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    Summary
    EudraCT Number:2014-004155-32
    Sponsor's Protocol Code Number:3090A1-301
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2015-04-06
    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 number2014-004155-32
    A.3Full title of the trial
    An Open-Label, Single-Arm, Safety and Efficacy Study of Recombinant Human Factor IX (rFIX; BeneFIX ) in Children Less Than 6 Years of Age With Severe Hemophilia B
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study Evaluating rFIX; BeneFIX in Severe Hemophilia B
    A.4.1Sponsor's protocol code number3090A1-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00037557
    A.5.4Other Identifiers
    Name:AliasNumber:B1821035
    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 SponsorWyeth Research
    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 supportWyeth Research
    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
    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.5.4Telephone number18007181021
    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 Limited
    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 solution 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.3.9.2Current sponsor code3090A1
    D.3.9.3Other descriptive namerecombinant coagulation factor IX
    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.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNONACOG ALFA
    D.3.9.1CAS number 181054-95-5
    D.3.9.2Current sponsor code3090A1
    D.3.9.3Other descriptive namerecombinant coagulation factor IX
    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 number500
    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 coagulation factor IX, (nonacog alfa), which is a recombinant DNA-based protein therapeutic which has structural and functional characteristics comparable to endogenous factor IX.
    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 B
    E.1.1.1Medical condition in easily understood language
    Disease in which clotting factor IX (FIX) is deficient or inactive. Subjects with this condition have an increased risk to bleed compared to unaffected individuals. Hemophilia B
    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 10060614
    E.1.2Term Hemophilia B (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 characterize the safety and efficacy of rFIX in children less than 6 years of age with severe hemophilia B in the setting of acute bleeding episodes, prophylaxis, and/or surgery.
    E.2.2Secondary objectives of the trial
    To measure the incremental recovery of rFIX in children following a 75 international unit (IU) per kilogram(kg) bolus infusion.
    To describe the immunogenicity/neoantigenicity of rFIX in patients regardless of previous FIX treatment by monitoring for inhibitor development (BIA) during the trial.
    To perform surveillance for other events of interest: thrombogenicity, hemorrhage/lack of effect, allergic-type manifestations, and red blood cell (RBC) agglutination.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Severe hemophilia B (plasma FIX activity level [FIX:C] less than or equal to [=<]1 percent [%]).
    2. Less than 5 years of age (in order to complete study treatment before age 6).
    3. In the investigator’s judgment, the subject and/or caregiver will be compliant to study procedures.
    4. The parent or legal guardian has voluntarily signed and dated written informed consent before any screening procedures.
    E.4Principal exclusion criteria
    1. The subject has a currently detectable FIX inhibitor (or history of an inhibitor) defined as greater than or equal to [>=] 0.6 Bethesda Units (BU). A family history of inhibitors will not exclude the subjects.
    2. Impaired hepatic function defined as greater than (>) 2.5 * Upper limit of normal (ULN) of Alanine aminotransferase (ALT), Aspartate aminotransferase (AST) and/or total bilirubin, except
    in documented hyperbilirubinemia of the newborn.
    3. Impaired renal function (serum creatinine >1.25 *ULN).
    4. Albumin less than (<) Lower limit of normal (LLN).
    5. Platelet count <100,000 per millimetre cube (mm^3).
    6. Abnormal prothrombin time (>1.25 * ULN).
    7. Known hypersensitivity to protein products or agents related to the test article, eg, hamster proteins.
    8. Documented HIV infection; or active infection with hepatitis.
    9. Any genetic coagulation disorder other than hemophilia B (with the exception of vitamin K-dependent coagulation deficiency of the newborn).
    10. Received any investigational drug within 30 days before entering the study.
    11. Any condition (medical or social) that, in the investigator's judgment, makes participation in the study not advisable.
    E.5 End points
    E.5.1Primary end point(s)
    1)Dose of Recombinant Human Factor IX (rFIX)
    2) Number of Recombinant Human Factor IX (rFIX) Infusions
    3) Response to Treatment
    4) Dose of Breakthrough Bleeding Episodes per 30-day Period During Prophylaxis Therapy
    5) Number of Infusions of Breakthrough Bleeding Episodes per 30-day Period During Prophylaxis Therapy
    6) Number of Breakthrough Bleeding Episodes per 30-day Period During Prophylaxis Therapy
    7) Dose of rFIX During the Operative Period
    8) Response of rFIX During the Operative Period
    9) Physician’s Global Assessment of Efficacy
    10) Overall Number of Subjects With Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs)
    E.5.1.1Timepoint(s) of evaluation of this end point
    1) Baseline up to Month 12
    2) Baseline up to Month 12
    3) 24 Hours after infusion
    4) Baseline up to Month 12
    5) Baseline up to Month 12
    6) Baseline up to Month 12
    7) Before preoperative rFIX infusion up to between 15 to 30 minutes following completion of the infusion
    8) Before preoperative rFIX infusion up to between 15 to 30 minutes following completion of the infusion
    9) Month 1 up to 12
    10) Baseline up to Month 12
    E.5.2Secondary end point(s)
    None
    E.5.2.1Timepoint(s) of evaluation of this end point
    None
    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 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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 No
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    France
    Spain
    Sweden
    United Kingdom
    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.8.9.2In all countries concerned by the trial years5
    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: 25
    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: 15
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    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 under 6 years of age
    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 25
    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: United States
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