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    Summary
    EudraCT Number:2019-004210-33
    Sponsor's Protocol Code Number:SIG-001-121
    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:2020-03-03
    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 number2019-004210-33
    A.3Full title of the trial
    A Phase 1/2 Open-Label, Dose-Escalation, Safety, Tolerability, and Efficacy Study of SIG-001 in Adult Patients with Severe or Moderately-Severe Haemophilia A Without Inhibitors
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A
    A.4.1Sponsor's protocol code numberSIG-001-121
    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 SponsorSigilon 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 supportSigilon Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSigilon Therapeutics, Inc.
    B.5.2Functional name of contact pointClinical Trials
    B.5.3 Address:
    B.5.3.1Street Address100 Binney St, Suite 600
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02142
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrials@sigilon.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 nameBDD-hFVIII Producing Spheres
    D.3.2Product code SIG-001
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntraperitoneal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNS14001
    D.3.9.2Current sponsor codeS14001
    D.3.9.3Other descriptive nameBDD-hFVIII producing spheres
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number42600
    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 No
    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
    Haemophilia A
    E.1.1.1Medical condition in easily understood language
    Haemophilia A is a genetic deficiency in clotting factor VIII, which causes increased bleeding and usually affects males.
    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 10018937
    E.1.2Term Haemophilia A
    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
    To evaluate the safety and tolerability of up to 3 dose levels of SIG-001 administered laparoscopically into the intraperitoneal space.
    E.2.2Secondary objectives of the trial
    To evaluate development of FVIII inhibitors after administration of SIG-001
    To characterize FVIII activity levels after administration of SIG-001
    To assess frequency of bleeding episodes following SIG-001 administration
    To assess effects of SIG-001 on usage of FVIII products
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males aged 18 years or older
    2. Diagnosis of Haemophilia A as follows:
    a) Severe (<1% FVIII activity) receiving FVIII prophylaxis or on-demand treatment; or
    b) Moderately-severe (≥1% - ≤2% FVIII activity) receiving FVIII prophylaxis
    3. Greater than 150 exposure days to treatment with FVIII products
    4. Fertile males (not surgically sterilized) who have sexual partners that are women of childbearing potential must be willing to use a barrier method of contraception (e.g. condoms) for at least 90 days post-SIG-001 administration
    5. Normal levels of von Willebrand factor (VWF) antigen (≥50 IU/dL)
    6. Able and willing to provide informed consent
    7. Willing to withdraw from FVIII prophylaxis during specified periods in the study
    E.4Principal exclusion criteria
    1. Morbid obesity defined as body mass index (BMI) ≥35
    2. Current FVIII inhibitors (>0.6 NBU/mL) or prior Immune Tolerance Induction (ITI)
    Note: Patients with a history of low and/or transient inhibitors require Sponsor review and approval.
    3. History of allergic reaction or anaphylaxis to recombinant FVIII products, alginate (including seaweed and algae), or barium and barium products (including barium contrast agents)
    4. Evidence of any bleeding disorder in addition to haemophilia A
    5. Abnormal laboratory values, as follows:
    a) Platelet count <100 × 109/L
    b) Creatinine ≥1.5 mg/dL
    c) Haemoglobin <11 g/dL
    d) Elevated levels (i.e. greater than 2 × upper limit of normal (ULN) of aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin
    Note: Patients with a known history of Gilbert’s disease and bilirubin levels above ULN are not excluded.
    6. Active infection with Hepatitis B or Hepatitis C virus, defined as a positive HBsAg, HBeAg, HBV DNA, or HCV RNA results, or currently managed with antiviral medications for Hepatitis B or C
    7. Uncontrolled HIV infection, defined as CD4+ counts ≤200/μL or by a viral load of >200 copies/mL
    8. Active alcoholism or drug addiction during the 12 months before the screening visit
    9. Active malignancy or history of malignancy in the 5 years prior to study entry, exclusive of surgically removed non-melanoma skin cancer
    10. Participation in another investigational medicine or device study within 90 days of screening or 5 investigational product half-lives, whichever is longer
    11. Prior administration of a gene therapy product
    12. Treatment with emicizumab less than 6 months prior to screening.
    Note: Patients with emicizumab exposure may be eligible before 6 months of prior exposure if FVIII activity assessed by one stage assay is back to pre-emicizumab treatment levels.
    13. Unable to tolerate general anaesthesia required for the laparoscopic procedure
    14. History of:
    a) Abdominal adhesions due to prior large, median laparotomy(ies)
    Note: Uncomplicated prior laparoscopic procedures are not exclusionary (e.g. cholecystectomy, laparoscopic appendectomy or diagnostic laparoscopy).
    b) Septic peritonitis
    c) Intraperitoneal mesh
    d) Inflammatory Bowel Disease (IBD), including ulcerative colitis or Crohn’s disease
    15. Other comorbidities that in the opinion of the Investigator increase the risk of abdominal adhesions
    16. Any disease or medical condition that in the Investigator’s opinion is a contraindication for anaesthesia or the laparoscopic procedure (e.g. prior significant cardiovascular events)
    17. In the Investigator’s judgment, the patient is unlikely to complete all protocol-required study visits, procedures, or comply with the study requirements for participation
    E.5 End points
    E.5.1Primary end point(s)
    Clinically significant changes from baseline in vital signs, clinical laboratory tests, and treatment emergent adverse events (TEAE)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Vitals – Screening, SIG-001 Administration, Follow-Up up to 5 years.
    TEAEs – Screening, SIG-001 Administration, Follow-Up up to 5 years.
    See Table 2 Schedule of Assessment in the Clinical Study Protocol for further details.
    E.5.2Secondary end point(s)
    Factor VIII inhibitor titers (Nijmegen Bethesda Units [NBU])
    FVIII activity levels (one-stage and chromogenic assays)
    Bleeding rate (annualized; ABR)
    Total use of factor VIII therapies (annualized)
    E.5.2.1Timepoint(s) of evaluation of this end point
    FVIII inhibitor titers - Screening 1, Follow Up up to 5 years.
    FVIII activity levels - Screening 1, SIG-001 Administration, Follow-Up up to 5 years.
    ABR and annualized FVIII therapy use - assessed prospectively throughout the study.
    See Table 2 Schedule of Assessment in the Clinical Study Protocol for further details.
    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 No
    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.8.2.4Number of treatment arms in the trial1
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    Germany
    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
    The End-of-Trial date will be based on the final data collection date for the entire study, which is the date of the last Follow-up visit.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    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 years6
    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: 16
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 state11
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 1
    F.4.2.2In the whole clinical trial 18
    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)
    Patients will be followed for approximately 5 years post SIG-001 administration for safety and efficacy.
    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 Decision2020-05-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-06-11
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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