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    Summary
    EudraCT Number:2013-000261-36
    Sponsor's Protocol Code Number:FGTW-1004
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-04-04
    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 ConcernedFrance - ANSM
    A.2EudraCT number2013-000261-36
    A.3Full title of the trial
    Clinical pharmacology, efficacy and safety study of FGTW in paediatric patients with severe congenital fibrinogen deficiency
    Etude de pharmacologie clinique, d’efficacité et de tolérance du FGTW chez des patients pédiatriques présentant un déficit congénital sévère en fibrinogène
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A fibrinogen concentrate study in paediatric patients with inherited fibrinogen deficiency
    A.4.1Sponsor's protocol code numberFGTW-1004
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/196/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorLFB Biotechnologies
    B.1.3.4CountryFrance
    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 supportLFB Biotechnologies
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLFB Biotechnologies
    B.5.2Functional name of contact pointClinical trial information desk
    B.5.3 Address:
    B.5.3.1Street Address3 avenue des Tropiques
    B.5.3.2Town/ cityCourtaboeuf
    B.5.3.3Post code92942
    B.5.3.4CountryFrance
    B.5.4Telephone number33169827010
    B.5.5Fax number33169827272
    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 CLOTTAFACT 1.5 g/100ml, powder and solvent for infusion
    D.2.1.1.2Name of the Marketing Authorisation holderLFB-BIOMEDICAMENTS
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 nameClottafact
    D.3.2Product code FGTW
    D.3.4Pharmaceutical form Powder and solvent for solution for infusion
    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 Yes
    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 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
    Afibrinogenaemia or severe hypofibrinogenaemia
    E.1.1.1Medical condition in easily understood language
    Inherited fibrinogen deficiency
    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 16.1
    E.1.2Level LLT
    E.1.2Classification code 10052651
    E.1.2Term Afibrinogenaemia
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level PT
    E.1.2Classification code 10051125
    E.1.2Term Hypofibrinogenaemia
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    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 the study is to assess the efficacy of FGTW as a replacement therapy in paediatric patients with afibrinogenaemia or severe hypofibrinogenaemia:
    ▪ in preventing excessive bleeding in patients undergoing a surgical procedure,
    ▪ in treating bleeding of non-surgical origin
    E.2.2Secondary objectives of the trial
    The secondary objectives are:
    - to assess the pharmacokinetic (PK) and pharmacodynamic (PD) properties of FGTW.
    - to assess the safety of FGTW
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed and dated informed consent form by parents or a legal representative
    2. Age 12 years old
    3. Patients with inherited afibrinogenaemia (fibrinogen antigen levels 0.2 g/L and unclottable PT, aPTT and TT) or severe hypofibrinogenaemia defined as fibrinogen antigen levels 0.5 g/L and activity to antigen ratio ≈ 1
    4. Negative results on HCG-based pregnancy test at the selection visit, before PK/PD part and before each event to be treated for females of childbearing potential (presence of menstruation)
    5. Covered by healthcare insurance in accordance with local requirements
    E.4Principal exclusion criteria
    1. Dysfibrinogenaemia
    2. Acquired fibrinogen deficiency
    3. Suspected present or past anticoagulation inhibitor
    4. Personal history of venous or arterial thrombosis or thromboembolic event
    5. Co-morbidity with other/unrelated coagulopathies
    6. Administration of any fibrinogen concentrate or fibrinogen containing blood product during the last 15 days (applicable only if fibrinogen levels to be measured at inclusion)
    7. Platelet count less than 100 x 109/L
    8. Serum creatinine > 2 times the upper limit of normal
    9. AST and / or ALT > 5 times the upper limit of normal
    10. Known history of hypersensitivity or other severe reaction to any component of the investigational medicinal product
    11. Any other condition which, in the opinion of the investigator, could interfere with the study results or would not be in the best interest of the patient.
    12. Participation in another drug clinical trial at the same time or within the previous 30 days
    13. Anticipated poor compliance with study procedures
    14. Legal incapacity or limited capacity
    15. Permanent treatment with antithrombotic or anti-platelet agents such as heparins, anti-IIa or anti-Xa agents, aspirin, clopidogrel and NSAIDs.
    Additional exclusion criteria specific to PK/PD Part
    1. Patients with hypofibrinogenaemia defined as fibrinogen antigen levels 0.5 g/L and activity to antigen ratio ≈ 1
    2. Significant bleeding(s) within 7 days prior to the start of the PK/PD part that could have an impact on the patient's plasma volume
    3. Administration of any fibrinogen concentrate or fibrinogen containing blood product during the last 15 days prior to the start of the PK/PD part
    4. Treatment with antithrombotic or anti-platelet agents such as heparins, anti-IIa or anti-Xa agents, aspirin, clopidogrel and NSAIDs within 10 days prior to the start of the PK/PD part.
    5. Elective surgery within 14 days after administration of FGTW for PK/PD part
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint will be the investigator’s overall assessment of the efficacy of FGTW on haemostasis at the end of each treatment episode (at 24h-48h after treatment when the patient is not hospitalised, or at the day of hospital discharge when the patient is hospitalised).
    Surgical Event:
    The investigator’s overall assessment of the efficacy of FGTW on haemostasis will be rated using the following 4-point scale :
    • Excellent: bleeding similar to that expected in a normal subjects,
    • Good: mild excessive bleeding,
    • Moderate: moderate but controlled excessive bleeding,
    • None: severe, uncontrolled bleeding.
    and will take into account the following parameters (when applicable):
    - Assessment of haemostatic efficacy of FGTW rated by the surgeon or a qualified physician at the end of the surgical procedure and on a daily basis using the same 4-point scale described above,
    - Blood loss (comparison of predicted and actual volumes), unless it cannot be measured (e.g. dental surgery),
    - Haemoglobin levels and blood product transfusion units used according to standard care,
    - Daily medical examination,
    - Amounts of administered FGTW.

