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    Summary
    EudraCT Number:2019-000667-24
    Sponsor's Protocol Code Number:ST-920-201
    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:2019-03-19
    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 number2019-000667-24
    A.3Full title of the trial
    A Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase I/II Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects with Fabry Disease
    A.4.1Sponsor's protocol code numberST-920-201
    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 SponsorSangamo 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 supportSangamo Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSangamo Therapeutics, Inc.
    B.5.2Functional name of contact pointST-920-201 Information Desk
    B.5.3 Address:
    B.5.3.1Street Address7000 Marina Blvd.
    B.5.3.2Town/ cityBrisbane, CA
    B.5.3.3Post code94005
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16282527545
    B.5.5Fax number+15103237388
    B.5.6E-mailST920201-Notifcation@sangamo.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameRecombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A
    D.3.2Product code ST-920
    D.3.4Pharmaceutical form Solution for injection/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 INNNot yet assigned
    D.3.9.2Current sponsor codeST-920
    D.3.9.3Other descriptive nameAdeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number5000000000000 to 25000000000000
    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 No
    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
    Fabry Disease (X-linked lysosomal storage disease)
    E.1.1.1Medical condition in easily understood language
    Fabry disease
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10016016
    E.1.2Term Fabry's disease
    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 assess the safety and tolerability of ST-920
    E.2.2Secondary objectives of the trial
    Secondary Objectives:
    •To assess the pharmacodynamics of α-Gal A and the presence of its substrates in plasma over time
    •To assess impact of ST-920 on ERT administration required for subjects on ERT
    •To assess the impact of ST-920 on renal function
    •To evaluate ST-920 vector DNA shedding over time
    Exploratory Objectives:
    • To assess clinical impact of ST-920 on classical Fabry disease
    • To assess the pharmacodynamics of α-Gal A and the presence of its substrates in urine and tissue over time
    • To assess the pharmacokinetics of α-Gal A over time
    • To assess immune response to rAAV2/6 and α-Gal A
    • To evaluate the impact of pre-existing antibodies to α-Gal A on the enzymatic activity levels of α-Gal A produced by ST-920
    • To assess the impact of ST-920 on substrate deposition in the kidney (via renal biopsy) in ERT-naïve and ERT-pseudo-naïve (defined as not having received ERT treatment during the 6 months prior to consent) subjects
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all of the following criteria to be included in the study:
    1) Subjects with documented diagnosis of classical Fabry disease as defined by <5% α-Gal A activity in either plasma or leukocytes and one or more of the following symptomatic characteristics of classical Fabry disease: i) cornea verticillata, ii) acroparesthesia, iii) anhidrosis, iv) angiokeratoma.
    For subjects who do not have a documented diagnostic α-Gal A activity level, a blood sample should be taken to measure α-Gal A activity levels (in plasma and/or leukocytes).
    For those subjects who are on ERT, this blood draw must be taken at least 13 days after their last ERT infusion (trough).
    i. If the subject’s level of α-Gal A activity is > 5% and the subject is on ERT, this level of enzyme activity may be due to residual α-Gal A activity from the last ERT infusion. In this case, the diagnosis of classical Fabry disease may be confirmed if the following three criteria are fulfilled:
    a. two or more of the following documented symptomatic characteristics of classical Fabry: cornea verticillata, acroparesthesia, anhidrosis, angiokeratoma. If there is documented clustered periumbilicial angiokeratoma, this symptom alone is sufficient as it is a pathognomonic sign of classical Fabry disease;
    b. a mutation that is indicative of classical Fabry (i.e. listed in a database, such as http://dbfgp.org); and
    c. the α-Gal A activity at trough is below the lower limit of the normal range of the assay.
    2. Subjects who are on ERT (14 days [± 3 days] regimen); or are ERT-naïve; or are ERT-pseudo-naïve (defined as not having received ERT treatment in the 6 months prior to consent).
    3. For subjects receiving ERT, ERT must have been administered at a stable dose for at least 6 months (defined as not having missed more than 3 doses of ERT during the 6 months prior to consent) and regimen (14 days ± 3 days for at least 3 months prior to enrollment).
    4. Male subjects ≥ 18 years of age
    5. Sexually mature subjects must agree to use a condom and refrain from sperm donation from the time of ST-920 administration until a minimum of 3 consecutive semen samples are negative for rAAV2/6 after administration of ST-920 and a minimum of 90 days after ST-920 administration
    6. Signed, written informed consent of the subject
    E.4Principal exclusion criteria
    Subjects who meet any of the following criteria will be excluded from participating in the study:
    1. Known to be unresponsive to ERT in the opinion of the Site Investigator and Medical Monitor (e.g., no documented substrate level decrease on ERT)
    2. Current treatment with migalastat (Galafold™) or prior treatment within 3 months of informed consent
    3. Positive neutralizing antibody response to AAV6
    4. Intercurrent illness expected to impair evaluation of safety or efficacy during the observation period of the study in the opinion of the Site Investigator or Medical Monitor
    5. eGFR ≤ 60 ml/min/1.73m^2
    6. New York Heart Association Class III or higher
    7. Active infection with hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) or an infection with tuberculosis (TB)
    8. History of liver disease such as secondary steatosis, non-alcoholic steatohepatitis (NASH) and cirrhosis, cholangitis or biliary disease within 6 months of informed consent; except for Gilbert’s syndrome
    9. Abnormal circulating AFP
    10. For subjects receiving ERT, recent or continued hypersensitivity response to ERT treatment within 6 months prior to consent, as manifested by significant infusion reaction to ERT in the opinion of the Site Investigator and Medical Monitor
    11. One or more of the following: i. Albumin ≤ 3.5 g/dL ii. Total bilirubin > upper limit of normal (ULN) and direct bilirubin ≥ 0.5 mg/dL iii. Alkaline phosphatase (ALP) > 2.0 x ULN iv. Alanine aminotransferase (ALT) > 1.5 x ULN
    12. Current or history of systemic (IV or oral) immunomodulatory agent or steroid use in the past 6 months (topical treatment is allowed, e.g. asthma or eczema). Occasional use of systemic steroid may be allowed after discussion with the Medical Monitor.
