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    Summary
    EudraCT Number:2019-001283-30
    Sponsor's Protocol Code Number:SPK-3006-101
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-01-20
    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 ConcernedGermany - PEI
    A.2EudraCT number2019-001283-30
    A.3Full title of the trial
    Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease (type II glycogen storage disease)
    A.4.1Sponsor's protocol code numberSPK-3006-101
    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
    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
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSpark Therapeutics
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address3737 Market Street, Suite 1300
    B.5.3.2Town/ cityPhiladelphia
    B.5.3.3Post codePA 19104
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrials@sparktx.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/3/19/2175
    D.3 Description of the IMP
    D.3.2Product code SPK-3006
    D.3.4Pharmaceutical form Suspension 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 INNSPK-3006
    D.3.9.2Current sponsor codeSPK-3006
    D.3.9.4EV Substance CodeSUB199354
    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
    Pompe Disease (also known as glycogen storage disease type II)
    E.1.1.1Medical condition in easily understood language
    Pompe Disease
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10053185
    E.1.2Term Glycogen storage disease type II
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate safety and tolerability of a single intravenous dose of SPK-3006 administered at escalating dose levels to participants with clinically moderate late-onset Pompe disease (LOPD).
    E.2.2Secondary objectives of the trial
    To evaluate potential efficacy and bioactivity of SPK-3006.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Be able to understand the purpose and risks of the study and provide
    signed and dated informed consent and authorization to use protected
    health information (PHI) in accordance with national and local privacy
    regulations;
    2. Are male or female ≥18 years of age with a confirmed diagnosis (e.g.,
    GAA genetic testing) of LOPD, or based on a documented deficiency of
    GAA enzyme activity;
    3. Have received a marketed ERT for at least the previous 24 months and
    maintained a stable dose, frequency and compliance for the past 6
    months with no dose variation and no longer improving on ERT;
    4. Have clinically moderate, LOPD characteristics;
    a. Able to walk ≥75 meters on the 6MWT (assistive devices permitted)
    but less than 500 meters assessed at two timepoints prior to initiating
    immunosuppression (Day -6) with <10% variance between the
    assessments. If the variance is ≥10%, a third timepoint will be
    collected;
    b. Percentage of the predicted FVC ≥30% and ≤80% in the upright
    position;
    5. Agree to use reliable contraception for a minimum of 6 months after
    administration of SPK-3006 or 12 weeks after the final dose of sirolimus,
    whichever occurs later. Female candidates of child-bearing potential
    must have a negative pregnancy test prior to initiating
    immunosuppression (Day -6) and on Day 0 prior to administration of
    SPK-3006. See Section 13 (Appendix 1) for guidance on reliable
    contraception and the definition of women of child-bearing potential
    (WOCBP);
    6. Agree to refrain from blood, plasma, platelets, egg or sperm and organ
    donation after receiving SPK-3006.
    E.4Principal exclusion criteria
    1. Active hepatitis B and/or C
    All candidates must be screened for both active hepatitis B and C,
    regardless of prior known history.
    a. Screening for hepatitis B
    All candidates must have a single sample at Screening for each of the
    following tests: hepatitis B surface antigen (HBsAg), total hepatitis B
    core antibody (anti-HBc), and a nucleic acid test for hepatitis B virus
    DNA (HBV-DNA viral assay)
    i. A candidate is not eligible if either HBsAg is positive or HBV-DNA is
    positive/detectable.
    ii. A candidate is eligible if the anti-HBc is positive and both HBsAg and
    HBV-DNA are negative, as this would be consistent with a prior infection
    of hepatitis B. Anti-HBc must be obtained in all candidates to
    discriminate between acute infection and possible reactivation of
    hepatitis B during the trial in candidates with no prior history of
    hepatitis B.
    iii. A candidate who is currently undergoing antiviral therapy for chronic
    hepatitis B is not eligible.
    b. Screening for hepatitis C
    i. A candidate who is currently undergoing antiviral therapy for chronic
    hepatitis C is not eligible.
    ii. All other candidates, including those who have never been treated or
    who have completed antiviral therapy for chronic hepatitis C must have a
    nucleic acid test for hepatitis C viral RNA (HCV-RNA single load assay) at
    Screening
    • A candidate is not eligible if the HCV-RNA load assay is
    positive/detectable.
    • Candidates treated with anti-viral therapy for chronic hepatitis C, must
    have completed anti-viral therapy at least 6 months prior to Screening
    and have a negative HCV-RNA at Screening.
    • Candidates with a documented or self-reported history of hepatitis C
    must have a single negative HCV-RNA at Screening
    2. Significant underlying liver disease. A candidate is not eligible if any
    of the following pre-existing diagnoses, which are indicative of
    significant underlying liver disease, are present in the medical record
    • Liver cirrhosis
    • Portal hypertension
    • Hepatic encephalopathy
    • Gamma-glutamyl transferase (GGT) >1.2X ULN adjusted for age and
    gender; or
    • Bilirubin >1.2X ULN adjusted for age and gender. Candidates with
    asymptomatic elevated bilirubin (e.g., Gilbert syndrome) can be
    considered after discussion with the Medical Monitor.
