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

NCT ID: NCT00879541

Last Updated: 2011-02-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 81 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-02-28
• Study Completion Date: 2010-10-31

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].

Conditions

Hemophilia A

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study 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.

Group Type: OTHER

Biostate® [SP]

Intervention Type: BIOLOGICAL

Single bolus intravenous dose of 50 IU/kg

PK Biostate® [RP]

Part 1: PK subjects are randomized to receive Biostate® \[RP\] either on Day 1 or Day 8.

Group Type: OTHER

Biostate® [RP]

Intervention Type: BIOLOGICAL

Single bolus intravenous dose of 50 IU/kg.

Efficacy

Part 2: This arm includes all subjects during the efficacy component of the study.

Group Type: EXPERIMENTAL

Biostate® [SP]

Intervention Type: BIOLOGICAL

The dose is dependent on the reason for use and may consist of repeated bolus doses as required to manage haemophilia condition.

Interventions

Biostate® [SP]

Single bolus intravenous dose of 50 IU/kg

Intervention Type: 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.

Intervention Type: BIOLOGICAL

Biostate® [RP]

Single bolus intravenous dose of 50 IU/kg.

Intervention Type: BIOLOGICAL

Other Intervention Names

Human Coagulation Factor VIII / von Willebrand Factor; Human Coagulation Factor VIII / von Willebrand Factor; Biostate®; Human Coagulation Factor VIII / von Willebrand Factor

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

• Active bleeding
• Body weight > 100 kg

• 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

• Minimum Eligible Age: 12 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Parexel

INDUSTRY

Sponsor Role: collaborator

CSL Behring

INDUSTRY

Sponsor Role: lead

Responsible Party

CSL Behring

Locations

Study Site

Plovdiv, , Bulgaria

Study Site

Sofia, , Bulgaria

Study Site

Varna, , Bulgaria

Study Site

Skopje, , North Macedonia

Study Site

Bialystok, , Poland

Study Site

Gdansk, , Poland

Study Site

Krakow, , Poland

Study Site

Lublin, , Poland

Study Site

Poznan, , Poland

Study Site

Warsaw, , Poland

Study Site

Wroclaw, , Poland

Study Site

Barnaul, , Russia

Study Site

Kirov, , Russia

Study Site

Moscow, , Russia

Countries

Bulgaria; North Macedonia; Poland; Russia

Other Identifiers

1472

Identifier Type: OTHER

Identifier Source: secondary_id

CSLCT-BIO-07-47

Identifier Type: -

Identifier Source: org_study_id