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Completed

NCT Number: NCT00879541

Study of a pd vWF/FVIII, 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].

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

12 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Study Site, Plovdiv, Bulgaria

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Biostate® [SP]

Biological

Single bolus intravenous dose of 50 IU/kg

Other names: Human Coagulation Factor VIII / von Willebrand Factor

Biostate® [RP]

Biological

Single bolus intravenous dose of 50 IU/kg.

Other names: Biostate®, Human Coagulation Factor VIII / von Willebrand Factor

Primary outcomes

  1. Haemostatic efficacy

    Time frame: Monthly, until final study visit

  2. Number of treatments/units required to resolve any bleeding event

    Time frame: From Day 1 until final study visit

  3. FVIII concentrate usage (number of infusions, IU/kg per event, per month, and per year)

    Time frame: From Day 1 until final study visit

  4. Assessment of blood loss during any surgical procedure

    Time frame: From Day 1 until final study visit

  5. Pharmacokinetics of FVIII activity

    Time frame: Up to 48 hours following infusions (Part 1 and Part 3 only)

Secondary outcomes

  1. The nature, frequency and incidence of adverse events

    Time frame: From Day 1 until final study visit

  2. Development of FVIII inhibitors

    Time frame: From Day 1 until final study visit

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Collaborators

  • Parexel

Registry information

Official study title

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

Important dates

Study start
2009
Primary completion
2010
Study completion
2010
First posted
Apr 10, 2009
Registry last updated
Feb 11, 2011

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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