All Currently Recruiting Bleeding Disorders Studies

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 
Nicole Myers, Research Nurse, 412-209-7261 nmyers@vitalant.org 
Megan Rode, Research Coordinator, 412-209-7563 mrode@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

Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A 

Overview: This open-label study is funded by ASC Therapeutics and is being done to investigate the safety and efficacy of an investigational drug, ASC-618, which is an Adenovirus- Associated Viral Vector-Mediated Gene Therapy of Human Factor VIII in Severe and Moderately Severe Hemophilia A. 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 #: NCT04676048

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

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

rFVIIIFc-VWF-XTEN; 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.

More information can be found on https://clinicaltrials.gov/  NCT #: NCT04759131

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 menstrual bleeding (HMB) (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

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.

 

Upcoming Bleeding Disorders Studies