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    The EU Clinical Trials Register currently displays   41501   clinical trials with a EudraCT protocol, of which   6826   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .
     
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    Summary
    EudraCT Number:2018-002904-14
    Sponsor's Protocol Code Number:C14101
    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:2018-10-04
    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 number2018-002904-14
    A.3Full title of the trial
    A Phase I/II, Open Label, Proof of Concept Study to investigate Tolerability and Safety of Treatment with Recombinant Human C1 Inhibitor in Patients with Preeclampsia.
    Een fase 1/2 open label, bewijs van concept studie om de tolerantie en veiligheid van behandeling met recombinant human C1 remmer in patienten met pre-eclampsie te oderzoeken
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the tolerance and safety of treatment in patients with preeclampsia
    Een studie om de tolerantie en veiligheid van behandeling van patienten met zwangerschapsvergiftiging te onderzoeken.
    A.3.2Name or abbreviated title of the trial where available
    Preeclampsia Study
    Pre-eclampsie Studie
    A.4.1Sponsor's protocol code numberC14101
    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 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 pointClinical Development Department
    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+31715247400
    B.5.6E-maila.simon@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 AuthorisationEuropean Union
    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.3.11 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
    Preeclampsia
    Pre-eclampsie
    E.1.1.1Medical condition in easily understood language
    Preeclampsia
    Zwangerschapsvergiftiging
    E.1.1.2Therapeutic area Diseases [C] - Female diseases of the urinary and reproductive systems and pregancy complications [C13]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary:
    To evaluate the tolerability and safety of the treatment with rhC1INH on top of Standard Care, for patients with preeclampsia.


    primair:
    Het evalueren van de tolerantie en veiligheid van de behandeling met rhC1INH samen met standaard behandeling voor patienten met pre-eclampsie
    E.2.2Secondary objectives of the trial
    Secondary:
    To evaluate the efficacy of treatment with rhC1INH on top of Standard Care, for patients with preeclampsia.
    Secundair:
    Het evalueren van de effectiviteit van de behandeling met rhC1INH samen met standaard behandeling voor patienten met pre-eclampsie
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Pregnant females, aged ≥ 18 years
    2. Singleton pregnancy
    3. Between 25 and 35 weeks of gestation
    4. Diagnosis of pre-eclampsia defined as hypertension developing after 20 weeks gestation and the coexistence of one or more of the following new onset conditions:
    a. Proteinuria (spot urine protein/creatinine ≥30 mg/mmol [0.3 mg/mg] or ≥300 mg/day or at least 1 g/l [‘2+’] on dipstick testing)
    b. Other maternal organ dysfunction:
    - renal insufficiency (creatinine ≥90 Umol/l)
    - liver involvement (elevated transaminases – at least twice upper limit of normal ± right upper quadrant or epigastric abdominal pain)
    - neurological complications (examples include eclampsia, altered mental status, blindness, stroke, or more commonly hyperreflexia when accompanied by clonus, severe headaches when accompanied by hyperreflexia, persistent visual scotomata)
    - hematological complications (thrombocytopenia – platelet count below 150,000/dL, DIC, hemolysis))
    c. Uteroplacental dysfunction
    - fetal growth restriction
    5. Provided written informed consent.
    6. Willingness and ability to comply with all protocol procedures.
    1. Zwangere vrouwen, leeftijd ≥ 18 jaar
    2. Eenling zwangerschap
    3. Tussen 25 en 35 weken zwangerschap
    4. Diagnose van pre-eclampsie gedefinieerd als hypertensie die zich na 20 weken zwangerschap ontwikkelt met daarnaast de presentative van een van de volgende nieuwe aandoeningen die nog niet eerder aanwezig waren:
    een. Proteïnurie (urineproteïne / creatinine ≥30 mg / mmol [0,3 mg / mg] of ≥300 mg / dag of minstens 1 g / l ['2+'] bij het testen van de urine)
    b. Andere maternale orgaanstoornissen:
    - nierinsufficiëntie (creatinine ≥90 Umol / l)
    - gerelateerd aan de lever (verhoogde transaminasen - minstens tweemaal bovengrens van normaal ± rechtsbovenkwadrant of epigastrische buikpijn)
    - neurologische complicaties (voorbeelden zijn eclampsie, veranderde mentale toestand, blindheid, beroerte, of vaker hyperreflexie wanneer dit gepaard gaat met clonus, ernstige hoofdpijn wanneer dit gepaard gaat met hyperreflexie, aanhoudende visuele scotomata)
    - hematologische complicaties (trombocytopenie - aantal bloedplaatjes onder 150.000 / dL, DIC, hemolyse))
