Liver Biopsy In Haemophilia Gene Therapy
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexMale
Age18-80
SponsorUniversity College, London
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.
Eligibility criteria
Qualifiers
Male and aged 18 to 80 years old
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)
Disqualifiers
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 of subject safety or efficacy result.
Patients with abnormal kidney function (estimated GFR <50ml/min)
Patients with a known allergy to iodine-based intravenous contrast agents
Trial design
Treatments tested in this trial
- Liver biopsy
Treatment groups
10 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov