Status:

COMPLETED

Study of Biostate® in Children With Von Willebrand Disease

Lead Sponsor:

CSL Behring

Collaborating Sponsors:

Parexel

Conditions:

Von Willebrand Disease

Eligibility:

All Genders

Up to 12 years

Phase:

PHASE3

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) i...

Eligibility Criteria

Inclusion

  • 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

  • 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

Key Trial Info

Start Date :

August 1 2010

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

August 1 2013

Estimated Enrollment :

17 Patients enrolled

Trial Details

Trial ID

NCT01213446

Start Date

August 1 2010

End Date

August 1 2013

Last Update

October 3 2017

Active Locations (7)

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Page 1 of 2 (7 locations)

1

Study site

Homyel, Belarus, 246040

2

Study site

Minsk, Belarus, 223040

3

Study site

Tbilisi, Georgia, 0179

4

Study site

Bremen, Germany, 28177