- ongoing, recruitingHuman Pharmacology (Phase I)- Other
A study to test a medicine (fitusiran) for preventing bleeds in people with severe hemophilia who previously received preventive treatment with emicizumab
This study is testing a new medicine, fitusiran, for severe haemophilia A. It's for adult men who have bleeding episodes despite taking another preventative treatment called emicizumab. The aim is to see if fitusiran can help reduce these bleeding events.
Italy · Spain - active not recruitingPHASE3
Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A
This study is looking at a new medicine called efanesoctocog alfa (BIVV001) for people with haemophilia A. It aims to see if it's safe and effective over a long time in preventing and treating bleeds, improving joint health and quality of life.
United States · Argentina · Australia - 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 - recruitingPHASE4
Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery
This study looks at how well Nuwiq, a treatment for hemophilia A, works to prevent bleeding in women and girls aged 12 and over who have hemophilia A and are having major surgery. It focuses on how safe and effective Nuwiq is in these situations.
United States · Finland · France - recruitingPHASE4
Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa
This study looks at how well a medicine called efanesoctocog alfa works over a long time to keep joints healthy in people with haemophilia A. It aims to understand its real-world benefits in preventing bleeding into joints.
Croatia · Czechia · France - active not recruiting
A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
This 4-year study looks at how well Elocta medicine helps people with haemophilia A keep their joints healthy. It observes patients already using Elocta in their daily lives to see its long-term effects on preventing joint problems.
Czechia · Estonia · Finland - 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 - active not recruitingPHASE1, PHASE2
Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"
This study is testing a new gene therapy called BAY 2599023 (DTX201) for men with severe haemophilia A. It aims to help their bodies make the missing clotting factor VIII by introducing a healthy gene, potentially reducing bleeding episodes.
United States · Bulgaria · France - active not recruitingPHASE1, PHASE2
Gene Therapy for Haemophilia A.
This study is testing a new gene therapy for severe Haemophilia A. It aims to see if a one-time treatment can safely help the body make its own clotting factor, potentially reducing the need for regular injections.
United States · United Kingdom - authorisedTherapeutic confirmatory (Phase III)
C0371017 - A PHASE 3, NON-INVESTIGATIONAL PRODUCT, MULTI COUNTRY COHORT STUDY TO DESCRIBE THE LONG-TERM SAFETY AND EFFECTIVENESS OF A PRIOR SINGLE-DOSE TREATMENT WITH INVESTIGATIVE GIROCTOCOGENE FITELPARVOVEC OR FIDANACOGENE ELAPARVOVEC IN PARTICIPANTS WITH HEMOPHILIA A OR HEMOPHILIA B, RESPECTIVELY
This study looks at the long-term safety and how well new gene therapies work for people with haemophilia A or B. Researchers want to see if these treatments prevent bleeding and if there are any side effects over time, like blood clots or liver problems. It's for adult males.
Italy · Germany · France - active not recruitingPHASE3
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
This study is testing a new gene therapy, PF-07055480, for adult men with moderate to severe haemophilia A. It aims to see if this one-time treatment can safely reduce bleeding by helping the body make more of the clotting factor it needs.
United States · Australia · Brazil - enrolling by invitation
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
This study is checking on men with severe Haemophilia A who previously received a treatment called BMN 270. We want to see how safe and effective this treatment remains over a long time, beyond five years after they first received it.
United States · Australia · Belgium