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    Summary
    EudraCT Number:2019-000447-26
    Sponsor's Protocol Code Number:SPK-8011-301
    National Competent Authority:Finland - Fimea
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2019-08-29
    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 ConcernedFinland - Fimea
    A.2EudraCT number2019-000447-26
    A.3Full title of the trial
    An Open-label, Non-investigational Product, Multi-center, Lead-in
    Study to Evaluate Prospective Bleeding and Infusion Data of Current
    FVIII Replacement Therapy in Adult Males with Hemophilia A
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An observational Study in Males with Hemophilia A
    A.4.1Sponsor's protocol code numberSPK-8011-301
    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 SponsorSpark Therapeutics, 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 supportSpark Therapeutics, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSpark Therapeutics, Inc
    B.5.2Functional name of contact pointSr. Clinical Project Manager
    B.5.3 Address:
    B.5.3.1Street Address3737 Market St
    B.5.3.2Town/ cityPhiladelphia
    B.5.3.3Post codePA 19104
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1215309-4101
    B.5.6E-mailmarcelyne.joseney-antoine@sparktx.com
    D. IMP Identification
    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
    Hemophilia A
    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 of this lead-in study is to establish the frequency of bleeding events in patients with severe hemophilia A receiving prophylactic or on demand infusions of factor VIII replacement therapy as standard of care. In addition, key characteristics of the bleeding events and FVIII infusions, as well as patient reported
    utcomes and medically important adverse events, will also be collected.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Able and willing to provide written informed consent
    2. Males ≥18 years of age
    3. Clinically severe hemophilia A, defined as:
    a. <1% (<1 IU/dL) endogenous FVIII activity levels as documented by a certified laboratory; OR
    b. 1-2% (1-2 IU/dL) endogenous FVIII activity levels and > 10 bleeding events per year
    (in the last 52 weeks prior to screening); OR
    c. 1-2% (1-2 IU/dL) endogenous FVIII activity levels and on regular continuous prophylaxis, defined as the intent to treat for 52 weeks/year and receiving a minimum of an a priori defined frequency of infusions for at least 45 weeks (85%) of the year under consideration
    4. Participants on a prophylaxis regimen must have been on FVIII replacement therapy for a minimum of 2 months prior to Screening with the intention to remain on trophylaxis regimen for the
    duration of the study
    5. Previous exposure to FVIII therapy (i.e., ≥ 150 documented exposure days to a FVIII protein product such as recombinant, plasmaderived or extended half-life FVIII product)
    6. No prior history of hypersensitivity or anaphylaxis associated with any FVIII or intravenous immunoglobulin administration
    7. No measurable inhibitor against FVIII at Screening or within the past 24 months, (i.e., < 0.6 Bethesda Units); no confirmed history of clinically significant FVIII inhibitor and no clinical signs or symptoms of decreased response to FVIII administration (Note: Family history of inhibitors is not exclusionary, nor will documentation of a single measurement of Bethesda titer of >0.6 BU that is not accompanied by clinical evidence of failure to respond to infused FVIII concentrate)
    8. Willing to consider participation and treatment in a future Spark hemophilia A gene therapy study.
    E.4Principal exclusion criteria
    1. Documented active hepatitis B or C within the past 12 months of Screening, as defined by:
    a. Hepatitis B with positive results of Hepatitis B surface Antigen (HBsAg) or detectable HBV-DNA viral load.
    i. Participants with positive HBsAB (evidence of vaccination) or positive HBcAB (previous exposure to Hep B virus) with a negative viral load are eligible
    b. Documented hepatitis C positive antibody (anti-HCV) with a detectable HCV-RNA viral load.
    i. Subjects with a negative anti-HCV or positive anti-HCV with a negative HCV-RNA viral load are eligible
    All participants, except for those with historical results within the past 12 months of screening, will be required to undergo a diagnostic valuation of hepatitis status to participate in the study.
