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New treatment option for Von Willebrand Disease (VWD)
Official title A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)
ClinicalTrials.gov ID: NCT05582993
What this study is testing
What is Vonicog Alfa?
Vonicog Alfa is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for von willebrand disease (vwd).
Also referred to as TAK-577, Recombinant von Willebrand Factor (rVWF).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 17
You may be able to join if
- The participant has a documented diagnosis of severe VWD (baseline von Willebrand factor ristocetin cofactor activity [VWF:RCo] \<20 international...
- The participant is \<18 years of age at the time of screening.
- Prescreening treatment requirements:
- The participant has been receiving OD therapy with VWF products for at least 12 months (for participants \>=2 years of age) prior to screening, has...
- The participant has been receiving prophylactic treatment with pdVWF products for at least 12 months prior to screening (for participants \>=2 years...
You likely can't join if
- The participant has been diagnosed with pseudo VWD or another hereditary or acquired coagulation disorder other than VWD (example, qualitative and...
- 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...
- The participant has a medical history of a thromboembolic event.
See the full eligibility criteria
Who can join
- The participant has a documented diagnosis of severe VWD (baseline von Willebrand factor ristocetin cofactor activity [VWF:RCo] \<20 international units per deciliter [IU/dL]) with a history of replacement therapy with...
- The participant is \<18 years of age at the time of screening.
- Prescreening treatment requirements:
- The participant has been receiving OD therapy with VWF products for at least 12 months (for participants \>=2 years of age) prior to screening, has experienced at least 1 VWF-treated bleeding event during (excluding...
- The participant has been receiving prophylactic treatment with pdVWF products for at least 12 months prior to screening (for participants \>=2 years of age) and switching to prophylaxis with vonicog alfa (rVWF) is...
- For participants \<2 years of age, the required duration for prior OD therapy with VWF products or for prior prophylactic treatment with pdVWF products is at least 6 months. Prior OD participants \<2 years of age should...
- For participants \>=2 years of age, the participant has available records that reliably evaluate type, frequency, severity, and treatment of BEs for at least 12 months preceding enrollment. For participants \<2 years of...
- If \>=12 years old at the time of screening, the participant has a body mass index (BMI) \>=15 but \ =2 to \ =5th and \ =5th and \<95th percentile based on gender (for clinical charts provided by CDC, refer to...
- Female participants of childbearing potential (that is, had onset of menses/reached puberty) must have a negative blood/urine pregnancy test result at screening and agree to employ highly effective birth control...
- The participant has voluntarily provided assent (if appropriate) and the legally authorized representative(s) has provided informed consent.
- The participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol, which should also be confirmed based on a prescreening evaluation held between the...
What rules you out
- 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...
- 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.
- 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...
- 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...
- 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...
- 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Peoria, Illinois, United States
- Indianapolis, Indiana, United States
- Iowa City, Iowa, United States
- Detroit, Michigan, United States
- East Lansing, Michigan, United States
- Minneapolis, Minnesota, United States
- Las Vegas, Nevada, United States
- New Brunswick, New Jersey, United States
- New York, New York, United States
- Charleston, South Carolina, United States
- Lille, France
- Lyon, France
- Dublin, Ireland
- Naples, Italy
- Turin (Torino), Italy
- Narita, Chiba, Japan
- Kashihara, Nara, Japan
- Beppu, Oita Prefecture, Japan
- Nagasaki, Japan
+ 1 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Peoria, Illinois, United States; Indianapolis, Indiana, United States; Iowa City, Iowa, United States; Detroit, Michigan, United States; East Lansing, Michigan, United States and 15 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.