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

Lilly

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Hemophilia A

Treatments: Combination Product: SIG-001

Study type: Interventional

Funder types: Industry

Identifiers

NCT04541628

SIG-001-121

Details and patient eligibility

About

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.

Enrollment: 3 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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3 participants in 1 patient group

SIG-001

Experimental group

Description:

Participants received a single dose of 50 milliliter (mL), 78.5 mL and 133 mL of SIG-001 spheres \[an encapsulated allogeneic cell therapy genetically modified with a non-viral vector to produce B-Domain Deleted Human Factor VIII (BDD-hFVIII) producing Spheres\] administered laparoscopically into the peritoneal cavity.

Treatment:

Combination Product: SIG-001