Liver biopsy after gene therapy for hemophilia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This study looks at liver tissue from people with hemophilia who previously had gene therapy. The goal is to understand how the gene therapy affected the liver, which may help improve future treatments.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 to 80 years old.
- You must have been in a specific gene therapy trial for hemophilia (FIX or FVIII) at St. Jude or a related trial in London.
- You need to be able to give permission and follow study rules.
- If you have a liver biopsy as part of this study, you must have normal blood clotting (no low platelets, no high INR) and no bleeding problems other than hemophilia.
- You must not have allergies to iodine contrast or anesthesia, and your kidneys must work well enough for the biopsy.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This Phase 1/2 study tests a one-time gene therapy (ASC618) to help the body make clotting factor for people with hemophilia A. It may reduce bleeding and the need for factor infusions, but you must meet several safety and eligibility checks.
This trial tests a new gene therapy (REGV131-LNP1265) that aims to let your body produce its own factor IX, reducing or stopping the need for regular infusions. It is for children and adults with severe hemophilia B who are already using factor IX replacement therapy.
This trial tests a gene therapy to help your body produce more of the clotting protein (factor VIII) it's missing. It may reduce or stop the need for regular factor infusions.
This trial tests a one-time gene therapy that uses a modified virus (lentivirus) to deliver a working copy of the factor VIII gene to your cells. The goal is to help your body produce factor VIII on its own, reducing or eliminating the need for factor VIII infusions.
This Phase 3 trial is checking how safe and effective two gene therapies are for people with hemophilia A or hemophilia B. It only includes people who already received the study medicines through specific Pfizer studies, so it’s mainly for that existing participant group.
This trial tests a one-time gene therapy treatment (GS001) to help people with hemophilia A make more of the clotting factor they lack. If it works and is safe for you, it could improve how well blood clots form over time.
Hear when a new Hemophilia A trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.