- recruitingPHASE3
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
This study is looking at a new treatment called CSL222 for teenage boys with severe or moderately severe haemophilia B. It's a single injection aimed at improving how their blood clots and reducing bleeding episodes. Researchers want to check if it's safe and effective.
United States · Austria · Belgium - enrolling by invitation
Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
This study is checking on men with haemophilia B who previously received a gene therapy called etranacogene dezaparvovec. It aims to understand the long-term safety and how well the treatment continues to work over time after their original studies finished.
United States · Belgium · Denmark - active not recruitingPHASE2
Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B
This study looks at a new treatment called PF-06838435 for people with haemophilia B. It checks if the treatment is safe and works well over a long time. It also explores a higher dose for new participants.
United States · Australia · Canada - recruiting
An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
This study looks at how well a new gene therapy, HEMGENIX, works and if it's safe for people with haemophilia B in the long term. It also compares HEMGENIX to standard Factor IX treatment to understand its effects better.
United States · Austria · Denmark - recruitingPHASE3
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
This study is testing a new medicine, marstacimab, for children and teenagers aged 1 to 17 with haemophilia A or B. It aims to see if the medicine can safely and effectively reduce bleeding episodes when given as a weekly injection under the skin.
Argentina · Australia · Austria - recruitingPHASE1, PHASE2
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
This study looks at a new gene therapy called REGV131-LNP1265 for people with haemophilia B. It aims to see if it's safe and helps the body make its own clotting factor, potentially reducing the need for regular treatments.
United States · Australia · Canada - recruitingPHASE3
Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
This study looks at a gene therapy called CSL222 for adults with Hemophilia B. It specifically checks if the treatment helps reduce bleeding in people who already have certain natural antibodies to the gene therapy virus.
United States · Australia · Canada - recruitingPHASE3
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
This study looks at the long-term safety and effectiveness of a new medicine, marstacimab, for people with haemophilia A or B. It includes patients who already took part in earlier studies of this medicine.
United States · Argentina · Australia - recruiting
World Bleeding Disorders Registry
The World Bleeding Disorders Registry collects anonymous health information from people with bleeding disorders like haemophilia. It helps doctors learn more about these conditions and improve care worldwide. Joining means sharing your health details to help others, with your permission.
Canada - active not recruitingPHASE3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
This study looks at a new gene therapy, PF-06838435, for adult men with moderately severe to severe haemophilia B. It aims to see if the treatment can reduce bleeding better than usual care and if it's safe.
United States · Australia · Brazil - ongoing, recruitingTherapeutic exploratory (Phase II)
A Global, Open-label Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Hemophilia B With Inhibitors (PRESent-3)
This study is looking at a new medicine called SerpinPC for men with a specific type of Haemophilia B who have developed 'inhibitors'. We want to see if SerpinPC can reduce bleeding episodes and if it's safe to use.
Spain · France · Italy - active not recruiting
A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
This study looks at how Haemophilia B affects men who already use regular FACTOR IX treatment. It aims to gather information about their bleeding, how their treatment works, and how it impacts their life, without testing new medicines. This helps researchers learn more for the future.
United States · Canada · Germany