Clinical Trials and Research
The Hemophilia Research Program is directed at advancing new therapies, evaluating novel aspects in hemophilia care, and creating new knowledge regarding the evolving needs of the aging patient with hemophilia. The program is comprised of both clinical trials and translational & basic research in hemophilia. Our patients have the opportunity to participate in cutting-edge research and contribute to scientific and medical knowledge in a variety of innovative ways.
Our center stages clinical trials aimed at studying novel clotting factor preparations. The trials are industry-sponsored, multi-center trials carried out in a number of centers throughout the United States and around the world. Patient participation helps to develop new treatment options that may be more effective to stop bleeding, or provide longer lasting bleed protection. Trial implementation (from site initiation to first patient included) can take many months as study protocols are rigorously examined for their validity and safety by our center and by the UCSD Human Research Protection Program (HRPP).
There is no cost for the studies or clotting factor preparations, and participants are reimbursed for time, effort, and travel costs. To learn more, call the Hemophilia Treatment Center, 619-471-0335, Monday to Friday, 8:30 a.m. – 4:30 p.m.
Clinical trials currently open for enrollment
- For patients with severe Hemophilia A: A Phase II/III, multicenter, partially randomized, open label trial investigating safety and efficacy of on-demand and prophylactic treatment with BAY 94-9027 in Severe Hemophilia A.
Sponsor: Bayer Healthcare
- For patients with Hemophilia B: A Phase I/II Open-Label, Single Ascending Dose Trial of a Self-Complementing Optimized Adeno--associated Virus Serotype 8 Factor IX Gene Therapy (AskBio009) in Adults with Hemophilia B
- For patients with Hemophilia A: A Phase I/III Open-label, Multicenter, Crossover Safety, Efficacy and Pharmacokinetic Study of Recombinant Coagulation Factor VIII (rFVIII) Compared to RecombinantHuman Antihaemophilic Factor VIII (rFVIII; INN: octocog alfa) in Subjects with Hemophilia A, and a Repeat PK, Safety and Efficacy Study
- For patients with Hemophilia A or B: An Ascending Single Dose Study To Evaluate The Safety , Tolerability And Pharmacokinetics/PharmacoDynamics of PF-05280602, A Recombinant Factor VIIa Variant (813D), In Adult Hemophilia A and B Subjects With Or Without Inhibitors
Clinical trials in preparation
- A Phase 2b, Multicenter, Open-Label Study to Investigate the Efficacy and Safety of Sofosbuvir/Ledipasvir Fixed-Dose Combination and Sofosbuvir + Ribavirin for Subjects with Chronic Hepatitis C Virus (HCV) and Inherited Bleeding Disorders. Protocol ID# GS-US-334-1274
For all patients with bleeding disorders:
Completed clinical trials
Translational and Basic Research
Dr. von Drygalski's research interests involve basic, translational, and clinical science in the field of hemostasis and thrombosis. Additionally, Dr. von Drygalski and her team are interested in furthering the understanding of morbidities such as joint disease and cardiovascular disease in adult patients with hemophilia.
Heart health is emerging as important for patients with hemophilia. For many decades, it was believed that patients with hemophilia were protected against heart disease and stroke as their "blood is naturally thin". Patients with hemophilia were considered "hypocoagulable". The aging population with hemophilia teaches us that this paradigm may not be true. Through our research efforts, we recently discovered that patients with hemophilia suffer more frequently from dangerously high blood pressure than the general population. (Read article.) This is of major concern as high blood pressure increases the risk for intracranial bleeding (stroke). Reasons for the prevalence of high blood pressure are poorly understood; as such, we are currently investigating this. Our center has launched a cross-sectional and longitudinal study in collaboration with the Division of Preventive Medicine at UC San Diego to assess heart health and risk factors for heart disease in hemophilia. This study receives funding support from Baxter Biosciences. Patients of all ages with hemophilia are welcome to join and contribute to new knowledge which ultimately will inform the medical community, result in better prevention and treatment strategies, and improve health outcomes of patients with hemophilia.
Joint health and "Hemophilic Arthropathy in Adulthood"
Joint health and "Hemophilic Arthropathy in Adulthood" is another emerging aspect of hemophilia health care and well-being. Hemophilic arthropathy is debilitating and therefore requires prevention and appropriate treatment. Unfortunately, even in the modern era of prophylactic clotting factor replacement therapy, the development of joint disease often cannot be avoided entirely. Reasons for this are somewhat unclear, but one may hypothesize that some patients’ joints react more sensitively to bleeding than others (i.e. few bleeds may suffice to cause permanent harm), that bleeding sometimes goes unnoticed (subclinical bleeding), or that other mechanisms related to bone health contribute to an unknown extent. We previously demonstrated that Musculoskeletal Ultrasound (MSKUS) has a high sensitivity to distinguish between bleeding and inflammation during painful episodes. (Read article.) We are about to launch a study investigating joint disease through MSKUS, Magnetic Resonance Imaging (MRI), functional joint health assessments, and biomarker studies in relation to personal metabolism of clotting factor preparations. We will do so in collaboration with our colleagues in Rheumatology and Radiology at UCSD as well as basic researchers at The Scripps Research Institute to investigate molecular aspects of hemophilic arthropathy. The ultimate goal is to shed light on the pathophysiology and progression of hemophilic arthropathy as the basis for the development of modern and direct intervention strategies to reduce the vicious cycle of bleeding, inflammation, pain and progression of disease.This study receives funding support from Biogen Idec. Patients of all ages with hemophilia are welcome to join and contribute to new knowledge which ultimately will inform the medical community, result in better prevention and treatment strategies, and improve outcomes of patients with hemophilia.
New bypassing strategies for inhibitors
Dr. von Drygalski is interested in novel bypassing strategies for patients with hemophilia and inhibitors. She was the 2012 recipient of a Bayer Early Career Investigator Award which allows her to study "FVa activity augmentation as a novel bypassing strategy in hemophilia" in collaboration with basic scientists at The Scripps Research Institute. Read more. The research team engineered a novel FVa molecule, named superFVa, which was found to have superior hemostatic properties in hemophilic plasma and controls bleeding in hemophilic mice. (Read article.) The team is now studying this novel molecule in hemophilia with inhibitors.
Molecular advances in hemophilic joint disease
Dr. von Drygalski is also interested in molecular aspects of hemophilic joint disease on a basic scientific level. One aspect involves a specific anti-fibrinolytic and anti-inflammatory protein called Thrombin Activatable Fibrinolysis Inhibitor (TAFI). To investigate how TAFI may contribute to joint disease and to study how strategies involving TAFI could contribute to decreased joint bleeding and inflammation, Dr. von Drygalski was awarded a Career Development Award by the National Hemophilia Foundation in 2013. This award was sponsored by Novo Nordisk. Read more.
Dr. von Drygalski works in close collaboration with basic scientists at the Scripps Research Institute. Look into their labs:
The team approach provides a unique platform to study the cross-talk between bleeding and inflammation in the joint, novel bypassing strategies for patients with inhibitors, immune tolerization, and to identify novel approaches for the treatment and prevention of bleeding and hemophilic arthropathy.