Brief Title
Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia
Official Title
A Global Epidemiologic Study to Determine the Prevalence of Neutralizing Antibodies and Related Adaptive Immune Responses to Adeno-Associated Virus (AAV) in Adults With Hemophilia
Brief Summary
Assess the seroprevalence of neutralizing antibodies (NAb) to AAV in adults with severe hemophilia A (coagulation factor VIII [FVIII] <1%) or moderately severe to severe hemophilia B (coagulation factor IX [FIX] ≤2%).
Study Type
Observational
Primary Outcome
Prevalence of neutralizing antibodies (NAb) - Baseline visit
Secondary Outcome
Prevalence of NAb to AAV including AAV2 and AAV8
Condition
Hemophilia A
Intervention
Non-treatment, seroprevalence
Study Arms / Comparison Groups
Hemophilia A
Description: Participants with hemophilia A
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Other
Estimated Enrollment
250
Start Date
June 14, 2017
Completion Date
April 30, 2022
Primary Completion Date
April 30, 2022
Eligibility Criteria
Inclusion Criteria: 1. Participant is male between 18 and 75 years old at the time of screening. 2. Established severe hemophilia A (plasma Factor VIII (FVIII) activity <1%) or B (plasma Factor IX (FIX) activity ≤2%). 3. Provision of signed informed consent form (ICF). 4. Participant is willing and able to comply with the requirements of the protocol. Exclusion Criteria: 1. Bleeding disorder(s) other than hemophilia A or B. 2. Personal laboratory evidence of having developed inhibitors to FVIII or FIX protein at any time (≥0.6 Bethesda Units [BU] on any single test). 3. Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy, or monoclonal antibody therapy. 4. Received systemic antiviral and/or interferon therapy within 30 days of blood draw, with the exception of treatment for human immunodeficiency virus (HIV). 5. Currently receiving ribavirin and/or interferon based therapy for active hepatitis C virus (HCV). 6. Received immunoglobulin therapy or plasma infusion within 120 days of the blood draw. 7. Has a known immune deficiency other than HIV. 8. Has lymphocyte or plasma cell malignancies. 9. Participant is a family member or employee of the investigator.
Gender
Male
Ages
18 Years - 75 Years
Accepts Healthy Volunteers
No
Contacts
Study Director, ,
Location Countries
Austria
Location Countries
Austria
Administrative Informations
NCT ID
NCT03185897
Organization ID
201601
Responsible Party
Sponsor
Study Sponsor
Baxalta now part of Shire
Study Sponsor
Study Director, Study Director, Shire
Verification Date
December 2019