NCT01224808

Brief Summary

The aim of the Von Willebrand Disease (VWD) therapy is to treat and prevent bleeding episodes due to abnormal platelet adhesion and abnormal blood coagulation as a result of low or abnormal Von Willebrand Factor (VWF) and/or Factor VIII (FVIII) levels. The long-term efficacy and safety of a VWF/FVIII concentrate, Biostate, will be investigated in children, adolescents, and adults with 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
20

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Oct 2010

Typical duration for phase_3

Geographic Reach
5 countries

6 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

First Submitted

Initial submission to the registry

June 8, 2010

Completed
4 months until next milestone

Study Start

First participant enrolled

October 1, 2010

Completed
19 days until next milestone

First Posted

Study publicly available on registry

October 20, 2010

Completed
3.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2014

Completed
Last Updated

October 3, 2017

Status Verified

October 1, 2017

Enrollment Period

3.4 years

First QC Date

June 8, 2010

Last Update Submit

October 2, 2017

Conditions

Von Willebrand Disease

Keywords

von Willebrand Disease

Outcome Measures

Primary Outcomes (1)

  • Haemostatic efficacy

    Up to 32 months

Secondary Outcomes (8)

  • Development of FVIII inhibitors

    Up to 32 months

  • Development of VWF inhibitors

    Up to 32 months

  • Frequency of Adverse events (AEs) per subject

    32 months

  • Severity of AEs per subject

    32 months

  • Severity of AEs per infusion

    32 months

  • +3 more secondary outcomes

Study Arms (1)

Experimental

EXPERIMENTAL
Biological: Biostate

Interventions

BiostateBIOLOGICAL

Single bolus doses, administered intravenously. Frequency and dose will be determined by the Investigator based on the subjects clinical condition, previous VWF concentrate requirements, response to therapy, weight and reason for usage.

Experimental

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Have completed Study CSLCT-BIO-08-52 (Assessment of Efficacy and Safety of Biostate in Paediatric Subjects with Von Willebrand Disease) or Study CSLCT-BIO-08-54 (Assessment of Efficacy and Safety of Biostate in Adolescent or Adult Subjects with Von Willebrand Disease).
  • The subject and/or his/her legal guardian understand(s) the nature of the study and has/have given written informed consent to participate in the study and is/are willing to comply with the protocol.

You may not qualify if:

  • Early discontinuation of a subject from the main studies CSLCT-BIO-08-52 or CSLCT-BIO-08-54.
  • Mental condition rendering the subject (or the subject's legal guardian) unable to understand the nature, scope and possible consequences of the study.
  • Any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct of the study.
  • Are not willing and/or not able to comply with the study requirements.
  • Employee at the study site, or spouse/partner or relative of the Investigator or Subinvestigators.
  • Female subjects of childbearing potential either not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study, or not sexually abstinent for the entire duration of the study, or not surgically sterile.
  • Intention to become pregnant during the course of the study.
  • Pregnancy, or nursing mother.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Study Site

Sofia, Bulgaria

Location

Study Site

Bremen, Germany

Location

Study Site

Warsaw, Poland

Location

Study Site

Wroclaw, Poland

Location

Study Site

Barnaul, Russia

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

June 8, 2010

First Posted

October 20, 2010

Study Start

October 1, 2010

Primary Completion

March 1, 2014

Study Completion

March 1, 2014

Last Updated

October 3, 2017

Record last verified: 2017-10

Locations

DE(1)UA(1)BU(1)RU(1)PL(2)