Study to investigate the study drug (WILATE) in patients with Von Willebrand Disease.

Phase 1

Conditions: Von Willebrand disease, type 3, type 2 (except 2N), or severe type 1
MedDRA version: 20.0Level: LLTClassification code 10055168Term: Von Willebrand's factor deficiencySystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2018-004675-13-HU

Lead Sponsor: Octapharma AG

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 28

Inclusion Criteria

1.Patients aged =6 years at the time of screening
2.VWD type 1 (baseline von Willebrand factor activity [VWF:Ac], ristocetin-based, <30 IU/dL), 2A, 2B, 2M, or 3 according to medical history requiring substitution therapy with a VWF-containing product to control bleeding
3.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 requiring treatment with a VWF-containing product
4.Availability of records to reliably evaluate type, frequency, and treatment of BEs for 6 months of on-demand treatment before screening
5.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 contraception is used, the medication class should remain unchanged for the duration of the study
6.Voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted

Are the trial subjects under 18? yes
Number of subjects for this age range: 2
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 21
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 3

Exclusion Criteria

1.Patients currently on prophylaxis or having received prophylaxis within 6 months before screening (as well as patients having received treatment once a month for menstrual bleeding, but not for any other bleeding)
2.History, or current suspicion, of VWF or FVIII inhibitors
3.Medical history of a thromboembolic event within 1 year before enrolment
4.Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine >120 µmol/L)
5.Platelet count <100,000/mL at screening (except for VWD type 2B)
6.Patients receiving, or scheduled to receive, immunomodulating drugs (other than an-tiretroviral chemotherapy), such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs
7.Pregnant or breast-feeding at the time of enrolment
8.Change in hormonal contraception within 6 months before enrolment
9.Cervical or uterine conditions causing abnormal uterine bleeding (including infection, dysplasia)
10.Treatment with any investigational medicinal product (IMP) in another interventional clinical study currently or within 4 weeks before enrolment
11.Other coagulation disorders or bleeding disorders due to anatomical reasons
12.Known hypersensitivity to any of the components of the study drug

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of this study is to determine the efficacy of Wilate in the prophylactic treatment of previously treated patients with type 3, type 2 (except 2N), or severe type 1 VWD;Secondary Objective: The secondary objectives of this study are to:<br> - Assess the VWF:Ac and VWF:Ag incremental IVR of Wilate over time (at baseline and at 3, 6, 9, and 12 months of treatment)<br> - Assess the safety and tolerability of Wilate<br>;Primary end point(s): The primary endpoint of this study is to demonstrate that the total annualised bleeding rate (TABR) during prophylactic treatment with Wilate lowers the patients’ TABR observed during on-demand treatment by more than 50%.;Timepoint(s) of evaluation of this end point: Baseline, 3, 6, 9 and 12 months<br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Spontaneous annualised bleeding rate (SABR)<br> - Wilate consumption data (VWF/FVIII IU/kg per month per patient) for prophylaxis<br> - Incremental VWF:Ac and VWF:Ag IVR of Wilate over time (at baseline and at 3, 6, 9, and 12 months of treatment)<br> - Safety and tolerability of Wilate by monitoring adverse events (AEs) throughout the study;Timepoint(s) of evaluation of this end point: Baseline, 3, 6, 9 and 12 months

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