Study of a Plasma-Derived Von Willebrand Factor/Factor VIII Concentrate (vWF/FVIII), Biostate®, in Subjects With Haemophilia A
Tracking InformationStart Date ICMJE | February 2009 |
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Estimated Primary Completion Date | July 2010 (final data collection date for primary outcome measure) |
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Current Primary Outcome Measures ICMJE (submitted: April 9, 2009) | - Haemostatic efficacy [ Time Frame: Monthly, until final study visit ] [ Designated as safety issue: No ]
- Number of treatments/units required to resolve any bleeding event [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
- FVIII concentrate usage (number of infusions, IU/kg per event, per month, and per year) [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
- Assessment of blood loss during any surgical procedure [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
- Pharmacokinetics of FVIII activity [ Time Frame: Up to 48 hours following infusions (Part 1 and Part 3 only) ] [ Designated as safety issue: No ]
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Original Primary Outcome Measures ICMJE | Same as current |
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Change History | Complete list of historical versions of study NCT00879541 on ClinicalTrials.gov Archive Site |
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Current Secondary Outcome Measures ICMJE (submitted: April 9, 2009) | - The nature, frequency and incidence of adverse events [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: Yes ]
- Development of FVIII inhibitors [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: Yes ]
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Original Secondary Outcome Measures ICMJE | Same as current |
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Descriptive InformationBrief Title ICMJE | Study of a Plasma-Derived Von Willebrand Factor/Factor VIII Concentrate (vWF/FVIII), Biostate®, in Subjects With Haemophilia A |
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Official Title ICMJE | A Phase II, Multicentre, Double-Blinded, Randomised, Cross-Over Study to Evaluate Efficacy, Safety and Pharmacokinetics of Biostate® in Subjects With Haemophilia A. |
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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]. |
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Detailed Description | |
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Study Phase | Phase II |
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Study Type ICMJE | Interventional |
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Study Design ICMJE | Treatment, Randomized, Double Blind (Subject, Investigator), Crossover Assignment, Safety/Efficacy Study |
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Condition ICMJE | Hemophilia A |
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Intervention ICMJE | - Biological: Biostate® [SP]
Single bolus intravenous dose of 50 IU/kg Other Name: Human Coagulation Factor VIII / von Willebrand Factor - Biological: Biostate® [SP]
The dose is dependent on the reason for use and may consist of repeated bolus doses as required to manage haemophilia condition. Other Name: Human Coagulation Factor VIII / von Willebrand Factor - Biological: Biostate® [RP]
Single bolus intravenous dose of 50 IU/kg. Other Names: - Biostate®
- Human Coagulation Factor VIII / von Willebrand Factor
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Study Arms / Comparison Groups | - PK Biostate® [SP]
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. Intervention: Biological: Biostate® [SP] - PK Biostate® [RP]
Part 1: PK subjects are randomized to receive Biostate® [RP] either on Day 1 or Day 8. Intervention: Biological: Biostate® [RP] - Efficacy: Experimental
Part 2: This arm includes all subjects during the efficacy component of the study. Intervention: Biological: Biostate® [SP]
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Recruitment InformationEstimated Enrollment ICMJE | 62 |
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Estimated Completion Date | July 2010 |
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Estimated Primary Completion Date | July 2010 (final data collection date for primary outcome measure) |
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Eligibility Criteria ICMJE | 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
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Gender | Male |
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Ages | 12 Years and older |
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Accepts Healthy Volunteers | No |
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Contacts ICMJE | |
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Location Countries ICMJE | Bulgaria, Poland, Russian Federation |
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Administrative InformationNCT ID ICMJE | NCT00879541 |
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Responsible Party | Global Head Clinical Research & Development, CSL Behring |
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Study ID Numbers ICMJE | 1476, CSLCT-BIO-07-47 |
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Study Sponsor ICMJE | CSL Behring |
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Collaborators ICMJE | Parexel |
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Investigators ICMJE | |
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Information Provided By | CSL Behring |
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