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    Summary
    EudraCT Number:2017-004805-42
    Sponsor's Protocol Code Number:SB-FIX-1501
    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:2018-01-04
    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 number2017-004805-42
    A.3Full title of the trial
    A Phase I, Open-Label, Ascending Dose Study to Assess the Safety and Tolerability of AAV2/6 Factor IX Gene Therapy via Zinc Finger Nuclease (ZFN) mediated targeted integration of SB-FIX in Subjects with Severe Haemophilia B
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase 1 study to evaluate the safety and tolerability of AAV2/6 Factor IX gene therapy in patients with severe Haemophilia B
    A.4.1Sponsor's protocol code numberSB-FIX-1501
    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 pointSB-FIX-1501 Information Desk
    B.5.3 Address:
    B.5.3.1Street Address501 Canal Blvd., Suite A100
    B.5.3.2Town/ cityRichmond CA
    B.5.3.3Post code94804
    B.5.3.4CountryUnited States
    B.5.4Telephone number+15109706000
    B.5.5Fax number+15103237388
    B.5.6E-mailSBFIX1501-Notification@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 namerAAV2/6 Left ZFN Vector
    D.3.2Product code SB-42906
    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 INNNot yet assigned
    D.3.9.1CAS number 2143578-14-5
    D.3.9.2Current sponsor codeSB-42906
    D.3.9.3Other descriptive nameAdeno-associated virus serotype 2/6 encoding Left side-zinc finger nuclease (ZFN1), rAAV6.2.2.2 hAlb ZFN 1 Vector
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10000000000000
    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.IMP: 2
    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 namerAAV2/6 Right ZFN Vector
    D.3.2Product code SB-43043
    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 INNNot yet assigned
    D.3.9.1CAS number 2143578-30-5
    D.3.9.2Current sponsor codeSB-43043
    D.3.9.3Other descriptive nameAdeno-associated virus serotype 2/6 encoding Right side-zinc finger nuclease (ZFN2), rAAV6.2.2.2 hAlb ZFN 2 Vector
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10000000000000
    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.IMP: 3
    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 namerAAV2/6 FIX Donor
    D.3.2Product code FIX DONOR
    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 INNNot yet assigned
    D.3.9.1CAS number 2143579-02-4
    D.3.9.2Current sponsor codeSB-F9 donor
    D.3.9.3Other descriptive nameHuman Factor 9 gene donor vector, rAAV6.2.2.2 Factor IX Donor Vector
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10000000000000
    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
    Severe Haemophilia B
    E.1.1.1Medical condition in easily understood language
    Bleeding disorder
    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 10018939
    E.1.2Term Haemophilia B (Factor IX)
    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 safety and tolerability of SB-FIX.
    E.2.2Secondary objectives of the trial
    1. Change from baseline in FIX antigen and activity levels
    2. Change from baseline in use of Factor IX replacement therapy
    3. Change from baseline in frequency and severity of bleeding episodes
    4. Immune response to FIX
    5. Presence and shedding of AAV2/6 vector DNA by PCR in plasma, saliva, urine, stool and semen
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusion Criteria
    1. Signed informed consent
    2. Male ≥18 years of age and older (adult cohort); Male 12-17 years of age (pediatric cohort)
    3. Severe heamophilia B (native circulating FIX activity <1%, with or without cross reactive material)
    4. FIX mutations confirmed by FIX genome sequencing
    5. With 150 or more exposure days to FIX concentrates
    6. Sexually active subject must agree to use double barrier contraceptive (one of them being a condom) or abstinence and all subjects must refrain from donating their sperm until at least 2 consecutive semen samples after SB-FIX are negative for AAV 2/6 and for a minimum of 90 days after SB-FIX administration
    E.4Principal exclusion criteria
    Exclusion Criteria
    1. Presence of neutralizing antibodies to AAV 2/6
    2. History of hypersensitivity response or allergic reaction to FIX or FIX products
    3. Currently receiving long acting FIX replacement therapy and unwilling to switch to short acting FIX infusions
    4. FIX mutations known to be associated with FIX inhibitors, including those with a large gene deletion
    5. Polymorphisms in the ZFN target region of the albumin locus
    6. Presence of any liver mass on MRI or equivalent imaging technology, or elevated alpha-fetoprotein (AFP)
    7. Any contraindication to the use of corticosteroids for immunosuppression
