Clinical Trials and Research: Hemophilia and Thrombosis Treatment Center
The Hemophilia Research Program is directed to advance new therapies, evaluate novel aspects in hemophilia care, and create new knowledge regarding the evolving needs of the aging patient with hemophilia. The program comprises clinical trials as well as translational and basic research in hemophilia. Our patients have the opportunity to participate in cutting-edge research and contribute to knowledge in a variety of innovative ways.
Our center stages clinical trials to study 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 by developing 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, since 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 you 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.
Trials currently in preparation
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
Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A
Sponsor: Biogen-Idec Hemophilia
Pivotal Study (Pharmacokinetics (PK), Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B
Sponsor: Baxter Healthcare Corporation
Translational and Basic Research
Dr. von Drygalski's research interests involve basic, translational and clinical science in the field of hemostasis and thrombosis. In addition, 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 the concept prevailed that patients with hemophilia are protected against cardiovascular disease and stroke since 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. However, we understand little about risk factors of heart disease, high blood pressure and stroke in hemophilia.
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. All patients with hemophilia at all ages 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.
Joint Health and "Hemophilic Arthropathy in Adulthood"
These are also emerging as important new aspects of hemophilia health care. The objective is to evaluate the contributions of bleeding, microbleeding and joint inflammation on progression of joint disease. To this end patients are invited to donate blood samples, joint fluid samples, tissue and urine for biomarker research and biobanking of specimen. Results from those studies can for example be correlated with joint imaging studies, clotting factor consumption and functional joint assessments to improve our understanding of the pathophysiology and progression of hemophilic arthropathy. The ultimate goal is to find beneficial intervention strategies. These studies are done in collaboration with rheumatology, radiology, UCSD bioengineering and a basic arthritis laboratory at The Scripps Research Institute.
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 will allow 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.