    13. Contraindication to use of corticosteroids for immunosuppression 14. History of malignancy except for non-melanoma skin cancer
    15. History of alcohol or substance abuse
    16. Participation in prior investigational interventional drug or medical device study within the last 3 months prior to consent (with the exception of implantable loop recorders as in the RaILRoAD trial)
    17. Prior treatment with a gene therapy product
    18. Known hypersensitivity to components of ST-920 formulation
    19. Any other reason that, in the opinion of the Site Investigator or Medical Monitor, would render the subject unsuitable for participation in the study
    E.5 End points
    E.5.1Primary end point(s)
    • Incidence of treatment-emergent adverse events (TEAEs) Additional safety evaluations will include:
    o Routine hematology, chemistry, and liver tests, vital signs, ECG and ECHO
    o Serial alpha fetoprotein (AFP) testing and MRI of liver (or equivalent imaging modality) to monitor for any liver mass
    E.5.1.1Timepoint(s) of evaluation of this end point
    -AE Assessment: Screening, Baseline(BL), Day 1, 2, 8 , Weeks (Wks) 2, 4, 6, 8, 12, 16, 20, 24, 28, 32,36, 40, 44, 48, 52 (EOS) & Early Termination Visit (ETV)
    -Clinical laboratory tests: Screening, BL, Day 1, 2, Weeks 2, 4, 6, 8, 12, 16, 20, 24, 36, 52 & ETV
    -Liver Panel: Screening, BL, twice weekly during the first 20 weeks after ST-920 infusion while the subject is on prednisone or other equipotent steroid, Weekly for 4 Wks (Wks 21, 22, 23, 24 [±2 days]) following discontinuation of immunosuppression & then monthly thereafter
    -Vital Signs: Screening, BL, Day 1, 2, 8, Wks 2, 4, 6, 8, 12, 16, 20, 24, 36, 52 & ETV
    -ECG: Screening,BL, Day 8, Wks 24, 52 & ETV
    -ECHO: Screening, EOS & ETV
    -AFP testing: Screening, Wks 4, 8, 12, 24, 52 & ETV
    -MRI of liver: Screening, Wks 24, 52 & ETV
    E.5.2Secondary end point(s)
    Secondary Endpoints:
    •Change from baseline at specific time points over the 1-year study period in:
    o α-Gal A activity in plasma
    o Gb3 levels in plasma
    o Lyso-Gb3 levels in plasma
    o Frequency of Fabrazyme® (or equivalent ERT) infusion
    o Estimated glomerular filtration rate (eGFR) using the CKD-EPI formula
    • ST-920 vector clearance measured by level of vector genome in blood, saliva, urine, stool, and semen

    Exploratory Endpoints:
    •Change from baseline at specific time points over the 1-year study period in:
    o Left ventricular mass measured by cardiac magnetic resonance imaging (MRI)
    o Total protein and albumin to creatinine ratios in urine
    o α-Gal A levels measured in tissues
    o Substrate levels measured in tissues and urine
    o Biomarkers of renal function in urine
    o Neuropathic pain measured by the Brief Pain Inventory (BPI)
    o Frequency of pain medication use
    o Gastrointestinal (GI) symptoms measured by the GI symptoms rating scale
    o Mainz Severity Score Index (MSSI)
    o Quality of life (QOL) patient-reported outcome measured by the SF-36 questionnaire
    • Antibody response to rAAV2/6 and α-Gal A and cell-mediated immune response to rAAV2/6 Substrate deposition in the kidney (via renal biopsy) in ERT-naïve and ERT-pseudo-naïve subjects
    E.5.2.1Timepoint(s) of evaluation of this end point
    -α-Gal A, Gb3 & Lyso-Gb3 levels: Twice weekly during the first 20 wks after ST-920 infusion, then week 24, 28, 32, 36,40, 44, 48, 52 & ETV
    -ERT administration log: Before & after ST-920 infusion (All timepoints except day 2)
    -eGFR: Screening, BL, Day 8, Wk 4, 8,12,16,20,24,28,32,36,40,44,48,52 & ETV
    -AAV2/6 clearance: BL, Day 8, Wk 2,4,8,12,16,20,24,36,52
    Exploratory Endpoints:
    Cardiac MRI, GI Symptoms rating scale, MSSI, QOL: BL,Wk 24,52 &ETV
    Protein & albumin to creatinine ratio in urine, Neuropathic pain: BL,Wk 12,24,36,52 &ETV
    Frequency of pain medication use: All intervals except Day 1,2& 8
    AAV Immunogenicity: BL,Wk 4,12,24,36,52(Additional samples when liver toxicity is suspected)
    Immune response to α-Gal A: Screening,BL,Wk 4,8,12,16,24,36,52 &ETV
    Renal Biopsy: BL &Wk 24
    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 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 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.8.3 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 concerned4
    E.8.5The trial involves multiple Member States No
    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 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 years0
    E.8.9.1In the Member State concerned months17
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months17
    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: 30
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 30
    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)
    Upon completion of this study, subjects will be strongly encouraged to participate in a separate long-term follow-up study for upto 4 years to monitor the long term safety of the study treatment.
    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 Opinion2019-11-12
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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