Clinical trial NCT01224808: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Von Willebrand Disease
Keywords: von Willebrand Disease
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2010-10-20
Enrollment: 20
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Haemostatic efficacy
timeFrame: Up to 32 months
Secondary outcomes:
measure: Development of FVIII inhibitors
timeFrame: Up to 32 months
measure: Development of VWF inhibitors
timeFrame: Up to 32 months
measure: Frequency of Adverse events (AEs) per subject
timeFrame: 32 months
measure: Severity of AEs per subject
timeFrame: 32 months
measure: Severity of AEs per infusion
timeFrame: 32 months
measure: Causality of AEs per subject
timeFrame: 32 months
measure: Causality of AEs per infusion
timeFrame: 32 months
measure: Frequency of Adverse events (AEs) per infusion
timeFrame: 32 months
Other outcomes:
Locations:
city: Sofia
country: Bulgaria
facility: Study Site
city: Bremen
country: Germany
facility: Study Site
city: Warsaw
country: Poland
facility: Study Site
city: Wroclaw
country: Poland
facility: Study Site
city: Barnaul
country: Russian Federation
facility: Study Site
city: Lviv
country: Ukraine
facility: Study Site
Study officials:
affiliation: CSL Behring
name: Program Director, Clinical R&D
role: STUDY_DIRECTOR
References: