At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Liver biopsy (procedure)
- How long the study runs
- Study runs about 35 months (dates as stated)
- About the drug or intervention
- Liver biopsy — procedure: The study population is patients with either haemophilia A or B who have previously been administered gene therapy treatment in one of three specific gene therapy clinical trials.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 18 Years to 80 Years
- Who
- Male
- Number of participants
- 10
- Started
- 2022-08-05
- Last checked
- 2024-12
Plain English Summary
What is this study?
- • Testing a new treatment for hemophilia b, severe
- • NA - 10 participants
- • To perform a liver biopsy in haemophilia A and B patients with endogenous FVIII:C/FIX:C expression at \>1% any time after gene transfer following AAV mediated gene transfer
Who can take part?
- • Ages 18 Years to 80 Years
- • Diagnosed with hemophilia b, severe
- • Male only
Where?
- • London - Royal Free Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
To perform a liver biopsy in haemophilia A and B patients with endogenous FVIII:C/FIX:C expression at \>1% any time after gene transfer following AAV mediated gene transfer. This is to obtain tissue for analysis, to understand if FIX/FVIII transgenic protein expression is mediated by AAV proviral DNA that is integrated into the host cell DNA or if stable expression in humans is mediated by episomal maintained AAV genome.
More detail
To better understand the consequences of AAV gene transfer patients will be recruited to undergo a liver biopsy. Patients will have endogenous FVIII:C/FIX:C expression at \>1% any time after gene transfer following AAV mediated gene transfer. Analysis of biopsy samples will: * Provide a clearer insight into the AAV life cycle in human liver * Define the number of human hepatocytes that are transduced * Improve understanding at the human hepatocyte level of long-term consequences of AAV mediated transgene expression from the liver that will include (i) changes in the pattern of gene expression in human hepatocytes following AAV mediated gene transfer, (ii) information on the epigenetic signature in the liver following AAV mediated gene transfer and how this changes with time and (iii) the consequences of transgene expression in hepatocytes. This study will provide new data addressing several unknowns with AAV mediated gene transfer in humans that will better inform on safety and efficacy following AAV gene transfer for patients who have already participated in gene therapy studies as well as those considering this treatment option.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years - 80 Years
- Who can join: Male only
What the study is looking for
- ✓Male and aged 18 to 80 years old
- ✓Patients who were enrolled and treated in one of the following clinical trials at Royal Free Hospital:
- ✓AGT4HB (EudraCT No 2005-005711-17) - FIX AAV gene therapy trial (Sponsor: St Jude Children's Research Hospital)
- ✓GO-8 (EudraCT No 2016-000925-38) - FVIII AAV gene therapy trial (Sponsor: UCL)
- ✓Patients with endogenous FVIII:C/FIX:C expression at \>1% any time after gene transfer, associated with normal...
Who cannot take part
- ✗Patients with a blood clotting cell count measured at \<140 x109/L
- ✗Patients with abnormal kidney function (estimated GFR \<50ml/min)
- ✗Patients with a known allergy to iodine-based intravenous contrast agents
- ✗Patients with a known allergy to local or general anaesthetic
- ✗Patients with a known reaction to FVIII/FIX concentrate infusions
See the full criteria
Where Is This Study? (1 UK site)
Royal Free Hospital
London NW3 2QG, United Kingdom
How to Get in Touch
Paul Batty
Sponsor contactCONTACT