Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43851   clinical trials with a EudraCT protocol, of which   7283   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2018-003086-33
    Sponsor's Protocol Code Number:C0371002
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-08-20
    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 ConcernedUK - MHRA
    A.2EudraCT number2018-003086-33
    A.3Full title of the trial
    Phase 3, open label, single arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-Padua) in adult male participants with moderately severe to severe hemophilia B (FIX:C <=2%) (BeneGene-2)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2)
    A.3.2Name or abbreviated title of the trial where available
    BeneGene-2
    A.4.1Sponsor's protocol code numberC0371002
    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 Centre
    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+18007181021
    B.5.6E-mailClinicalTrials.gov_Inquiries@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/3/18/2090
    D.3 Description of the IMP
    D.3.1Product namerAAV-Spark100-hFIX-Padua
    D.3.2Product code PF-06838435
    D.3.4Pharmaceutical form Solution for infusion
    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 INNFIDANACOGENE ELAPARVOVEC
    D.3.9.2Current sponsor codePF-06838435 (SPK-9001)
    D.3.9.4EV Substance CodeSUB193007
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3E13
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    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 to moderately severe hemophilia B <=2%
    E.1.1.1Medical condition in easily understood language
    Hemophilia B, or Christmas disease, results from a deficiency of blood coagulation 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 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 demonstrate the efficacy of a single infusion of PF-06838435 in male participants >=18 years of age with moderately severe to severe hemophilia B (FIX:C <=2%).
    E.2.2Secondary objectives of the trial
    - To demonstrate that the use of exogenous FIX is significantly reduced post PF-06838435 infusion.
    - To compare additional efficacy parameters post PF-06838435 infusion to baseline including use of exogenous FIX, information on bleeding events, and patient reported outcomes
    - Safety and tolerability of PF-06838435
    - Efficacy assessment over the 6 years of the study.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Participants must have completed at least 6 months of routine FIX prophylaxis
    therapy during the lead-in study (C0371004) prior to providing consent at the screening visit for this study.
    2. Participants who have documented moderately severe to severe hemophilia B, defined as
    FIX:C <=2%.
    3. Participants must agree to suspend prophylaxis therapy for hemophilia B after
    administration of the IP. FIX replacement therapy is allowed as needed
    4. Acceptable screening laboratory values as follows:
    Hemoglobin >=11 g/dL;
    Platelets >=100,000 cells/mL;
    Creatinine ≤ 2.0 mg/dL.
    5. Sex: Male. Contraceptive use by men should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
    Male participants are eligible to participate if they agree to the following for at least
    time required for 3 consecutive ejaculate samples to test negative for vector shedding: Refrain from donating sperm. PLUS either: Be abstinent from heterosexual or homosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent.
    OR Must agree to use contraception/barrier as detailed below:
    Agree to use male condom when engaging in any activity that allows for
    passage of ejaculate to another person
    6. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
    E.4Principal exclusion criteria
    1. Anti-AAV-Spark100 neutralizing antibodies (nAb) titer≥1:1 (i.e., positive for nAb), performed by a central laboratory during screening.
    2. Prior history of inhibitor to FIX or positive inhibitor testing as measured by the
    central laboratory >=0.6 Bethesda Units (BU) during screening. Clinical signs or
    symptoms of decreased response to FIX.
    3. Known hypersensitivity to FIX replacement product or intravenous immunoglobulin administration.
    4. History of chronic infection or other chronic disease that investigator deems as an
    unacceptable risk.
    5. Any concurrent clinically significant major disease or condition that the investigator deems unsuitable for participation or other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal deation or behavior (including alcoholism) or laboratory abnormality that may ncrease the risk associated with study participation or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
    6. Alanine transaminase (ALT), aspartate aminotransferase (AST), alkaline phosphatase(ALP) >2x upper limit of normal (ULN)based on central laboratory results.
    7. Bilirubin >1.5xULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is
    fractionated and direct bilirubin <35%) based on central laboratory results.
