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Recruiting Phase 3 NCT02932618

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)

Trial Parameters

Condition Von Willebrand Disease
Sponsor Baxalta now part of Shire
Study Type INTERVENTIONAL
Phase Phase 3
Enrollment 31
Sex ALL
Min Age N/A
Max Age 17 Years
Start Date 2017-11-06
Completion 2026-03-31
Interventions
Vonicog alfaAntihemophilic Factor (Recombinant)

Brief Summary

The main aim of the study is to check effectiveness, side effects, and tolerability of vonicog alfa (recombinant von Willebrand factor \[rVWF\]), with or without ADVATE, in the treatment and control of nonsurgical bleeding events in pediatric participants (less than (\<)18 years of age) with severe hereditary von Willebrand disease (VWD). The participants will be treated with vonicog alfa for 12-18 months. Their von Willebrand Disease will be treated by their doctor according to their doctor's usual clinical practice. During the study, participants will be followed up at clinics or over telephone calls.

Eligibility Criteria

Inclusion Criteria: * Diagnosis of severe von Willebrand disease (VWD) (defined as von Willebrand factor: ristocetin cofactor \[VWF:RCo\] less than \[\<\] 20 percent \[%\]): * Type 1 (VWF:RCo \<20 International Units per deciliter \[IU/dL\]); or * Type 2A (VWF:RCo \<20 IU/dL), Type 2B (as diagnosed by genotype), Type 2N (Factor VIII coagulation activity \[FVIII:C\] \<10 % and historically documented genetics), Type 2M; or * Type 3 (VWF:Ag less than or equal to \[=\<\] 3 IU/dL). * Age 0 to \<18 years at the time of Screening. * The participant has provided assent (if appropriate) and legally authorized representative(s) has provided informed consent. * If female of childbearing potential, participant presents with a negative serum pregnancy test. * If applicable, participant agrees to employ adequate birth control measures for the duration of the study. * The participant and/or the legally authorized representative are willing and able to comply with the requirements of the protocol, wh

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