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Extension Study of Biostate in Subjects With Von Willebrand Disease

Primary Purpose

Von Willebrand Disease

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Biostate

About this trial

This is an interventional treatment trial for Von Willebrand Disease focused on measuring von Willebrand Disease

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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.

Sites / Locations

Study Site
Study Site
Study Site
Study Site
Study Site
Study Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Experimental

Arm Description

Outcomes

Primary Outcome Measures

Haemostatic efficacy

Secondary Outcome Measures

Development of FVIII inhibitors

Development of VWF inhibitors

Frequency of Adverse events (AEs) per subject

Severity of AEs per subject

Severity of AEs per infusion

Causality of AEs per subject

Causality of AEs per infusion

Frequency of Adverse events (AEs) per infusion

Full Information

NCT ID

NCT01224808

First Posted

June 8, 2010

Last Updated

October 2, 2017

Sponsor

CSL Behring

1. Study Identification

Unique Protocol Identification Number

NCT01224808

Brief Title

Extension Study of Biostate in Subjects With Von Willebrand Disease

Official Title

An Open-Label, Multi-Centre Extension Study to Assess the Efficacy and Safety of Biostate® in Paediatric, Adolescent, and Adult Subjects With Von Willebrand Disease Who Completed Clinical Studies CSLCT-BIO-08-52 or CSLCTBIO-08-54

Study Type

Interventional

2. Study Status

Record Verification Date

October 2017

Overall Recruitment Status

Completed

Study Start Date

October 2010 (undefined)

Primary Completion Date

March 2014 (Actual)

Study Completion Date

March 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

CSL Behring

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Von Willebrand Disease

Keywords

von Willebrand Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

20 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Experimental

Arm Type

Experimental

Intervention Type

Biological

Intervention Name(s)

Biostate

Intervention Description

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.

Primary Outcome Measure Information:

Title

Haemostatic efficacy

Time Frame

Up to 32 months

Secondary Outcome Measure Information:

Title

Development of FVIII inhibitors

Time Frame

Up to 32 months

Title

Development of VWF inhibitors

Time Frame

Up to 32 months

Title

Frequency of Adverse events (AEs) per subject

Time Frame

32 months

Title

Severity of AEs per subject

Time Frame

32 months

Title

Severity of AEs per infusion

Time Frame

32 months

Title

Causality of AEs per subject

Time Frame

32 months

Title

Causality of AEs per infusion

Time Frame

32 months

Title

Frequency of Adverse events (AEs) per infusion

Time Frame

32 months

10. Eligibility

Sex

All

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Program Director, Clinical R&D

Organizational Affiliation

CSL Behring

Official's Role

Study Director

Facility Information:

Facility Name

Study Site

City

Sofia

Country

Bulgaria

Facility Name

Study Site

City

Bremen

Country

Germany

Facility Name

Study Site

City

Warsaw

Country

Poland

Facility Name

Study Site

City

Wroclaw

Country

Poland

Facility Name

Study Site

City

Barnaul

Country

Russian Federation

Facility Name

Study Site

City

Lviv

Country

Ukraine

12. IPD Sharing Statement

Links:

URL

http://www.cslbehring.com/clinical-trials/contact-us.htm?registryRefNum=NCT01224808&registryName=ctgov

Description

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