Hemophilia A Clinical Trial
— PASsOfficial title:
Inhibitor Development in Patients With Hemophilia A Undergoing Surgery
Verified date | April 2017 |
Source | Emory University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
Hemophilia A is a genetic deficiency of factor VIII that causes blood to clot too slowly. The disease is classified based on how much factor VIII is in the blood. People with mild or moderate hemophilia A have low, but detectable, blood levels of factor VIII and bleed with trauma or surgery. At the time of surgery, they need to receive factor VIII replacement by infusion into the vein so that blood can clot normally and abnormal bleeding can be avoided. A complication of hemophilia A is the development of an antibody that binds factor VIII and makes the factor VIII infused for treatment not work properly. This antibody is called an inhibitor. In mild and moderate hemophilia A, inhibitors are not common, but have been reported to occur after intensive factor VIII infusions, as may occur at the time of surgery. This study is designed to observe people with mild and moderate hemophilia A who are having surgery. Information on the surgery, treatments given, bleeding, and infection will be gathered. Also, blood will be drawn to determine how the immune system is reacting to the factor VIII. No specific treatments will be given as part of this study. We will use the information to determine what influences inhibitor development. A better understanding of inhibitor development will help medical providers do things to avoid inhibitor development in this population or researchers to design new treatments.
Status | Completed |
Enrollment | 30 |
Est. completion date | March 2016 |
Est. primary completion date | February 2016 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | N/A and older |
Eligibility |
Inclusion Criteria: - Males with mild/moderate hemophilia A (fVIII activity 1-40%) - Planned surgical intervention which is anticipated to require 5 consecutive days of fVIII replacement therapy (These can be outpatient or inpatient treatment days.) - Weight >22.5 kg (To assure that volumes of blood to be drawn for study purposes are safe.) Exclusion Criteria: - Past history of an inhibitor (inhibitor titer >0.4 BU/ml) - HIV infection with CD4 count <400/ul - Currently receiving immunosuppressive medication(s) - Unable to tolerate quantity of blood to be drawn - Current or past diagnosis autoimmune disorder - Current or past diagnosis of immune deficiency disorder other than HIV |
Country | Name | City | State |
---|---|---|---|
United States | Emory University Comprehensive Hemophilia Treatment Center | Atlanta | Georgia |
United States | University of Colorado, Hemophilia and Thrombosis Center | Aurora | Colorado |
United States | University of North Carolina | Chapel Hill | North Carolina |
United States | The University of Texas Health Science Center at Houston | Houston | Texas |
United States | Indiana Hemophilia and Thrombosis Center | Indianapolis | Indiana |
United States | University of Pittsburgh and Hemophilia Center of Pennsylvania | Pittsburgh | Pennsylvania |
United States | Oregon Health & Science University | Portland | Oregon |
Lead Sponsor | Collaborator |
---|---|
Emory University | National Heart, Lung, and Blood Institute (NHLBI), National Institutes of Health (NIH) |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Inhibitor development (inhibitor titer > 0.4 BU/ml) | Primary Study Endpoint: Inhibitor development (inhibitor titer > 0.4 BU/ml) by post-operative (POD) day 90. Three months or 90 days was selected as the primary end point based on data collected in the case-control study where 17/18 cases had developed their inhibitor within 12 weeks of their intensive fVIII treatment and only 1 case developed the inhibitor >16 weeks after the intensive fVIII treatment. | postopereratvie date 90 |
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