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

Stopped early · Phase 1/Phase 2

Conditions studied: Hemophilia A

In brief

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.

Key facts

Study ID

NCT04541628

Run by

Eli Lilly and Company

People needed

3

Starts

2020-09-28

Expected to finish

2023-01-09

Last updated by the study team

2024-09-04

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Clinical Study Site — Indianapolis, Indiana, United States
• Clinical Study Site — Boston, Massachusetts, United States
• Clinical Study Site — Seattle, Washington, United States
• Clinical Study Site — London, United Kingdom
• Clinical Study Site — Manchester, United Kingdom
• Clinical Study Site — Southampton, United Kingdom

Full record on ClinicalTrials.gov

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