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
Enrollment
242
Non-treatment study examining the prevalence of preexisting immunity to adeno-associated virus (AAV)
Orthopaedic Hemophilia Treatment Center
Los Angeles, California, United States
Prevalence of neutralizing antibodies (NAb) - Baseline visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Time frame: Baseline visit
Prevalence of neutralizing antibodies (NAb) - Year 1 Visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Time frame: Year 1 visit
Prevalence of neutralizing antibodies (NAb) - Year 2 Visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Time frame: Year 2 visit
Prevalence of neutralizing antibodies (NAb) - Year 3 Visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Time frame: Year 3 visit
Prevalence of NAb to AAV including AAV2 and AAV8
Prevalence of neutralizing antibodies (NAb) to adeno-associated virus (AAV) including AAV2 and AAV8
Time frame: Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit
Prevalence of binding antibodies to AAV, including AAV8 and AAV2
Prevalence of binding antibodies to adeno-associated virus (AAV), including AAV8 and AAV2
Time frame: Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit
T-cell mediated immune response to AAV8
T-cell mediated immune response to AAV8
Time frame: Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit
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