EudraCT number2011-000987-92
    A.3Full title of the trial
    Open-label, phase II, single arm study to evaluate the safety, immunogenicity, pharmacokinetics and efficacy of recombinant human C1 inhibitor for the treatment of acute attacks in pediatric patients with hereditary angioedema, from 2 up to and including 13 years of age
    Otevřená jednoramenná studie fáze II hodnotící bezpečnost, imunogenitu, farmakokinetiku a účinnost rekombinantního lidského inhibitoru C1 pro léčbu akutních záchvatů hereditárního angioedému u dětských pacientů od 2 do 13 let věku včetně
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety of Ruconest in children with HAE
    A.3.2Name or abbreviated title of the trial where available
    Safety of Ruconest in 2-13 year old HAE patients
    A.4.1Sponsor's protocol code numberC11209
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/103/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPharming Technologies B.V.
    B.1.3.4CountryNetherlands
    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 supportPharming Technologies B.V.
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPharming Technologies B.V.
    B.5.2Functional name of contact pointTessa Heimap - van Rossenberg
    B.5.3 Address:
    B.5.3.1Street AddressDarwinweg 24
    B.5.3.2Town/ cityLeiden
    B.5.3.3Post code2333 CR
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31715247425
    B.5.5Fax number+31715247445
    B.5.6E-mailt.heikamp@pharming.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 Ruconest
    D.2.1.1.2Name of the Marketing Authorisation holderPharming Group N.V
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 nameRuconest
    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.3.8INN - Proposed INNCONESTAT ALFA
    D.3.9.1CAS number 80295-38-1
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number50 U/kg BW-4200 U
    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
    Attacks associated with hereditary angioedema (HAE) in pediatric population 2-13 years of age.
    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 18.1
    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 assess the clinical safety, immunogenicity and tolerability of Ruconest in the treatment of acute angioedema attacks in 2-13 year old HAE patients.
    E.2.2Secondary objectives of the trial
    To assess the pharmacokinetics (PK) and pharmacodynamics (PD) of Ruconest in the treatment of acute angioedema attacks in 2-13 year old HAE patients.

    To assess the efficacy of Ruconest in the treatment of acute angioedema attacks in 2-13 year old HAE patients.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Screening
    • From 2 up to and including 13 years of age
    • Clinical and laboratory confirmed diagnosis of HAE (baseline C1INH activity <50% of normal)
    • Signed written informed consent (parental permission) signed by the legal guardian(s)

    Treatment
    • Clinical symptoms of an acute HAE attack
    • Onset of eligible symptoms within 5 hours from the moment at which medical evaluation to determine eligibility has occurred
    • IS score for at least one anatomical location at the time of initial evaluation of at least 3 (moderate severity or greater) without signs of spontaneous regression
    • 24h or more have passed since the patient’s last study treatment
    E.4Principal exclusion criteria
    Screening
    • A diagnosis of acquired C1INH deficiency (AAE)
    • A medical history of allergy to rabbits or rabbit-derived products (including rhC1INH, antisera), or positive anti-rabbit epithelium (dander) IgE test (cut off>0.35 kU/L in ImmunoCap® assay (Phadia, Sweden) or equivalent)
    • Treatment with investigational drug in another clinical study in the last 30 days
    • Any clinical significant abnormality in the physical examination and/or the routine laboratory assessments, that in the opinion of the Investigator makes the patient unsuitable for participation in the study
    • Any condition or treatment that in the opinion of the investigator might interfere with the evaluation of the study objectives

    Treatment
    • Any changes since screening that would exclude patient based on above exclusion criteria.
    • 10 HAE attacks were previously treated with study medication.
    • Suspicion for an alternate explanation of the symptoms other than an acute HAE attack.
    • Use of any disallowed concomitant medication since onset of acute HAE attack
    E.5 End points
    E.5.1Primary end point(s)
    Assessment of safety and tolerability.
    E.5.1.1Timepoint(s) of evaluation of this end point
    This will be evaluated by continuous recording adverse events.
    E.5.2Secondary end point(s)
    Time to beginning of relief, time to minimal symptoms, time to complete resolution.
    Pharmacokinetic and pharmacodynamic parameters (C1INH activity and C4 in plasma) during treatment for the first attack.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The clinical endpoints will be evaluated following the treatment of an attack. Blood samples for PK and PD will be taken at 5 minutes and between 2-4 hours after the Ruconest injection at the first attack.
    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 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 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.8.3 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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA14
    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
    Israel
    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 years0
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    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: 20
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) No
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Paediatric Patients not able to give legally effective informed consent. Parents/legal guardians will be informed and asked about their consent on behalf of the minor. The will of the minor will be respected.
    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 30
    F.4.2.2In the whole clinical trial 40
    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 be treated according to medical routine.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-08-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-10-02
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-07-17
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