- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00879541
Study of a pd vWF/FVIII, Biostate®, in Subjects With Haemophilia A
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
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Plovdiv, Bulgaria
- Study Site
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Sofia, Bulgaria
- Study Site
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Varna, Bulgaria
- Study Site
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Skopje, Macedonia, The Former Yugoslav Republic of
- Study Site
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Bialystok, Poland
- Study Site
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Gdansk, Poland
- Study Site
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Krakow, Poland
- Study Site
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Lublin, Poland
- Study Site
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Poznan, Poland
- Study Site
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Warszawa, Poland
- Study Site
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Wroclaw, Poland
- Study Site
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Barnaul, Russian Federation
- Study Site
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Kirov, Russian Federation
- Study Site
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Moscow, Russian Federation
- Study Site
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
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
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 |
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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:
The dose is dependent on the reason for use and may consist of repeated bolus doses as required to manage haemophilia condition.
Other Names:
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Other: PK Biostate® [RP]
Part 1: PK subjects are randomized to receive Biostate® [RP] either on Day 1 or Day 8.
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Single bolus intravenous dose of 50 IU/kg.
Other Names:
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Experimental: Efficacy
Part 2: This arm includes all subjects during the efficacy component of the study.
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Single bolus intravenous dose of 50 IU/kg
Other Names:
The dose is dependent on the reason for use and may consist of repeated bolus doses as required to manage haemophilia condition.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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Haemostatic efficacy
Time Frame: Monthly, until final study visit
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Monthly, until final study visit
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Number of treatments/units required to resolve any bleeding event
Time Frame: From Day 1 until final study visit
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From Day 1 until final study visit
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FVIII concentrate usage (number of infusions, IU/kg per event, per month, and per year)
Time Frame: From Day 1 until final study visit
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From Day 1 until final study visit
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Assessment of blood loss during any surgical procedure
Time Frame: From Day 1 until final study visit
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From Day 1 until final study visit
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Pharmacokinetics of FVIII activity
Time Frame: Up to 48 hours following infusions (Part 1 and Part 3 only)
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Up to 48 hours following infusions (Part 1 and Part 3 only)
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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The nature, frequency and incidence of adverse events
Time Frame: From Day 1 until final study visit
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From Day 1 until final study visit
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Development of FVIII inhibitors
Time Frame: From Day 1 until final study visit
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From Day 1 until final study visit
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Collaborators and Investigators
Sponsor
Collaborators
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- CSLCT-BIO-07-47
- 1472 (Other Identifier: CSL Behring)
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
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Christoph KönigsRoche Pharma AG; Chugai Pharma Germany GmbHRecruitingSevere Hemophilia A | Severe Hemophilia A With Inhibitor | Severe Hemophilia A Without InhibitorGermany
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GWT-TUD GmbHHannover Medical School; Hoffmann-La RocheCompleted
-
Kathelijn FischerRadboud University Medical Center; University Medical Center Groningen; Maastricht... and other collaboratorsRecruitingAdolescent | Child | Hemophilia A With Inhibitor | Adult | Hemophilia A Without Inhibitor | Hemophilia A, SevereNetherlands
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Hoffmann-La RocheActive, not recruitingSevere Hemophilia A | Moderate Hemophilia ABrazil, Germany, Italy, Spain, United States, Turkey, United Kingdom, Tunisia, Canada, Hungary, Ireland, Morocco, Serbia
-
Catalyst BiosciencesCompletedHemophilia A | Hemophilia B | Hemophilia A With Inhibitor | Hemophilia B With Inhibitor | Hemophilia A Without Inhibitor | Hemophilia B Without InhibitorBulgaria, Russian Federation
-
JW PharmaceuticalRecruitingHemophilia A With Inhibitor | Hemophilia A Without InhibitorKorea, Republic of
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PfizerCompletedFactor VIII Deficiency, Congenital | Hemophilia A, Congenital | Factor 8 Deficiency, Congenital | Autosomal Hemophilia A | Classic Hemophilia
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BioMarin PharmaceuticalRecruitingHemophilia A With Inhibitor | Hemophilia A With Anti Factor VIIIUnited States, United Kingdom, Taiwan, Korea, Republic of, Brazil, Italy
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American Thrombosis and Hemostasis NetworkTakeda; CSL Behring; OctapharmaCompletedHemophilia A | Hemophilia B | Hemophilia | Hemophilia A With Inhibitor | Haemophilia | Hemophilia B With Inhibitor | Haemophilia A Without Inhibitor | Haemophilia B Without InhibitorUnited States
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BayerCompletedHemophilia A; Hemophilia BIsrael
Clinical Trials on Biostate® [SP]
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CSL BehringCompletedVon Willebrand DiseaseFrance
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CSL BehringCompletedVon Willebrand DiseaseBulgaria, Germany, Poland, Russian Federation, Ukraine
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CSL BehringParexelCompletedVon Willebrand DiseaseUkraine, Lebanon, Germany, Belarus, Georgia, Guatemala
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Hacettepe UniversityCompletedIron Deficiency AnemiaTurkey
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CSL BehringTerminatedHemophilia ARussian Federation, Germany, Italy, Austria, Greece
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Federal University of Health Science of Porto AlegreCompletedCardiovascular DiseasesBrazil
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CSL BehringParexelCompletedHemophilia AUkraine, Lebanon, Belarus, Georgia, Guatemala, Mexico
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Case Comprehensive Cancer CenterSuspendedMalignant Neoplasm of ProstateUnited States
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CSL BehringParexelCompletedVon Willebrand DiseaseBulgaria, Poland, Russian Federation, Ukraine
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Fotona d.o.o.UnknownStress Urinary IncontinenceCanada