NCT05582993 A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)
| NCT ID | NCT05582993 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Takeda |
| Condition | Von Willebrand Disease (VWD) |
| Study Type | INTERVENTIONAL |
| Enrollment | 24 participants |
| Start Date | 2024-11-06 |
| Primary Completion | 2030-04-11 |
Trial Parameters
Eligibility Fast-Check
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Brief Summary
The main aim of the study is to evaluate the effectiveness of prophylaxis with vonicog alfa (recombinant von Willebrand factor \[rVWF\]) in children. This study will enroll those participants who have been previously treated with VWF product or with a plasma-derived VWF (pdVWF) product. In this study, participants will be treated with vonicog alfa (rVWF) for 12 months. During the study, participants will visit the study clinic 5 times after treatment initiation.
Eligibility Criteria
1. The participant has a documented diagnosis of severe VWD (baseline von Willebrand factor ristocetin cofactor activity \[VWF:RCo\] \<20 international units per deciliter \[IU/dL\]) with a history of replacement therapy with VWF concentrate required to control bleeding and a diagnosis of VWD type 1, type 2 (2A, 2B, 2M, 2N), or type 3. Diagnosis is confirmed, as applicable, by genetic testing and/or by multimer analysis, which may be documented in participant's history or at screening. 2. The participant is \<18 years of age at the time of screening. 3. Prescreening treatment requirements: 1. The participant has been receiving OD therapy with VWF products for at least 12 months (for participants \>=2 years of age) prior to screening, has experienced at least 1 VWF-treated bleeding event during (excluding menorrhagia/heavy menstrual bleeding \[HMB\], as applicable) in the last 12 months, and prophylactic treatment is recommended by the investigator (Prior OD participants); or 2. The par