Clinical study to assess how well wilate works in the regular treatment of young children with severe von Willebrand disease

Phase 3

• Conditions: Von Willebrand disease; Haematological Disorders

• Registration Number: ISRCTN11217735
• Lead Sponsor: Octapharma (Austria)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-08-23
• Last Update Posted: 18 March 2024
• Study Completion: 2024-12-31

Recruitment & Eligibility

• Status: Ongoing
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

1. Patients aged <6 years at the time of screening
2. Type 3 (at least four patients), severe type 2 (except 2N) or severe type 1 VWD (and of which with VWF:RCo <20%) according to medical history, requiring substitution therapy with a VWF-containing product
3. Minimum body weight 12.5 kg at the time of screening
4. Voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted (obtained from the patient’s parent(s)/legal guardian(s))

Exclusion Criteria

1. History, or current suspicion of VWF or FVIII inhibitors
2. Injection of DDAVP or VWF-containing product within 72 hours prior to inclusion
3. Medical history of a thromboembolic event
4. Platelet count <100,000/µl at screening (except for VWD type 2B)
5. Patients receiving, or scheduled to receive, immunosuppressant drugs (other than antiretroviral chemotherapy), such as prednisone (equivalent to >10 mg/day), or similar drugs
6. Treatment with any investigational medicinal product (IMP) in another interventional clinical study currently or within four weeks before enrolment
7. Other coagulation disorders or bleeding disorders
8. Known hypersensitivity to any of the components of the study drug

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
The total annualised bleeding rate (tABR) during prophylactic treatment with wilate: the tABR will be calculated as the total number of spontaneous bleeds, traumatic bleeds, and other bleeds occurring in the time period between the first prophylactic dose of wilate and the Study Completion Visit, divided by the duration (in years) between the first prophylactic dose of wilate and the Study Completion Visit. Bleeding episodes occurring within surgery periods will be excluded from the calculation of tABR. In addition to the estimates of the tABR exploratory 95% CIs will be calculated from a Poisson regression model