Boston Hemophilia Center | Research & Innovation

For many children with rare or hard-to-treat conditions, research provides an opportunity for patients, families, providers and scientists to better understand the disease, how to manage it, and potentially to develop new treatment options. There are dozens of clinical trials currently open and listed on clinicaltrials.gov for patients with bleeding disorders. We offer clinical studies in hemophilia, von Willebrand disease and young women with bleeding disorders that are developed and lead at our Center, as well as those sponsored by other institutions and pharmaceutical companies..

Gene therapy trials in Hemophilia

Gene Therapy provides an opportunity to provide the body with instructions on how to make a particularly protein. In the case of hemophilia, these proteins are factor VIII or factor IX.

Observational Studies

Hemophilia

  • P-TAP Hemo: Pharmacokinetic Tailored Approach to Prophylaxis in Hemophilia is a prospective study investigates the utility of integrating pharmacokinetic profiles in to prophylactic patient factor VIII replacement regimens. 
  • ATHN 2: Switching Study, This longitudinal, observational study aims to define the inhibitor incidence and strength (based on Bethesda Units) for previously treated patients with hemophilia A or B switching to a new factor replacement product.
  • eTHINK: Evolving Treatment of Hemophilia’s Impact on Neurodevelopment, Intelligence and Other Cognitive Functions
  • B-natural: Natural history study of factor IX treatment and Complications, This investigator-initiated academic collaboration is a prospective longitudinal, observational study to characterize the hemophilia B siblings or hemophilia B patients with inhibitors in terms of their medical history, patterns of bleeding, clinical management, quality of life, and complications including the development of joint disease, inhibitory antibodies to FIX, use of immune tolerance induction (ITI) 
  • CDC Public Health Surveillance for Bleeding Disorders: The purpose of this project is to monitor the health status of people with hemophilia and other bleeding disorders. This will help HTCs and CDC better understand the health issues of people with bleeding disorders.

Women with bleeding disorders 

  • ATHENA: Characterizing the impact and treatment of reproductive tract bleeding on women and post-menarchal girls with bleeding disorders

Publications highlighting prior clinical trials and clinical-care innovations

Hemophilia

Von Willebrand Disease (VWD)

Women with bleeding disorders