Popular Trials
Virus Therapy
Gene Therapy for Hemophilia A
This trial tests a gene therapy that uses a harmless virus to deliver a healthy gene to adults with severe haemophilia A. The goal is to help their bodies produce a missing blood-clotting protein, potentially curing their condition. Gene therapy for hemophilia has been explored for many years, with several programs in advanced stages.
Gene Therapy
Gene Therapy for Hemophilia A
This trial is testing a new gene therapy for hemophilia A patients. It uses a harmless virus to help liver cells produce a missing protein needed for blood clotting. This could reduce the need for frequent injections. The therapy aims to provide a long-term solution by enabling the body to produce the necessary protein on its own.
Recombinant Factor VIII
Efanesoctocog Alfa for Hemophilia A
This trial is looking at the long-term safety of a drug called BIVV001 in people with hemophilia A. It will also look at how well the drug works to prevent and treat bleeding episodes, and the drug's effect on joint health and quality of life.
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Trials for Hemophilia B Patients
Coagulation Factor Replacement Therapy
Prophylactic Replacement Therapy for Hemophilia B
This trial is designed to see how well current prophylactic treatments work in hemophilia A and B patients before they receive gene therapy in a Phase 3 study. So far, only hemophilia B patients are being enrolled.
Monoclonal Antibodies
Marstacimab for Hemophilia
This trial tests the safety & effectiveness of a new medicine to prevent bleeding episodes in pediatric hemophilia patients. Participants will get weekly subcutaneous shots & visit the study site 10 times over 14 months. Some visits may be done at home.
Monoclonal Antibodies
Marstacimab for Hemophilia
This trial is an extension of a previous study to assess the long-term safety and efficacy of a prophylactic treatment for hemophilia A and B. Approximately 145 participants will receive injections of the marstacimab drug once weekly for the duration of the study.
Coagulation Factor Replacement Therapy
SerpinPC for Severe Hemophilia
This trial tests a new injection called SerpinPC to prevent bleeding in people with severe hemophilia A or B. It aims to see if SerpinPC is safe and effective, especially for those who don't respond well to other treatments. SerpinPC works by helping the blood clot better. Serpins are being explored as potential treatments for hemophilia by rebalancing coagulation.
Trials for HB Patients
Coagulation Factor Replacement Therapy
Prophylactic Replacement Therapy for Hemophilia B
This trial is designed to see how well current prophylactic treatments work in hemophilia A and B patients before they receive gene therapy in a Phase 3 study. So far, only hemophilia B patients are being enrolled.
Monoclonal Antibodies
Marstacimab for Hemophilia
This trial tests the safety & effectiveness of a new medicine to prevent bleeding episodes in pediatric hemophilia patients. Participants will get weekly subcutaneous shots & visit the study site 10 times over 14 months. Some visits may be done at home.
Monoclonal Antibodies
Marstacimab for Hemophilia
This trial is an extension of a previous study to assess the long-term safety and efficacy of a prophylactic treatment for hemophilia A and B. Approximately 145 participants will receive injections of the marstacimab drug once weekly for the duration of the study.
Coagulation Factor Replacement Therapy
SerpinPC for Severe Hemophilia
This trial tests a new injection called SerpinPC to prevent bleeding in people with severe hemophilia A or B. It aims to see if SerpinPC is safe and effective, especially for those who don't respond well to other treatments. SerpinPC works by helping the blood clot better. Serpins are being explored as potential treatments for hemophilia by rebalancing coagulation.
Phase 3 Trials
Gene Therapy
Gene Therapy for Hemophilia A
This trial tests a new gene therapy combined with anti-inflammatory medication for patients with severe hemophilia A. The treatment aims to help their bodies produce a necessary clotting protein and reduce inflammation.
Monoclonal Antibodies
Emicizumab for Hemophilia A
This trial tests emicizumab, a medication that helps blood clot, in people with mild or moderate hemophilia A. It works by mimicking a missing protein to improve blood clotting. Emicizumab has been approved for treatment of hemophilia A patients.
Coagulation Factor Replacement Therapy
Prophylactic Replacement Therapy for Hemophilia B
This trial is designed to see how well current prophylactic treatments work in hemophilia A and B patients before they receive gene therapy in a Phase 3 study. So far, only hemophilia B patients are being enrolled.
Gene Therapy
Gene Therapy for Hemophilia A
This trial is testing BMN 270, a new treatment for hemophilia A, to see if it can reduce bleeding episodes better than current treatments. It works by helping the body make more of a protein needed for blood clotting. BMN 270 has shown therapeutic levels of FVIII in mice and primates and normalization of bleeding in hemophilic mice.
Trials With No Placebo
Gene Therapy
Gene Therapy for Hemophilia A
This trial tests a new gene therapy combined with anti-inflammatory medication for patients with severe hemophilia A. The treatment aims to help their bodies produce a necessary clotting protein and reduce inflammation.
Monoclonal Antibodies
Emicizumab for Hemophilia A
This trial tests emicizumab, a medication that helps blood clot, in people with mild or moderate hemophilia A. It works by mimicking a missing protein to improve blood clotting. Emicizumab has been approved for treatment of hemophilia A patients.
Gene Therapy
Gene Therapy for Hemophilia A
This trial is testing a new gene therapy for hemophilia A. A virus is used to introduce a gene that creates a protein, Factor VIII, which helps the blood to clot. This protein is normally lacking in people with hemophilia A.
