Severe hemophilia A (<1% FVIII:C) MedDRA version: 20.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850
Conditions
Interventions
Trade Name: Wilate
Product Name: Wilate
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Wilate
Current Sponsor code: Wilate
Other descriptive name: HUMAN COAGUL
Sponsors
Octapharma AG
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Severe hemophilia A (200/µL) 5. Good documentation of the historical bleeding rate (at least for the 6 months pre-ceding study start) 6. Voluntarily given, fully informed written and signed consent obtained by the pa-tient (or parent/legal guardian in case of adolescents) before any study-related pro-cedures are conducted Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 40 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: 1. Any coagulation disorders other than hemophilia A 2. History of FVIII inhibitor activity (=0.6 BU) or detectable FVIII inhibitory anti-bodies (=0.6 BU using the Nijmegen modification of the Bethesda assay) at screening, as determined by the central laboratory 3. Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine >120 µmol/L) 4. Patients receiving or scheduled to receive immunomodulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs 5. Treatment with any investigational medicinal product in another interventional clinical study currently or within 4 weeks before enrollment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to determine the efficacy of Wilate in the prophylactic treatment of previously treated patients (PTP) with severe hemophilia A. ;Secondary Objective: The secondary objectives of this study are to: • Determine the efficacy of Wilate in the treatment of breakthrough bleeding episodes (BEs) • Determine the efficacy of Wilate in surgical prophylaxis • Calculate the FVIII:C pharmacokinetics (PK) for Wilate at baseline • Calculate the FVIII:C incremental IVR of Wilate over time (at baseline, and at 3 and 6 months of treatment) • Assess the association between AB0 blood type and the FVIII:C half-life of Wilate • Assess the association between the VWF:Ag concentration and the FVIII:C half-life of Wilate • Assess the safety and tolerability of Wilate • Assess the immunogenicity of Wilate ;Primary end point(s): The primary endpoint of this study is a 50% reduction of the total annualized bleeding rate (TABR) observed in the GENA-01 study, with a total of 58.1 BEs per patient per year.;Timepoint(s) of evaluation of this end point: After the end of the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoints of this study are the: 1. Spontaneous annualized bleeding rate (SABR) 2. Efficacy of Wilate in the treatment of breakthrough BEs based on the proportion of BEs successfully treated with Wilate 3. Descriptive efficacy of Wilate in surgical prophylaxis 4. Wilate consumption data (FVIII IU/kg per week per patient) for prophylaxis ;Timepoint(s) of evaluation of this end point: After the end of the study | — |
Countries
Bulgaria, Hungary, Poland, Russian Federation
Contacts
Public ContactClinical Trial Manager
Octapharma AG
Outcome results
None listed