RECRUITING

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

Conditions

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

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

Quick Facts

Study Start:2024-11-06

Study Completion:2030-04-11

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05582993

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 17 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
Age 18 years or older Willing and able to provide informed consent Able to understand and follow study procedures Stable medical condition	1. 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). 2. The participant has a history or presence of a VWF inhibitor at screening. 3. The participant has a history or presence of an factor VIII (FVIII) inhibitor with a titer \>=0.6 Bethesda units per milliliter (/mL). 4. The participant has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins. 5. The participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies, or animal allergies. 6. The participant has a medical history of a thromboembolic event. 7. The participant is human immunodeficiency virus (HIV)-positive with an absolute helper T cell (CD4) count \<200 per cubic millimeter or microliter (/mm\^3). 8. 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. 9. The participant has been diagnosed with renal disease, with a serum creatinine level \>=2.5 milligram per deciliter (mg/dL). 10. 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). 11. 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). 12. The participant is pregnant or lactating at the time of enrollment. 13. The participant has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia). 14. 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. 15. The participant has not received OD or prophylactic treatment with a VWF product prior to this study. 16. The participant has a progressive fatal disease and/or life expectancy of less than 15 months. 17. 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. 18. 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. 19. 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.

Inclusion Criteria

Exclusion Criteria

Age 18 years or older
Willing and able to provide informed consent
Able to understand and follow study procedures
Stable medical condition

1. 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).
2. The participant has a history or presence of a VWF inhibitor at screening.
3. The participant has a history or presence of an factor VIII (FVIII) inhibitor with a titer \>=0.6 Bethesda units per milliliter (/mL).
4. The participant has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins.
5. The participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies, or animal allergies.
6. The participant has a medical history of a thromboembolic event.
7. The participant is human immunodeficiency virus (HIV)-positive with an absolute helper T cell (CD4) count \<200 per cubic millimeter or microliter (/mm\^3).
8. 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.
9. The participant has been diagnosed with renal disease, with a serum creatinine level \>=2.5 milligram per deciliter (mg/dL).
10. 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).
11. 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).
12. The participant is pregnant or lactating at the time of enrollment.
13. The participant has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).
14. 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.
15. The participant has not received OD or prophylactic treatment with a VWF product prior to this study.
16. The participant has a progressive fatal disease and/or life expectancy of less than 15 months.
17. 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.
18. 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.
19. 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.

Contacts and Locations

Study Contact

Takeda Contact

CONTACT

+1-877-825-3327

medinfoUS@takeda.com

Principal Investigator

Study Director

STUDY_DIRECTOR

Takeda

Study Locations (Sites)

Childrens Hospital of Michigan

Detroit, Michigan, 48201

United States

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

Minneapolis, Minnesota, 55404

United States

New York - Presbyterian/Weill Cornell Medical Center

New York, New York, 10021

United States

Medical University of South Carolina (MUSC)

Charleston, South Carolina, 29425

United States

Collaborators and Investigators

Sponsor: Takeda

Study Director, STUDY_DIRECTOR, Takeda

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-11-06

Study Completion Date2030-04-11

Study Record Updates

Study Start Date2024-11-06

Study Completion Date2030-04-11

Terms related to this study

Additional Relevant MeSH Terms

Von Willebrand Disease (VWD)