Severe Haemophilia A MedDRA version: 17.0 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII) System Organ Class: 100000004850
Conditions
Interventions
Product Name: Human cell line recombinant factor VIII
Product Code: human-cl rhFVIII
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: N.A.
CAS Number: N.A.
Curre
Sponsors
Octapharma AG
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Evaluable completion of study GENA-03 by having a study participation period of 6 months, provided that prophylaxis with Human-cl rhFVIII is continued without intermediate interruption. 2. Voluntarily given, fully informed written and signed consent obtained from the parents (or legal guardians) before any study-related procedures are conducted. The need for obtaining assent will depend on the subjects’ developmental stage and intellectual capacity. 3. Capability to understand and comply with the relevant aspects of the study. Are the trial subjects under 18? yes Number of subjects for this age range: 50 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. Development of FVIII inhibitors (=0.6 Bethesda units [BU]) in the course of study GENA-03. 2. Any severe liver or kidney disease (alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels >5 times of upper limit of normal, creatinine >120 µmol/L).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the long-term immunogenicity and tolerability of Human-cl rhFVIII in previously treated children with severe Haemophilia A.;Secondary Objective: To determine the long-term efficacy of Human-cl rhFVIII in the prophylaxis and in the treatment of (breakthrough) BEs, and in surgical prophylaxis in previously treated children with severe Haemophilia A.;Primary end point(s): Long-term immunogenicity and tolerability are the primary endpoints of the study.;Timepoint(s) of evaluation of this end point: Inhibitor activity will be determined at tri-monthly intervals until study completion. Simultaneously, anti-rhFVIII antibodies will be measured. The same parameters will also be determined in case an inhibitor development is suspected. The sampling for inhibitor and antibody measurements should be performed not less than 48 hours after the previous administration of any FVIII product. In the case of positive inhibitor results, an inhibitor retesting using a second separately drawn sample should be performed. Long-term clinical tolerability will be assessed by monitoring Adverse Events (AEs) throughout the entire study duration. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Long-Term Efficacy of Prophylactical Treatment 2. Long-Term Efficacy of Treatment of Bleeding Episodes 3. Efficacy in Surgical Prophylaxis;Timepoint(s) of evaluation of this end point: 1. Efficacy is to be assessed by evaluating the frequency of IMP injections, and to the prophylactic IMP doses needed. The number of spontaneous breakthrough BEs will be evaluated. Overall study drug consumption data (FVIII [IU/kg]), per month and per year), per subject and in total, will be analysed. 2. Efficacy is to be assessed by the subject/subject’s parents (or legal guardians) at the end of a BE (in collaboration with the Investigator in case of on-site treatment). 3. Efficacy will be assessed by the surgeon at the end of surgery (i.e. after last suture), as well as postoperatively (i.e. at date of discharge or on postoperative Day 6, whichever occurs later) by both the surgeon and the haematologist. | — |
Countries
Czech Republic, France, Poland, Romania, Russian Federation, Turkey, United Kingdom
Contacts
Public ContactClinical Research & Development Hae
Octapharma Pharmazeutika Prod.Ges.m.b.H.
Outcome results
None listed