    8. Currently receiving antiviral therapy for hepatitis B or C or with history of active hepatitis B or hepatitis C or HIV-1 or HIV 1/2 antibody positive. To be considered HCV-negative after an active HCV infection, viral assays in two samples collected at least six months apart must be negative
    9. Chronic anaemia, leukopenia, or thrombocytopenia
    10. Past medical history of active tuberculosis or systemic fungal disease
    11. Symptomatic cardiovascular disease as a co-morbid condition
    12. Markers of hepatic inflammation or overt or occult cirrhosis as evidenced by one or more of the following:
    • Albumin ≤3.5 g/dL (35g/L)
    • Total bilirubin >1.5 x ULN and direct bilirubin ≥0.5 mg/dL (8.55 µmol/L)
    • Alkaline phosphatase >2.0 x ULN
    • ALT or AST >2.0 x ULN
    13. History of chronic renal disease or creatinine ≥ 1.5 mg/dL (133 µmol/L)
    14. Systemic (iv or oral) immunomodulatory agent or steroid use (topical treatment is allowed, e.g. for asthma or eczema) within 3 months prior to Screening
    15. History of chronic infection or other chronic disorder considered an unacceptable risk
    16. History of malignancy except for treated basal cell or squamous cell carcinoma
    17. History of alcohol or substance abuse
    18. Previously received gene therapy product
    19. Participation in prior investigational drug or medical device study within the previous 3 months
    20. History of therapeutic non-adherence
    21. Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for participation in the study
    E.5 End points
    E.5.1Primary end point(s)
    Primary:
    Safety and tolerability will be assessed by the grading of AEs/SAEs and change in laboratory evaluations from baseline
    E.5.1.1Timepoint(s) of evaluation of this end point
    1) AE assessment
    Baseline, Day 0, Day 1, Day 7, weeks 2, 4, 6, 8, 10, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, months 15, 18, 21,24, 27, 30, 33, 36 (EOS) & Early Termination Visit
    2) Laboratory evaluations
    -CBC: Baseline, Day 0, Day 1, weeks 4, 8, 12, 28,52, months 15, 18, 21,24, 27, 30, 33, 36/EOS & Early Termination Visit
    -Serum Chemistry, Metabolic Panel: All time points except day 7
    -Coagulation screen: Baseline, Day 0, weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, months 15, 18, 21,24, 27, 30, 33, 36/EOS & Early Termination Visit
    -Liver Panel: Tested twice per week from baseline through week 12, then tested at wks 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, months 15, 18, 21, 24, 27, 30, 33, 36/EOS & early termination
    E.5.2Secondary end point(s)
    Secondary:
    1. Changes in FIX antigen and FIX activity levels from baseline over time
    2. Change from baseline in the number of FIX units infused per week
    3. Change from baseline in number and severity of bleeding episodes
    4. Changes in neutralizing antibodies to FIX from baseline over time
    5. Presence and shedding of AAV2/6 vector DNA, by PCR in plasma, saliva, urine, stool, and semen over time
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. FIX antigen: Baseline (BL), Day 1, Day 7, Wks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, Months 15, 18, 21, 24, 27, 30, 33, 36 (EOS) & Early Termination Visit
    2. FIX activity: BL, Day 1, Day 7, Weekly (Wk 2-Wk 12), Wk 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, Months 15, 18, 21, 24, 27, 30, 33, 36 & Early Termination Visit
    3. Bleeding episodes & FIX concentrates usage: From BL through end of the study including early termination visit
    4. FIX inhibitor level: BL, Day 1, Wks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, Months 15, 18, 21,24, 27, 30, 33, 36 & Early Termination Visit
    5. Presence & shedding in AAV2/6 vector DNA by PCR in plasma, saliva, urine, stool & semen over time: BL, Day 1, Day 7, WKs 2, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 & Early termination visit
    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 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) No
    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 concerned8
    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 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
    LVLS
    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 months0
    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 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 Yes
    F.1.1Number of subjects for this age range: 3
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 2
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 16
    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 asked to participate in a separate long-term follow-up study 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 Decision2018-02-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-05
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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