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-HR

Lead Sponsor: Octapharma AG

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

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:RCo] <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 AND having experienced at least 6 BEs (excluding menstrual bleeds) over a period of 6 months, with at least 2 of these BEs treated with a VWF containing
product AND having records available to reliably evaluate the type, frequency, and treatment of BEs in this 6-month period
OR
Having switched to prophylactic treatment with a VWF-containing product within the past 2 years AND having records available to reliably evaluate the type, frequency, and treatment of BEs over a period of 6 months of on-demand
treatment
4.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
5.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: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 15
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 3

Exclusion Criteria

1.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
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. Body weight <20 kg at screening
7.Patients receiving, or scheduled to receive, immunosuppressant drugs (other than an-tiretroviral chemotherapy), such as prednisone (equivalent to >10 mg/day), or similar drugs
8.Pregnant or breast-feeding at the time of 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 incremental IVR of Wilate for VWF:Ac and FVIII:C over time<br> - Determine the pharmacokinetics (PK) of WIlate for VWF:Ac and FVIII:C in paediatric patients aged 6 to 16 years<br> - Assess the safety and tolerability of Wilate<br>- Determine Wilate consumption data<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, 1, 2, 3, 6, 9 and 12 months<br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Spontaneous annualised bleeding rate (SABR) calculated in analogy with TABR<br> - Incremental IVR of Wilate for VWF:Ac (VWF:RCo and VWF:GP1bM) and FVIII:C (OS and CHR) over time (at baseline and at 1, 2, 3, 6, 9, and 12 months of treatment).<br>- For paediatric patients, baseline PK profile characteristics of VWF:Ac (VWF:RCo), and FVIII:C (OS and CHR) based on blood samples taken pre-dose and 1, 3, 9, 24, 48, and 72 hours after dosing<br> - Safety and tolerability of Wilate by monitoring adverse events (AEs) throughout the study<br>- Wilate consumption data (VWF/FVIII IU/kg per month per patient) for<br>prophylaxis;Timepoint(s) of evaluation of this end point: Baseline, 1, 2, 3, 6, 9 and 12 months

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