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    Summary
    EudraCT Number:2006-003148-51
    Sponsor's Protocol Code Number:F13CD-1725
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2008-09-23
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2006-003148-51
    A.3Full title of the trial
    A Multi-Centre, Open-Label, Single-Arm and Multiple Dosing Trial on Efficacy and Safety of Monthly Replacement Therapy with Recombinant Factor XIII (rFXIII) in Subjects with Congenital Factor XIII Deficiency.
    Studio multicentrico, in aperto, singolo braccio e con somministrazioni multiple per valutare efficacia e sicurezza della terapia sostitutiva mensile con Fattore XIII Ricombinante(rFXIII)nei soggetti con deficit congenito di Fattore XIII.
    A.3.2Name or abbreviated title of the trial where available
    F13CD-1725
    F13CD-1725
    A.4.1Sponsor's protocol code numberF13CD-1725
    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 SponsorNOVO NORDISK
    B.1.3.4CountryItaly
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/03/179
    D.3 Description of the IMP
    D.3.1Product nameRecombinant Factor XIII
    D.3.2Product code F13CD
    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 INNCoagulation factor XIII
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.3Concentration number5
    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 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 Yes
    D.3.11.13.1Other medicinal product typeProdotto da DNA Ricombinante
    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
    Congenital Factor XIII Deficency
    Deficit congenito di Fattore XIII
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10016083
    E.1.2Term Factor XIII deficiency
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10061992
    E.1.2Term Haemophilia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level HLT
    E.1.2Classification code 10009737
    E.1.2Term Coagulation factor deficiencies
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of monthly replacement therapy with rFXIII on prevention of bleeding episodes in subjects with congenital FXIII deficiency.
    Valutare l'efficacia della terapia di sostituzione mensile con il Fattore XIII Ricombinante nella prevenzione di episodi di sanguinamento in pazienti con deficit congenito del Fattore XIII.
    E.2.2Secondary objectives of the trial
    -Percentage of subjects without 'bleeding episodes requiring treatment” with a FXIII containing product during the rFXIII treatment period -Rate (number per subject year) of spontaneous ”bleeding episodes requiring treatment” with a FXIII containing product during the rFXIII treatment period -Rate (number per subject year) of traumatic ”bleeding episodes requiring treatment” with a FXIII containing product during the rFXIII treatment period -Rate (number per subject year) of intracranial ”bleeding episodes requiring treatment” with a FXIII containing product during the rFXIII treatment period -Percentage of subjects having a normal clot solubility one hour after rFXIII administration and 28 days after rFXIII administration -Level of FXIII activity one hour after rFXIII administration and 28 days after rFXIII administration -Number of subjects withdrawn from the trial due to lack of efficacy of rFXIII treatment -AEs -Number of subjects with rFXIII antibody development -Number
    -% di sogg senza ''episodi di sanguinamento richiedenti tratt'' con prodotto contenente FXIII durante il periodo di tratt con rFXIII-Frequenza di ''episodi di sanguinamento richiedenti tratt'' spontanei con prodotto contenente FXIII durante il periodo di tratt con rFXIII.-Frequenza di ''episodi di sanguinamento richiedenti tratt'' traumatici con prodotto contenente FXIII durante il periodo di tratt con rFXIII.-Frequenza di ''episodi di sanguinamento richiedenti tratt'' intracranici con prodotto contenente FXIII durante il periodo di tratt con rFXIII.-Percentuale di sogg aventi normale profilo di solubilità del coagulo un'ora dopo la sommin di rFXIII e 28 gg dopo la sommin di rFXIII.-Livello di attività del FXIII un'ora dopo la sommin di rFXIII e 28 gg dopo la sommin di rFXIII.-Numero di sogg ritirati dallo studio per mancanza di eff nel tratt con rFXIII.-Eventi Avversi-Numero di sogg con sviluppo di anticorpi per il rFXIII-Numero di sogg con sviluppo di inibitori per il rFXIII.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Informed consent obtained before any trial-related activities (Trial-related activities are any procedure that would not have been performed during normal management of the subject). 2.Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit). 3.Treatment with regular FXIII replacement therapy initiated at least 6 months prior to screening and one of the following : a documented history of &#8805;1 treatment-requiring bleeding episode prior to initiation of regular replacement therapy or a documented family history of FXIII congenital deficiency (only for subjects on regular replacement therapy prior to screening). 4.Documented history of &#8805; 2 bleeding episodes requiring treatment with FXIII containing blood products within the last 12 months prior to screening (only for subjects receiving on-demand treatment prior to screening). 5.Subjects with age &#8805; 6 years and a weight &#8805;20 kg. 6.If female and of child-bearing potential: negative pregnancy test at screening.
