Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease VWD) Patients <6 Years of Age

• Status: Recruiting
• Trial end date: 2023-01-01
• Target enrollment: 0
• Participant gender: All

Summary

The WIL-33 study aims to determine the efficacy, pharmacokinetics, immunogenicity and safety of wilate as routine prophylaxis in up to 12 paediatric patients (eight evaluable) with severe von Willebrand Disease VWD (defined as screening von Willebrand factor ristocetin cofactor activity [VWF:RCo] <20%) under the age of 6 years, over a period of 12 months.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Octapharma

Criteria

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 (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