    All candidates who do not have the pre-existing diagnoses listed above
    must have the following assessments performed at Screening
    • Measurement of serum albumin. A candidate is not eligible if the serum
    albumin level is below the testing laboratory's lower limit of normal
    • Liver fibrosis = stage 3. The following results are indicative of fibrosis
    >= stage 3 and exclude the candidate from participation
    - FibroScan, with a score > 8.3 kPa units
    - FibroTest/ FibroSURE with a result > 0.48
    - AST-Platelet Ratio Index (APRI) >1;
    Of note, if results from more than one modality for evaluation of liver
    fibrosis are available (e.g., results from both FibroScan and FibroSURE
    are available) the result from FibroScan should be consulted for
    determination of liver fibrosis as it will take precedence over other
    modalities.
    3. Human immunodeficiency virus (HIV) infection
    4. Prior hypersensitivity to rhGAA
    5. Pre-existing AAV-Spark100 neutralizing antibody titers >1:1
    6. High titer antibody responses to rhGAA (anti-GAA >1:30,000)
    7. Participation in a clinical study with an investigational drug in the past
    6 months (vaccination studies are accepted; observation studies are
    accepted after discussion with the Medical Monitor);
    8. Requires any invasive ventilation or requires noninvasive ventilation
    while awake and upright;
    9. Any change to respiratory muscle strength training within 90 days
    prior to informed consent (for participants receiving respiratory muscle
    strength training) or initiation of respiratory muscle strength training
    prior to Day 0
    10. Received any prior vector or gene transfer agent
    11. Concomitant use of medication that is contraindicated with sirolimus
    (see Section 19.6.4.1), such as, medication known to be a strong or
    moderate inducer or inhibitor of CYP3A4 – excluded only during the
    sirolimus immunosuppressive period
    12. Live vaccines within 30 days prior to informed consent to at least 52
    weeks after receiving SPK-3006 infusion
    13. Use of systemic immunosuppressive agents (e.g., corticosteroids)
    within 30 days prior to informed consent and to Day 0 prior to SPK-3006
    administration
    14. Active malignancy (except non-melanoma skin cancer)
    15. History of liver cancer
    16. Pregnant or nursing women
    17. Any evidence of an active infection at the time of SPK-3006 infusion
    18. Known allergy or hypersensitivity to SPK-3006 investigational
    product or sirolimus
    19. Any concurrent clinically significant condition that would not allow
    the potential participant to complete the follow-up examinations during
    the course of the study, or other condition that, in the opinion of the
    Investigator and/or Medical Monitor, and/or Sponsor makes the
    candidate unsuitable for participation in the study
    E.5 End points
    E.5.1Primary end point(s)
    • Incidence of AEs and SAEs, clinically significant abnormal laboratory
    values, change in vital signs, change in PE, vector shedding in bodily
    fluids, liver function tests
    • Immune responses against the SPk100 capsid (neutralizing
    antibodies) and GAA transgene product (binding and neutralizing
    antibodies)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Evaluation of AEs and SAEs will be ongoing throughout the study.

    Laboratory assessments including shedding and liver function tests will occur as detailed in the schedule of assessments in protocol SPK-3006-101.
    E.5.2Secondary end point(s)
    • Changes from baseline in the Six-Minute Walk Test (6MWT)
    • Changes from baseline in the Forced Vital Capacity (FVC)
    • Peak and steady-state vector-derived GAA enzyme levels assessed by
    total GAA protein and activity measured in circulation
    • Biomarkers of muscle injury and glycogen accumulation (creatine
    kinase [CK], urine glucose tetrasaccharide [Hex 4])
    E.5.2.1Timepoint(s) of evaluation of this end point
    Designated assessments will be collected as detailed in the schedule of assessments in protocol SPK-3006-101.
    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 Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    E.7.1.1First administration to humans Yes
    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 Yes
    E.8.1.7.1Other trial design description
    Dose-escalation
    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 EEA14
    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
    United States
    Denmark
    France
    Germany
    Italy
    Netherlands
    Spain
    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 all phases of the study including the Month 60 (Year 5)/EOS
    visit procedures. The end of the study is defined as the date of the last
    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 years5
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months6
    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: 20
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    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 14
    F.4.2.2In the whole clinical trial 20
    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)
    SPK-3006 is currently being investigated as a single dose treatment. Once the trial is over and the defined long-term follow-up (LTFU) is complete, participants will continue to receive care for Pompe
    disease by their treating physician.
    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 Decision2020-08-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-07-22
    P. End of Trial
    P.End of Trial StatusOngoing
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