drugset / Trial / NCT04953884
Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease (VWD) Patients <6 Years of Age
NaSingle-groupOpen-labelTreatment
Summary
The WIL-33 study aimed 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.
Timeline
- Start
- 2021-09-22
- Primary completion
- 2024-12-16
- Completion
- 2024-12-16
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Wilate | Protein / enzyme biologic | 20 iu/kg | — |
| Subject | Wilate | Protein / enzyme biologic | 30 iu/kg | — |
| Subject | Wilate | Protein / enzyme biologic | 40 iu/kg | — |
| Subject | Wilate | Protein / enzyme biologic | 50 iu/kg | — |
| Subject | Wilate | Protein / enzyme biologic | 80 iu/kg | — |