An Observational Study of Vonicog Alfa (rVWF) in Pediatric Participants With Von Willebrand Disease (vWD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: vonicog alfa (rVWF).
- Who it may be relevant to
- Registry conditions: Von Willebrand Disease (vWD). Basic parameters: up to 17 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Japan
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Vonvendi Intravenous Specified Drug Use-results Survey "Pediatric Administration"
Overview
This study is conducted in Japan of vonicog alfa (rVWF) used to treat pediatric participants with Von Willebrand Disease (vWD). The main aim of the study is to evaluate adverse drug reaction and effectiveness of vonicog alfa (rVWF). During the study, pediatric participants with vWD will be administered with rVWF under routine normal practice. The investigators will evaluate adverse events due to rVWF for 1 year from the start of drug administration. The study sponsor will not be involved in how the participants are administered but will be recorded what happens during the study.
Interventions
- Drug vonicog alfa (rVWF)
rVWF administered by intravenous injection.
Primary outcome measures
- Number of Participants who Experience at Least One Adverse Drug Reactions (ADRs) [Time frame: Up to 1 year]
Secondary outcome measures (4)
- Hemostatic Efficacy Assessed by Hemostatic Efficacy Rating Scale [Time frame: 1 year]
- Hemostatic Efficacy during Perioperative Periods Assessed by Hemostatic Efficacy Rating Scale [Time frame: 1 year]
- Number of Infusions per Bleeding Episode [Time frame: 1 year]
- Number of Infusions during Perioperative Periods [Time frame: 1 year]
Eligibility criteria
Inclusion criteria
- Under 18 years old with vWD.
- Participants who are treated with rVWF for the purpose of hemostatic treatment and management during bleeding episodes or perioperative periods.
- Participants who have prescription or administration after the approval date of rVWF for pediatric use in Japan.
Exclusion criteria
\- Patients who are participating in clinical trials of rVWF.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Japan · 1 center
- Takeda selected site — Tokyo
Identifiers
NCT: NCT07404644 · TAK-577-4010 · jRCT2031250712