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    Summary
    EudraCT Number:2011-002419-27
    Sponsor's Protocol Code Number:FORMA-02
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-07-03
    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 ConcernedBulgarian Drug Agency
    A.2EudraCT number2011-002419-27
    A.3Full title of the trial
    Prospective, open-label, uncontrolled, Phase III study to assess the efficacy and safety of Octafibrin for on-demand treatment of acute bleeding and to prevent bleeding during and after surgery in subjects with congenital fibrinogen deficiency.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of the effectiveness and safety of Octafibrin, a replacement for fibrinogen, in patients with inherited fibrinogen deficiency causing episodes of bleeding.
    A.3.2Name or abbreviated title of the trial where available
    Octafibrin in Congenital Fibrinogen Deficiency - FORMA-02
    A.4.1Sponsor's protocol code numberFORMA-02
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02267226
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/001/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorOctapharma AG
    B.1.3.4CountrySwitzerland
    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 supportOctapharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationQED Clinical Services Ltd
    B.5.2Functional name of contact pointProject Management
    B.5.3 Address:
    B.5.3.1Street AddressThe Learning House, Snowdon Drive, Winterhill
    B.5.3.2Town/ cityMilton keynes
    B.5.3.3Post codeMK61BP
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number441908251480
    B.5.5Fax number441908251499
    B.5.6E-mailJLaney@qed-clinical.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 No
    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 nameOctafibrin
    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 INNFibrinogen (as clottable protein)
    D.3.9.1CAS number 9001-32-5
    D.3.9.3Other descriptive nameHuman fibrinogen
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    Congenital fibrinogen deficiency.
    E.1.1.1Medical condition in easily understood language
    Inherited defect in the blood clotting mechanism.
    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 17.1
    E.1.2Level LLT
    E.1.2Classification code 10066357
    E.1.2Term Congenital hypofibrinogenemia
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10066356
    E.1.2Term Congenital hypofibrinogenaemia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The study aims to demonstrate the efficacy of Octafibrin for on-demand treatment of acute bleeding episodes (spontaneous or after trauma).
    E.2.2Secondary objectives of the trial
    • To show an association between the overall clinical assessment of the effectiveness of Octafibrin and the laboratory endpoint ‘clot strength’ or ‘clot firmness’ (referred to as ‘maximum clot firmness’ [MCF] in this protocol) that was used as a "surrogate" measure of effectiveness in the previous study FORMA-01.
    • To achieve a peak target level of fibrinogen in plasma of 100 mg/dL in minor bleeds and 150 mg/dL for major bleeds 1 hour post-infusion.
    • To determine the response to Octafibrin based on incremental in vivo recovery (IVR).
    • To demonstrate the efficacy of Octafibrin in preventing bleeding during and after surgery.
    • To assess the safety of Octafibrin in subjects with congenital fibrinogen deficiency, including immunogenicity, thromboembolic complications, and early signs of allergic or hypersensitivity reactions.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects who meet all of the following criteria are eligible for the study:
    1. Aged ≥12 years (only 18 and above in Russia)
    2. Documented diagnosis of congenital fibrinogen deficiency, expected to require on-demand treatment for bleeding or surgical prophylaxis:
    – Fibrinogen deficiency manifested as afibrinogenaemia or severe hypofibrinogenaemia.
    – Historical plasma fibrinogen activity of<50 mg/dL or levels below the limit of detection of the local assay method.
    3. Expected to have an acute bleeding episode (spontaneous or after trauma) or planning to undergo elective surgery.
    4. Informed consent signed by the subject or legal guardian.
    E.4Principal exclusion criteria
    Subjects who meet any of the following criteria are not eligible for the study:
    1. Life expectancy <6 months.
    2. Bleeding disorder other than congenital fibrinogen deficiency, including dysfibrinogenaemia.
    3. Prophylactic treatment with a fibrinogen concentrate.
    4. Treatment with:
    – Any fibrinogen concentrate or other fibrinogen-containing blood product within 2 weeks prior to start of treatment for the bleeding episode or surgery
    – Any coagulation-active drug (i.e., non-steroidal anti-inflammatory drugs, warfarin, coumarin derivatives, platelet aggregation inhibitors) within 1 week prior to start of treatment for the bleeding episode or surgery, or as a planned or expected medication during the time period from Day 1 until 24 hours (i.e., 1 day) after the last Octafibrin infusion.
    5. Presence or history of:
    – Hypersensitivity to study medication
    – Deep vein thrombosis or pulmonary embolism within 1 year prior to start of treatment for the bleeding episode or surgery
    – Arterial thrombosis within 1 year prior to start of treatment for the bleeding episode or surgery
    – Hypersensitivity to human plasma proteins
    – Oesophageal varicose bleeding
    – End-stage liver disease (i.e., Child-Pugh score B or C).
    6. Pregnant women within the first 20 weeks of gestation.
    7. Currently breast-feeding.
    8. Known positive HIV infection with a viral load >200 particles/μL or >400,000 copies/mL.
    9. Polytrauma 1 year prior to start of treatment for the bleeding episode or surgery.
    10. Diagnosis or suspicion of a neutralising anti-fibrinogen inhibitor currently or at any time in the past.
    11. Acute or chronic medical condition which may, in the opinion of investigator, affect the conduct of the study, including
    – Subjects receiving immune-modulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs at study start
    – Subjects having evidence or a history (within the previous 12 months) of abuse of any drug licit or illicit substance.
    12. Participation in another interventional clinical study currently or during the past 4 weeks.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the overall clinical assessment of haemostatic efficacy in acute bleeding at 24 hours (i.e., 1 day) after last infusion or the end of the treatment observation period (whichever comes last) of the IMP for the first bleeding episode of each patient.
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 hours (i.e., 1 day) after last infusion or the end of the treatment observation period (whichever comes last) of the IMP for the first bleeding episode of each patient.
    E.5.2Secondary end point(s)
    Clot strength (MCF)

    Peak target level of fibrinogen in plasma 1 hour post-infusion

    Octafibrin use - The dose of the IMP used per day and in total.

    In-vivo recovery (IVR)- Response and classical IVR will be calculated for each infusion of each subject.

    Surgical prophylaxis
    Efficacy of Octafibrin in surgical prophylaxis will be assessed intra-operatively (at the end of surgery = after last suture) by the surgeon and post-operatively by the haematologist using two 4-point efficacy scales. The overall surgical efficacy will be adjudicated by the IDMEAC.

    Safety analysis
    E.5.2.1Timepoint(s) of evaluation of this end point
    - MCF before 1st infusion and 1h after the end of the 1st and last infusion of each bleeding episode
    - Fibrinogen levels 1 hour after infusion of the investigational medicinal product (IMP
    - In vivo recovery (IVR)for each infusion
    - Octafibrin use per day and in total
    - Efficacy of Octafibrin in surgical prophylaxis will be assessed intra-operatively by the surgeon and post-operatively by the haematologist
    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 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 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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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
    Bulgaria
    Egypt
    India
    Lebanon
    Morocco
    Russian Federation
    Saudi Arabia
    Turkey
    United Kingdom
    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
    Last Patient Last Visit.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days1
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days1
    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: 4
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 19
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 14
    F.4.2.2In the whole clinical trial 24
    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)
    On completion of the study patients will return to Standard of Care treatment as considered appropriate by their haematologist.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-09-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-07-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-01-23
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