NCT04817462Recruiting
Liver Biopsy In Haemophilia Gene Therapy
Source: ClinicalTrials.gov · Last updated 20 Dec 2024 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- First submitted
- 23 Feb 2021
- Start date
- 5 Aug 2022
- Primary completion date
- 1 Jul 2025
- Lead sponsor
- University College, London
- Diseases
- Haemophilia
- NCT number
- NCT04817462
What you commit to
- Study type: Interventional
- Planned participants: 10
- From 5 Aug 2022 to the primary completion date, 1 Jul 2025
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Presence of FVIII or FIX inhibitor (done within 14 weeks of biopsy)
Other illness
- 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 a known allergy to iodine-based intravenous contrast agents
- Patients with a known allergy to local or general anaesthetic
- Evidence of any bleeding disorder not related to haemophilia A or B
Lab values
- Patients with a platelet count measured at \<140 x109/L
- Patients with abnormal kidney function (estimated GFR \<50ml/min)
Other
- Patients with a known reaction to FVIII/FIX concentrate infusions
- Patients unable and unwilling to provide and sign an informed consent.
Who 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)
- 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.
Age in the registry: 18 years to 80 years · Sex: Male · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Royal Free Hospital Coordinator of this site? Confirm its status | London | United Kingdom | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.