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    Summary
    EudraCT Number:2019-004451-37
    Sponsor's Protocol Code Number:C3731003
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2020-10-08
    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 ConcernedGermany - PEI
    A.2EudraCT number2019-004451-37
    A.3Full title of the trial
    Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants with Moderately Severe to Severe Hemophilia A (FVIII:C≤1%)
    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 VIII Gene Therapy With PF-07055480 in Adult Males With Moderately Severe to Severe Hemophilia A
    A.4.1Sponsor's protocol code numberC3731003
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04370054
    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+1 800 7181021
    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/3/17/1874
    D.3 Description of the IMP
    D.3.1Product namegiroctocogene fitelparvovec
    D.3.2Product code PF-07055480
    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 INNgiroctocogene fitelparvovec
    D.3.9.2Current sponsor codePF-07055480
    D.3.9.4EV Substance CodeSUB203742
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1E13
    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
    hemophilia A
    E.1.1.1Medical condition in easily understood language
    inherited condition caused by a lack of Factor VIII which is required for blood to clot and stop any bleeding
    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 10060613
    E.1.2Term Hemophilia A (Factor VIII)
    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
    Evaluate the efficacy of a single infusion of PF-07055480 in participants ≥18 and <65 years of age with moderately severe to severe hemophilia A (FVIII C ≤ 1%).
    E.2.2Secondary objectives of the trial
    - To demonstrate that the use of exogenous Factor VIII (FVIII) is significantly reduced post PF-07055480 infusion.
    - To assess additional efficacy parameters post PF-07055480 infusion including FVIII activity level, use of exogenous FVIII, information on bleeding events and patient-reported outcomes (PROs).
    - Estimate the durability of efficacy up to 5 years after PF-07055480 infusion.
    - To estimate the safety and tolerability of PF-07055480, including immunogenicity, for the study duration of 5 years after PF-07055480 infusion.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    - Optional vector shedding sub-study, 10 Dec 2019, Version 1: To further characterize the kinetics of vector shedding.

    - Optional ultrasounds of the joints sub-study, 10 Dec 2019, Version 1: To compare and evaluate joint health post PF-07055480 infusion to baseline via ultrasounds.

    - Optional X-rays of the joints sub-study, 11 Aug 2020, Version 1: To compare joint health post PF-07055480 infusion to baseline and evaluate long-term joint outcomes via X-rays.
    E.3Principal inclusion criteria
    • Males who have been followed on routine Factor VIII prophylaxis therapy in the lead-in study (C0371004) and have ≥ 150 documented exposure days to a Factor VIII protein product
    • Moderately severe to severe hemophilia A (Factor VIII activity ≤ 1%)
    • Suspension of FVIII prophylaxis therapy post study drug infusion
    E.4Principal exclusion criteria
    • Anti-AAV6 neutralizing antibodies
    • History of inhibitor to Factor VIII
    • Laboratory values at screening visit that are abnormal or outside acceptable study limits
    • Significant and/or unstable liver disease, biliary disease, significant liver fibrosis
    • Planned surgical procedure requiring Factor VIII surgical prophylactic factor treatment 12 months from screening visit
    • Active hepatitis B or C
    •Serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with either Cluster of Differentiation 4 positive (CD4+) cell count ≤200 mm3 or viral load >20 copies/mL
    E.5 End points
    E.5.1Primary end point(s)
    Total annualized bleeding rate (ABR, spontaneous and traumatic bleedings, treated and untreated) from Week 12 through 15 months
    following PF-07055480 infusion versus Total ABR on prior Factor VIII
    (FVIII) prophylaxis replacement regimen.
    E.5.1.1Timepoint(s) of evaluation of this end point
    from Week 12 to 15 months (primary analysis) post study drug infusion
    E.5.2Secondary end point(s)
    - Factor VIII (FVIII) activity level >5% at 15 months following infusion
    of PF-07055480.
    - ABR (spontaneous and traumatic treated bleedings) from Week 12
    through 15 months following PF-07055480 infusion versus ABR on prior FVIII prophylaxis replacement regimen.
    - Annualized infusion rate (AIR) of exogenous FVIII from Week 12
    through 15 months following infusion of PF-07055480 versus AIR on
    prior FVIII prophylaxis replacement regimen.
    - FVIII activity level from Week 12 through 15 months following infusion of PF-07055480.

