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Von Willebrand Disease (VWD)Phase 3NCT05582993

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

A Phase 3 study testing Vonicog Alfa and ADVATE in people with Von Willebrand Disease (VWD), run by Takeda at 23 sites in 6 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages Up to 17 years
Conditions the study is for
Von Willebrand Disease (VWD)
What is being tested?
Vonicog Alfa and ADVATE
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
23 sitesin 6 countries
Study dates
6 Nov 2024to 11 Apr 2030
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

None listed.

Who cannot join

  • 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.
Show all 19Show fewer
  • The participant is human immunodeficiency virus (HIV)-positive with an absolute helper T cell (CD4) count <200 per cubic millimeter or microliter (/mm^3).
  • The participant has been diagnosed with significant liver disease per the investigator's medical assessment of the participant's current condition or medical history or as evidenced by, but not limited to, any of the following: serum alanine aminotransferase (ALT) greater than 5 times the upper limit of normal (ULN), hypoalbuminemia, portal vein hypertension (example, presence of otherwise unexplained splenomegaly, history of esophageal varices), or liver cirrhosis classified as Child-Pugh class B or C.
  • The participant has been diagnosed with renal disease, with a serum creatinine level >=2.5 milligram per deciliter (mg/dL).
  • The participant has a platelet count <100,000/mL at screening (because participants with type 2B VWD are considered eligible for this study, for participants with type 2B VWD, platelet count[s] at screening will be evaluated in consultation with the sponsor, taking into consideration historical trends in platelet counts and the investigator's medical assessment of the participants condition).
  • The participant has been treated with an immunomodulatory drug, excluding topical treatment (example, ointments, nasal sprays), within 30 days prior to signing the informed consent (or assent, if appropriate).
  • The participant is pregnant or lactating at the time of enrollment.
  • The participant has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).
  • The participant has participated in another clinical study involving another IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
  • The participant has not received OD or prophylactic treatment with a VWF product prior to this study.
  • The participant has a progressive fatal disease and/or life expectancy of less than 15 months.
  • The participant is unable to complete screening procedures and/or comply with the requirements of the protocol in the opinion of the investigator, based on the joint prescreening evaluation held between the investigator and the sponsor.
  • The participant has a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study and/or evidence of an uncooperative attitude.
  • The participant is member of the study team or in a dependent relationship with one of the study team members, which includes close relatives (that is, children, partner/spouse, siblings, and parents) as well as employees.

Trial locations and contacts

23 sites in 6 countries. Contact the study team or a site near you to ask about taking part.

Takeda Contact contact

+1-877-825-3327

medinfoUS@takeda.com

23 sites in 6 countries

United States12 sites, 12 recruiting

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35223

    Site Contact, 205-638-9285, cbstolz@uabmc.edu

    Christina Bemrich-Stolz

    Recruiting

  • Medical University of South Carolina (MUSC)

    Charleston, South Carolina, 29425

    Site Contact, 843-792-2957, bergmans@musc.edu

    Shayla Bergmann

    Recruiting

  • Childrens Hospital of Michigan

    Detroit, Michigan, 48201

    Site Contact, 313-745-5515, Rajpu1ma@cmich.edu

    Madhvi Rajpurkar

    Recruiting

  • Michigan State University Center for Bleeding Disorders & Clotting Disorders

    East Lansing, Michigan, 48824

    Site Contact, 517-353-9385, soodaari@msu.edu

    David Cervi

    Not yet recruiting

  • Riley Hospital for Children Indiana University Health

    Indianapolis, Indiana, 46202

    Site Contact, 317-944-2143, kmfitzpa@iu.edu

    Kerry Hege

    Recruiting

  • University of Iowa Hospitals & Clinics PARENT

    Iowa City, Iowa, 52242

    Site Contact, (319) 335-6688, Janice-staber@uiowa.edu

    Janice Staber

    Recruiting

  • Cure 4 the Kids

    Las Vegas, Nevada, 89135

    Site Contact, 702-732-1493, afoord@cure4thekids.org

    Aimee Foord

    Recruiting

  • Children's Health Care d/b/a Children's Minnesota

    Minneapolis, Minnesota, 55404

    Site Contact, 612-813-5940, Susan.kuldanek@childrensmn.org

    Susan Kuldanek

    Recruiting

  • Rutgers - Robert Wood Johnson Medical School

    New Brunswick, New Jersey, 08901

    Site Contact, 732-235-8864, murphysu@cinj.rutgers.edu

    Susan Murphy

    Recruiting

  • New York - Presbyterian/Weill Cornell Medical Center

    New York, New York, 10021

    Site Contact, (212) 746-3978, cam9061@med.cornell.edu

    McGuinn Catherine

    Recruiting

  • Arnold Palmer Childrens Hosptial

    Orlando, Florida, 32806

    Site Contact, +1 (321) 841-2748, amina.rafique@orlandohealth.com

    Amina Rafique

    Not yet recruiting

  • Bleeding and Clotting Disorders Institute

    Peoria, Illinois, 61614

    Site Contact, 309-692-5337, jroberts@ilbcdi.org

    Jonathan Roberts

    Recruiting

Other trials in United States

Japan4 sites, 4 recruiting

  • NHO Beppu Medical Center

    Beppu, Oita Prefecture, 874-0011

    Site Contact, +81(977)67-1111, koga.hiroshi.ab@mail.hosp.go.jp

    Hiroshi Koga

    Recruiting

  • Nara Medical University Hospital

    Kashihara, Nara, 634-8522

    Site Contact, 81-744-22-3051, roc-noga@naramed-u.ac.jp

    Keiji Nogami

    Recruiting

  • Nagasaki University Hospital

    Nagasaki, 852-8501

    Site Contact, +81958197200, funakoshi@nagasaki-u.ac.jp

    Yasutomo Funakoshi

    Recruiting

  • Japanese Red Cross Narita Hospital

    Narita, Chiba, 286-8523

    Site Contact, +81-476-22-2311, nogunari@naritasekijyuji.jp

    Yasushi Noguchi

    Recruiting

Other trials in Japan

France2 sites, 2 recruiting

  • Hemostase Clinique - Institut Cœur-Poumons (4eme étage aile est) Bureau 419

    Lille, 59037

    Site Contact, +33 3 20 44 48 45, sophie.susen@chu-lille.fr

    Sophie Susen

    Recruiting

  • Hopital Edouard Herriot - CHU Lyon

    Lyon, 69677

    Site Contact, 04 72 11 88 10, sandrine.meunier@chu-lyon.fr

    Sandrine Meunier

    Recruiting

Other trials in France

Italy2 sites

  • Azienda Ospedaliera Pediatrica Santobono Pausillipon

    Naples, 80123

    Completed

  • Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino

    Turin (Torino), 10126

    Completed

Other trials in Italy

United Kingdom2 sites, 2 recruiting

  • Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital for Children NHS Foundation Trust

    London, WC1N 3HR

    Site Contact, Mary.mathias@gosh.nhs.uk

    Mary Mathias

    Recruiting

  • Oxford University Hospitals NHS Foundation Trust

    Oxford, OX3 9DU

    Site Contact, +44 1865 226-652, Saket.Badle@ouh.nhs.uk

    Saket Badle

    Recruiting

Other trials in United Kingdom

Ireland1 site, 1 recruiting

  • Children's Health Ireland

    Dublin, D12N512

    Site Contact, (353) 01 409 6913, beatrice.nolan@childrenshealthireland.ie

    Beatrice Nolan

    Recruiting

Other trials in Ireland

About this study

Official title: A Phase 3, Prospective, Open-label, Uncontrolled, Multicenter Study on Efficacy and Safety of Prophylaxis With Vonicog Alfa (rVWF) in Children Diagnosed With Severe Von Willebrand Disease

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.

Being tested
Vonicog Alfa, ADVATE
Medical subject headings
von Willebrand Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Vonicog AlfaBiological

    Also called TAK-577, Recombinant von Willebrand Factor (rVWF), Vonvendi-Veyvondi

    Vonicog Alfa administered by intravenous injection.

  • ADVATEBiological

    Also called Recombinant Factor VIII (rFVIII), Octocog Alfa

    ADVATE administered by intravenous injection.

Study groups in NCT05582993
Study groupRoleWhat they receive
Cohort 1: Participants With Age >=12 to <18 yearsExperimentalParticipants with age greater than or equal to (>=) 12 to less than (<) 18 years who have received on-demand (OD) therapy or prophylactic treatment with a pdVWF product will receive vonicog alfa (rVWF) with an initial dose selected within the range of 40 to 60 international units per kilogram (IU/kg) vonicog alfa (rVWF), intravenous infusions, twice-weekly for 12 months. Participants may receive vonicog alfa (rVWF) with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management).
Receives: Biological: Vonicog Alfa; Biological: ADVATE
Cohort 2: Participants With Age >=6 to <12 yearsExperimentalParticipants with age >=6 to <12 years who have received OD therapy of VWF product or prophylactic treatment with a pdVWF product will receive vonicog alfa (rVWF) with an initial dose selected within the range of 40 to 60 IU/kg vonicog alfa (rVWF), intravenous infusions, twice-weekly for 12 months. Participants may receive vonicog alfa (rVWF) with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management).
Receives: Biological: Vonicog Alfa; Biological: ADVATE
Cohort 3: Participants With Age <6 yearsExperimentalParticipants with age <6 years who have received OD therapy of VWF product or prophylactic treatment with a pdVWF product will receive vonicog alfa (rVWF) with an initial dose selected within the range of 40 to 60 IU/kg vonicog alfa (rVWF), intravenous infusions, twice-weekly for 12 months. Participants may receive vonicog alfa (rVWF) with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management).
Receives: Biological: Vonicog Alfa; Biological: ADVATE

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    ABR during the study compared to historical ABR for each participant for both spontaneous and traumatic bleeding episodes as classified by the investigator during prophylactic treatment with vonicog alfa (rVWF) will be reported.

    Measured: 12 months

Secondary outcomes (38)

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Number of participants with TEAEs and SAEs will be reported.

    Measured: 12 months

  2. Number of Participants With TEAEs by Severity

    Number of participants with severity of TEAE will be reported.

    Measured: 12 months

  3. Number of Participants With TEAEs and SAEs by Causality

    Number of participants with causality related TEAEs and SAEs will be reported.

    Measured: 12 months

  4. Number of Participants With Thromboembolic Events, Hypersensitivity Reactions and Infusion-Related Reactions (IRR)

    Number of participants with thromboembolic events, hypersensitivity reactions and IRR will be reported.

    Measured: 12 months

  5. Number of Participants Who Develop Neutralizing Antibodies to VWF and Factor VIII (FVIII)

    Number of participants who develop neutralizing antibodies to VWF and FVIII will be reported.

    Measured: 12 months

  6. Number of Participants Who Develop Binding Antibodies to VWF and FVIII

    Number of participants who develop total binding antibodies to VWF and FVIII will be reported.

    Measured: 12 months

  7. Number of Participants With Clinically Significant Change From Baseline Values in Vital Sign Parameters

    Number of participants with clinically significant change from baseline values in vital sign parameters per investigator assessment will be reported.

    Measured: 12 months

  8. Number of Participants With Clinically Significant Change From Baseline Values in Laboratory Parameters

    Number of participants with clinically significant change from baseline values in laboratory parameters per investigator assessment will be reported.

    Measured: 12 months

  9. Number of Participants With Categorized ABR

    ABR categorized as 0, 0-2, 2-5, or >5 bleeding episodes during vonicog alfa (rVWF) prophylaxis. Number of participants with categorized ABR will be reported.

    Measured: 12 months

  10. Number of Participants Previously Receiving On-demand Treatment who will Achieve ABR Percent Reduction Success

    ABR percent reduction success is defined as at least 25% reduction of ABR during vonicog alfa (rVWF) prophylaxis relative to the participant's own historical ABR during OD treatment prior to enrollment in this study. Number of participants previously receiving on-demand treatment who will achieve ABR percent reduction success will be reported.

    Measured: 12 months

  11. Number of pdVWF Switch Participants With Spontaneous ABR Preservation Success

    ABR preservation success is defined as achieving an ABR for spontaneous bleeding episodes during vonicog alfa (rVWF) prophylaxis that is no greater than the participant's own historical ABR during prophylactic treatment with pdVWF prior to enrollment in this study. Number of pdVWF switch participants with spontaneous ABR preservation success will be reported.

    Measured: 12 months

  12. Number of Spontaneous ABR Historically and While on Prophylactic Treatment With Vonicog Alfa (rVWF) by Location of Bleeding

    Number of spontaneous ABR historically and while on prophylactic treatment with vonicog alfa (rVWF) by location of bleeding will be reported.

    Measured: 12 months

  13. ABR for Bleeding Episodes by Bleeding Cause Historically and While on Prophylactic Treatment With Vonicog Alfa (rVWF)

    ABR for bleeding episodes by bleeding cause historically and while on prophylactic treatment with vonicog alfa (rVWF) will be reported.

    Measured: 12 months

  14. Total Number of Infusions Administered Per Week During Prophylactic Treatment With Vonicog Alfa (rVWF)

    Total number of infusions administered per week during prophylactic treatment with vonicog alfa (rVWF) will be reported.

    Measured: 12 months

  15. Average Number of Infusions Per Week During Prophylactic Treatment With Vonicog Alfa (rVWF)

    Average number of infusions per week during prophylactic treatment with vonicog alfa (rVWF) will be reported.

    Measured: 12 months

  16. Total Weight Adjusted Consumption of Vonicog Alfa (rVWF) Per Month During Prophylactic Treatment

    Total weight adjusted consumption of vonicog alfa (rVWF) per month during prophylactic treatment will be reported.

    Measured: 12 months

  17. Overall Hemostatic Efficacy Rating of Breakthrough Bleed Treatment at Resolution of the Bleeding Episode

    Overall hemostatic efficacy rating of breakthrough bleed treatment at resolution of the bleeding episode will be reported.