    c. Uteroplacentale disfunctie
    - foetale groeivermindering
    5. Mits schriftelijke geïnformeerde toestemming.
    6. Bereidheid en het vermogen om te voldoen aan alle protocolprocedures.
    E.4Principal exclusion criteria
    1. Impending Fetal death compromise – absent end-diastolic umbilical artery flow, pathological CTG
    2. Any known fetal abnormality
    3. Patients diagnosed with hereditary angioedema (HAE)
    4. Medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
    5. Treatment with any investigational drug during the current pregnancy
    6. Patient with known chronic hypertension requiring antihypertensive treatment
    7. Patient with abnormalities in their clotting system.
    8. Patient with known renal and/or hepatic abnormality
    9. Patient with a history of malignancy (except if determined cured or on remission for at least 5 years)
    10. Medical history of any organ transplants
    11. Uncontrolled diabetes, defined as HbA1C > 7%
    12. Currently treated with statins
    13. Any other condition or treatment that, in the opinion of the Investigator, might interfere with the evaluation of study objectives
    1. Dreigend Foetale sterfte-compromis - afwezigheid van end-diastolische arteriële stroom, pathologisch CTG
    2. Een bekende foetale afwijking
    3. Patiënten met de diagnose erfelijk angio-oedeem (HAE)
    4. Medische voorgeschiedenis van allergie voor konijnen of van konijnen afgeleide producten (inclusief rhC1INH)
    5. Behandeling met een onderzoeksmedicijn tijdens de huidige zwangerschap
    6. Patiënt bekend met chronische hypertensie waarvoor behandeling met bloedrukverlagers nodig is.
    7. Patiënt met afwijkingen in hun stollingssysteem.
    8. Patiënt met bekende nier- en / of leverafwijking
    9. Patiënt met een historie van kwaadaardige tumoren(behalve indien genezen verklaard of in remissie gedurende ten minste 5 jaar)
    10. Medische geschiedenis van transplantaties van organen
    11. Ongecontroleerde diabetes, gedefinieerd als HbA1C> 7%
    12. Momenteel behandeld met statines
    13. Elke andere voorwaarde of behandeling die, naar de mening van de Onderzoeker, de evaluatie van de studiedoelstellingen zou kunnen verstoren
    E.5 End points
    E.5.1Primary end point(s)
    - Incidence and severity of adverse events
    - Number and percentage of patients who discontinue study drug or withdraw from the study
    - Incidentie en ernst van bijwerkingen
    - Aantal en percentage patiënten die de studiemedicatie staken of zich uit de studie terugtrekken
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the study
    Gedurende de studie
    E.5.2Secondary end point(s)
    - Time from start of rhC1INH to day of delivery
    - Proportion of patients reaching gestation week 37
    - Tijd vanaf het begin van behandeling met rhC1INH tot de dag van geboorte
    - Percentage patiënten dat de zwangerschaps week 37 bereikt
    E.5.2.1Timepoint(s) of evaluation of this end point
    At birth
    Bij de geboorte
    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 Yes
    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.8.3 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 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
    Australia
    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
    Last subject last visit
    Laatste patient, laatste bezoek voor de studie
    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 months9
    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: 15
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male No
    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 Yes
    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 15
    F.4.2.2In the whole clinical trial 30
    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)
    None, end of pregnancy is end of treatment
    Geen, einde zwangerschap is einde van de behandeling
    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 Decision2018-10-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-06-03
    P. End of Trial
    P.End of Trial StatusOngoing
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