Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease

NCT ID: NCT00941616

Last Updated: 2017-10-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 22 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-06-30
• Study Completion Date: 2012-02-29

Brief Summary

The aim of this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD).

Pharmacokinetic Component:

PK parameters will be determined from a subgroup of subjects. Subjects who complete the PK component will subsequently continue in the efficacy component of the study, either continuing on a previously established prophylaxis regimen or continuing to receive on-demand treatment with the occurrence of non-surgical bleeding (NSB) events.

Efficacy Component:

Three treatment arms are defined for the efficacy component of the study. (1) Subjects who are currently being treated on a set prophylaxis regimen with a VWF product at the time of study entry will be enrolled in the "Prophylaxis" arm. (2) Subjects not being treated on a set prophylaxis regimen at the time of study entry who require a VWF product for the treatment of NSB events will be enrolled in the "On-demand" arm and commence using Biostate in the treatment of NSB events. (3) Subjects enrolled in the "On-demand" arm have the possibility to enter the "Cross-over to Prophylaxis" arm to receive an additional 12 months of prophylactic treatment.

Conditions

Von Willebrand Disease

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

PK

Includes subjects participating in the pharmacokinetic component of the study.

Group Type: EXPERIMENTAL

Biostate®

Intervention Type: BIOLOGICAL

80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180

Prophylaxis

Includes subjects receiving 12 months of prophylactic therapy.

Group Type: EXPERIMENTAL

Biostate®

Intervention Type: BIOLOGICAL

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.

On-demand

Includes subjects receiving 12 months of on-demand treatment.

Group Type: EXPERIMENTAL

Biostate®

Intervention Type: BIOLOGICAL

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.

Cross-over to prophylaxis

Includes subjects completing 12 months of on-demand treatment (the "On-demand" arm) who cross-over to prophylactic therapy for an additional 12-month period.

Group Type: EXPERIMENTAL

Biostate®

Intervention Type: BIOLOGICAL

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.

Interventions

Biostate®

80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180

Intervention Type: BIOLOGICAL

Biostate®

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.

Intervention Type: BIOLOGICAL

Other Intervention Names

Human Coagulation Factor VIII / von Willebrand Factor; Human Coagulation Factor VIII / von Willebrand Factor

Eligibility Criteria

Inclusion Criteria

• Diagnosed with VWD
• Desmopressin acetate (DDAVP) treatment is ineffective or contraindicated or not available
• Evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B) within 10 years prior to their first dose of Biostate®
• Written informed consent given

Exclusion Criteria

• Actively bleeding immediately prior to initial PK period
• Have received DDAVP or a VWF product in the 5 days prior to their first dose of study product
• Have Type 2B, 2N or 2M VWD

• Requiring a VWF product for a planned surgical procedure at enrolment
• Have received aspirin or other non-steroidal anti-inflammatory drugs within 7 days prior to their first dose of study product
• Known history of, or are suspected to have, VWF or FVIII inhibitors
• Suffering an 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 Biostate®, VWF/FVIII concentrates, or human albumin
• Impaired liver function at screening
• Evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit
• Participation in a clinical study or use of an investigational compound in the 3 months preceding the first day of study drug administration, or plans to enter such a study during the study period.
• Females who are pregnant, breast-feeding or who have a positive pregnancy test at screening

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Parexel

INDUSTRY

Sponsor Role: collaborator

CSL Behring

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Program Director, Clinical R&D

Role: STUDY_DIRECTOR

CSL Behring

Locations

Study Site

Sofia, , Bulgaria

Study Site

Warsaw, , Poland

Study Site

Wroclaw, , Poland

Study Site

Barnaul, , Russia

Study Site

Lviv, , Ukraine

Countries

Bulgaria; Poland; Russia; Ukraine

Other Identifiers

1481

Identifier Type: OTHER

Identifier Source: secondary_id

2008-004922-18

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CSLCT-BIO-08-54

Identifier Type: -

Identifier Source: org_study_id