search
Back to results

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

Primary Purpose

Hemophilia A

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Biostate® [SP]
Biostate® [SP]
Biostate® [RP]
Sponsored by
CSL Behring
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring Hemophilia A

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Other

Other

Experimental

Arm Label

PK Biostate® [SP]

PK Biostate® [RP]

Efficacy

Arm 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.

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

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

Outcomes

Primary Outcome Measures

Haemostatic efficacy
Number of treatments/units required to resolve any bleeding event
FVIII concentrate usage (number of infusions, IU/kg per event, per month, and per year)
Assessment of blood loss during any surgical procedure
Pharmacokinetics of FVIII activity

Secondary Outcome Measures

The nature, frequency and incidence of adverse events
Development of FVIII inhibitors

Full Information

First Posted
April 9, 2009
Last Updated
February 10, 2011
Sponsor
CSL Behring
Collaborators
Parexel
search

1. Study Identification

Unique Protocol Identification Number
NCT00879541
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.
Study Type
Interventional

2. Study Status

Record Verification Date
February 2011
Overall Recruitment Status
Completed
Study Start Date
February 2009 (undefined)
Primary Completion Date
October 2010 (Actual)
Study Completion Date
October 2010 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
CSL Behring
Collaborators
Parexel

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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].

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
Keywords
Hemophilia A

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
81 (Actual)

8. Arms, Groups, and Interventions

Arm Title
PK Biostate® [SP]
Arm Type
Other
Arm 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.
Arm Title
PK Biostate® [RP]
Arm Type
Other
Arm Description
Part 1: PK subjects are randomized to receive Biostate® [RP] either on Day 1 or Day 8.
Arm Title
Efficacy
Arm Type
Experimental
Arm Description
Part 2: This arm includes all subjects during the efficacy component of the study.
Intervention Type
Biological
Intervention Name(s)
Biostate® [SP]
Other Intervention Name(s)
Human Coagulation Factor VIII / von Willebrand Factor
Intervention Description
Single bolus intravenous dose of 50 IU/kg
Intervention Type
Biological
Intervention Name(s)
Biostate® [SP]
Other Intervention Name(s)
Human Coagulation Factor VIII / von Willebrand Factor
Intervention Description
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
Intervention Name(s)
Biostate® [RP]
Other Intervention Name(s)
Biostate®, Human Coagulation Factor VIII / von Willebrand Factor
Intervention Description
Single bolus intravenous dose of 50 IU/kg.
Primary Outcome Measure Information:
Title
Haemostatic efficacy
Time Frame
Monthly, until final study visit
Title
Number of treatments/units required to resolve any bleeding event
Time Frame
From Day 1 until final study visit
Title
FVIII concentrate usage (number of infusions, IU/kg per event, per month, and per year)
Time Frame
From Day 1 until final study visit
Title
Assessment of blood loss during any surgical procedure
Time Frame
From Day 1 until final study visit
Title
Pharmacokinetics of FVIII activity
Time Frame
Up to 48 hours following infusions (Part 1 and Part 3 only)
Secondary Outcome Measure Information:
Title
The nature, frequency and incidence of adverse events
Time Frame
From Day 1 until final study visit
Title
Development of FVIII inhibitors
Time Frame
From Day 1 until final study visit

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
Study Site
City
Plovdiv
Country
Bulgaria
Facility Name
Study Site
City
Sofia
Country
Bulgaria
Facility Name
Study Site
City
Varna
Country
Bulgaria
Facility Name
Study Site
City
Skopje
Country
Macedonia, The Former Yugoslav Republic of
Facility Name
Study Site
City
Bialystok
Country
Poland
Facility Name
Study Site
City
Gdansk
Country
Poland
Facility Name
Study Site
City
Krakow
Country
Poland
Facility Name
Study Site
City
Lublin
Country
Poland
Facility Name
Study Site
City
Poznan
Country
Poland
Facility Name
Study Site
City
Warszawa
Country
Poland
Facility Name
Study Site
City
Wroclaw
Country
Poland
Facility Name
Study Site
City
Barnaul
Country
Russian Federation
Facility Name
Study Site
City
Kirov
Country
Russian Federation
Facility Name
Study Site
City
Moscow
Country
Russian Federation

12. IPD Sharing Statement

Links:
URL
http://www.cslbehring.com/clinical-trials/contact-us.htm?registryRefNum=NCT00879541&registryName=ctgov
Description
Click here to request more information about this study

Learn more about this trial

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

We'll reach out to this number within 24 hrs