Trial | NCT04541628  Report issue

Title

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A
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)

  • Phase

    Phase 1/Phase 2

  • Study Type

    Interventional

  • Status

    Terminated

  • Study Participants

    3
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 Started
Sep 28
2020
Primary Completion
Oct 28
2022
Study Completion
Oct 28
2022
Last Update
Nov 10
2022

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.

SIG-001 Experimental

B-Domain Deleted Human Factor VIII (BDD-hFVIII) Producing Spheres

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
No Results Posted