NCT01213446

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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
17

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Aug 2010

Typical duration for phase_3

Geographic Reach
6 countries

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2010

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

October 1, 2010

Completed
3 days until next milestone

First Posted

Study publicly available on registry

October 4, 2010

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2013

Completed
Last Updated

October 3, 2017

Status Verified

October 1, 2017

Enrollment Period

3 years

First QC Date

October 1, 2010

Last Update Submit

October 2, 2017

Conditions

Von Willebrand Disease

Keywords

Von Willebrand Disease

Outcome Measures

Primary Outcomes (19)

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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

Secondary Outcomes (8)

  • Frequency of adverse events (AEs) per infusion

    13 months

  • Severity of AEs per infusion

    13 months

  • Severity of AEs per subject

    13 months

  • Relatedness of AEs per infusion

    13 months

  • Relatedness of AEs per subject

    13 months

  • +3 more secondary outcomes

Study Arms (1)

Biostate

EXPERIMENTAL
Biological: Biostate

Interventions

BiostateBIOLOGICAL

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.

Biostate

Eligibility Criteria

AgeUp to 12 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Study site

Homyel, 246040, Belarus

Location

Study site

Minsk, 223040, Belarus

Location

Study site

Tbilisi, 0179, Georgia

Location

Study site

Bremen, 28177, Germany

Location

Study site

Guatemala City, CP, 01010, Guatemala

Location

Study site

Beirut, Lebanon

Location

Study Site

Lviv, Ukraine

Location

MeSH Terms

Conditions

von Willebrand Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersBlood Platelet DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Program Director, Clinical R&D

    CSL Behring

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 1, 2010

First Posted

October 4, 2010

Study Start

August 1, 2010

Primary Completion

August 1, 2013

Study Completion

August 1, 2013

Last Updated

October 3, 2017

Record last verified: 2017-10

Locations

GE(1)UA(1)DE(1)BE(2)GU(1)LE(1)