RecruitingPhase 3

A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)

United States, France, Ireland24 participantsStarted 2024-11-06

Plain-language summary

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.

Who can participate

Age range

17 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

. 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.
. The participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies, or animal allergies.
. The participant has a medical history of a thromboembolic event.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Annualized Bleeding Rate (ABR) for Spontaneous or Traumatic Bleeding Episodes as Assessed by Investigator During Prophylactic Treatment With Vonicog Alfa (rVWF)

Timeframe: 12 months

Trial details

NCT IDNCT05582993

SponsorTakeda

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2030-04-11

Contact for this trial

Takeda Contact

+1-877-825-3327 medinfoUS@takeda.com

View on ClinicalTrials.gov More Von Willebrand Disease (VWD) trials