An Observational Study of Vonicog Alfa (rVWF) in Pediatric Participants With Von Willebrand Disease (vWD)

Vonvendi Intravenous Specified Drug Use-results Survey "Pediatric Administration"

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.

Study Overview

• Status: Recruiting
• Conditions: Von Willebrand Disease (vWD)
• Intervention / Treatment: Drug: vonicog alfa (rVWF)

• Study Type: Observational
• Enrollment (Estimated): 13

Contacts and Locations

• Study Contact: Name: Takeda Contact; Phone Number: +1-877-825-3327; Email: medinfoUS@takeda.com

Study Locations

• Japan
  • Tokyo
    • Tokyo, Tokyo, Japan
      • Recruiting
      • Takeda Selected Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

The population of this study are all participants who meet the inclusion/exclusion criteria.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
vonicog alfa (rVWF) Group; Participants with Von Willebrand Disease (vWD) who received vonicog alfa (rVWF) in accordance with package insert.	Drug: vonicog alfa (rVWF); rVWF administered by intravenous injection.; Other Names: TAK-577; Recombinant von Willebrand Factor (rVWF); Vonvendi Intravenous

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants who Experience at Least One Adverse Drug Reactions (ADRs)	Adverse Event (AE) refers to any undesirable medical occurrence in a patient administered a drug, regardless of causal relationship. This includes any unfavorable or unintended sign (including abnormal laboratory findings), symptom, or disease occurring during administration, regardless of causal relationship. Adverse drug reaction (ADR) refers to AE related to administered drug.	Up to 1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hemostatic Efficacy Assessed by Hemostatic Efficacy Rating Scale	Hemostatic efficacy for bleeding episodes will be assessed by a predefined 4-point rating scale (Excellent, Good, Moderate, None).	1 year
Hemostatic Efficacy during Perioperative Periods Assessed by Hemostatic Efficacy Rating Scale	Hemostatic efficacy during perioperative periods will be assessed by a predefined 4-point rating scale (Excellent, Good, Moderate, None).	1 year
Number of Infusions per Bleeding Episode		1 year
Number of Infusions during Perioperative Periods		1 year

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

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Study record dates

Study Major Dates

• Study Start (Actual): February 19, 2026
• Primary Completion (Estimated): May 30, 2031
• Study Completion (Estimated): May 30, 2031

Study Registration Dates

• First Submitted: February 5, 2026
• First Submitted That Met QC Criteria: February 5, 2026
• First Posted (Actual): February 11, 2026

Study Record Updates

• Last Update Posted (Actual): February 23, 2026
• Last Update Submitted That Met QC Criteria: February 19, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: Von Willebrand Disease (vWD)
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; von Willebrand Diseases
• Other Study ID Numbers: TAK-577-4010; jRCT2031250712 (Registry Identifier: jRCT)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites).

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No