Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD

Octapharma

Status and phase

Completed

Phase 3

Conditions

Von Willebrand Diseases

Treatments

Drug: Wilate

Study type

Interventional

Funder types

Industry

Identifiers

NCT04052698

WIL-31

Details and patient eligibility

About

This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy of Wilate in previously treated patients with VWD undergoing regular prophylaxis.

Enrollment

43 patients

Sex

All

Ages

6+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

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

Aged ≥6 years at the time of screening
VWD type 1 (baseline von Willebrand factor activity [VWF:Ristocetin Co-factor (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 on-demand treatment with a VWF-containing product with at least 1, and an average of ≥2, documented spontaneous BEs per month in the last 6 months, with at least 2 of these BEs requiring treatment with a VWF-containing product
Availability of records to reliably evaluate type, frequency, and treatment of BEs for at least 6 months of on-demand treatment before screening
Female patients of child-bearing potential must have a negative urine pregnancy test at screening and agree to use adequate birth control measures; in case hormonal contra-ception is used, the medication class should remain unchanged for the duration of the study
All patients to provide voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted

Exclusion criteria

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

Having received on-demand or prophylactic treatment with a VWF-containing product but having no records available to reliably evaluate the type, frequency, and treatment of BEs over a period of at least 6 months of on-demand treatment
History, or current suspicion, of VWF or FVIII inhibitors
Medical history of a thromboembolic event within 1 year before enrolment
Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate trans-aminase [ASAT] levels >5 times of upper limit of normal, creatinine >120 µmol/L)
Platelet count <100,000/µL at screening (except for VWD type 2B)
Body weight <20 kg at screening
Patients receiving, or scheduled to receive, immunosuppressant drugs (other than an-tiretroviral chemotherapy), such as prednisone (equivalent to >10 mg/day), or similar drugs
Pregnant or breast-feeding at the time of enrolment
Cervical or uterine conditions causing abnormal uterine bleeding (including infection, dysplasia)
Treatment with any IMP in another interventional clinical study currently or within 4 weeks before enrolment
Other coagulation disorders or bleeding disorders due to anatomical reasons
Known hypersensitivity to any of the components of the study drug