Severe Hemophilia-A (< 1% FVIII:C) MedDRA version: 13.1 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Bayer HealthCare AG
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Part A 1. Male, age = 12 years. Enrollment will begin with subjects >6-12 years before it is opened to all age groups. 2. Severe hemophilia A defined as 50 ED with any FVIII concentrate, currently treated with Kogenate FS/Bayer 4. No current evidence of inhibitor antibody measured using the Nijmegen modified Bethesda assay [=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Parts A and B 1. Any individual with another bleeding disorder that is different from Hemophilia A (eg, von Willebrand disease, Hemophilia B) 2. Any individual with thrombocytopenia (platelet count 2x upper limit of normal or AST/ALT > 5x upper limit of normal 4. Any individual without a documented negative inhibitor test based on medical records or screening laboratory test (except for PUPs) 5. Any individual who is receiving chemotherapy, immune modulatory drugs (IVIG, cyclosporine, chronic use of oral or i.v. corticosteroids), has participated in another FVIII study within the last month, or received another experimental drug within the last 3 months. 6. Any individual who requires any pre-medication to tolerate FVIII treatment (eg, antihistamines). 7. Any individual who is unwilling to comply with study visits or other protocol requirements, for example (eg. prophylaxis treatment) or is not suitable for participation in this study for any reason, according to the Investigator’s judgment. 8. Known hypersensitivity to active substance, mouse or hamster protein. 9. Previous participation in this study Part B only (PUPs): 10. First treatment with BAY 81-8973 for high risk bleeding situations (e.g. surgery, intracranial bleed), or requiring intensive or prolonged treatment. 11. Unable to tolerate volume of blood draws required for study participation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Continuous throughout duration of study;Secondary Objective: The secondary objectives are - To assess the safety and efficacy of BAY 81-8973 during surgeries. - To assess incremental recovery of BAY 81-8973. - To assess pharmacokinetic parameters in a subset of children. ;Primary end point(s): The primary variable is the annualized number of total bleeds (sum of spontaneous bleeds and traumatic bleeds) during prophylaxis that occur within 48 h of the last prophylaxis infusion. Both joint and non-joint bleeding will be assessed. ;Main Objective: The primary objective is to evaluate the safety and efficacy of the treatment with BAY 81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Subject/parents’ assessment of the response of treatment of bleeding events which is assessed as excellent, good, moderately well or poorly. • Annualized number of total bleeds (sum of spontaneous and trauma bleeds) during prophylaxis treatment • Assessment of adequacy of haemostasis during surgical interventions • Number of infusions for the treatment of a bleed • Consumption of FVIII ;Timepoint(s) of evaluation of this end point: Continuous throughout duration of study | — |
Countries
Austria, Bulgaria, Canada, Denmark, Hungary, Ireland, Israel, Italy, Latvia, Lithuania, Norway, Poland, Romania, Serbia, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactCTP Team / Ref: "EU CTR" / S102 -
Bayer HealthCare AG
Outcome results
None listed