Study of Biostate® in Children With Von Willebrand Disease

Von Willebrand Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01213446
• Other study ID #: CSLCT-BIO-08-52
• Secondary ID: 2009-017753-3414
• Status: Completed
• Phase: Phase 3
• First received: October 1, 2010
• Last updated: October 2, 2017
• Start date: August 2010
• Est. completion date: August 2013

Study information

• Verified date: October 2017
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 17
• Est. completion date: August 2013
• Est. primary completion date: August 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 12 Years

Eligibility

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

Related Conditions & MeSH terms

• Von Willebrand Disease
• Von Willebrand Diseases

Intervention

Biological:
• Biostate
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.

Locations

Country	Name	City	State
Belarus	Study site	Homel
Belarus	Study site	Minsk
Georgia	Study site	Tbilisi
Germany	Study site	Bremen
Guatemala	Study site	Guatemala	CP
Lebanon	Study site	Beirut
Ukraine	Study Site	Lviv

Sponsors (2)

Lead Sponsor	Collaborator
CSL Behring	Parexel

Countries where clinical trial is conducted

Belarus,  Georgia,  Germany,  Guatemala,  Lebanon,  Ukraine, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Haemostatic efficacy		From Day 1 until final study visit
Primary	Incremental Recovery of VWF		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
Primary	Incremental Recovery of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Half-life of VWF		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
Primary	Half-life of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Area under the concentration curve (AUC) of VWF		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
Primary	AUC of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Maximum plasma concentration (Cmax) of VWF		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
Primary	Maximum plasma concentration (Cmax) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Minimum plasma concentration (Cmin) of VWF		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
Primary	Minimum plasma concentration (Cmin) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Time to maximum concentration (tmax) of VWF		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
Primary	Time to maximum concentration (tmax) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Mean residence time (MRT) of VWF		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
Primary	Mean residence time (MRT) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Clearance (CL) of VWF		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
Primary	Clearance (CL) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Volume of distribution of steady state (Vss) of VWF		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
Primary	Volume of distribution of steady state (Vss) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Secondary	Frequency of adverse events (AEs) per infusion		13 months
Secondary	Severity of AEs per infusion		13 months
Secondary	Severity of AEs per subject		13 months
Secondary	Relatedness of AEs per infusion		13 months
Secondary	Relatedness of AEs per subject		13 months
Secondary	Development of VWF inhibitors		Sample taken at baseline, then every 3 months up to 12 months
Secondary	Development of FVIII inhibitors		Sample taken at baseline, then every 3 months up to 12 months
Secondary	Frequency of adverse events (AEs) per subject		13 months

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