Hemophilia gene therapy registry
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This registry collects information from people with hemophilia who have had gene therapy, to track safety and how well it works over time. By joining, you help improve future treatments for others.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have hemophilia
- You have already received a gene therapy product for hemophilia
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 trial is a research registry that enrolls children with hemophilia A or B to collect health information. It may help researchers better understand bleeding risk and treatment needs over time.
This gene therapy trial uses a modified virus to deliver a working copy of the gene that makes factor IX, the protein missing in hemophilia B. The goal is to help your body produce enough factor IX to reduce or prevent bleeding episodes, potentially reducing the need for regular factor IX infusions.
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 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.
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.
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