View clinical trials related to Hemophilia A.
Filter by:In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector). Gene therapy is a very new medical technique being used in a number of clinical studies for diseases such as cancer and cystic fibrosis. At this time, the U.S. Food and Drug Administration has approved no gene transfer products for commercial use. To date, 8 subjects have received AAV vector in the muscle for a hemophilia B trial by intramuscular injection, and, to date, 6 subjects have been treated with AAV vector in the current hemophilia B liver trial. Eleven cystic fibrosis subjects have received AAV vector into their nasal sinuses or lungs to date. In this study, AAV human Factor IX vector will be injected into the liver using a catheter inserted into a large blood vessel (called the proper hepatic artery or the right hepatic artery).
The purpose of this study is to determine the relative bioavailability of ReFacto AF as compared to ReFacto, when each is administered as 2-minute bolus infusions.
To identify the causative mutations in previously untreated patients with hemophilia A enrolled in the ReFacto® clinical safety and efficacy study CTN 93-R833-0XX/C9741-28, using two established hemophilia mutation testing laboratories (one in Europe and one in North America).
To characterize the safety and efficacy of rFIX in children less than 6 years of age with severe hemophilia B in the setting of acute bleeding episodes, prophylaxis, and/or surgery. This study will provide an opportunity for systematic observation of treatment with rFIX in children less than 6 years of age regardless of prior FIX treatment. Younger patients exhibit a different pharmacokinetic profile and therefore may respond differently to rFIX infusions when compared with older children and adults. This evaluation will provide data from which recommendations can be made regarding rFIX dosing and treatment of these patients. Surveillance for certain observations that have been made in patients treated with rFIX in the clinical and postmarketing setting will be performed, including inhibitor development, thrombogenicity, FIX recovery/lack of effect, allergic-type manifestations, and RBC agglutination. Comparisons will be derived from published reports and communications describing experience with other FIX products and protein therapeutics in general.
To characterize the safety and efficacy of ReFacto AF in treating acute bleeding episodes during prophylaxis treatment, including neoantigenicity.
To define the natural history, immunologic, and genetic factors that influence the clinical outcome of hepatitis C in a cohort of hemophilic siblings.
To determine the risk factors associated with inhibitor formation in hemophilia A and to study the mechanism of tolerance in the murine hemophilia A model.
To examine mechanisms of individual differences in the progression of HIV infection in hemophiliacs.
To elucidate the role of hepatitis delta virus (HDV) in the development of chronic liver disease in patients with hemophilia.
To determine the prevalence of hepatitis delta virus (HDV) in a large cohort of hemophiliacs and to elucidate the role of HDV in the development and progression of liver disease in this population.