Please feel free to contact any of our Research Staff for any questions on a study you are interested in hearing more about.
Our research team email is: dl-slt_hcwp_research@vitalant.org
Debbie Vehec, Research Nurse, 412-209-7564 dvehec@vitalant.org
Christiana Ekekwe, Research Nurse, 412-209-7292 cekekwe@vitalant.org
Brandon Lawryk, Research Coordinator, 412-209-7563 blawryk@vitalant.org
Gene Therapy Studies:
Gene-transfer, open-label, dose-escalation study of SPK-8011-101 [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 gene 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
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
Overview: This study is funded by Spark Therapeutics and is being done to investigate the safety and efficacy of an investigational drug, SPK-8016, which is a gene 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 #: NCT03734588
Clinical Drug Studies:
A Phase 1, Open-Label Study to Assess the Pharmacokinetics, and Safety and Tolerability of a Single Intravenous Injection of rFVIIIFc-VWF-XTEN (BIVV001) in Adults with type 2N and 3 von Willebrand disease (VWD) BIVV001 PK Study
Overview: This study is being conducted to assess the safety and tolerability of a single IV dose of BIVV001 in adult patients with type 2N and 3 VWD
More information can be found on https://clinicaltrials.gov/ NCT #: NCT 04770935
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
Non-Drug 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 collection of blood and health information.
Additional Information/Links:
http://www.athn.org/
The ATHN 9 Study- A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Severe Von Willebrand Disease (VWD)
Overview: The overarching objective of this longitudinal, observational and prospective study is to characterize the safety and effectiveness of factor replacement in participants enrolled in the ATHNDataset with clinically severe congenital VWD (VWF:Ag, VWF:GPlbM or VWF:RCo), ≤30% or ≤40% of normal, with severe bleeding phenotype defined as requiring recurrent use of factor concentrates).
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 document genotype data on the population of RCD patients being cared for in U.S. HTCs.
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 in people with hemophilia and chronic liver disease.
http://www.athn.org/
ATHN PoP Profile Study- 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.
HCWP/ITxM Hemostasis and Thrombosis Research Registry
Overview: This is a research registry and repository 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.
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.
The von Willebrand Disease Aging and Bleeding Correlation Study: VWD ABC Study
Overview: This multi-center study will determine 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 determine this, the study will examine medical history, blood and genetic tests for VWD and bleeding and collect information about bleeds from study 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 compares 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 the risk of bleeding in people with a history of type 1 VWD and current decreased VWF levels receiving VWD treatment during surgery.
Pregnancy Loss in von Willebrand Disease: a Multi-Center Retrospective Cohort Study
Overview: The purpose of the study is to determine bleeding and other complications during pregnancy for patients who have von Willebrand disease.