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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00941616
Other study ID # CSLCT-BIO-08-54
Secondary ID 14812008-004922-
Status Completed
Phase Phase 2/Phase 3
First received July 15, 2009
Last updated October 2, 2017
Start date June 2009
Est. completion date February 2012

Study information

Verified date October 2017
Source CSL Behring
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Completed
Enrollment 22
Est. completion date February 2012
Est. primary completion date February 2012
Accepts healthy volunteers No
Gender All
Age group 12 Years and older
Eligibility 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 (for participation in the PK component):

- 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

Exclusion Criteria (for all subjects):

- 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

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
Biostate®
80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
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.

Locations

Country Name City State
Bulgaria Study Site Sofia
Poland Study Site Warsaw
Poland Study Site Wroclaw
Russian Federation Study Site Barnaul
Ukraine Study Site Lviv

Sponsors (2)

Lead Sponsor Collaborator
CSL Behring Parexel

Countries where clinical trial is conducted

Bulgaria,  Poland,  Russian Federation,  Ukraine, 

Outcome

Type Measure Description Time frame Safety issue
Primary Haemostatic efficacy at time of non-surgical bleeding (NSB) event From Day 1 until final study visit
Primary Haemostatic efficacy overall Monthly (prophylactic therapy) or once every 3 months (for on-demand use)
Primary Number of treatments with blood product transfusions required to resolve any bleeding event From Day 1 until final study visit
Primary vWF/FVIII concentrate usage (number of infusions, IU/kg per dose, per event, per month and per year) From Day 1 until final study visit
Primary Assessment of blood loss during any surgical procedure From Day 1 until final study visit
Primary Number of spontaneous or traumatic NSB events From Day 1 until final study visit
Primary Pharmacokinetic parameters for vWF and FVIII (PK arm only) Up to 72 hours following infusions on Day 1 and approximately Day 180
Secondary Development of FVIII inhibitors From Day 1 until final study visit
Secondary Development of vWF inhibitors From Day 1 until final study visit
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Active, not recruiting NCT04953884 - Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease VWD) Patients <6 Years of Age Phase 3