Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease
This study has been completed.
Sponsor:
CSL Behring
Collaborator:
Parexel
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT00941616
First received: July 15, 2009
Last updated: April 4, 2013
Last verified: April 2013
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Purpose
The aim of this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD).
Pharmacokinetic Component:
PK parameters will be determined from a subgroup of subjects. Subjects who complete the PK component will subsequently continue in the efficacy component of the study, either continuing on a previously established prophylaxis regimen or continuing to receive on-demand treatment with the occurrence of non-surgical bleeding (NSB) events.
Efficacy Component:
Three treatment arms are defined for the efficacy component of the study. (1) Subjects who are currently being treated on a set prophylaxis regimen with a VWF product at the time of study entry will be enrolled in the "Prophylaxis" arm. (2) Subjects not being treated on a set prophylaxis regimen at the time of study entry who require a VWF product for the treatment of NSB events will be enrolled in the "On-demand" arm and commence using Biostate in the treatment of NSB events. (3) Subjects enrolled in the "On-demand" arm have the possibility to enter the "Cross-over to Prophylaxis" arm to receive an additional 12 months of prophylactic treatment.
| Condition | Intervention | Phase |
|---|---|---|
|
Von Willebrand Disease |
Biological: Biostate® |
Phase 2 Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | An Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy and Safety of Biostate® in Subjects With Von Willebrand Disease. |
Resource links provided by NLM:
Genetics Home Reference related topics:
von Willebrand disease
MedlinePlus related topics:
Hemophilia
U.S. FDA Resources
Further study details as provided by CSL Behring:
Primary Outcome Measures:
- Haemostatic efficacy at time of non-surgical bleeding (NSB) event [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
- Haemostatic efficacy overall [ Time Frame: Monthly (prophylactic therapy) or once every 3 months (for on-demand use) ] [ Designated as safety issue: No ]
- Number of treatments with blood product transfusions required to resolve any bleeding event [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
- vWF/FVIII concentrate usage (number of infusions, IU/kg per dose, 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 ]
- Number of spontaneous or traumatic NSB events [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
- Pharmacokinetic parameters for vWF and FVIII (PK arm only) [ Time Frame: Up to 72 hours following infusions on Day 1 and approximately Day 180 ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Development of FVIII inhibitors [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: Yes ]
- Development of vWF inhibitors [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: Yes ]
| Enrollment: | 22 |
| Study Start Date: | June 2009 |
| Study Completion Date: | February 2012 |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: PK
Includes subjects participating in the pharmacokinetic component of the study.
|
Biological: Biostate®
80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
Other Name: Human Coagulation Factor VIII / von Willebrand Factor
|
|
Experimental: Prophylaxis
Includes subjects receiving 12 months of prophylactic therapy.
|
Biological: Biostate®
Frequency and dose will be determined by the Investigator based on the subjects clinical condition, previous VWF concentrate requirements, response to therapy, weight and reason for usage.
Other Name: Human Coagulation Factor VIII / von Willebrand Factor
|
|
Experimental: On-demand
Includes subjects receiving 12 months of on-demand treatment.
|
Biological: Biostate®
Frequency and dose will be determined by the Investigator based on the subjects clinical condition, previous VWF concentrate requirements, response to therapy, weight and reason for usage.
Other Name: Human Coagulation Factor VIII / von Willebrand Factor
|
|
Experimental: Cross-over to prophylaxis
Includes subjects completing 12 months of on-demand treatment (the "On-demand" arm) who cross-over to prophylactic therapy for an additional 12-month period.
|
Biological: Biostate®
Frequency and dose will be determined by the Investigator based on the subjects clinical condition, previous VWF concentrate requirements, response to therapy, weight and reason for usage.
Other Name: Human Coagulation Factor VIII / von Willebrand Factor
|
Eligibility| Ages Eligible for Study: | 12 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Diagnosed with VWD
- Desmopressin acetate (DDAVP) treatment is ineffective or contraindicated or not available
- Evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B) within 10 years prior to their first dose of Biostate®
- Written informed consent given
Exclusion Criteria (for participation in the PK component):
- Actively bleeding immediately prior to initial PK period
- Have received DDAVP or a VWF product in the 5 days prior to their first dose of study product
- Have Type 2B, 2N or 2M VWD
Exclusion Criteria (for all subjects):
- Requiring a VWF product for a planned surgical procedure at enrolment
- Have received aspirin or other non-steroidal anti-inflammatory drugs within 7 days prior to their first dose of study product
- Known history of, or are suspected to have, VWF or FVIII inhibitors
- Suffering an acute or chronic medical condition, other than VWD, which may affect the conduct of the study
- Known or suspected hypersensitivity or previous evidence of severe side effects to Biostate®, VWF/FVIII concentrates, or human albumin
- Impaired liver function at screening
- Evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit
- Participation in a clinical study or use of an investigational compound in the 3 months preceding the first day of study drug administration, or plans to enter such a study during the study period.
- Females who are pregnant, breast-feeding or who have a positive pregnancy test at screening
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00941616
Locations
| Bulgaria | |
| Study Site | |
| Sofia, Bulgaria | |
| Poland | |
| Study Site | |
| Warsaw, Poland | |
| Study Site | |
| Wroclaw, Poland | |
| Russian Federation | |
| Study Site | |
| Barnaul, Russian Federation | |
| Ukraine | |
| Study Site | |
| Lviv, Ukraine, 79044 | |
Sponsors and Collaborators
CSL Behring
Parexel
Investigators
| Study Director: | Program Director, Clinical R&D | CSL Behring |
More Information
Additional Information:
No publications provided
| Responsible Party: | CSL Behring |
| ClinicalTrials.gov Identifier: | NCT00941616 History of Changes |
| Other Study ID Numbers: | CSLCT-BIO-08-54, 1481, 2008-004922-18 |
| Study First Received: | July 15, 2009 |
| Last Updated: | April 4, 2013 |
| Health Authority: | Russia: Ministry of Health of the Russian Federation Bulgaria: Bulgarian Drug Agency Brazil: National Health Surveillance Agency Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products Ukraine: State Pharmacological Center - Ministry of Health |
Keywords provided by CSL Behring:
|
Von Willebrand Disease |
Additional relevant MeSH terms:
|
Von Willebrand Diseases Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases Coagulation Protein Disorders Blood Platelet Disorders Hemorrhagic Disorders |
Genetic Diseases, Inborn Factor VIII Coagulants Hematologic Agents Therapeutic Uses Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 23, 2013