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    EudraCT Number:2019-001283-30
    Sponsor's Protocol Code Number:SPK-3006-101
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-11-08
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    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
    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. cell therapy medicinal product No
    D. therapy medical product Yes
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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. 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. Males and Females ≥18 years of age with a confirmed diagnosis (i.e. genetic testing) of late-onset Pompe disease, based on a documented deficiency of GAA enzyme activity and GAA genotyping;
    3. Received 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;
    4. Have clinically moderate, late-onset Pompe disease characteristics;
    a. Able to walk ≥75 meters on the 6-Minute Walk Test (assistive devices permitted) but less than 500 meters assessed at 2 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 6 months after administration of SPK-3006. 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
    6. Agree to refrain from blood, plasma, platelets, egg or sperm donation during the study period
    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: HBsAg (Hepatitis B surface antigen), anti-HBc (Total Hepatitis B core antibody), and an HBV-DNA viral assay (nucleic acid test for hepatitis B virus DNA).
    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:
    iv. A candidate who is currently undergoing antiviral therapy for chronic hepatitis C is not eligible.
    v. All other candidates, including those who have never been treated or who have completed antiviral therapy for chronic hepatitis C must have a single HCV-RNA load assay (also referred to as a nucleic acid test [NAT] for HCV RNA) at screening.
    • A candidate is not eligible if his 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 the time of screening.
    • Candidates with a documented or self-reported history of HCV must have a single negative HCV-RNA at time of 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; or
    • Hepatic encephalopathy
    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 infection;
    4. Prior hypersensitivity to GAA;
    5. Pre-existing anti-AAV neutralizing antibody titers >1:1;
    6. High titer antibody responses to rhGAA (anti-GAA >1:30,000);
    7. Participation in a clinical study with investigational drug in the past six months (vaccination studies are accepted; observation studies are accepted after discussion with the sponsor 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 of the protocol), 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 and for the duration of the study;
    13. Use of systemic immunosuppressive agents (e.g., corticosteroids) within 30 days prior to informed consent and after informed consent 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 Sponsor, makes the candidate unsuitable for participation in the study;
    20. Unable or unwilling to comply with the visit schedule and study assessments described in the clinical protocol.
    E.5 End points
    E.5.1Primary end point(s)
    • Incidence of adverse and serious adverse events (AEs/SAES), clinically significant abnormal laboratory values, change in vital signs, change in physical examination, vector shedding in bodily fluids, liver function tests
    • Immune responses against AAV capsid and GAA transgene product
    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 plasma
    • Biomarkers of muscle injury and glycogen accumulation (CK, Urine 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. 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. trial design description
    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 EEA12
    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 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
    The end of the study is defined as the date of the last visit of the last participant in the study (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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    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: 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)
    When the trial is over, participants continue to receive care for Pompe disease by their treating physician. A long-term follow-up (LTFU) study for up to an additional 4 years observation is planned for all participants dosed with SPK-3006. After the observation period in SPK-3006-101, participants will be asked to participate in the LTFU study (under separate protocol and informed consent). If an LTFU study is not enrolling at a planned W52 visit, the participant may remain in SPK-3006-101.
    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 Decision2020-05-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-01-06
    P. End of Trial
    P.End of Trial StatusOngoing
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