    Bleeding Event Study:
    The investigator’s overall assessment of the efficacy of FGTW on haemostasis will be rated using the following 4-point scale :
    • Excellent: bleeding stopped immediately,
    • Good: bleeding stopped within the expected time period,
    • Moderate: bleeding was controlled with difficulty,
    • None: uncontrolled bleeding or need of other haemostatic treatments.
    and will take into account the following parameters (when applicable):
    - Daily assessment of haemostatic efficacy of FGTW rated by a qualified physician using the same 4-point scale described above,
    - Haemoglobin levels and blood product transfusion units used according to standard care,
    - Daily medical examination,
    - Amounts of administered FGTW.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At the end of each treatment episode:
    - at 24h-48h after treatment when the patient is not hospitalised,
    - at the day of hospital discharge when the patient is hospitalised.
    E.5.2Secondary end point(s)
    Efficacy:
    - Daily assessment of haemostatic efficacy of FGTW using the 4-point surgical/bleeding scales described above,
    - Amounts of administered FGTW,
    - Plasma levels of fibrinogen activity after infusion,
    - Time to wound healing, if applicable,
    - ROTEM® FIBTEM assay parameters in frozen plasma.

    Clinical Pharmacology:
    - Fibrinogen plasma concentrations determined by the Clauss method and the nephelometric method.
    - Pharmacodynamic profile will be assessed by measuring FIBTEM-MCF (ROTEM®) over time in frozen plasma. TT, PT, aPTT will also be measured.
    - Based on a prior PK model obtained from another clinical study with afibrinogenaemic adults, individual fibrinogen values will be reconstructed by Bayesian analysis, and primary PK model parameters will be estimated (e.g. CL (L/h), and Vss (L)). Secondary PK parameters will be derived: Cmax, tmax, AUC, and terminal half-life.
    -Incremental recovery will also be calculated.

    Safety:
    Safety will be assessed throughout the study until the last visit of the patient by incidence, nature, severity, seriousness, outcome, relationship to IMP of adverse events and changes in physical examination findings, clinical laboratory tests, and vital sign measurements.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The timepoints of evaluation of secondary endpoints are variable (the individual timepoints and periods are described in the synopsis and flow charts of the study protocol).
    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 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) Yes
    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
    Lebanon
    Morocco
    Turkey
    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 visit of the last patient
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months11
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    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: 12
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 3
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 8
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    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 No
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception Yes
    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 No
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Paediatric patients below 13 years old
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 2
    F.4.2.2In the whole clinical trial 12
    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)
    CLOTTAFACT 1.5 g/ 100 ml is registered in France
    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 Decision2013-11-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-09-06
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-12-11
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