Study of Biostate® in Children With Von Willebrand Disease

Phase 3

Completed

• Conditions: Von Willebrand Disease
• Interventions: Biological: Biostate

• Registration Number: NCT01213446
• Lead Sponsor: CSL Behring

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-08
• Study First Submitted: 2010-10-01
• Study First Submitted QC: 2010-10-01
• Study First Posted: 2010-10-04
• Primary Completion: 2013-08
• Study Completion: 2013-08
• Status Verified: 2017-10
• Last Update Submitted: 2017-10-02
• Last Update Posted: 2017-10-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 17

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Biostate	Biostate	-

Primary Outcome Measures

Name	Time	Method
Haemostatic efficacy	From Day 1 until final study visit
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
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
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
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
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
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
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
Incremental Recovery of FVIII	Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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
Half-life of FVIII	Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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
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
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
AUC of FVIII	Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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
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
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
Clearance (CL) of FVIII	Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1

Secondary Outcome Measures

Name	Time	Method
Development of FVIII inhibitors	Sample taken at baseline, then every 3 months up to 12 months
Frequency of adverse events (AEs) per subject	13 months
Severity of AEs per infusion	13 months
Severity of AEs per subject	13 months
Frequency of adverse events (AEs) per infusion	13 months
Relatedness of AEs per infusion	13 months
Relatedness of AEs per subject	13 months
Development of VWF inhibitors	Sample taken at baseline, then every 3 months up to 12 months

Trial Locations

Locations (2)

Study site; 🇱🇧 Beirut, Lebanon

Study Site; 🇺🇦 Lviv, Ukraine