Indiana Hemophilia and Thrombosis Center | Indianapolis, IN
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About
The main aim of the study is to check effectiveness, side effects, and tolerability of 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 rVWF 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.
Enrollment
Sex
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Inclusion criteria
Diagnosis of severe von Willebrand disease (VWD) (defined as von Willebrand factor: ristocetin cofactor [VWF:RCo] less than [<] 20 percent [%]):
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, which should also be confirmed based on a pre-screening evaluation held between the Investigator and the Sponsor, to ensure no eminent risk is present that could challenge the participants compliance with the study requirements.
Additional inclusion criteria for both previously treated participants and participants undergoing surgery are as follows:
Additional inclusion criterion for previously untreated participants are as follows:
Exclusion criteria
Primary purpose
Allocation
Interventional model
Masking
34 participants in 3 patient groups
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Takeda Contact
Data sourced from clinicaltrials.gov
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