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    Summary
    EudraCT Number:2016-005083-34
    Sponsor's Protocol Code Number:CAPI2017NL10
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-04-06
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2016-005083-34
    A.3Full title of the trial
    Conestat alfa as prophylactic treatment for idiopathic non-histaminergic acquired angioedema
    Conestat alfa (Ruconest®) behandeling voor het voorkómen van angio-oedeem aanvallen bij patiënten met angio-oedeem met onbekende oorzaak
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Conestat alfa as prophylactic treatment for idiopathic non-histaminergic acquired angioedema
    Conestat alfa (Ruconest®) behandeling voor het voorkómen van angio-oedeem aanvallen bij patiënten met angio-oedeem met onbekende oorzaak
    A.3.2Name or abbreviated title of the trial where available
    Conestat alfa prophylaxis for InH-AAE (CAPI)
    Conestat alfa profylaxe voor InH-AAE (CAPI)
    A.4.1Sponsor's protocol code numberCAPI2017NL10
    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 SponsorUniversity Medical Center Utrecht
    B.1.3.4CountryNetherlands
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportPharming Group N.V.,
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity Medical Center Utrecht
    B.5.2Functional name of contact pointZonne Hofman
    B.5.3 Address:
    B.5.3.1Street AddressHeidelberglaan 100
    B.5.3.2Town/ cityUtrecht
    B.5.3.3Post code3584CX
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31887551808
    B.5.5Fax number+31887557388
    B.5.6E-mailz.l.m.hofman-2@umcutrecht.nl
    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 2100 U powder for solution for injection
    D.2.1.1.2Name of the Marketing Authorisation holderPharming Group N.V.
    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 numberORPHA70668
    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 bolus use (Noncurrent)
    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
    The medical condition to be investigated is idiopathic non-histaminergic angioedema (InH-AAE). This disease is characterized by recurrent episodes with swelling of the subcutis and mucous membranes. Patient with InH-AAE have by defenition no wheals and are nonresponsive to anti-histamine therapy.
    Het te onderzoeken ziektebeeld is idiopatisch non-histaminerg verworven angio-oedeem (InH-AAE). Deze aandoening wordt gekenmerkt door recidiverende episodes met zwellingen in de subcutis en mucosa. InH-AAE patienten hebben per definitie geen urticaria en reageren niet op antihistaminica.
    E.1.1.1Medical condition in easily understood language
    The medical condition to be investigated is idiopathic non-histaminergic angioedema (InH-AAE). This disease is characterized by recurrent episodes with swelling of the skin and mucous membranes.
    Het te onderzoeken ziektebeeld is idiopatisch non-histaminerg verworven angio-oedeem (InH-AAE). Deze aandoening wordt gekenmerkt door recidiverende episodes met zwellingen in de subcutis en mucosa.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    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 exploratory study is to test if prophylactic treatment with conestat alfa decreases the frequency of angioedema attacks in patients with idiopathic non-histaminergic acquired angioedema.
    Het primaire doel van deze exploratieve studie is om te testen of profylactische behandeling met conestat alfa de aanvalsfrequentie van angio-oedeem by patiënten met idiopatisch non-histaminerg verworven angio-oedeem verminderd.
    E.2.2Secondary objectives of the trial
    To test if prophylactic use of conestat alfa decreases disease activity in patients with InH-AAE.

    To test if prophylactic use of conestat alfa improves the quality of life in patients with InH-AAE.

    To establish the safety of prophylactic use of Conestat alfa in patients with InH-AAE.

    To examine potential biomarkers for disease activity, response to treatment and predictive value for response to treatment.
    Het effect van profylactisch conestat alfa gebruik bij patiënten met InH-AAE op ziekte activiteit.

    Het effect van profylactisch conestat alfa gebruik bij patiënten met InH-AAE op kwaliteit van leven.

    Vaststellen van de veiligheid van profylactisch gebruik van conestat alfa bij patiënten met InH-AAE.

    Onderzoek naar potentiele biomarkers voor ziekte activiteit, therapierespons, en predictie van therapierespons.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Age ≥ 18 years.

    Diagnosis idiopathic non-histaminergic acquired angioedema (InH-AAE) were “non-histaminergic” is defined as following: insufficient effect of treatment with antihistamines up to 4 times the standard dose (step 2 in CSU-treatment regimen), defined as having breakthrough attacks.

    Minimal mean attack rate of 2 per month during the past six months despite treatment; with at least one attack in the month prior to inclusion.

    Normal C4 levels.