Virus Therapy
Gene Therapy for Hemophilia A
This trial is testing a new gene therapy to treat hemophilia A by delivering a healthy copy of the gene that's missing or defective in people with the disease. Researchers want to find the optimal dose of the therapy so that the body can produce enough clotting factor on its own.
Coagulation Factor Replacement Therapy
Prophylactic Replacement Therapy for Hemophilia B
This trial is designed to see how well current prophylactic treatments work in hemophilia A and B patients before they receive gene therapy in a Phase 3 study. So far, only hemophilia B patients are being enrolled.
View More Related Trials
Frequently Asked Questions
Introduction to hemophilia a
What are the top hospitals conducting hemophilia a research?
Hemophilia A, a rare bleeding disorder caused by a deficiency in clotting factor VIII, is receiving significant attention from leading hospitals around the world. In Chicago, Rush University Medical Center stands out among its peers with five active clinical trials dedicated to hemophilia A and an impressive track record of 14 completed trials since initiating its first investigation in 2006. Across the border in Canada, McMaster University Medical Centre at Hamilton Health Sciences has also made remarkable strides in hemophilia A research. Despite being relatively new to the field with only five all-time trials conducted since 2017, their commitment to innovation is evident through their ongoing efforts.
In Toronto's medical landscape, St. Michael's Hospital plays a pivotal role in advancing knowledge and treatment options for patients with hemophilia A. With an equal number of ongoing trials as their counterparts mentioned above and eight previously conducted studies since embarking on their initial trial back in 2008, they demonstrate unwavering dedication towards improving patient outcomes.
Meanwhile, across the Atlantic Ocean lies Arkansas Children's Hospital situated in Little Rock—a prominent institution actively involved in hemophilia A research too. Sharing similar statistics with other top hospitals regarding their current involvement with five ongoing clinical tests while having contributed nine completed investigations so far (since starting out ten years ago), this hospital showcases how geographical boundaries do not limit scientific advancements.
Lastly but certainly not least crucial is Novo Nordisk Investigational Site located within Hamilton - a facility that has become an influential force within hemophilia A research despite beginning its journey just seven years ago; it now boasts five active clinical trials along with twelve past undertakings.
These hospitals represent beacons of hope for those living with hemophilia A as they tirelessly work towards better understanding this complex disorder and developing novel treatment approaches that can improve patients' quality of life significantly This collective effort underscores our unwavering commitment to unraveling the mysteries surrounding this condition while ensuring brighter prospects for individuals affected by hemophilia A across the globe.
Which are the best cities for hemophilia a clinical trials?
When it comes to hemophilia A clinical trials, several cities have emerged as key hubs for research and innovation. Los Angeles, California leads the way with 30 active trials investigating treatments like efanesoctocog alfa (BIVV001), Damoctocog alfa-pegol (Jivi, BAY94-9027), and BAX 888. Hamilton, Ontario closely follows with 27 ongoing studies focused on promising options such as PF-06741086 and PF-06838435 (formerly SPK-9001). Additionally, Sacramento, California plays a significant role in advancing hemophilia A research with its 17 active trials exploring therapies like SB-525 (PF-07055480) and valoctocogene roxaparvovec. These cities provide individuals with hemophilia A access to cutting-edge clinical trials that pave the way for improved treatment options and better quality of life.
Which are the top treatments for hemophilia a being explored in clinical trials?
Exciting advancements are being made in clinical trials for the treatment of hemophilia A. Leading the charge is emicizumab, a drug currently being explored in four active trials and recognized for its potential in 18 all-time hemophilia A trials since its introduction in 2015. Another promising contender is PF-06741086, which has shown promise with two ongoing trials and five all-time studies since 2017. Valoctocogene roxaparvovec also holds great potential, with two current trials and three all-time studies since 2015. Additionally, concizumab and fitusiran have each gained attention with their participation in two active trials and six/all past time-honored efforts (starting from 2015) , along with fitusiran's inclusion in four previous investigations beginning as recently as 2018. These groundbreaking therapies offer renewed hope to individuals living with hemophilia A.
What are the most recent clinical trials for hemophilia a?
Exciting advancements are being made in the field of hemophilia A, with recent clinical trials offering potential breakthroughs in treatment options. One such trial is studying the efficacy of aPCC-emicizumab, which has reached Phase 3 and became available on 9/1/2023. Additionally, Coagulation Factor VIIa (Recombinant) is also being investigated as a potential therapeutic option for individuals with hemophilia A. This treatment has entered Phase 3 and became available on 9/1/2023. Another promising study focuses on NNC0365-3769 (Mim8) PPX, a novel intervention that holds great promise for those affected by this condition. This innovative approach reached Phase 3 and became available on 6/26/2023. These comprehensive clinical trials provide hope for improved treatments to enhance the quality of life for individuals living with hemophilia A.
What hemophilia a clinical trials were recently completed?
Several recent clinical trials have concluded, advancing our understanding and treatment options for hemophilia A. In February 2021, Bioverativ (a Sanofi company) completed a trial investigating the efficacy of efanesoctocog alfa (BIVV001). Additionally, in November 2019, another trial evaluating the same therapy was finalized. Earlier studies include a trial by Genzyme (a Sanofi Company), which wrapped up in July 2018, examining Fitusiran as a potential treatment. Furthermore, Hoffmann-La Roche conducted two trials on Emicizumab that were completed in January 2017 and September 2016 respectively. These significant accomplishments highlight ongoing efforts to enhance management strategies for individuals with hemophilia A.