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Trial record 1 of 1 for:    SIG-001-121
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Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

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ClinicalTrials.gov Identifier: NCT04541628
Recruitment Status : Suspended (Temporary enrolment halt)
First Posted : September 9, 2020
Last Update Posted : August 19, 2021
Sponsor:
Information provided by (Responsible Party):
Sigilon Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE August 21, 2020
First Posted Date  ICMJE September 9, 2020
Last Update Posted Date August 19, 2021
Actual Study Start Date  ICMJE September 28, 2020
Estimated Primary Completion Date September 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 1, 2020)
  • Number and proportion of participants with treatment emergent adverse events (TEAEs) overall and by dose/cohort. [ Time Frame: From SIG-001 administration until study completion (5 years) ]
  • Number and proportion of participants with serious treatment emergent adverse events (TEAEs) overall and by dose/cohort. [ Time Frame: From SIG-001 administration until study completion (5 years) ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 1, 2020)
  • Number and proportion of participants with inhibitor titer values assessed by Nijmegen Bethesda Assay overall and by dose/cohort. [ Time Frame: From SIG-001 administration until study completion (5 years) ]
  • FVIII activity levels assessed by one-stage and chromogenic assays overall and by dose/cohort. [ Time Frame: From SIG-001 administration until study completion (5 years) ]
  • Annualized Bleeding Rate (ABR) for all bleeds following SIG-001 administration presented by bleed type and location. [ Time Frame: From SIG-001 administration until study completion (5 years) ]
  • Total number of replacement FVIII therapies administered overall and by dose/cohort. [ Time Frame: From SIG-001 administration until study completion (5 years) ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A
Official Title  ICMJE 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 (SIG-001-121)
Brief Summary SIG-001-121 is a first-in-human (FIH), phase 1/2, multi-centre, open-label, dose escalation study to assess the safety, tolerability, and preliminary efficacy of SIG-001 in adults with severe or moderately severe haemophilia A without inhibitors. Up to three dose cohorts (3 patients each) are planned. Cohort expansions (up to 3 additional patients) may be triggered to collect additional information about safety and efficacy.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hemophilia A
Intervention  ICMJE Combination Product: SIG-001
Laparoscopic administration of SIG-001 spheres, an encapsulated allogeneic cell therapy genetically modified with a non-viral vector to produce BDD-hFVIII.
Study Arms  ICMJE Experimental: SIG-001
B-Domain Deleted Human Factor VIII (BDD-hFVIII) Producing Spheres
Intervention: Combination Product: SIG-001
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Suspended
Estimated Enrollment  ICMJE
 (submitted: September 1, 2020)
18
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 2026
Estimated Primary Completion Date September 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Males aged 18 years or older
  • Diagnosis of Haemophilia A defined as ≤2% FVIII activity
  • Greater than 150 exposure days to treatment with FVIII products
  • Use of reliable barrier contraception if applicable
  • Normal levels of von Willebrand factor (VWF) antigen
  • Able and willing to provide informed consent
  • Willing to withdraw from FVIII prophylaxis during specified periods in the study

Exclusion Criteria:

  • Body mass index (BMI) ≥35
  • Current FVIII inhibitors (>0.6 Nijmegen Bethesda Units/mL) or prior Immune Tolerance Induction (ITI)
  • History of allergic reaction or anaphylaxis to recombinant FVIII products or SIG-001 components
  • Evidence of any bleeding disorder in addition to haemophilia A
  • Abnormal laboratory values as defined in the protocol
  • Active infection with Hepatitis B or Hepatitis C virus or currently managed with antiviral medications for Hepatitis B or C
  • Uncontrolled HIV infection
  • Active alcoholism or drug addiction during the 12 months before the screening visit
  • Active malignancy or history of malignancy in the 5 years prior to study entry
  • Participation in another investigational medicine or device study
  • Prior administration of a gene therapy product
  • Significant underlying disease or comorbidities that are a contraindication for general anaesthesia or laparoscopic procedure
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04541628
Other Study ID Numbers  ICMJE SIG-001-121
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Sigilon Therapeutics, Inc.
Study Sponsor  ICMJE Sigilon Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Sigilon Therapeutics, Inc.
Verification Date August 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP