Clinical trial NCT00941616: Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease
Official title: An Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy and Safety of Biostate® in Subjects With Von Willebrand Disease.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Von Willebrand Disease
Keywords: Von Willebrand Disease
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
Collaborators: name: Parexel
First posted: 2009-07-17
Enrollment: 22
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Haemostatic efficacy at time of non-surgical bleeding (NSB) event
timeFrame: From Day 1 until final study visit
measure: Haemostatic efficacy overall
timeFrame: Monthly (prophylactic therapy) or once every 3 months (for on-demand use)
measure: Number of treatments with blood product transfusions required to resolve any bleeding event
timeFrame: From Day 1 until final study visit
measure: vWF/FVIII concentrate usage (number of infusions, IU/kg per dose, per event, per month and per year)
timeFrame: From Day 1 until final study visit
measure: Assessment of blood loss during any surgical procedure
timeFrame: From Day 1 until final study visit
measure: Number of spontaneous or traumatic NSB events
timeFrame: From Day 1 until final study visit
measure: Pharmacokinetic parameters for vWF and FVIII (PK arm only)
timeFrame: Up to 72 hours following infusions on Day 1 and approximately Day 180
Secondary outcomes:
measure: Development of FVIII inhibitors
timeFrame: From Day 1 until final study visit
measure: Development of vWF inhibitors
timeFrame: From Day 1 until final study visit
Other outcomes:
Locations:
city: Sofia
country: Bulgaria
facility: Study Site
city: Warsaw
country: Poland
facility: Study Site
city: Wroclaw
country: Poland
facility: Study Site
city: Barnaul
country: Russian Federation
facility: Study Site
city: Lviv
country: Ukraine
facility: Study Site
Study officials:
affiliation: CSL Behring
name: Program Director, Clinical R&D
role: STUDY_DIRECTOR
References: