Shared Decision-Making for Hemophilia Gene Therapy
Dr. Courtney Thornburg and her research team at Rady Children’s Hospital-San Diego (RCHSD) Hemophilia Treatment Center are conducting a research study about shared decision-making for hemophilia gene therapy. The study is funded by the National Hemophilia Foundation Innovative Investigator Award and BioMarin Pharmaceuticals.
The study is designed to develop shared decision-making tools that can be used in clinical discussions with patients with hemophilia who may be eligible for gene therapy. Dr. Thornburg and her research team developed a prototype of the Clinician Decision Box tool, and they are looking for feedback from HTC healthcare providers prior to pilot testing with patients. The research involves reviewing the tool and providing feedback during a 45-60 minute interview by telephone or video conference with a member of the research team. Participants who complete the interview will receive a $100 electronic gift card to compensate for their time.
If you would like to participate, please contact our team via e-mail at: [email protected] or via phone at: 858-966-1700 x 227376 or complete this four-question form to provide your contact information to the study team.
Clinical Research Study for Immune-Mediated Thrombotic Thrombocytopenic Purpura
The VITA Study is looking for adults ages 18 or older who are diagnosed with immune-mediated thrombotic thrombocytopenic purpura (iTTP) and experiencing an acute episode. The purpose of the study is to evaluate the safety and effectiveness of an investigational study drug, called TAK-755, in combination with little to no plasma exchange (PEX). The study drug is available at the institutions listed below. To learn more, please contact a participating site or click on the Clinicaltrials.gov link below.
University of Minnesota Med CAR
Minneapolis, Minnesota, United States, 55455
Contact: Site Contact 612-626-3757
Duke University Medical Center
Durham, North Carolina, United States, 27710
Contact: Site Contact 919-684-5350
Leo Jenkins Cancer Center/ECU School of Medicine
Greenville, North Carolina, United States 27834
Contact: Site Contact 252-744-3556
Ohio State University
Columbus, Ohio, United States 43210
Contact: Site Contact 614-293-2887
The PREDICT study investigators are conducting a Phase 4 interventional trial to assess the effect of using a new risk score for persons with hemophilia A transitioning from a standard half life (SHL) FVIII to an extended half life FVIII product (Jivi® [antihemophilic factor (recombinant), PEGylated-aucl]). The study may help to determine the most appropriate prophylaxis dosing regimen when switching factor VIII products.
This is a 6-month study which is currently recruiting and open to males and females ≥12 years of age with hemophilia A of any disease severity on prophylaxis for which study drug is provided. The study design requires few visits and blood draws. Its outcome is intended to provide helpful information to the community on the care and management of patients with hemophilia A. If you are interested in becoming a treatment site or wish to refer a patient, please contact Bayer using the information below.
PREDICT Study Website
For more information about this study or to submit a request to speak to a medical expert, please visit Prophylaxis regimen for hemophilia A patients (bayer.com).
In addition, you may also contact Jessica Charlet at [email protected] or telephone (412) 660-6440.