Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment

Octapharma

Status: Completed

Conditions: Von Willebrand Diseases

Treatments: Drug: Von Willebrand Factor-Containing Product

Study type: Observational

Funder types: Industry

Identifiers

NCT04053699

WIL-29

Details and patient eligibility

About

The purpose of this study is to prospectively obtain reliable data on the bleeding and treatment pattern of patients with VWD undergoing on-demand treatment with a VWF-containing product over a period of 6 months. The data obtained will be used as a basis for historical comparisons with the bleeding and treatment pattern obtained from a clinical study on the efficacy of prophylactic treatment with a VWF/FVIII concentrate.

Enrollment: 56 patients

Sex: All

Ages: 66+ months old

Volunteers: No Healthy Volunteers

Inclusion criteria

Patients who meet all of the following criteria are eligible for the study:

• Male or female patients aged ≥5.5 years at the time of enrolment
• VWD type 1 (baseline von Willebrand factor activity [VWF:RCo], <30 IU/dL), 2A, 2B, 2M, or 3 according to medical history requiring substitution therapy with a VWF-containing product to control bleeding
• Currently receiving frequent on-demand treatment with a VWF-containing product
• In female patients of child-bearing potential using hormonal contraception, the medication class should remain unchanged for the duration of their study participation
• Voluntarily given, fully informed written and signed consent obtained before collection of any patient data

Exclusion criteria

Patients who meet any of the following criteria are not eligible for the study:

• Patients currently on prophylaxis for VWD (except for perioperative prophylaxis) as well as patients having received treatment once a month for menstrual bleeding, but not for any other bleeds
• Patients whose VWD treatment is planned to be switched from on-demand to prophylactic treatment in the next 6 months
• History, or current suspicion, of VWF or FVIII inhibitors
• Medical history of a thromboembolic event within 6 months before enrolment
• Severe liver or kidney diseases as described in the medical records
• Female patients with an existing or suspected pregnancy or who are breast-feeding at the time of enrolment
• Change in hormonal contraception within 6 months before enrolment
• Cervical or uterine conditions causing abnormal uterine bleeding (including infection or dysplasia)
• Other coagulation disorders or bleeding disorders due to anatomical reasons
• Participation in an interventional clinical study during the 6-month of study period
• Inability to complete the patient diary to reliably evaluate the type, frequency, and treatment of BEs during the 6-month study period

Trial design

56 participants in 1 patient group

Patients undergoing treatment with a VWF-containing product

Description:

Patients with type 3, type 2 (except 2N), or severe type 1 VWD undergoing routine on-demand treatment with a VWF-containing product over a period of 6 months

Treatment:

Drug: Von Willebrand Factor-Containing Product