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Study of Biostate® in Children With Von Willebrand Disease

NCT ID: NCT01213446

Last Updated: 2017-10-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

17 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-08-31

Study Completion Date

2013-08-31

Brief Summary

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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

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Conditions

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Von Willebrand Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Biostate

Group Type EXPERIMENTAL

Biostate

Intervention Type BIOLOGICAL

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.

Interventions

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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.

Intervention Type BIOLOGICAL

Eligibility Criteria

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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
Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Parexel

INDUSTRY

Sponsor Role collaborator

CSL Behring

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Program Director, Clinical R&D

Role: STUDY_DIRECTOR

CSL Behring

Locations

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Study site

Homyel, , Belarus

Site Status

Study site

Minsk, , Belarus

Site Status

Study site

Tbilisi, , Georgia

Site Status

Study site

Bremen, , Germany

Site Status

Study site

Guatemala City, CP, Guatemala

Site Status

Study site

Beirut, , Lebanon

Site Status

Study Site

Lviv, , Ukraine

Site Status

Countries

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Belarus Georgia Germany Guatemala Lebanon Ukraine

Other Identifiers

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2009-017753-34

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

1494

Identifier Type: OTHER

Identifier Source: secondary_id

CSLCT-BIO-08-52

Identifier Type: -

Identifier Source: org_study_id