A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)
ConditionVon Willebrand Disease (VWD)
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
AgeUp to 17
SponsorTakeda
About this trial
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
Qualifiers
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.
The participant is <18 years of age at the time of screening.
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
The participant has been receiving prophylactic treatment with pdVWF products for at least 12 months prior to screening (for participants >=2 years of age) and switching to prophylaxis with vonicog alfa (rVWF) is recommended by the investigator (Switch participants).
Disqualifiers
The participant has been diagnosed with pseudo VWD or another hereditary or acquired coagulation disorder other than VWD (example, qualitative and quantitative platelet disorders or elevated prothrombin time/international normalized ratio 1.4).
The participant has a history or presence of a VWF inhibitor at screening.
The participant has a history or presence of an factor VIII (FVIII) inhibitor with a titer >=0.6 Bethesda units per milliliter (/mL).
The participant has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins.
Trial design
Treatments tested in this trial
- Vonicog Alfa
- ADVATE
Treatment groups
24 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov