Clinical trial NCT01213446: Study of Biostate® in Children With Von Willebrand Disease
Official title: A Phase III Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy, and Safety of Biostate® in Paediatric Subjects With Von Willebrand Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Von Willebrand Disease
Keywords: Von Willebrand Disease
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
Collaborators: name: Parexel
First posted: 2010-10-04
Enrollment: 17
Sex: ALL
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (VWD) in whom treatment with a VWF product is required for prophylactic therapy, haemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event.


Eligibility criteria: Inclusion Criteria:

* Male and female subjects between 0 and \<12 years of age
* Diagnosed with VWD Type 1, 2A, or 3
* Desmopressin acetate (DDAVP) treatment is ineffective, contraindicated, or not available for subject
* von Willebrand factor: ristocetin cofactor (VWF:RCo) is \<20% at screening or the subject has a history of VWF:RCo \<10%
* 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 immunization
* Written informed consent given

Exclusion Criteria:

* Active bleeding immediately prior to initial PK period
* Received treatment with DDAVP or a VWF concentrate product for their VWD in the 5 days prior to their first study treatment
* Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of commencing the PK period.
* Known history or suspicion of having VWF or FVIII inhibitors
* 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 other FVIII/VWF concentrates
* Participation in a clinical study or use of an investigational compound in another study in the 3 months preceding study start
* Unwillingness and/or inability to comply with the study requirements

Primary outcomes:
measure: Haemostatic efficacy
timeFrame: From Day 1 until final study visit
measure: Incremental Recovery of VWF
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
measure: Incremental Recovery of FVIII
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
measure: Half-life of VWF
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
measure: Half-life of FVIII
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
measure: Area under the concentration curve (AUC) of VWF
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
measure: AUC of FVIII
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
measure: Maximum plasma concentration (Cmax) of VWF
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
measure: Maximum plasma concentration (Cmax) of FVIII
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
measure: Minimum plasma concentration (Cmin) of VWF
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
measure: Minimum plasma concentration (Cmin) of FVIII
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
measure: Time to maximum concentration (tmax) of VWF
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
measure: Time to maximum concentration (tmax) of FVIII
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
measure: Mean residence time (MRT) of VWF
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
measure: Mean residence time (MRT) of FVIII
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
measure: Clearance (CL) of VWF
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
measure: Clearance (CL) of FVIII
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
measure: Volume of distribution of steady state (Vss) of VWF
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
measure: Volume of distribution of steady state (Vss) of FVIII
timeFrame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Secondary outcomes:
measure: Frequency of adverse events (AEs) per infusion
timeFrame: 13 months
measure: Severity of AEs per infusion
timeFrame: 13 months
measure: Severity of AEs per subject
timeFrame: 13 months
measure: Relatedness of AEs per infusion
timeFrame: 13 months
measure: Relatedness of AEs per subject
timeFrame: 13 months
measure: Development of VWF inhibitors
timeFrame: Sample taken at baseline, then every 3 months up to 12 months
measure: Development of FVIII inhibitors
timeFrame: Sample taken at baseline, then every 3 months up to 12 months
measure: Frequency of adverse events (AEs) per subject
timeFrame: 13 months
Other outcomes:
Locations:
city: Homel
country: Belarus
facility: Study site
city: Minsk
country: Belarus
facility: Study site
city: Tbilisi
country: Georgia
facility: Study site
city: Bremen
country: Germany
facility: Study site
city: Guatemala
country: Guatemala
facility: Study site
state: CP
city: Beirut
country: Lebanon
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: