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New treatment option for Hemophilia B, Severe
Official title Liver Biopsy In Haemophilia Gene Therapy
ClinicalTrials.gov ID: NCT04817462
What this study is testing
- What it's testing
- 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.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 80, men only
You may be able to join if
- 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)
- FLT180a-01 (EudraCT: 2017-000852-24) - FIX AAV gene therapy trial ((Sponsor: UCL) [now enrolled in long term follow up study FLT180a-04 (EudraCT No...
You likely can't join if
- Patients with a platelet count measured at \<140 x109/L
- Any condition that, in the opinion of the investigator or Sponsor would prevent the patient from fully complying with the requirements of the study...
- 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 eligibility criteria
- 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)
- FLT180a-01 (EudraCT: 2017-000852-24) - FIX AAV gene therapy trial ((Sponsor: UCL) [now enrolled in long term follow up study FLT180a-04 (EudraCT No 2017-005080-40) (Sponsor: Freeline Therapeutics Ltd)
- Patients with endogenous FVIII:C/FIX:C expression at \>1% any time after gene transfer, associated with normal prothrombin (PT) and thrombin times (TT) as determined in a coagulation assay.
- Patients with a platelet count measured at \<140 x109/L
- Any condition that, in the opinion of the investigator or Sponsor would prevent the patient from fully complying with the requirements of the study and/or would influence or interfere with evaluation and interpretation...
- 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
- Presence of FVIII or FIX inhibitor (done within 14 weeks of biopsy)
- Evidence of any bleeding disorder not related to haemophilia A or B
- Patients unable and unwilling to provide and sign an informed consent.
The study team makes the final eligibility decision.
Where it's taking place
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 18 years to 80 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include London, United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.