Find recruiting clinical trials for haemophilia in the UK — including haemophilia A, haemophilia B, and patients with inhibitors. Gene therapy is transforming care. See pathways and where trials fit in.
Free to use — Live data from ClinicalTrials.gov — Updated hourly
Answer a few questions about your condition and we'll match you to the most relevant clinical trials.
See where clinical trials fit into your treatment journey
Regular factor replacement
Standard: Recombinant Factor VIII (Haemophilia A) or Factor IX (Haemophilia B)
Less frequent dosing
Standard: Efmoroctocog alfa, Eftranonacog alfa, or other EHL products
Bypassing factor altogether
Standard: Emicizumab (Hemlibra) — bispecific antibody mimicking Factor VIIIa
One-time curative treatment
Standard: AAV-based gene transfer (Valoctocogene roxaparvovec for Haem A, Etranacogene dezaparvovec for Haem B)
Deficiency of Factor VIII. About 80% of haemophilia cases. Standard treatment is regular Factor VIII infusions, but Emicizumab and gene therapy are changing the landscape.
Deficiency of Factor IX. Less common but treatment advances including extended half-life factors and gene therapy are significant.
About 30% of severe Haemophilia A patients develop inhibitors (antibodies) against replacement factor. Emicizumab has been transformative for this group.
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Gene therapy aims to enable the body to produce its own clotting factor. Early results show significant factor level increases lasting 3-5+ years. Research is ongoing into long-term durability and whether re-treatment is possible.
Emicizumab is a bispecific antibody that mimics Factor VIII function. It is approved on the NHS but trials are exploring longer-acting formulations and use in patients without inhibitors.
Gene therapy trials for haemophilia have primarily enrolled adults. However, paediatric trials are beginning as safety data matures. Discuss with your haemophilia centre what options are available for children.
Use our search above to find trials that match your condition and location. Review the eligibility criteria carefully.
Talk to your GP or specialist about any trials you are interested in. They can help determine if a trial is appropriate for you.
Reach out to the trial team directly using the contact information on the ClinicalTrials.gov listing. They will guide you through screening.
If you meet the criteria and decide to participate, you will go through informed consent and begin the trial process.