The ATHN Data Set, a limited data set related to Bleeding and Clotting Disorders
Overview: This is a national registry study funded by the American Thrombosis and Hemostasis Network. Patients of all ages, seen at HCWP, who have been diagnosed with a bleeding or clotting disorder, will be invited to participate. The study involves only collection of health information.
The ATHN Serological Surveillance Study
Overview: The goal of this study is to monitor health indicators among populations with bleeding disorders in order to assess trends over time, measure rates of and risk factors for complications, identify high risk populations for prevention and identify issues that require research. The study involves only collection of blood and health information.
The ATHN 7, Hemophilia Natural History Study
Overview: The goal of this study is to determine the safety of non-factor products, bypassing agents or clotting factor replacement products when used for participants with hemophilia with or without inhibitors.. The study involves only collection of blood and health information.
CDC Public Health Surveillance for Bleeding Disorders – Registry for Bleeding Disorders Surveillance
Overview: With funding from the CDC to the American Thrombosis and Hemostasis Network, the goal of this study is to monitor health indicators among populations with bleeding disorders in order to assess trends over time, measure rates of and risk factors for complications, identify high risk populations for prevention and identify issues that require research.
An Observational Study of Postoperative Deep Venous Thrombosis (DVT) in Hemophiliacs Undergoing Major Orthopedic Surgery
Overview: This observational study is sponsored by the University of North Carolina at Chapel Hill, with funding from CSL Behring Foundation for Research and Advancement of Patient Health. The study will enroll adult males with hemophilia A or B who are scheduled to undergo total hip or knee replacements. The purpose of this research study is to learn more about rates of ultrasound detectable blood clots and potentially provide prevention guidelines in this setting.
A Phase IV trial in patients with hemophilia A, Phase IV, Multicenter, Single-arm, Open-label Study of Emicizumab prophylaxis in patients with hemophilia A with or without inhibitors undergoing minor surgical procedures
Overview: This study will evaluate the safety and efficacy of emicizumab prophylaxis in patients with hemophilia A (PwHA; with or without inhibitors) that are undergoing minor surgical procedures without additional prophylaxis with bypassing agents (BPA; for patients with inhibitors) or factor VIII (FVIII; for patients without inhibitors).
Genotype and Adeno-Associated Virus (AAV) Immune Status of Patients with Inherited Blood Diseases
Overview: This study is funded by the Hemophilia Center of Western PA and is a pre-screening study for participation in gene transfer studies. This study will enroll adult males with Hemophilia A and B to (1) have genetic testing done for hemophilia and (2) test for immunity to a virus (known as AAV) that is used to make several gene therapy drugs. Those who are discovered not immune to AAV through participation in this study may be able to enroll in a gene therapy trial at the center.
HCWP/ITxM Hemostasis and Thrombosis Research Registry
Overview: This is a research registry and repository is sponsored by the Hemophilia Center of Western PA. The study will enroll adults and children with bleeding disorders who receive care at our center. The purpose of the study is to (1) allow us to contact patients about potential research opportunities and (2) permit information and yearly blood samples to be collected for research.
Weight-Based Dosing in Hemophilia A: A randomized, controlled, open-label, crossover trial to measure factor VIII recovery following factor VIII concentrate dosing based on total body weight, ideal body weight, and lean body mass
Overview: This study is sponsored by the Hemophilia Center of Western PA. The study will enroll adult males with hemophilia A to test whether bleeds are effectively controlled on lower doses of factor VIII if the dosage is based on lean body mass and ideal body weight (what a person should weigh) rather than on what a person actually weighs.
OMICS Studies to Develop New Therapies for Hemophilia
Overview: The goal of this research is to utilize the tools of precision medicine, including platelet and vascular (blood vessel) genomics (study of genes and gene function), and the microbiome (microorganisms such as bacteria and their genetic material that are present in or on the human body) to help understand the relationship between hemophilia phenotype (severity) and complications such as inhibitor formation.
Design of Less Immunogenic Factor VIII Proteins
Overview: The goal of this research is to look at Hemophilia A patients who have developed an immune response to their VIII treatments. In this study we would like to understand why some people make inhibitors to Factor VIII.
Gene-transfer, open-label, dose-escalation study of SPK-8011 [adeno-associated viral vector with B-domain deleted human factor VIII gene] in individuals with hemophilia A
Overview: This study is funded by Spark Therapeutics and is being done to investigate the safety and efficacy of an investigational drug, SPK-8011, which is a genetic therapy that is being tested for use in patients with hemophilia . The drug is given as an IV medication that is expected to help the body to make its own factor.
Normalized von Willebrand factor levels in type 1 von Willebrand disease and risk of bleeding with invasive procedures
Overview: This study is being done to compare the risk of bleeding in patients with a history of type 1 VWD and current normal VWF levels not receiving VWD treatment during surgery and people with a history of type 1 VWD and current decreased VWF levels receiving VWD treatment during surgery.
Prospective, Randomized, Crossover Trial Comparing Recombinant Von Willebrand Factor (rVWF) Combined with Tranexamic Acid (TA) vs. Tranexamic Acid (TA) Alone to Minimize Menorrhagia in Women with Von Willebrand Disease: The VWD Minimize Study
Overview: The purpose of this research study is to compare the use of recombinant von Willebrand factor (rVWF) and tranexamic acid (TA, Lysteda®) in women with von Willebrand Disease (VWD) who have heavy monthly periods (also called menorrhagia).
Pregnancy Loss in von Willebrand Disease: a Multi-Centre Retrospective Cohort Study
Overview: The purpose of the study is to see if there are any problems during pregnancy for patients who have von Willebrand disease.