A Phase 3, Prospective, Open-label, Uncontrolled, Multicenter Study on Efficacy and Safety of Prophylaxis With Vonicog Alfa (rVWF) in Children Diagnosed With Severe Von Willebrand Disease
Takeda
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
- Age range
- Up to 17 years
- Sex
- All
- Healthy volunteers
- No
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:…
Interventions
- BiologicalVonicog Alfa
Vonicog Alfa administered by intravenous injection.
- BiologicalADVATE
ADVATE administered by intravenous injection.
Locations (15)
- University of Alabama at BirminghamBirmingham, Alabama
- Bleeding and Clotting Disorders InstitutePeoria, Illinois
- Riley Hospital for Children Indiana University HealthIndianapolis, Indiana
- University of Iowa Hospitals & Clinics PARENTIowa City, Iowa
- Childrens Hospital of MichiganDetroit, Michigan
- Children's Health Care d/b/a Children's MinnesotaMinneapolis, Minnesota