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

Hemophilia A Clinical Trial

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00879541
• Other study ID #: CSLCT-BIO-07-47
• Secondary ID: 1472
• Status: Completed
• Phase: Phase 2
• First received: April 9, 2009
• Last updated: February 10, 2011
• Start date: February 2009
• Est. completion date: October 2010

Study information

• Verified date: February 2011
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Health authority: Russia: FSI Scientific Center of Expertise of Medical ApplicationBulgaria: Bulgarian Drug AgencyPoland: Office for Registration of Medicinal Products, Medical Devices and Biocidal ProductsMacedonia: Ministry of Health
• Study type: Interventional

Clinical Trial 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].

Recruitment information / eligibility

• Status: Completed
• Enrollment: 81
• Est. completion date: October 2010
• Est. primary completion date: October 2010
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years and older

Eligibility

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 Design

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

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• Biostate® [SP]
Single bolus intravenous dose of 50 IU/kg
• Biostate® [SP]
The dose is dependent on the reason for use and may consist of repeated bolus doses as required to manage haemophilia condition.
• Biostate® [RP]
Single bolus intravenous dose of 50 IU/kg.

Locations

Country	Name	City	State
Bulgaria	Study Site	Plovdiv
Bulgaria	Study Site	Sofia
Bulgaria	Study Site	Varna
Macedonia, The Former Yugoslav R	Study Site	Skopje
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
Russian Federation	Study Site	Barnaul
Russian Federation	Study Site	Kirov
Russian Federation	Study Site	Moscow

Sponsors (2)

Lead Sponsor	Collaborator
CSL Behring	Parexel

Countries where clinical trial is conducted

Bulgaria,  Macedonia, The Former Yugoslav Republic of,  Poland,  Russian Federation, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Haemostatic efficacy		Monthly, until final study visit	No
Primary	Number of treatments/units required to resolve any bleeding event		From Day 1 until final study visit	No
Primary	FVIII concentrate usage (number of infusions, IU/kg per event, per month, and per year)		From Day 1 until final study visit	No
Primary	Assessment of blood loss during any surgical procedure		From Day 1 until final study visit	No
Primary	Pharmacokinetics of FVIII activity		Up to 48 hours following infusions (Part 1 and Part 3 only)	No
Secondary	The nature, frequency and incidence of adverse events		From Day 1 until final study visit	Yes
Secondary	Development of FVIII inhibitors		From Day 1 until final study visit	Yes

External Links

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