Brief Title
Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A
Official Title
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.
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
Number and proportion of participants with treatment emergent adverse events (TEAEs) overall and by dose/cohort.
Secondary Outcome
Number and proportion of participants with inhibitor titer values assessed by Nijmegen Bethesda Assay overall and by dose/cohort.
Condition
Hemophilia A
Intervention
SIG-001
Study Arms / Comparison Groups
SIG-001
Description: B-Domain Deleted Human Factor VIII (BDD-hFVIII) Producing Spheres
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Combination Product
Estimated Enrollment
18
Start Date
September 28, 2020
Completion Date
September 2026
Primary Completion Date
September 2026
Eligibility Criteria
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
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
, 1-617-586-2822, [email protected]
Location Countries
United Kingdom
Location Countries
United Kingdom
Administrative Informations
NCT ID
NCT04541628
Organization ID
SIG-001-121
Responsible Party
Sponsor
Study Sponsor
Sigilon Therapeutics, Inc.
Study Sponsor
, ,
Verification Date
October 2020