    1.Firma del Consenso Informato prima di ogni attivita' relativa allo studio. 2.Diagnosi di deficit congenito della subunita' A del Fattore XIII (confermata da esame di genotipizzazione durante la visita di screening) 3.Trattamento con regolare terapia di sostituzione del fattore XIII, iniziato da almeno 6 mesi prima della visita di screening e uno dei seguenti: una storia documentata di uno o piu' episodi di sanguinamento richiedenti trattamento prima dell'inizio di regolare terapia di sostituzione o una anamnesi di deficit congenito del Fattore XIII (solo per soggetti riceventi regolare terapia di sostituzione prima dello screening). 4.Anamnesi documentata di almeno due o piu' episodi di sanguinamento richiedenti trattamento con prodotti emoderivati contenenti FXIII nei dodici mesi precedenti la visita di screening (solo per soggetti riceventi trattamento ''on demand'' prima della visita di screening). 5.Soggetti con eta' uguale o maggiore di anni 6 di eta' e con peso maggiore od uguale di 20 Kg. 6.Test di gravidanza negativo alla visita di screening per soggetti di sesso femminile in eta' fertile.
    E.4Principal exclusion criteria
    1.Known neutralizing antibodies (inhibitors) towards FXIII. 2.Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency. 3.Documented history of two treatment-requiring bleeding episodes per year during previous regular replacement therapy with FXIII containing blood products (FFP, pd FXIII and cryoprecipitate). 4.Platelet count (thrombocytes) < 75 × 109/L. 5.Known or suspected allergy to trial product(s) or related products. 6.Previous participation in this trial. 7.Subject has received treatment with any investigational drug within 30 days of trial enrolment, except pdFXIII. 8.Planned major surgery during the trial period. Catheter, ports and dental extractions do not count as surgeries and will not exclude the subject. 9.Renal insufficiency defined as current dialysis therapy. 10.Any history of confirmed venous or arterial thrombo-embolic events. 11.Subject has received any anti-thrombotic or anti-platelet drugs within 7 days of trial enrollment. 12.Subject has medical, social or psychosocial factors expected to impact compliance or safety. 13.Any disease or condition which, judged by the Investigator, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome. 14.Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation in participating in the trial. 15.Females of childbearing potential who are pregnant, breastfeeding or intend to become pregnant or are not using adequate contraceptive methods (adequate contraceptive measures as required by local law or practice) from the time of enrollment to completion of all follow-up trial visits
    1.Conosciuta presenza di inibitori contro il FXIII. 2.Qualsiasi altro disordine della coagulazione congenito o acquisito altro rispetto al deficit di Fattore XIII. 3.Anamnesi documentata di piu' di due episodi di sanguinamento richiedenti trattamento in un anno durante precedente terapia sostitutiva con prodotti emoderivati contenenti Fattore XIII (FFP, pd FXIII e crioprecipitati) 4. Conta piastrinica 5. Sospetta o Conosciuta allergia ai prodotti dello studio o a prodotti ad esso correlati. 6. Precedente partecipazione a questo stesso trial 7. Il Soggetto ha ricevuto un trattamento con qualsiasi prodotto sperimentale nei 30 giorni precedenti l'arruolamento in questo studio, con l'eccezzione del pdFXIII. 8. Operazione chirurgica pianificata nel periodo dello studio. Cateteri,ports e estrazioni dentali non rientrano nella definizione di chirurgia e pertanto escludendo il soggetto. 9. Insufficienza Renale definita come concomitante terapia di dialisi. 10. Qualsiasi pregresso di confermato evento trombo embolico arterioso o venoso. 11. Soggetto ha ricevuto un qualsiasi farmaco antitrombotico o antipiastrinico nei 7 giorni precedenti l'arruolamento nello studio. 12. Il Soggetto possiede caratteristiche mediche, sociali o psicosociali che potrebbero inficiare con la corretta adesione allo studio o alla sua sicurezza. 13. Qualsiasi malattia o condizione che, a giudizio dello Sperimentatore, potrebbero costituire un rischio potenziale per il Soggetto, interferendo con la partecipazione allo studio o con la conclusione dello studio. 14. Incapacita' mentale,riluttanza o presenza di barriere linguistiche che possano precludere l'adeguata comprensione o cooperazione alla partecipazione allo studio durante la partecipazione in questo studio. 15. Donne in gravidanza, che stiano allattando o che intendano iniziare una gravidanza, o che non usino adeguati metodi di contraccezione dal momento dell'arruolamento al completamento di tutte le visite di Follow-up.
    E.5 End points
    E.5.1Primary end point(s)
    Rate (number per subject year) of 'bleeding episodes requiring treatment' with a FXIII containing product during the rFXIII treatment period
    Tasso di ''episodi di sanguinamento richiedenti trattamento'' con prodotto contenente FXIII durante il periodo di trattamento con rFXIII.
    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 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) 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 No
    E.8.1.2Open No
    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 Yes
    E.8.2.3.1Comparator description
    Tasso di Sanguinamento in Terapia on demand
    E.8.2.4Number of treatment arms in the trial1
    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 EEA19
    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
    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 months15
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months21
    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: 0
    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.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2008-09-23. Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Minori
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    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)
    Alla fine dello studio sara' proposto ai soggetti partecipanti di proseguire con uno studio aggiuntivo di Follow-up con l'utilizzo del prodotto in sperimentazione.
    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 Decision2008-07-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2008-07-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2010-04-15
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