An Open-label, Multi-centre Study to Assess the Efficacy and Safety of Biostate® in Patients With von Willebrand's Disease (VWD)

• Conditions: Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]; Von Willebrand's disease (VWD); MedDRA version: 17.1Level: PTClassification code 10047715Term: Von Willebrand's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders

• Registration Number: EUCTR2014-005401-20-Outside-EU/EEA
• Lead Sponsor: CSL Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-01-30
• Last Update Posted: 2 February 2015

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 23

Inclusion Criteria

Patients who fulfill the following criteria are eligible for inclusion into this study:

a) Patients with a diagnosis VWD who require prophylactic therapy, have a non-surgery bleed, or are undergoing surgery.

b) Patients who would routinely receive a plasma derived FVIII/VWF product as treatment for their VWD.

c) Patients over 3 years of age and =14 kg.
Inclusion of children < 13 years of age into the surgical and non-surgical bleed arms of the study will be at the discretion of the Investigator. The Investigator will take into consideration the blood profile of the patient and the required blood draw volume, bearing in mind that 17 ml of blood
is to be drawn prior to each administration of Biostate® and that in accordance with the National Institute of Health Clinical Centre Guidelines, a paediatric blood draw should not exceed 3ml/kg, or 7ml/kg in a 6 week period.

d) Patients and/or their legally acceptable representative must give written informed consent to participate in the study and must understand the nature of the study and must be willing to comply with all protocol requirements.

In addition, patients receiving prophylactic therapy or being managed for elective surgery must also fulfill the following criterion:

e) Patients must have been vaccinated against hepatitis A and B (unless protective antibodies are present resulting from a previously experienced infection). Documentation of immunisation (or protective antibodies resulting from a previous infection) must be present at baseline.

Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 9
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 13

Exclusion Criteria

The following patients are not eligible for inclusion into this study:

a) Patients with a known history of adverse drug reactions to factor VIII/VWF concentrates.

b) Patients with a known or suspected VWF or FVIII inhibitor.

c) Patients who are currently taking non-steroidal anti-inflammatory drugs (NSAIDs) which in the opinion of the Investigator would affect the efficacy and safety outcomes of the study.

d) Patients with evidence or a history within the previous 12 months of abuse of any drug substance, licit or illicit, which in the opinion of the Investigator may affect the ability of the patient to comply with all protocol requirements.

e) Patients who have within 90 days prior to the study screening visit, participated in a clinical study or used an investigational compound (eg. a new chemical entity not registered for clinical use), or who are planning to enter such a different study during the study period.

f) Patients who are suffering from an acute or chronic medical condition, other than VWD, which may, in the opinion of the Investigator, affect the conduct of the study.

g) Patients with a positive pregnancy test.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified