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    Summary
    EudraCT Number:2017-001255-31
    Sponsor's Protocol Code Number:B7841003
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-11-09
    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 number2017-001255-31
    A.3Full title of the trial
    A MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS PF-06741086 IN SUBJECTS WITH SEVERE HEMOPHILIA
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS PF-06741086 IN SUBJECTS WITH SEVERE HEMOPHILIA
    A.4.1Sponsor's protocol code numberB7841003
    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., 235 East 42nd Street, New York, NY 10017
    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., 235 East 42nd Street, New York, NY 10017
    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 pointClinical Trials.gov Call center
    B.5.3 Address:
    B.5.3.1Street Address235 East 42nd Street
    B.5.3.2Town/ cityNew York
    B.5.3.3Post codeNY 10017
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1800718 1021
    B.5.5Fax number+1303739 1119
    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 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/3/16/1752
    D.3 Description of the IMP
    D.3.1Product namePF-06741086 100 mg/ml
    D.3.2Product code PF-06741086
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPF-06741086
    D.3.9.3Other descriptive namePF-06741086
    D.3.9.4EV Substance CodeSUB187130
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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.IMP: 2
    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/3/16/1752
    D.3 Description of the IMP
    D.3.1Product namePF-06741086 150 mg/ml
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPF-06741086
    D.3.9.3Other descriptive namePF-06741086
    D.3.9.4EV Substance CodeSUB187130
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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
    Severe Hemophilia A or B, with or without inhibitors to Factor VIII (FVIII) or Factor IX (FIX).
    E.1.1.1Medical condition in easily understood language
    Severe Hemophilia A or B, with or without inhibitors to Factor VIII (FVIII) or Factor IX (FIX).
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10053751
    E.1.2Term Hemophilia A with anti factor VIII
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10053754
    E.1.2Term Hemophilia B without inhibitors
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10053753
    E.1.2Term Hemophilia A without inhibitors
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10053752
    E.1.2Term Hemophilia B with anti factor IX
    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
    •To determine the safety and tolerability of long term treatment (up to 365 days) with PF 06741086 in severe hemophilia A and B subjects with
    or without inhibitors to FVIII or FIX.
    E.2.2Secondary objectives of the trial
    •To determine the efficacy of long term treatment with PF 06741086 in severe hemophilia A and B subjects with or without inhibitors to FVIII or FIX.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    All subjects must meet the following inclusion criteria to be eligible for
    enrollment into the study. Subjects continuing from B7841002 within 30
    days of the B7841003 Day 1 visit do not require screening procedures
    to confirm eligibility:
    1.Evidence of a personally signed and dated informed consent document
    indicating that the subject (or a legally acceptable
    representative/parent(s)/legal guardian) has been informed of all
    pertinent aspects of the study.
    2.Males ≥18 and <75 years of age.
    3.Body Mass Index (BMI) ≥17.5 and ≤30.5 kg/m2 and total body weight
    ≥50 and ≤100 kg.
    a. For adolescents, the BMI must be ≥14 kg/m2 and total body weight
    must be ≥30 kg.
    4.Diagnosis of severe hemophilia A or B (FVIII or FIX activity ≤1%).
    5.If receiving therapy for human immunodeficiency virus (HIV) or active
    hepatitis infection, have stable disease and be on a stable regimen at the
    time of study entry (ie, stable dosing for at least 3 months before
    consent).
    6.Willing and able to comply with scheduled visits, treatment plan,
    laboratory tests, and other study procedures.
    The following inclusion criteria only apply to de novo subjects (Cohorts 5
    and 6) only:
    1. Adolescent males ≥12 to <18 years of age are eligible
    2. Patients with an episodic (on-demand) treatment regimen who are
    willing and able to washout from Factor VIII (for at least 72 hours) or
    Factor IX (for at least 96 hours) replacement therapy, or bypass agent
    therapy (for rFVIIa and activated prothrombin complex concentrate
    [APCC]: at least 72 hours; for other products: at least 5 half-lives) prior
    to Screening laboratory assessments of factor activity and have no plans
    to institute prophylactic factor or bypass agent treatment during the
    study period.
    3. Had at least 6 acute bleeding episodes (spontaneous/traumatic)
    during the 6 month period prior to Screening. Surgical bleeding episodes
    do not apply to this criterion.
