All Currently Recruiting Bleeding Disorders Studies

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.
Additional Information/Links:
http://www.athn.org/

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.
Additional Information/Links:
http://www.athn.org/

The ATHN 10 Study- A Multicenter Project in which the American Thrombosis & Hemostasis Network (ATHN) will offer free genotyping to individuals with Rare Coagulation Disorders (RCD)

Overview: This is a multi-center project in which the American Thrombosis & Hemostasis Network (ATHN) will offer free genotyping to individuals with Rare Coagulation Disorders (RCD). The primary goal is to leverage the infrastructure at ATHN affiliated Hemophilia Treatment Centers (HTCs) and the laboratory services at CIBD to document genetic data on the population of RCD patients being cared for in U.S. HTCs.
Additional Information/Links:
http://www.athn.org/

The ATHN 11 Study- A multi-center observational study to evaluate long-term outcomes in hemophilia liver transplant recipients

Overview: This research is studying the long-term outcomes of liver transplants for people with hemophilia.
http://www.athn.org/

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: The DVT in Hemophilia (Mosh) Study

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.

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.

INHIBIT Phase III Inhibitor Clinical Trials Platform: Inhibitor Eradication Trial Comparing Eloctate Immune Tolerance Induction (ITI) plus Emicizumab vs. Eloctate ITI alone to Eradicate Inhibitor Formation in Individuals with Severe Hemophilia A

Overview: This is a multi-center randomized phase III clinical trial, the Inhibitor Eradication Trial, in which Eloctate ITI plus Emicizumab will be compared with Eloctate ITI alone to eradicate inhibitors in patients with severe hemophilia A This adaptive design is necessary as randomized trials in rare diseases are often not possible. The INHIBIT Clinical Trials Platform includes two linked trials, the Inhibitor Prevention Trial (Prevention Trial) and the Inhibitor Eradication Trial (Eradication Trial) that will be conducted at up to 41 U.S. hemophilia treatment centers (HTCs) affiliated with universities. The Inhibitor Eradication Trial is a 48-week randomized phase III trial, in which 90 previously treated patients (PTPs) with severe hemophilia A and high-responding inhibitors (anti-VIII > 0.6 B.U.), will be enrolled. Subjects will include individuals with severe hemophilia A who develop inhibitors during the linked Inhibitor Prevention Trial and adults or children at the same HTCs refractory to or never undergoing immune tolerance induction (ITI). Once enrolled, subjects who meet all the inclusion and none of the exclusion criteria, will be randomized to weekly Eloctate ITI plus weekly Emicizumab vs. weekly Eloctate ITI alone to eradicate inhibitor formation, defined as anti-FVIII<0.6 B.U.

More information can be found on https://clinicaltrials.gov/ NCT#: NCT04303572

INHIBIT Phase III Inhibitor Clinical Trials Platform: Inhibitor Prevention Trial Comparing Eloctate (rFVIIIFc) vs. Emicizumab (Hemlibra) to Prevent Inhibitor Formation in Individuals with Severe Hemophilia A

Overview: This is a multi-center randomized phase III clinical trial, the Inhibitor Prevention Trial, in which consecutive hemostatic agents will be compared using adaptive design to prevent inhibitors in patients with severe hemophilia A. This adaptive design is necessary as randomized trials in rare diseases are otherwise not possible. This adaptive design is necessary as randomized trials in rare diseases are otherwise not possible. The INHIBIT Clinical Trials Platform includes two linked trials, the Inhibitor Prevention Trial (Prevention Trial) and the Inhibitor Eradication Trial (Eradication Trial) that will be conducted at up to 41 U.S. hemophilia treatment centers (HTCs) affiliated with universities. The Inhibitor Prevention Trial is a 48-week randomized phase III trial, in which 66 previously untreated patients (PUPs) with severe hemophilia A will be enrolled. Subjects will include children from 4 months of age up to 4 years of age who have not been previously treated with clotting factor. Once enrolled, subjects who meet all the inclusion and none of the exclusion criteria will be randomized to preemptive weekly Eloctate (rFVIIIFc) vs. weekly Emicizumab (Hemlibra) to prevent inhibitor formation, defined as anti-FVIII >= 0.6 BU.

More information can be found on https://clinicaltrials.gov/ NCT#: NCT04303559

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: Pratt DLI-F8P Study

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.

rFVIIIFc-VWF-XTEND; BIVV001- EFC16295- A Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Pediatric Patients <12 Years of Age with Severe Hemophilia A

Overview: This study is being conducted to determine the safety, efficacy, and pharmacokinetics (PK) of BIVV001 administered as once-weekly (QW) prophylaxis treatment in previously treated pediatric patients <12 years of age with severe hemophilia A.

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: The SPARK 8011-101 Study

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.

More information can be found on https://clinicaltrials.gov/ NCT #: NCT03003533

The von Willebrand Disease Aging and Bleeding Correlation Study: VWD ABC Study

Overview: This study is being done to see if people with type 1 VWD experience age related increases in VWF levels and what effect this has on bleeding in this population. In order to discover this, the study will examine medical history, blood and genetic tests for VWD and bleeding and collect information about bleeds from the subjects.

VWD Aging & Surgery-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). More information can be found on https://clinicaltrials.gov/ NCT# NCT02606045

Prospective, Randomized Trial Comparing Recombinant von Willebrand Factor (rVWF) Plus Tranexamic Acid (TA) vs. rVWF Alone to Reduce Postpartum Hemorrhage in Women with von Willebrand Disease: The VWD-Woman Trial

Overview: This is a prospective, randomized single-center phase III pilot clinical trial comparing recombinant von Willebrand factor (rVWF, Vonvendi®) plus tranexamic acid (TA, Cyclokapron®) vs. rVWF alone to reduce postpartum hemorrhage (PPH) in women with von Willebrand disease (VWD).

More information can be found on https://clinicaltrials.gov/ NCT#: NCT04344860

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.

The Molecular and Clinical Biology of von Willebrand Disease: VWD PPG STUDY

Overview: This project is being done to compare bleeding symptoms (or lack of bleeding symptoms) to 1) routine laboratory tests used to diagnose VWD; 2) new genetic or specialized tests of how VWF works; 3) determine if other genes or proteins might cause you to have (or not have) bleeding symptoms or abnormal VWD lab tests.

Upcoming Bleeding Disorders Studies

The ATHN 7- A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Hemophilia

Overview: This research is studying the safety of the treatments used for people with hemophilia. The main purpose of this research is to study the use of bleeding disorder treatment products and their outcomes. This is very important as new Food and Drug Administration (FDA) approved treatment products for bleeding disorders are becoming available. We need to know the good and bad effects of these new treatments. The research is looking at any complications that may develop as you treat your bleeding disorder and how well you feel the treatment product is working.
http://www.athn.org/