Haemophilia A - a hereditary bleeding disorder characterised by a deficiency in the plasma protein known as coagulation Factor VIII (FVIII). MedDRA version: 9.1 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII)
Conditions
Interventions
Product Name: Biostate [SP]
Product Code: Biostate [SP]
Pharmaceutical Form: Powder and solvent for solution for infusion
Other descriptive name: HUMAN COAGULATION FACTOR VIII
Concentration unit: IU i
Sponsors
CSL Behring GmbH
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Subjects must meet all of the following inclusion criteria to be eligible to participate in this study: 1. Male subjects who are at least 12 years. 2. Subjects who have been diagnosed with Haemophilia A and have = 1% FVIII levels in the absence of factor replacement according to their medical history. 3. Subjects with evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunisation) within 10 years prior to Day 1 documented in the medical notes. 4. Subjects with at least 150 prior exposure days to a FVIII replacement product confirmed by their treating physician. 5. Subjects and/or their legally acceptable representatives who understand the nature of the study, and have given written informed consent to participate in the study. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Subjects who fulfil the following criteria will not ´be eligible for inclusion into this trial: For participation in the PK part: 1. Subjects who are actively bleeding. 2. Subjects with body weight > 100 kg For all subjects at Day 1: 3. Subjects who have received an infusion of any FVIII product, cryoprecipitate, whole blood, plasma, or DDAVP in the 4 days prior to Day 1. 4. Subjects with a known history of FVIII inhibitors, or have a FVIII inhibitor level of > 0.6 Bethesda Units (BU) at screening. 5. Subjects who have received aspirin or other Non-Steroidal Anti-Inflammatory Drugs (NSAIDs) within 7 days of administration of study product. 6. Subjects with CD4 lymphocytes 1.5 x upper limit of normal (ULN) and/or AST/ALT >2.5 x ULN (referring to limits of the laboratory that performs the determination) at screening. 8. Subjects suffering an acute or chronic medical condition, other than haemophilia A, which may, in the opinion of the Investigator, affect the conduct of the study. 9. Subjects suffering von Willebrand Disease with vWF:RCo level < 50 IU/dL at screening 10. Subjects having evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit. 11. Subjects with a known or suspected hypersensitivity or previous evidence of severe side effects to Biostate®, FVIII concentrates or human albumin. 12. Subjects who have participated in a clinical study or used an investigational compound (e.g. a new chemical entity not approved for clinical use) in the three months preceding the first day of study drug administration, or who are planning to enter such a study during the study period. 13. Subjects who are not willing and/or not able to comply with the study requirements.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 1. To assess the efficacy of Biostate® [SP] in subjects with Haemophilia A 2. To assess the comparability of the pharmacokinetics of Biostate® [RP] and Biostate® [SP] in subjects with Haemophilia A ;Secondary Objective: To assess the safety of Biostate® [SP] in subjects with Haemophilia A;Primary end point(s): Primary efficacy endpoint assessed during Part Two of the study. Thís awill include the following: - Assessment of haemostatic efficacy by the Investigator using the grading scale of Excellent, Good, Moderate, None and will be evaluated on a retrospective monthly basis, - Any blood product transfusion requirements, and the number of treatments/units required to resolve the event - FVIII levels concentrate usage: number of infusions, IU/kg per event, month and per year. - Assessment of blood loss during any surgical event FVIII levels will be assessed to determine the minimum pharmacokinetic parameters as follows: 1. Incremental recovery 2. Half life (T1/2) 3. Area under the concentration-time curve (AUC) 4. Mean residence time (MRT) 5. Volume of distribution at steady state (Vss) 6. Maximum plasma concentration (Cmax) 7. Time the maximum concentration occurs (Tmax) 8. Minimum plasma concentration (Cmin) 9. Total clearance of the drug from the body (CL = dose/AUC) | — |
Countries
Bulgaria
Outcome results
None listed