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    Summary
    EudraCT Number:2015-003027-61
    Sponsor's Protocol Code Number:B1821048
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2015-12-23
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2015-003027-61
    A.3Full title of the trial
    An open-label, single dose pharmacokinetic study of BeneFIX (nonacog alfa, recombinant factor IX) in male Chinese subjects with haemophilia B.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A single dose study of BeneFIX (nonacog alfa, recombinant factor IX) in male Chinese subjects with haemophilia B.
    A.4.1Sponsor's protocol code numberB1821048
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02213250
    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 SponsorPfizer Inc.
    B.1.3.4CountryUnited States
    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 supportPfizer Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPfizer Inc.
    B.5.2Functional name of contact point Clinical Trials.gov Call Center
    B.5.3 Address:
    B.5.3.1Street Address235 E 42nd Street
    B.5.3.2Town/ cityNew York
    B.5.3.3Post code10017
    B.5.3.4CountryUnited States
    B.5.6E-mailClinicalTrials.govCallCenter@pfizer.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 BeneFIX
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Ltd
    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 nameBeneFIX
    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 INNNONACOG ALFA
    D.3.9.1CAS number 181054-95-5
    D.3.9.4EV Substance CodeSUB03451MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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
    Haemophilia B
    E.1.1.1Medical condition in easily understood language
    Haemophilia B
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    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 is to characterise the single dose pharmacokinetic profiles of BeneFIX in male Chinese subjects with Haemophilia B
    E.2.2Secondary objectives of the trial
    To assess the safety and toleration of single dose BeneFIX in male Chinese subjects with Haemophilia B
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all of the following criteria;
    • Male Chinese subjects 6 years and older (weight ≥20 kg) with moderate to severe hemophilia B (Factor IX activity ≤2%)
    • Subjects should not have received infusion of any Factor IX products for at least 4 days before the administration of BeneFIX on Day 1
    • Subjects must be in a non-bleeding state before BeneFIX administration on Day 1
    • Evidence of personally or legally representative (legal only for pediatric subjects) signed and dated informed consent documentation indicating that the subject has been informed of all pertinent aspects of the study
    • Subjects who are willing and able to comply with scheduled visits, treatment plans, laboratory tests and other trial procedures.
    E.4Principal exclusion criteria
    Subjects presenting with any of the following will not be included in the study
    • Evidence or history of clinically significant haematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic or allergic (including drug allergies) disease or clinical findings at Screening.
    • Diagnosed with any other bleeding disorder in addition to Haemophilia B
    • Documented Human Immunodeficiency Virus positive patients
    • Current FIX inhibitor or history of FIX inhibitor (defined as ≥Upper Limit of Normal (ULN) of the reporting lab)
    • Subjects anticipating elective surgery that may be planned to occur in the 1 month following study entry
    • Treated with immunomodulatory therapy within 30 days
    • Treated with an investigational drug within 30 days or 5 half-lives preceding the first dose of the study drug
    • Subjects with known hypersensitivity to the active substance or to any of the excipients of BeneFIX
    • Subjects with known hypersensitivity to Chinese Hamster Ovary cell (CHO cell) proteins
    • Subjects with any of hepatic or renal impairment (ALT or AST >3x Upper Limit of Normal (ULN), total bilirubin >2x ULN, serum creatinine >2x ULN), Prothrombin Time >1.5x ULN, Platelet count <80,000 uL
    • Unwilling or unable to follow the terms of the protocol
    • Any condition which may compromise the subject’s ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation, in the opinion of the Investigator or Sponsor
    • A positive urine drug screen
    • History of regular alcohol consumption exceeding 14 drinks/week for men within 6 months of screening
    • Screening supine blood pressure ≥140 mm Hg (systolic) or ≥90 mm Hg (diastolic) following at least 5 minutes rest
    • 12-lead ECG showing QTc >450 msec or QRS >120 msec at screening
    • Blood donation of approximately 1 pint within 56 days prior to dosing
    • History of sensitivity to heparin or heparin-induced thrombocytopenia
    • Unwilling or unable to comply with the Lifestyle Guidelines
    • Other severe acute or chronic medical or psychiatric conditions or laboratory abnormality that may increase risk associated with study participation.
    E.5 End points
    E.5.1Primary end point(s)
    Pharmacokinetic Endpoints:
    •Primary study endpoints will be Cmax, AUClast, AUCinf, Tmax, CL, Vss, Kel, t1/2, MRT, incremental recovery of BeneFIX (as measured by FIX activity)
    Safety Endpoints:
    •Adverse events, laboratory abnormalities, vital signs, inhibitor development, allergic reactions, and thrombogenicity.
    E.5.1.1Timepoint(s) of evaluation of this end point
    0-96 hours post dose
    E.5.2Secondary end point(s)
    N/A
    E.5.2.1Timepoint(s) of evaluation of this end point
    N/A
    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 No
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Pharmacokinetic
    E.7.2Therapeutic exploratory (Phase II) No
    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 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    China
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months1
    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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 3
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 under the age of 18
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    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)
    NONE
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: China
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