- recruitingTherapeutic use (Phase IV)
A Long-Term Follow-Up Study in Subjects with Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial
This long-term study monitors adult men with severe haemophilia A who have previously received a gene therapy called Roctavian. Doctors want to see how well the treatment continues to work over time, check for any long-lasting side effects, and make sure that participants stay safe and well.
Italy · Belgium · France - 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 - recruitingPhase I and Phase II (Integrated)- First administration to humans
(19429) A phase 1/2 open-label safety and dose-finding study of BAY 2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human factor VIII, in adults with severe hemophilia A
This study is testing a new gene therapy, called BAY 2599023, for adult men with severe haemophilia A. It aims to see how safe the treatment is and find the right dose. The goal is to help their bodies make more of the clotting factor they need.
Netherlands · Germany · France - 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.
United States · Argentina · Australia - active not recruiting
A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A
This study looks at the long-term effects of ADYNOVI/ADYNOVATE medicine for people with haemophilia A. Researchers want to see how it works and if there are any side effects when used in everyday life.
United States · Bulgaria · Croatia - recruiting
Treatment of Hemophilia A Patients With FVIII Inhibitors
This study looks at how doctors manage Haemophilia A in men who develop ‘inhibitors’ to their treatment. It observes different treatments, including a new medicine called emicizumab, to see how well they work and if they are safe over up to five years.
United States · Germany - 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 - 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 - recruitingPHASE1
Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A
This study is looking at a new medicine called efanesoctocog alfa for people with severe haemophilia A. It compares how long this new medicine stays in the body compared to two other similar medicines currently used. The goal is to see if the new medicine lasts longer.
Bulgaria · Germany · Italy - recruitingPHASE1, PHASE2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
This study is testing a new medicine called NXT007 for people with moderate or severe haemophilia A. It's looking at how safe it is, how it works in the body, and if it helps reduce bleeding. Both adults, teenagers, and children can take part.
United States · Canada · Italy - active not recruiting
Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A
This study looks at how a medicine called Jivi (damoctocog alfa pegol) works for people with haemophilia A in their daily lives. It checks its long-term safety and how happy patients and doctors are with it.
United States · Belgium · Brazil - active not recruiting
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
This study looks at the long-term safety and effectiveness of new gene therapies, SPK-8011 and SPK-8016, for men with haemophilia A. It follows participants who have already received one of these treatments to see how they're doing over many years.
United States · Australia · Canada - active not 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 - 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 - active not recruiting
Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding
This study looks at how safe a medicine called Jivi is for people with haemophilia A over a long time. It checks patients who are already using Jivi to prevent bleeding, gathering information during their regular doctor visits.
Austria · Germany · Greece - active not recruiting
UPLC-MS/MS Monitoring of Emicizumab Therapy
This study looks at how levels of a medicine called emicizumab, used for severe haemophilia A, affect bleeding. Doctors want to see if checking medicine levels in the blood can help predict and prevent bleeding episodes in patients.
France - 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 - recruitingPHASE3
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
This study compares two treatments, NXT007 and emicizumab, for people aged 12 and over with haemophilia A. It aims to see how well they prevent bleeding, if they are safe, and how they work in the body.
United States · Argentina · Australia - 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 - recruitingPHASE3
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A
This study compares a new medicine, NXT007, with standard Factor VIII treatment for people with haemophilia A. It aims to see if NXT007 is safe and works well to prevent bleeding in adults and teenagers aged 12 and older.
United States · Argentina · Brazil - 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 - 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 - 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 - 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