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.
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.
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Trials for Hemophilia A Patients
Coagulation Factor Replacement Therapy
aPCC-Emicizumab Safety for Hemophilia A
This trial uses a special blood-clotting medicine along with emicizumab to help control bleeding in children and adults with hemophilia A who have inhibitors. The treatment works by helping the blood to clot better. Emicizumab is approved for regular use in patients with severe hemophilia A with and without inhibitors.
Gene Therapy
Gene Therapy for Hemophilia A
This trial tests a single treatment for adult males with severe hemophilia A. The treatment aims to help their bodies produce a necessary protein, reducing the need for regular preventive treatments. Research has been ongoing for a long time to enable the body to produce this protein and reduce bleeding episodes.
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.
Trials for Hemophilia B Patients
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.
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.
Gene Therapy
Factor IX Gene Therapy for Hemophilia B
This trial tests a new gene therapy treatment for adult men with severe hemophilia B. The treatment aims to help their bodies produce a crucial blood-clotting protein. Participants will receive one dose and be monitored over several years to see if it reduces their bleeding episodes. Gene therapy for hemophilia B has shown promising results in recent studies, suggesting potential long-term therapeutic production of the coagulant protein.
Phase 3 Trials
Coagulation Factor Replacement Therapy
aPCC-Emicizumab Safety for Hemophilia A
This trial uses a special blood-clotting medicine along with emicizumab to help control bleeding in children and adults with hemophilia A who have inhibitors. The treatment works by helping the blood to clot better. Emicizumab is approved for regular use in patients with severe hemophilia A with and without inhibitors.
Gene Therapy
Gene Therapy for Hemophilia A
This trial tests a single treatment for adult males with severe hemophilia A. The treatment aims to help their bodies produce a necessary protein, reducing the need for regular preventive treatments. Research has been ongoing for a long time to enable the body to produce this protein and reduce bleeding episodes.
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.
Trials With No Placebo
Coagulation Factor Replacement Therapy
aPCC-Emicizumab Safety for Hemophilia A
This trial uses a special blood-clotting medicine along with emicizumab to help control bleeding in children and adults with hemophilia A who have inhibitors. The treatment works by helping the blood to clot better. Emicizumab is approved for regular use in patients with severe hemophilia A with and without inhibitors.
Gene Therapy
Gene Therapy for Hemophilia A
This trial tests a single treatment for adult males with severe hemophilia A. The treatment aims to help their bodies produce a necessary protein, reducing the need for regular preventive treatments. Research has been ongoing for a long time to enable the body to produce this protein and reduce bleeding episodes.
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.
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Frequently Asked Questions
Introduction to Hemophilia
What are the top hospitals conducting Hemophilia research?
When it comes to advancing the treatment and care for individuals with Hemophilia, several hospitals are leading the way in clinical trials. In Chicago, Rush University Medical Center is at the forefront of research with five ongoing trials focused on this rare bleeding disorder. With a history dating back to 2006 when they conducted their first Hemophilia trial, Rush University has since accumulated a total of 14 studies dedicated to improving the lives of those affected by this condition. Meanwhile, McMaster University Medical Centre in Hamilton, Canada has made significant strides as well. Despite being relatively new to conducting Hemophilia trials since 2017, they have already launched five active investigations.
In Toronto's St. Michael's Hospital, researchers are also putting forth considerable effort with five ongoing Hemophilia trials and eight completed studies since delving into this field in 2008. Furthermore, Arkansas Children’s Hospital holds its place among the top institutions contributing to Hemophilia research where they currently have five active clinical trials and nine completed ones that commenced back in 2011.
On the West Coast of the United States lies another key player making a difference: The Orthopaedic Institute for Children located in Los Angeles. Although smaller in scale compared to others on this list, they have four ongoing Hemophilia trials and participated in an additional five previously conducted studies after initiating their first trial related to hemophiliac patients' welfare back around six years ago since2015.
These hospitals represent beacons of hope for individuals living with Hemophilia worldwide who face daily challenges due to uncontrolled bleeding tendencies triggered by even minor injuries or spontaneous occurrences within their bodies. By actively engaging in these clinical trials — investigating new therapies or improving existing treatments — medical professionals are working towards finding innovative approaches that could potentially transform lives positively while ensuring better management options for future generations impacted by this hereditary blood disorder
Which are the best cities for Hemophilia clinical trials?
In the realm of Hemophilia clinical trials, several cities are at the forefront of research and development. Los Angeles, California leads with 25 active trials focusing on promising treatments like efanesoctocog alfa (BIVV001), Damoctocog alfa-pegol (Jivi, BAY94-9027), and BAX 888. Hamilton, Ontario follows closely behind with 22 ongoing studies investigating therapies such as PF-06741086 and PF-06838435 (formerly SPK-9001). Additionally, Sacramento, California boasts 17 active trials exploring treatments like SB-525 (PF-07055480) and valoctocogene roxaparvovec. These cities offer individuals living with Hemophilia access to cutting-edge clinical trials that pave the way for advancements in care and improved quality of life.
Which are the top treatments for Hemophilia being explored in clinical trials?
Exciting advancements are being made in Hemophilia research, with several potential treatments showing promise in ongoing clinical trials. Leading the way is emicizumab, currently being explored in four active trials and 18 all-time Hemophilia studies since its introduction in 2015. Another contender worth mentioning is PF-06741086, which has garnered attention with two active and five all-time Hemophilia trials since its listing in 2017. Additionally, valoctocogene roxaparvovec and fitusiran have both demonstrated potential as they undergo testing in two active trials each. These innovative therapies bring hope for improved management of this genetic bleeding disorder that affects many individuals worldwide.
What are the most recent clinical trials for Hemophilia?
Exciting advancements are being made in the field of hemophilia, with recent clinical trials offering promising insights into potential treatment options. One notable study is investigating aPCC-emicizumab, a novel therapeutic approach for individuals with hemophilia. Alongside this, the efficacy of coagulation Factor VIIa (Recombinant) is also being evaluated as a potential treatment option. Additionally, marstacimab (PF-06741086), an innovative drug candidate, is undergoing Phase 3 trials to assess its effectiveness in managing hemophilia symptoms. These studies bring hope and anticipation for improved therapies that can enhance the quality of life for those living with this condition.
What Hemophilia clinical trials were recently completed?
Recently, several important clinical trials in the field of hemophilia have reached completion, offering potential advancements in treatment. In February 2021, a trial sponsored by Bioverativ, a Sanofi company, concluded its investigation into efanesoctocog alfa (BIVV001). This was followed by another trial for the same therapy completed in November 2019. Furthermore, Genzyme, a Sanofi Company, successfully finished their Fitusiran trial in July 2018. Earlier studies included an Emicizumab trial sponsored by Hoffmann-La Roche that ended in January 2017 and another Emicizumab trial completed in September 2016. These significant milestones highlight ongoing efforts to improve outcomes for individuals living with hemophilia and bring hope to patients worldwide.