    Measured: 12 months

  18. Number of Infusions of rVWF and ADVATE (rFVIII, octocog alfa) per Bleeding Episode

    Number of infusions of vonicog alfa (rVWF) and ADVATE per bleeding episode will be reported.

    Measured: 12 months

  19. Weight-adjusted Consumption of Vonicog Alfa (rVWF) and ADVATE per Bleeding Episode

    Weight-adjusted consumption of vonicog alfa (rVWF) and ADVATE per bleeding episode will be reported.

    Measured: 12 months

  20. Plasma Level of Vonicog Alfa (rVWF) based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco)

    Plasma level of vonicog alfa (rVWF) based on VWF:Rco will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  21. Plasma Level of Vonicog Alfa (rVWF) based on Von Willebrand Factor Antigen (VWF:Ag)

    Plasma level of vonicog alfa (rVWF) based on VWF:Ag will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  22. Plasma Level of Vonicog alfa (rVWF) based on Von Willebrand Factor Collagen Binding (VWF:CB)

    Plasma level of vonicog alfa (rVWF) based on VWF:CB will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  23. Plasma Level of Vonicog alfa (rVWF) based on Von Willebrand Factor Glycoprotein 1b Binding (VWF:GP1bM)

    Plasma level of vonicog alfa (rVWF) based on VWF:GP1bM will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  24. Plasma Level of Factor VIII Clotting (FVIII:C)

    Plasma level of FVIII:C will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  25. Incremental Recovery Based on VWF:Rco

    Incremental recovery based on VWF:Rco will be reported.

    Measured: 12 months

  26. Incremental Recovery Based on VWF:Ag

    Incremental recovery based on VWF:Ag will be reported.

    Measured: 12 months

  27. Incremental Recovery Based on VWF:CB

    Incremental recovery based on VWF:CB will be reported.

    Measured: 12 months

  28. Incremental Recovery Based on VWF:GP1bM

    Incremental recovery based on VWF:GP1bM will be reported.

    Measured: 12 months

  29. Ratio of Area Under the Plasma Level Versus Time Curve From Time 0 to End of the Partial Dosing Interval (AUC0- Tau;ss) and Area Under the Level Versus Time Curve From 0 to 96 Hours (AUC0-96; ss) for VWF:Rco

    Ratio of AUC0- Tau;ss/AUC0-96; ss for VWF:Rco will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  30. Terminal Half-life (T1/2) for VWF:Rco

    TT1/2 based on VWF:Rco will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  31. Maximum Plasma Level During the Partial Dosing Interval at Steady State (Cmax;ss) for VWF:Rco

    Cmax;ss for VWF:Rco will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  32. Minimum Time to Reach the Maximum Plasma Level at Steady State (Tmax;ss) for VWF:Rco

    Tmax;ss for VWF:Rco will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  33. Volume of Distribution at Steady State (Vss) for VWF:Rco

    Vss for VWF:Rco will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  34. Clearance (CL) for VWF:Rco

    CL for VWF:Rco will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  35. Maximum Plasma Level During the Partial Dosing Interval at Steady State (Cmax;ss) for FVIII:C

    Cmax;ss for FVIII:C will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  36. Minimum Time to Reach the Maximum Plasma Level at Steady State (Tmax;ss) for FVIII:C

    Tmax;ss for FVIII:C will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  37. Ratio of Area Under the Plasma Level Versus Time Curve From Time 0 to End of the Partial Dosing Interval (AUC0- Tau;ss) and Area Under the Level Versus Time Curve From 0 to 96 Hours (AUC0-96; ss) for FVIII:C

    Ratio of AUC0- Tau;ss and AUC0-96; ss for FVIII:C will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

  38. Area Under the Level Versus Time Curve From 0 to 96 Hours (AUC0-96; ss) for FVIII:C

    AUC0-96; ss for FVIII:C will be reported.

    Measured: Within 30 minutes pre-infusion and at multiple timepoints (up to 96 hours) post-infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Oct 2022
  2. Study start6 Nov 2024
  3. Primary completion11 Apr 2030 (estimated)
  4. Study completion11 Apr 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
24 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05582993
Sponsor's protocol ID
TAK-577-3001
Other IDs
2023-509877-22-00, 2023
Status last verified
Sep 2026
First submitted
14 Oct 2022
Last updated
9 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Vonicog Alfa and ADVATE in people with Von Willebrand Disease (VWD), run by Takeda at 23 sites in 6 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 0 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 23 sites in United States, Japan, France, Italy, United Kingdom and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Takeda Contact (+1-877-825-3327, medinfoUS@takeda.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Annualized Bleeding Rate (ABR) for Spontaneous or Traumatic Bleeding Episodes as Assessed by Investigator During Prophylactic Treatment With Vonicog Alfa (rVWF) (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05582993.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Sep 2026; AzTrials last refreshed its copy on 26 Oct 2026.

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