Haemophilia A is an X-linked recessive clotting disorder in which the clotting factor, factor VIII (FVIII), is deficient or inactive. Patients with low levels of FVIII have an increased tendency to bleed and is characterised by recurrent haemorrhages due to trauma, surgery or spontaneous haemorrhaging when the condition is severe.
Conditions
Interventions
Trade Name: ReFacto
Product Name: ReFacto
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: moroctocog alfa
Other descriptive name: recombinant coagulation factor
Sponsors
Wyeth Pharmaceuticals
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Inhibitor positive hemophilia A patients: 1. Written informed consent or assent, as applicable. 2. Patients with moderate or severe congenital hemophilia A (FVIII:C the upper limit of normal as defined by the laboratory performing the assay), attributable by the Investigator to the patient’s current FVIII product, at the time of study visit and a positive inhibitor result confirmed by the central laboratory. 4. Patients with exposure to at least two different FVIII products during their lifetime. 5. Patients with > 50 cumulative ED to FVIII products. 6. Patients able to comply with a 72-hour FVIII washout period. *de novo inhibitor is defined as no known history of FVIII inhibitor titer. Comparison group (inhibitor-free patients): 1. Written informed consent / assent, as applicable. 2. Patients with moderate or severe congenital hemophilia A (FVIII:C 50 cumulative exposure days to FVIII products. 5. Documented negative FVIII BIA at the time of the study visit (=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Inhibitor positive hemophilia A patients: 1. Patients with a negative FVIII inhibitor result ( the upper limit of normal as defined by the laboratory performing the assay) prior to their current inhibitor. 3. Patients who have not had at least 1 FVIII BIA performed during the last 5 years prior to their visit for this study. 4. Patients who have received FVIII immune tolerance therapy at any time. 5. Patients, who as of the study visit, have taken only one FVIII product during their lifetime. 6. For patients whose current inhibitor is attributable to treatment with ReFacto; treatment with ReFacto prior to their previous product. In other words patients who were previously treated with ReFacto, then switched to another product, then switched back to ReFacto. 7. Patients with immune disorders (e.g., HIV, myeloma, lymphoma). Comparison group (inhibitor-free patients): 1. Patients with a positive FVIII inhibitor result (> the upper limit of normal as defined by the laboratory performing the assay) at the time of the study visit. 2. Patients with a prior history of FVIII inhibitor (> the upper limit of normal as defined by the laboratory performing the assay). 3. Patients with immune disorders (e.g., HIV, myeloma, lymphoma).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To describe the patterns of antibodies and associated epitopes in a subset of patients with hemophilia A, who meet the protocol entry criteria, using the methodology described in the protocol.;Secondary Objective: N/A;Primary end point(s): The endpoint will be a descriptive presentation of epitope regions of FVIII in samples from the three patient groups, using the methodology described in the protocol. | — |
Countries
Belgium, Germany, Italy, Spain, United Kingdom
Outcome results
None listed