    4. Subjects enrolled as Factor VIII or Factor IX inhibitor patients must
    have a positive inhibitor test result (above the upper limit of normal) at
    the local laboratory and must receive a bypass agent as primary
    treatment for bleeding episodes. A positive inhibitor test result will be
    above the upper limit of normal for the assay. Inhibitor test results from
    up to 6 months prior to Day 1 may be used to meet this requirement.
    E.4Principal exclusion criteria
    Subjects with any of the following characteristics/conditions will not be included in the study:
    1.Females.
    2.Known coronary artery, thrombotic, or ischemic disease, or known severe, uncontrolled hypercholesterolemia.
    3.Known hemostatic defect other than hemophilia A or B.
    4.ATIII, Protein C, or Protein S deficiency, Factor V Leiden, Prothrombin 20210 mutation, or other known pro thrombotic condition.
    5.Regular, concomitant therapy with immunomodulating drugs (eg, intravenous immunoglobulin [IVIG], and routine systemic corticosteroids).
    6.Currently receiving treatment for acute bleeding episodes with APCC (eg, Factor Eight Inhibitor Bypass Agent [FEIBA]) and cannot substitute treatment with rFVIIa at a dose level of approximately 90 µg/kg for the duration of the study.
    7.Abnormal renal or hepatic function as defined by the following laboratory results at any time prior to Day 1:
    •Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels >3 times the upper limit of normal (ULN).
    •Total bilirubin level >2 mg/dL (>35 µmol/L).
    •Serum albumin < the lower limit of normal (LLN).
    •Serum creatinine level >1.25 times the ULN.
    8.Abnormal hematology values as defined by the following laboratory results at any time prior to Day 1:
    •Platelet count <100,000/µL.
    •Fibrinogen level < LLN.
    •Hemoglobin level <10 gm/dL.
    9.Abnormal coagulation activity as defined by the following laboratory results at any time prior to Day 1:
    •Prothrombin time (PT) >1.25 times the ULN.
    10.CD4 cell count ≤200/µL.
    11.Known hypersensitivity (eg. latex allergy) or allergic reaction to hamster protein.
    12.Known sensitivity to heparin or heparin induced thrombocytopenia.
    13.Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or subjects who are Pfizer employees, including their family members, directly involved in the conduct of the study.
    14.Participation in other studies involving investigational drug(s) (excluding PF 06741086) within 30 days prior to study entry and/or during study participation.
    15.Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.
    16.Fertile male subjects who are unwilling or unable to use a highly effective method of contraception as outlined in this protocol for the duration of the study and for at least 28 days after the last dose of investigational product.
    17.Had major surgery, as judged by the investigator, within 3 months prior to the study or have elective surgery planned during the study (applies only to de novo subjects).
    E.5 End points
    E.5.1Primary end point(s)
    •Frequency, severity and causal relationship of treatment emergent
    adverse events (TEAEs); Day 1 up to Day 393.
    •Frequency and magnitude of abnormal laboratory findings (including hematology, chemistry, urinalysis); Day 1 up to Day 393.
    •Changes from baseline in vital sign (blood pressure, pulse rate,
    temperature and respiration rate) measurements and physical
    examinations; Day 1 up to Day 393 and ECG Day 1 to Day 29 in de novo
    subjects only.
    •Frequency, severity and casual relationship of infusion and injection site reactions; Day 1 up to Day 393.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Timepoints are all listed in the list of primary endpoints.
    E.5.2Secondary end point(s)
    •Frequency and annualized rate of bleeding episodes; Day 1 up to Day 393.
    •Frequency of rescue (FVIII, or FIX) therapy for treatment of breakthrough bleeding episodes.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Timepoints are all listed in the list of secondary endpoints.
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    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 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.8.2.4Number of treatment arms in the trial4
    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 EEA6
    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
    Brazil
    Chile
    Croatia
    France
    Poland
    South Africa
    Spain
    Switzerland
    United States
    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
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months17
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months18
    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: 6
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 30
    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 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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    F.4.2.2In the whole clinical trial 36
    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
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-03-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-03-08
    P. End of Trial
    P.End of Trial StatusOngoing
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