    2. Currently on antiviral therapy to treat their hepatitis B or C;
    3. Documented diagnoses of significant liver disease within the past 6 months of
    screening, as defined by:
    • Portal hypertension, or
    • Chronic, persistent splenomegaly or
    • Hepatic encephalopathy, or
    Any participant without any of the pre-existing diagnoses above must have performed
    either a Fibroscan or a combination of Fibrotest & APRI, within 6 months prior to
    screening or at Screening:
    i. FibroScan, with a score >8.8kPa units
    ii. FibroTest/FibroSURE with a result >0.48
    iii. AST-Platelet Ratio Index (APRI) >1).
    4. Have serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm3.
    a. Participants who are HIV-positive and on current antiretroviral drug regimen for
    12 weeks, with a CD4 count (>200/mm3) and undetectable viral load (<50 gc/mL) at Screening, are eligible to enroll;
    b. Participants who are HIV-negative with historical results within 12 months prior to Screening will not be required to undergo diagnostic evaluation of HIV status to participate in the study.
    c. Participants without historical results within 12 months of screening will be required to undergo HIV screening to determine their HIV status prior to participation in the study.
    5. Anti-AAV-Spark 200 neutralizing antibody titers >1:1;
    6. Previously received a Sparkhemophilia A gene therapy product
    7. Previously dosed with any investigational or approved gene therapy product at any time
    8. Previously treated with an investigational drug within the last 12 weeks;
    9. Planned surgical procedure in the next 12 months requiring FVIII prophylactic
    treatment.
    10. Any history of chronic infection or other chronic disease, concurrent clinically significant major disease (such as liver abnormalities or type I diabetes) including active malignancy, except for non-melanoma skin cancer, any other condition or any other unspecified reasons that, in the opinion of the Investigator or Sponsor, makes the participant unsuitable for participation and dosing in a future clinical study for Spark's hemophilia A gene therapy.
    11. Unable or unwilling to comply with the schedule of visits and/or study assessments described in the clinical protocol.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy Endpoints:
    • Number of bleeding events, annualized (Annualized bleeding rate [ABR])
    • Location and severity of bleeds
    • Bleed type: spontaneous or traumatic
    • Number of treated and untreated bleeds
    • Number of bleeds into known any joints as well as bleeding into target joints, annualized
    • Number of prophylactic FVIII infusions per week
    • Dose and total FVIII consumption
    • Number of on-demand FVIII infusions required over the study duration
    • Annualized number of infusions (AIR)

    Safety Endpoints:
    • Incidence of medically important adverse events and serious adverse events

    Exploratory Endpoints:
    • Activities assessments
    • Quality-of-life assessments
    • Health-economic parameters
    E.5.1.1Timepoint(s) of evaluation of this end point
    All participants will be included in all data summaries to the extent that a participant provides Screening visit data and data from at least one post-screening evaluation.
    Number of FVIII infusions per participant will be calculated, dose level and FVIII products as well as annual FVIII consumption and bleeding events per participant. These will be used to calculate the annualized infusion rate and annualized bleeding rate.
    The target joint assessments, level of activity, quality of life and health-economic parameters will be summarized according to recommendations and guidelines cited in the literature. In general, this consists of summarizing individual items scores and a total, mean or otherwise-defined composite score across all and/or a defined subset of item scores.
    E.5.2Secondary end point(s)
    Not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    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 No
    E.6.6Pharmacokinetic No
    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 Yes
    E.6.13.1Other scope of the trial description
    This observational study is to evaluate prospectively the frequency and type of bleeding events occurring in patients with hemophilia A receiving prophylactic or on demand infusions of factor VIII replacement therapy as the standard of care. In addition, key characteristics of the bleeding events and FVIII infusions, as well as patient reported outcomes and medically important adverse events, will also be collected.
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Observational study without IMP
    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 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 EEA20
    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
    Austria
    Belgium
    Brazil
    Canada
    Finland
    France
    Germany
    Ireland
    Israel
    Italy
    South Africa
    Sweden
    Thailand
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    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: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 20
    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)
    This is an observational study which subjects will continuous their own standard of care during study. After subject has ended the participation in the trial, subject can continuous their own standard of care provided by their healthcare provider. The Sponsor may be conducting another clinical trial in the future that will test the effectiveness of an investigational therapy on treating hemophilia A (dosing study). Eligible patients can be enrolled in that study.
    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 Decision2019-10-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-23
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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