Study of a Plasma-Derived Von Willebrand Factor/Factor VIII Concentrate (vWF/FVIII), Biostate®, in Subjects With Von Willebrand Disease
Tracking InformationStart Date ICMJE | June 2009 |
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Estimated Primary Completion Date | November 2011 (final data collection date for primary outcome measure) |
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Current Primary Outcome Measures ICMJE (submitted: July 15, 2009) | - 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 ]
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Original Primary Outcome Measures ICMJE | Same as current |
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Change History | Complete list of historical versions of study NCT00941616 on ClinicalTrials.gov Archive Site |
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Current Secondary Outcome Measures ICMJE (submitted: July 15, 2009) | - 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 ]
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Original Secondary Outcome Measures ICMJE | Same as current |
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Descriptive InformationBrief Title ICMJE | Study of a Plasma-Derived Von Willebrand Factor/Factor VIII Concentrate (vWF/FVIII), Biostate®, in Subjects With Von Willebrand Disease |
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Official Title ICMJE | An Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy and Safety of Biostate® in Subjects With Von Willebrand Disease. |
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Brief Summary | 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. |
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Detailed Description | |
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Study Phase | Phase II, Phase III |
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Study Type ICMJE | Interventional |
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Study Design ICMJE | Treatment, Non-Randomized, Open Label, Parallel Assignment, Safety/Efficacy Study |
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Condition ICMJE | Von Willebrand Disease |
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Intervention ICMJE | - Biological: Biostate®
80 IU/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180 Other Name: Human Coagulation Factor VIII / von Willebrand Factor - 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
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Study Arms / Comparison Groups | - PK: Experimental
Includes subjects participating in the pharmacokinetic component of the study. Intervention: Biological: Biostate® - Prophylaxis: Experimental
Includes subjects receiving 12 months of prophylactic therapy. Intervention: Biological: Biostate® - On-demand: Experimental
Includes subjects receiving 12 months of on-demand treatment. Intervention: Biological: Biostate® - Cross-over to prophylaxis: Experimental
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. Intervention: Biological: Biostate®
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Recruitment InformationEstimated Enrollment ICMJE | 25 |
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Estimated Completion Date | November 2011 |
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Estimated Primary Completion Date | November 2011 (final data collection date for primary outcome measure) |
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Eligibility Criteria ICMJE | 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
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Gender | Both |
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Ages | 12 Years and older |
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Accepts Healthy Volunteers | No |
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Contacts ICMJE | |
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Location Countries ICMJE | Brazil, Bulgaria, Poland, Russian Federation, Ukraine |
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Administrative InformationNCT ID ICMJE | NCT00941616 |
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Responsible Party | Global Head Clinical Research & Development, CSL Behring |
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Study ID Numbers ICMJE | 1481, CSLCT-BIO-08-54, 2008-004922-18 |
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Study Sponsor ICMJE | CSL Behring |
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Collaborators ICMJE | Parexel |
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Investigators ICMJE | Study Director: | Program Director, Clinical R&D | CSL Behring | |
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Information Provided By | CSL Behring |
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