Compassionate Use of Concizumab for Hemophilia
Treatments studied
Part of Blood & lymphatic clinical trials.
This program provides access to a new experimental medicine called concizumab for people with hemophilia who cannot use standard treatments. It is designed for patients who have tried other options without success or cannot take them safely.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have congenital hemophilia (severe type A with factor VIII less than 1%, or moderate/severe type B with factor IX at or below 2%) without inhibitors, OR any severity of hemophilia with a documented history of inhibitors
- You cannot be treated satisfactorily with currently approved hemophilia medicines, due to reasons like inhibitors, allergic reactions, or poor vein access
- You are unable to enroll in standard clinical trials testing concizumab
- You must be able and willing to give informed consent and follow the program requirements
- You do not have a known severe allergy to concizumab or related medications
- You have no other serious medical conditions that would make the treatment unsafe for you
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 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 single dose of CSL222, a gene therapy, in adolescent boys with severe or moderately severe hemophilia B. The goal is to see if it can reduce or eliminate the need for regular factor IX infusions.
This trial tests a new gene therapy called SPK-8011QQ for adults with severe or moderately severe hemophilia A. The goal is to see if a single infusion can help your body produce its own factor VIII, reducing or stopping the need for regular factor VIII injections.
This trial tests if a new medicine, NXT007, is better than emicizumab at preventing bleeding episodes in people with hemophilia A. It may be an option for those with or without inhibitors who are willing to switch to a preventive treatment.
This trial will track how well Alhemo works and how safe it is for people with hemophilia A or B in everyday use. If you are 12 or older and your doctor has already decided to start you on Alhemo, this study will collect information to help doctors better understand the medicine's real-world benefits and risks.
This trial studies Hemlibra (emicizumab) in people with mild congenital hemophilia A to see how well it works and what safety issues may occur. It may help reduce bleeding in patients who meet the study’s specific health and safety requirements.
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