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

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeUp to 17
SponsorTakeda

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

None

Trial population

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

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • vonicog alfa (rVWF)

    Drug

    rVWF administered by intravenous injection.

Treatment groups

13 Participants
are divided into 1 treatment group
Group A: vonicog alfa (rVWF) Group1 intervention

Trial outcomes

Primary outcomes

1

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.

Time frame
Up to 1 year

Secondary outcomes

1

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).

Time frame
1 year
2

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).

Time frame
1 year
3

Number of Infusions per Bleeding Episode

Time frame
1 year
4

Number of Infusions during Perioperative Periods

Time frame
1 year

Other outcomes

Sponsors and contacts

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