    Written informed consent.
    Leeftijd ≥ 18 jaar

    Diagnose idiopathic non-histaminerg verworven angio-oedema (InH-AAE) waarbij “non-histaminerg” alsvolgt is gedefinieerd: onvoldoende effect van behandeling met antihistaminica met maximaal 4 keer de standaard dosis (stap 2 in CSU-behandelingsprotocol), gedefinieerd als het voorkomen van doorbraak aanvallen.

    Minimale aanvalsfrequentie van 2 per maand gedurende de afgelopen 6 maanden ondanks behandeling met anti-histaminica; met ten minste één angio-oedeem aanval in de maand voorafgaande aan studie-inclusie.

    Normale C4 levels.

    Getekend informed consent.
    E.4Principal exclusion criteria
    Presence of recurrent wheals/ urticaria accompanying angioedema.

    Diagnosis other than InH-AAE is deemed more likely.

    ACE-inhibitor use in the past 6 months.

    History suggesting allergy for rabbits or rabbit derived products (such as conestat alfa).

    Currently trying to conceive, pregnancy and women giving breastfeeding.

    Inability to comply with study and follow-up procedures.

    Presence of clinically significant conditions that could interfere with the interpretation of the study results and or compromise the safety of the patients.

    Participation in an investigational drug or device trial within the last 30 days prior to screening.
    Aanwezigheid van urticaria bij angio-oedeem.

    Een andere diagnose dan InH-AAE is meer waarschijnlijk.

    ACE-gebruik in de afgelopen 6 maanden.

    Voorgeschiedenis suggestief voor konijnenallergie of allergie voor van konijnen verkregen producten (zoals conestat alfa).

    Actieve zwangerschapswens, zwangerschap en momenteel borstvoeding gevend.

    Onvermogen te kunnen voldoen aan de studieprocedures.

    Aanwezigheid van klinisch significante aandoeningen die kunnen interfereren met de interpretatie van de studie en de veiligheid van de patiënt.

    Deelname aan een klinische geneesmiddelenstudie of hulpmiddelen studie in de afgelopen dagen.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of this study is the mean monthly attack frequency during the 8-week treatment period as reported by the patient on the AAS form.
    This primary parameter will be compared with the patient reported mean monthly attack frequency over the past 6 months (primary outcome) and the mean monthly attack frequency during the 8-week observational period.
    Het primaire eindpunt van deze studie is de gemiddelde maandelijkse aanvalsfrequentie gedurende de 8-weekse behandelperiode zoals gerapporteerd op het AAS formulier.
    Dit eindpunt zal worden vergeleken met de door de patiënt geraporteerde aanvalsfrequentie over de afgelopen 6 maanden (primary outcome), de gemiddelde maandelijkse aanvalsfrequentie tijdens de 8-weekse observatieperiode.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Daily by AAS & at initiation visit.
    Dagelijks met de AAS & tijdens de inclusie visit.
    E.5.2Secondary end point(s)
    The mean monthly disease severity as measured by AAS28 or AAS7.

    The quality of life measured by AE-QoL.

    Changes in the UCT score.

    Number of adverse and serious adverse events.

    Levels of biomarkers.
    De gemiddelde maandelijkse ziekteactiviteit gemeten met de AAS28 of AAS7.

    De kwaliteit van leven gemeten met de AE-QoL.

    Veranderingen in de UCT scores. Changes in the UCT score.

    Aantal adverse en serious adverse events.

    Levels van biomarkers.
    E.5.2.1Timepoint(s) of evaluation of this end point
    AAS: daily
    AE-QoL&UCT: montly
    Adverse events: Montly during obervation and twice weekly during the 8-weeks of therapy.
    Biomarkers: At the first visit (V1), last visit (V18) and during the treatment period at V2, V4, V9 and V17.
    AAS: dagelijks
    AE-QoL&UCT: maandelijks
    Adverse events: maandelijks tijdens observatie, tweewekelijks tijdens de behandelperiode.
    Biomarkers: bij het eerste bezoek (V1), laatste bezoek(V18) en tijdens de behandelperiode bij V2, V4, V9 en V17.
    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 Yes
    E.6.4Safety No
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    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.8.3 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 No
    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 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
    The end of the trial is defined by the last study visit of the last study subject.

    The study (or funding for the study) can be terminated by Pharming or by the investigator as described in the trial agreement.
    Het einde van de trial wordt gedefinieerd als het laatste bezoek van de laatste deelnemer.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    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: 10
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 state10
    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)
    Study participants will return to routine care.
    Deelnemers gaan terug naar reguliere zorg.
    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 Decision2017-04-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-08-23
    P. End of Trial
    P.End of Trial StatusOngoing
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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