Brief Title
Study of a pd vWF/FVIII, Biostate®, in Subjects With Haemophilia A
Official Title
A Phase II, Multicentre, Double-blinded, Randomised, Cross-over Study to Evaluate Efficacy, Safety and Pharmacokinetics of Biostate® in Subjects With Haemophilia A.
Brief Summary
The aim of this study are to - assess the efficacy of Biostate® [Study Product (SP)] in subjects with Haemophilia A - compare the pharmacokinetics of Biostate® [SP] with the previously marketed product Biostate® (here referred to as Biostate® [Reference Product (RP)]). This study is divided into 3 parts: Part 1: Cross-over pharmacokinetic (PK) component. PK subjects will be randomised to determine the order in which they receive the two study products. This part of the study is double-blinded. Part 2: Efficacy component. All subjects will receive Biostate® [SP] as required to manage their haemophilia condition for an estimated period of 6 months (or minimum of 50 exposure days) to assess efficacy and safety of the product. This part of the study is open-label. Part 3: Repeat pharmacokinetic assessment. Subjects who participated in Part 1 (PK component) will undergo a repeat PK assessment on Day 180 following administration of Biostate® [SP].
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Haemostatic efficacy
Secondary Outcome
The nature, frequency and incidence of adverse events
Condition
Hemophilia A
Intervention
Biostate® [SP]
Study Arms / Comparison Groups
PK Biostate® [SP]
Description: Part 1: PK subjects are randomized to receive Biostate® [SP] either on Day 1 or Day 8. Part 3: All PK subjects receive Biostate® [SP] on Day 180.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
81
Start Date
February 2009
Completion Date
October 2010
Primary Completion Date
October 2010
Eligibility Criteria
Inclusion Criteria: - Diagnosed with Haemophilia A with ≤ 1% Factor VIII (FVIII) levels in the absence of factor replacement - 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 - At least 150 days of prior exposure to a FVIII replacement product - Written informed consent given Exclusion Criteria (for participation in the pharmacokinetic (PK) component): - Active bleeding - Body weight > 100 kg Exclusion Criteria (for all subjects): - Receipt of an infusion of any FVIII product, cryoprecipitate, whole blood, plasma, or desmopressin acetate (DDAVP) in the 4 days prior to Day 1 - Known history of FVIII inhibitors, or FVIII inhibitor level > 0.6 Bethesda Units (BU) at screening - Receipt of aspirin or other Non-Steroidal Anti-Inflammatory Drugs (NSAIDs) within 7 days of administration of study product. - CD4 lymphocytes < 200/µL. Subjects wo are HIV-1 positive may be considered for the study if viral load ≤ 200 particles/µL at screening and all other eligibility criteria are met. - Impaired liver function ie. bilirubin >1.5 x upper limit of normal (ULN) and/or AST/ALT > 2.5 x ULN at screening. - Acute or chronic medical condition, other than haemophilia A, which may, in the opinion of the Investigator, affect the conduct of the study - von Willebrand Disease (VWD) with Von Willebrand Factor:Ristocetin Cofactor (vWF:RCo) level < 50 IU/dL at screening - Evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit - Known or suspected hypersensitivity or previous evidence of severe side effects to Biostate®, FVIII concentrates or human albumin - Participation in a clinical study or use of an investigational compound (e.g. a new chemical entity not approved for clinical use) in the 3 months preceding the first day of study drug administration, or plans to enter such a study during the study period - Not willing and/or not able to comply with study requirements
Gender
Male
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
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Location Countries
Bulgaria
Location Countries
Bulgaria
Administrative Informations
NCT ID
NCT00879541
Organization ID
CSLCT-BIO-07-47
Secondary IDs
1472
Study Sponsor
CSL Behring
Collaborators
Parexel
Study Sponsor
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Verification Date
February 2011