Study of a pd vWF/FVIII, Biostate®, in Subjects With Haemophilia A

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

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

, ,

Location Countries

Bulgaria

Location Countries

Bulgaria

Administrative Informations

NCT ID

NCT00879541

Organization ID

CSLCT-BIO-07-47

Secondary IDs

Study Sponsor

CSL Behring

Collaborators

 Parexel

Study Sponsor

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Verification Date

February 2011