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    Summary
    EudraCT Number:2009-010172-21
    Sponsor's Protocol Code Number:ENJOIH01
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2012-07-26
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2009-010172-21
    A.3Full title of the trial
    An Investigator-initiated study on rFVIIa prophylaxis in children with haemophilia A and inhibitors
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Investigator-initiated study on rFVIIa prophylaxis in children with haemophilia A and inhibitors
    A.4.1Sponsor's protocol code numberENJOIH01
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01105546
    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 SponsorFondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
    B.1.3.4CountryItaly
    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 supportNovo Nordisk A/S
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
    B.5.2Functional name of contact pointDr Elena Santagostino
    B.5.3 Address:
    B.5.3.1Street AddressVia Pace 9, Dept. of Internal Medicine and Medica, Specialties, IRCCS Maggiore Hospital
    B.5.3.2Town/ cityMilano
    B.5.3.3Post code 20122
    B.5.3.4CountryItaly
    B.5.4Telephone number+39-02-55035273
    B.5.5Fax number+30-02-55032072
    B.5.6E-mailHemophilia_ctr@policlinico.mi.it
    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 NovoSeven
    D.2.1.1.2Name of the Marketing Authorisation holderNovo Nordisk A/S
    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.3.4Pharmaceutical form Powder and solvent 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 INNEPTACOG ALFA (ACTIVATED)
    D.3.9.1CAS number 102786-61-8
    D.3.9.2Current sponsor coderFVIIa
    D.3.9.3Other descriptive nameRecombinant activated coagulation factor VII
    D.3.9.4EV Substance CodeSUB06591MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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 Yes
    D.3.11.13.1Other medicinal product typeRecombinant activated coagulation factor produced in baby hamster kidney cells
    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
    Hemophilia A
    E.1.1.1Medical condition in easily understood language
    Hemophilia A
    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 10056492
    E.1.2Term Haemophilia A with anti factor VIII
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objective of this study is to evaluate the efficacy and safety of a prophylactic regimen with rFVIIa in reducing the frequency of joint bleeds and the development of signs of joint damage including synovitis, as measured by the Hemophilia Joint Health Score (HJHS), in children with haemophilia A who develop high-responding inhibitors.
    E.2.2Secondary objectives of the trial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Male patients with haemophilia A who have been treated with FVIII on demand or on prophylaxis and who have developed inhibitors to FVIII.
    • Age < 8 years.
    • ≤ 2 years from the time of first inhibitor detection.
    • High-responding inhibitors (historical peak > 5 BU/mL) and known anamnestic response in case of negative inhibitor titre.
    • Patient is candidate for daily ITI treatment with doses of FVIII ranging from 50 IU/kg/day to 200 IU/kg/day.
    • Maximal two bleedings in the same joint within the last 6 months before entering the study or maximal six joint bleeds in the same joint within 2 years.
    • Adequate venous access for daily infusion and capable (patient or caregiver) of reconstituting and injecting the study drug (as judged by the Investigator).
    • Informed consent by parents or legal guardians.
    E.4Principal exclusion criteria
    • ITI already started.
    • Known or suspected hypersensitivity to the active substance, or to any of the excipients of the study drug, or to mouse, hamster or bovine protein.
    • Administration of any investigational product within 30 days prior to randomisation.
    • Other coagulation disorders than congenital haemophilia A.
    • Family history of thrombosis at an early age (< 40 years), known thrombophilia, any previous thrombosis including catheter-related deep vein thrombosis, previous neonatal thrombosis.
    • Known pseudo tumours.
    • Known severe liver disease.
    • Platelet count < 50,000 platelets/µL at screening.
    • Surgery within one month or planned major and/or orthopaedic surgery.
    E.5 End points
    E.5.1Primary end point(s)
    Total number of joint bleeds.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The last assessment of efficacy (clinical outcome) will take place at the final visit (Month 18, or earlier if the patient has achieved factor VIII recovery >= 25% or prematurely discontinues the study). For the evaluation of efficacy, clinical outcome measures (such as bleeding episodes) as of randomisation will be taken into account. Primary endpoint
    E.5.2Secondary end point(s)
    • Number of re-bleeds in the same joint (re-bleed is defined as bleed occurring in the same joint within 72 hours of last treatment given).
    • Time interval between haemorrhages in the same joint.
    • Number of non-joint bleeds.
    • Number of life threatening bleeds.
    • Number of limb-threatening bleeds.
    • Total number of all bleeds.
    • Joint status evaluated by the HJHS.
    • Days of hospitalisation, days lost from school/work (caregivers).
    • Adherence to the assigned treatment regimen.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The last assessment of efficacy (clinical outcome) will take place at the final visit (Month 18, or earlier if the patient achieves FVIII recovery  25% or prematurely discontinues the study).
    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 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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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
    On-demand regimen is compared to prophylactic regimen
    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.8.4.1Number 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 EEA18
    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
    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 years2
    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 years3
    E.8.9.2In all countries concerned by the trial months8
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 5
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 35
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 15
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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
    Children
    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 35
    F.4.2.2In the whole clinical trial 50
    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)
    FVIII prophylaxis for patients who will achieve inhibitor eradication by ITI (success= 70% of all ITI); treatment with bypassing agents (rFVIIa or aPCC) for patients who remain with high-responding inhibitors
    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 Decision2012-07-25
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2013-07-04
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