    - The following secondary parameters will be assessed from Week 12
    through 15 months after PF-07055480 infusion and compared with prior FVIII prophylaxis replacement regimen:
    • Annualized FVIII consumption.
    • Annualized bleeding rate (ABR) of specific type:
    o by cause (spontaneous or traumatic)
    o by location (in joints, in target joints, or in soft tissue).
    • Total ABR by cause and by location.
    • Percentage of participants without bleeds.

    - The following secondary parameters will be assessed by visit through 15 months after PF-07055480 infusion:
    • FVIII activity level.
    • Change from baseline in joint health as measured by the HJHS instrument.
    • Change from baseline in the following patient-reported outcome
    (PRO) endpoints:
    o Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)
    o Haemophilia Activities List (HAL).

    - The following parameters will be analysed yearly or by visit as
    appropriate:
    • ABR.
    • FVIII activity level.
    • AIR of exogenous FVIII.
    • Annualized FVIII consumption.
    • ABR of specific type:
    o by cause (spontaneous or traumatic).
    o by location (in joint, in target joints, or in soft tissue).
    • Total ABR.
    • Total ABR by cause and by location.
    • Percentage of participants without bleeds.
    • Change from baseline in joint health as measured by the HJHS
    instrument.
    • Change from baseline in PRO endpoints: Haem-A-QoL and HAL.

    In addition, ABR, Total ABR, and AIR will be analyzed throughout the 5 year study period.

    - Incidence and severity of adverse events (AEs).
    - Events of special interest (such as hypersensitivity reactions, clinically reported thrombotic events, and malignancy).
    - Immunogenicity:
    • Antibodies against adeno-associated virus 6 (AAV6) capsid protein
    (neutralizing antibodies [nAbs] and anti-drug antibodies [ADAs]).
    • T-cell responses against AAV6 capsid and against the transgene.
    • FVIII inhibitors.
    E.5.2.1Timepoint(s) of evaluation of this end point
    -FVIII activity level (proportion of participants >5%): at 15 months.
    -Total ABR: yearly and up to 5 years.
    -ABR: from Week 12 through 15 months, then yearly and up to 5 years.
    -FVIII activity level: from Week 12 through 15 months, then yearly and at specified visits.
    -AIR of exogenous FVIII, annualized FVIII consumption, percentage of
    participants without bleeds, ABR of specific type (by cause and by
    location), Total ABR by cause and by location: from Week 12 through 15 months, then yearly and up to 5 years.
    -Change from baseline in joint health as measured by the HJHS
    instrument, change from baseline in PRO endpoints (Haem-A-QoL and HAL): by visit through 15 months and up to 5 years.
    - Incidence and severity of AEs, events of special interest, immunogenicity: Up to 5 years.
    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 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 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 EEA11
    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
    Japan
    Korea, Republic of
    Saudi Arabia
    Taiwan
    United States
    France
    Sweden
    Spain
    Germany
    Greece
    Italy
    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
    A participant is considered to have completed the study if he/she has completed the entire study including end of study (EOS) visit (Week 260).
    The end of the study is defined as the date of the EOS visit of the last participant in the study.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years7
    E.8.9.2In all countries concerned by the trial months2
    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: 63
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 22
    F.4.2.2In the whole clinical trial 63
    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)
    No difference from the expected normal treatment of that condition.

    Please note that participants exiting C3731003 will be invited to enter a separate study to assess the long-term safety and effectiveness of giroctocogene fitelparvovec for the treatment of hemophilia A for a total of up to 10 additional years, corresponding to up to 15 years post-giroctocogene fitelparvovec infusion.
    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 Decision2021-09-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-05-14
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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