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

February 10, 2011 updated by: CSL Behring

A Phase II, Multicentre, Double-blinded, Randomised, Cross-over Study to Evaluate Efficacy, Safety and Pharmacokinetics of Biostate® in Subjects With Haemophilia A.

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 Overview

Study Type

Interventional

Enrollment (Actual)

81

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Plovdiv, Bulgaria
        • Study Site
      • Sofia, Bulgaria
        • Study Site
      • Varna, Bulgaria
        • Study Site
      • Skopje, Macedonia, The Former Yugoslav Republic of
        • Study Site
      • Bialystok, Poland
        • Study Site
      • Gdansk, Poland
        • Study Site
      • Krakow, Poland
        • Study Site
      • Lublin, Poland
        • Study Site
      • Poznan, Poland
        • Study Site
      • Warszawa, Poland
        • Study Site
      • Wroclaw, Poland
        • Study Site
      • Barnaul, Russian Federation
        • Study Site
      • Kirov, Russian Federation
        • Study Site
      • Moscow, Russian Federation
        • Study Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: 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.

Single bolus intravenous dose of 50 IU/kg
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor
The dose is dependent on the reason for use and may consist of repeated bolus doses as required to manage haemophilia condition.
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor
Other: PK Biostate® [RP]
Part 1: PK subjects are randomized to receive Biostate® [RP] either on Day 1 or Day 8.
Single bolus intravenous dose of 50 IU/kg.
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor
  • Biostate®
Experimental: Efficacy
Part 2: This arm includes all subjects during the efficacy component of the study.
Single bolus intravenous dose of 50 IU/kg
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor
The dose is dependent on the reason for use and may consist of repeated bolus doses as required to manage haemophilia condition.
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Haemostatic efficacy
Time Frame: Monthly, until final study visit
Monthly, until final study visit
Number of treatments/units required to resolve any bleeding event
Time Frame: From Day 1 until final study visit
From Day 1 until final study visit
FVIII concentrate usage (number of infusions, IU/kg per event, per month, and per year)
Time Frame: From Day 1 until final study visit
From Day 1 until final study visit
Assessment of blood loss during any surgical procedure
Time Frame: From Day 1 until final study visit
From Day 1 until final study visit
Pharmacokinetics of FVIII activity
Time Frame: Up to 48 hours following infusions (Part 1 and Part 3 only)
Up to 48 hours following infusions (Part 1 and Part 3 only)

Secondary Outcome Measures

Outcome Measure
Time Frame
The nature, frequency and incidence of adverse events
Time Frame: From Day 1 until final study visit
From Day 1 until final study visit
Development of FVIII inhibitors
Time Frame: From Day 1 until final study visit
From Day 1 until final study visit

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Collaborators

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2009

Primary Completion (Actual)

October 1, 2010

Study Completion (Actual)

October 1, 2010

Study Registration Dates

First Submitted

April 9, 2009

First Submitted That Met QC Criteria

April 9, 2009

First Posted (Estimate)

April 10, 2009

Study Record Updates

Last Update Posted (Estimate)

February 11, 2011

Last Update Submitted That Met QC Criteria

February 10, 2011

Last Verified

February 1, 2011

More Information

Terms related to this study

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Hemophilia A

Clinical Trials on Biostate® [SP]

3
Subscribe