    8. Current unstable liver or biliary disease per investigator assessment defined by the presence of ascites, hepatic encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis. NOTE: Stable chronic liver disease including Gilbert's syndrome, asymptomatic gallstones, is acceptable if the participant otherwise meets entry criteria). Note: Participants who have a central laboratory test value that is outside the range specified by the exclusion criteria may have the test repeated, by the central laboratory, to determine eligibility; however, the result must be available prior to Baseline Visit /Visit 2.
    9. Currently on antiviral therapy for hepatitis B or C.
    10. Any participants with a planned surgical procedure requiring FIX surgical prophylactic
    factor treatment in the next 12 months.
    11. Participants using therapies that are restricted. See Section 6.5.2 for therapies not
    allowed during study participation.
    12. Previously dosed in a gene therapy research trial at any time or in an interventional clinical study within the last 12 weeks, excluding participation in study C0371004.
    13. Active hepatitis B or C; HBsAg, HBV-DNA positivity, or HCV-RNA positivity.
    14. Significant liver disease, as defined by pre-existing diagnosis of portal hypertension, splenomegaly or hepatic encephalopathy. Additionally during screening, a serum albumin level below normal limits and/or significant liver fibrosis by any of the following diagnostic modalities: FibroScan score >8 kPa units, Fibro Test/FibroSURE >0.48 or AST-to-Platelet Ratio Index (APRI) >1.In the investigator's opinion, if there is concern regarding the FibroTest results due to a confounding medical history (e.g., proteinuria can impact FibroTest result), the investigator can perform a different assessment of liver fibrosis (e.g., FibroScan or APRI) during the screening period.*Please note: if a participant has a known history of Gilbert's syndrome, a FibroTest cannot be used for fibrosis testing. However, the participant could be tested using FibroScan or APRI.
    15. Serological evidence of HIV-1 or HIV-2 with CD4+ cell count ≤200 mm3 and/or viral load >20 copies/mL.
    16. 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 participants who are Pfizer employees, including their family members, directly
    involved in the conduct of the study.
    17. Unable to comply with scheduled visits, treatment plan, laboratory tests and other study procedures for up to six years post infusion of PF-06838435 in the
    investigator’s judgement.
    18. Sensitivity to heparin or heparin-induced thrombocytopenia.
    19. Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the investigator or the sponsor’s Medical Monitor,contraindicates participation in the study.
    E.5 End points
    E.5.1Primary end point(s)
    •ABR (first 12 month post infusion of PF-06838435) will be compared to pre infusion of PF-06838435 (under SOC FIX prophylaxis replacement regimen).
    •Steady state FIX:C (Week 12 to Month 12) will be compared to a threshold of 5%.
    E.5.1.1Timepoint(s) of evaluation of this end point
    month 12
    E.5.2Secondary end point(s)
    •AIR for FIX at first 12 month post infusion of PF-06838435 vs AIR during SOC FIX replacement regimen will be tested for superiority.
    •FIX consumption, first 12 months post infusion of PF-06838435, similar method as applied to AIR will used to do hypothesis testing on this endpoint.
    •Number of bleeding events of specific type first 12 months post infusion of PF- 06838435: spontaneous and traumatic, and untreated. Similar method applied to ABR will be used to test these endpoints.
    •Frequency of target joint bleeds first 12 months post infusion of PF- 06838435 will be summarized.
    •HJHS for first 12 months post infusion of PF-06838435 will be compared to baseline
    •PROs endpoints (Haem A Qol, Physical Health domain, HAL, Complex Lower Extremity Activities Component Score) 12 month post infusion of PF-06838435 will be summarized, and compared/to baseline to assess the improvement from baseline if baseline is available.
    E.5.2.1Timepoint(s) of evaluation of this end point
    first 12 months post infusion
    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 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 Yes
    E.6.11Pharmacogenomic Yes
    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 Yes
    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 Yes
    E.8.2.3.1Comparator description
    C0371004 lead-in observational study
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    Australia
    Brazil
    Canada
    France
    Germany
    Greece
    Israel
    Japan
    Saudi Arabia
    Sweden
    Taiwan
    Turkey
    United Kingdom
    United States
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 50
    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 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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 50
    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 Decision2019-11-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-01-28
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat Apr 20 01:05:15 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA