Study of Biostate® in Children With Von Willebrand Disease
Primary Purpose
Von Willebrand Disease
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Biostate
Sponsored by
About this trial
This is an interventional treatment trial for Von Willebrand Disease focused on measuring Von Willebrand Disease
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
Sites / Locations
- Study site
- Study site
- Study site
- Study site
- Study site
- Study site
- Study Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Biostate
Arm Description
Outcomes
Primary Outcome Measures
Haemostatic efficacy
Incremental Recovery of VWF
Incremental Recovery of FVIII
Half-life of VWF
Half-life of FVIII
Area under the concentration curve (AUC) of VWF
AUC of FVIII
Maximum plasma concentration (Cmax) of VWF
Maximum plasma concentration (Cmax) of FVIII
Minimum plasma concentration (Cmin) of VWF
Minimum plasma concentration (Cmin) of FVIII
Time to maximum concentration (tmax) of VWF
Time to maximum concentration (tmax) of FVIII
Mean residence time (MRT) of VWF
Mean residence time (MRT) of FVIII
Clearance (CL) of VWF
Clearance (CL) of FVIII
Volume of distribution of steady state (Vss) of VWF
Volume of distribution of steady state (Vss) of FVIII
Secondary Outcome Measures
Frequency of adverse events (AEs) per infusion
Severity of AEs per infusion
Severity of AEs per subject
Relatedness of AEs per infusion
Relatedness of AEs per subject
Development of VWF inhibitors
Development of FVIII inhibitors
Frequency of adverse events (AEs) per subject
Full Information
NCT ID
NCT01213446
First Posted
October 1, 2010
Last Updated
October 2, 2017
Sponsor
CSL Behring
Collaborators
Parexel
1. Study Identification
Unique Protocol Identification Number
NCT01213446
Brief Title
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
2. Study Status
Record Verification Date
October 2017
Overall Recruitment Status
Completed
Study Start Date
August 2010 (undefined)
Primary Completion Date
August 2013 (Actual)
Study Completion Date
August 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CSL Behring
Collaborators
Parexel
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Von Willebrand Disease
Keywords
Von Willebrand Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
17 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Biostate
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Biostate
Intervention Description
PK component: Single bolus infusion of 80 IU VWF:RCo/kg administered intravenously on Day 1, and approximately Day 180 in Type 3 VWD subjects only.
Efficacy component: Repeated bolus doses over 12 months as required to manage VWD condition.
Primary Outcome Measure Information:
Title
Haemostatic efficacy
Time Frame
From Day 1 until final study visit
Title
Incremental Recovery of VWF
Time Frame
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
Title
Incremental Recovery of FVIII
Time Frame
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Title
Half-life of VWF
Time Frame
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
Title
Half-life of FVIII
Time Frame
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Title
Area under the concentration curve (AUC) of VWF
Time Frame
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
Title
AUC of FVIII
Time Frame
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Title
Maximum plasma concentration (Cmax) of VWF
Time Frame
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
Title
Maximum plasma concentration (Cmax) of FVIII
Time Frame
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Title
Minimum plasma concentration (Cmin) of VWF
Time Frame
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
Title
Minimum plasma concentration (Cmin) of FVIII
Time Frame
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Title
Time to maximum concentration (tmax) of VWF
Time Frame
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
Title
Time to maximum concentration (tmax) of FVIII
Time Frame
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Title
Mean residence time (MRT) of VWF
Time Frame
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
Title
Mean residence time (MRT) of FVIII
Time Frame
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Title
Clearance (CL) of VWF
Time Frame
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
Title
Clearance (CL) of FVIII
Time Frame
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Title
Volume of distribution of steady state (Vss) of VWF
Time Frame
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
Title
Volume of distribution of steady state (Vss) of FVIII
Time Frame
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Secondary Outcome Measure Information:
Title
Frequency of adverse events (AEs) per infusion
Time Frame
13 months
Title
Severity of AEs per infusion
Time Frame
13 months
Title
Severity of AEs per subject
Time Frame
13 months
Title
Relatedness of AEs per infusion
Time Frame
13 months
Title
Relatedness of AEs per subject
Time Frame
13 months
Title
Development of VWF inhibitors
Time Frame
Sample taken at baseline, then every 3 months up to 12 months
Title
Development of FVIII inhibitors
Time Frame
Sample taken at baseline, then every 3 months up to 12 months
Title
Frequency of adverse events (AEs) per subject
Time Frame
13 months
10. Eligibility
Sex
All
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Program Director, Clinical R&D
Organizational Affiliation
CSL Behring
Official's Role
Study Director
Facility Information:
Facility Name
Study site
City
Homel
ZIP/Postal Code
246040
Country
Belarus
Facility Name
Study site
City
Minsk
ZIP/Postal Code
223040
Country
Belarus
Facility Name
Study site
City
Tbilisi
ZIP/Postal Code
0179
Country
Georgia
Facility Name
Study site
City
Bremen
ZIP/Postal Code
28177
Country
Germany
Facility Name
Study site
City
Guatemala
State/Province
CP
ZIP/Postal Code
01010
Country
Guatemala
Facility Name
Study site
City
Beirut
Country
Lebanon
Facility Name
Study Site
City
Lviv
Country
Ukraine
12. IPD Sharing Statement
Links:
URL
http://www.cslbehring.com/clinical-trials/contact-us.htm?registryRefNum=NCT01213446®istryName=ctgov
Description
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Study of Biostate® in Children With Von Willebrand Disease
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