Overview

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

Status:
Active, not recruiting
Trial end date:
2022-04-01
Target enrollment:
0
Participant gender:
All
Summary
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.
Phase:
Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Octapharma
Treatments:
Factor VIII
Criteria
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