Extension Study of Biostate in Subjects With Von Willebrand Disease

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Von Willebrand Disease

Intervention / Treatment
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* Biological: Biostate


Participation Criteria
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Eligibility Criteria
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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.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Experimental<br> | Biological: Biostate<br>* 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.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Haemostatic efficacy |  | Up to 32 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| Causality of AEs per subject |  | 32 months | 
| Causality of AEs per infusion |  | 32 months | 
| Frequency of Adverse events (AEs) per infusion |  | 32 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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von Willebrand Disease