Study of a pd vWF/FVIII, Biostate®, in Subjects With Haemophilia A - Full Text View

Purpose

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].

Condition	Intervention	Phase
Hemophilia A	Biological: Biostate® [SP] Biological: Biostate® [RP]	Phase 2

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Investigator) Primary Purpose: Treatment
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.

Resource links provided by NLM:

Genetics Home Reference related topics: hemophilia

MedlinePlus related topics: Hemophilia

Drug Information available for: Kogenate Moroctocog Alfa Advate

U.S. FDA Resources

Further study details as provided by CSL Behring:

Primary Outcome Measures:

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 ]

Secondary Outcome Measures:

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 ]

Enrollment:	81
Study Start Date:	February 2009
Study Completion Date:	October 2010
Primary Completion Date:	October 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
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.	Biological: Biostate® [SP] Single bolus intravenous dose of 50 IU/kg Other Name: Human Coagulation Factor VIII / von Willebrand Factor
PK Biostate® [RP] Part 1: PK subjects are randomized to receive Biostate® [RP] either on Day 1 or Day 8.	Biological: Biostate® [RP] Single bolus intravenous dose of 50 IU/kg. Other Names: Biostate® Human Coagulation Factor VIII / von Willebrand Factor
Experimental: Efficacy Part 2: This arm includes all subjects during the efficacy component of the study.	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

Eligibility

Ages Eligible for Study:	12 Years and older
Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00879541

Locations

Bulgaria
Study Site
Plovdiv, Bulgaria
Study Site
Sofia, Bulgaria
Study Site
Varna, Bulgaria
Macedonia, The Former Yugoslav Republic of
Study Site
Skopje, Macedonia, The Former Yugoslav Republic of
Poland
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
Russian Federation
Study Site
Barnaul, Russian Federation
Study Site
Kirov, Russian Federation
Study Site
Moscow, Russian Federation

Sponsors and Collaborators

CSL Behring

Parexel

More Information

Additional Information:

Click here to request more information about this study This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Global Head Clinical Research & Development, CSL Behring
ClinicalTrials.gov Identifier:	NCT00879541 History of Changes
Other Study ID Numbers:	CSLCT-BIO-07-47, 1472
Study First Received:	April 9, 2009
Last Updated:	February 10, 2011
Health Authority:	Russia: FSI Scientific Center of Expertise of Medical Application Bulgaria: Bulgarian Drug Agency Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products Macedonia: Ministry of Health

Keywords provided by CSL Behring:

Hemophilia A

Additional relevant MeSH terms:

Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders

Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 16, 2013