Von Willebrand Disease (VWD)
Conditions
Interventions
Cohort 1: Participants With Age >=12 to =) 12 to less than (=6 to =6 to <12 years who have received OD therapy of VWF product or prophylactic treatment with a pdVWF product will receive vonicog alfa (
Sponsors
Koumura Emiko
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.The participant has a documented diagnosis of severe VWD (baseline von Willebrand factor ristocetin cofactor activity [VWF:RCo] =2 years of age) prior to screening, has experienced at least 1 VWF-treated bleeding event during (excluding menorrhagia/heavy menstrual bleeding [HMB], as applicable) in the last 12 months, and prophylactic treatment is recommended by the investigator (Prior OD participants); or - 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 rVWF is recommended by the investigator (Switch participants). - 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 =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: https://www.cdc.gov/growthcharts/clinical_charts.htm). 6.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 measures for the duration of their participation in the study. 7.The participant has voluntarily provided assent (if appropriate) and the legally authorized representative(s) has provided informed consent. 8.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 investigator and the sponsor to ensure no eminent risk is present that could challenge the participant's compliance with the study requirements.
Exclusion criteria
Exclusion criteria: 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 =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, part
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1.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 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. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1.Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) Time Frame: 12 months 2.Number of Participants With TEAEs by Severity Time Frame: 12 months 3.Number of Participants With TEAEs and SAEs by Causality Time Frame: 12 months 4.Number of Participants With Thromboembolic Events, Hypersensitivity Reactions and Infusion-Related Reactions (IRR) Time Frame: 12 months 5.Number of Participants Who Develop Neutralizing Antibodies to VWF and Factor VIII (FVIII) Time Frame: 12 months 6.Number of Participants Who Develop Binding Antibodies to VWF and FVIII Time Frame: 12 months 7.Number of Participants With Clinically Significant Change From Baseline Values in Vital Sign Parameters Time Frame: 12 months Number of participants with clinically significant change from baseline values in vital sign parameters per investigator assessment will be reported. 8.Number of Participants With Clinically Significant Change From Baseline Values in Laboratory Parameters Time Frame: 12 months Number of participants with clinically significant change from baseline values in laboratory parameters per investigator assessment will be reported. 9.Number of Participants With Categorized ABR Time Frame: 12 months 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. 10.Number of Participants Previously Receiving On-demand Treatment who will Achieve ABR Percent Reduction Success Time Frame: 12 months 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. 11.Number of pdVWF Switch Participants With Spontaneous ABR Preservation Success Time Frame: 12 mont | — |
Countries
France, Ireland, Italy, Japan, United States
Contacts
Public ContactContact for Clinical Trial Information
1-1, Doshomachi 4-chome, Chuo-ku, Osaka
Outcome results
None listed