- 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 - 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 - 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 - 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 - 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 - 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)
Spain · France · Italy