Bleeding disorder Hemophilia A
Conditions
Interventions
None listed
Sponsors
University of Texas Health Science Center
Eligibility
Age
2 Years to 11 Years
Inclusion criteria
Inclusion criteria: 1. An informed consent, approved by the appropriate Institutional Review Board (IRB)/Independent Ethics Committee (IEC), has been administered, signed, and dated. 2. Subject has severe hemophilia A defined by a baseline FVIII:C =0.01 IU/ml the child must exit the study. 3. Subject weighs 3.5 kg or more at the time of his baseline study evaluation
Exclusion criteria
Exclusion criteria: 1. Subject has had prior exposure to clotting factor concentrates or blood products, including packed red blood cells (RBC), platelets, plasma, or cryoprecipitate. 2. Subject has a clinically significant chronic disease other than hemophilia A. 3. Subject is currently participating in another investigational drug study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary Endpoints: An inhibitor is defined by a Nijmegen test > 0.6 Bethesda units (BU) on two consecutive tests conducted in the central laboratory. During the first 50 days of exposure to a single FVIII product: - Analyze and quantify subclasses of anti-FVIII antibodies - Characterize FVIII-specific T-cells and changes which occur - Quantify total FOXP3-positive regulatory T-cells (Treg) - Assess RNA expression, transcript profile, and exon usage in relevant pathways - Identify F8 gene mutation and other known genomic predictors of inhibitor development - Record infection(s), immunization(s), bleeding episodes, and factor usage | — |
Secondary
| Measure | Time frame |
|---|---|
| N.a. | — |
Countries
Netherlands
Outcome results
None listed