[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100484050":3},{"organization":4,"armGroups":7,"interventions":23,"overallOfficials":40,"centralContacts":44,"locations":51,"responsibleParty":383,"collaborators":49,"id":385,"slug":386,"hasResults":387,"nctId":388,"briefTitle":389,"officialTitle":390,"acronym":49,"eligibilityCriteria":391,"healthyVolunteers":387,"sex":392,"minAge":49,"maxAge":393,"enrollmentInfo":394,"targetDuration":49,"studyType":397,"phases":398,"briefSummary":400,"conditions":401,"keywords":49,"overallStatus":54,"whyStopped":49,"lastUpdateSubmitDate":403,"lastUpdatePostDateStruct":404,"startDateStruct":407,"completionDateStruct":409,"leadSponsor":411,"locationsCount":412},{"fullName":5,"class":6},"Takeda","INDUSTRY",[8,15,19],{"label":9,"type":10,"description":11,"interventionNames":12},"Cohort 1: Participants With Age >=12 to \u003C18 years","EXPERIMENTAL","Participants with age greater than or equal to (\\>=) 12 to less than (\\\u003C) 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\u002Fkg) 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).",[13,14],"Biological: Vonicog Alfa","Biological: ADVATE",{"label":16,"type":10,"description":17,"interventionNames":18},"Cohort 2: Participants With Age >=6 to \u003C12 years","Participants with age \\>=6 to \\\u003C12 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\u002Fkg 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).",[13,14],{"label":20,"type":10,"description":21,"interventionNames":22},"Cohort 3: Participants With Age \u003C6 years","Participants with age \\\u003C6 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\u002Fkg 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).",[13,14],[24,33],{"type":25,"name":26,"description":27,"armGroupLabels":28,"otherNames":29},"BIOLOGICAL","Vonicog Alfa","Vonicog Alfa administered by intravenous injection.",[9,16,20],[30,31,32],"TAK-577","Recombinant von Willebrand Factor (rVWF)","Vonvendi-Veyvondi",{"type":25,"name":34,"description":35,"armGroupLabels":36,"otherNames":37},"ADVATE","ADVATE administered by intravenous injection.",[9,16,20],[38,39],"Recombinant Factor VIII (rFVIII)","Octocog Alfa",[41],{"name":42,"affiliation":5,"role":43},"Study Director","STUDY_DIRECTOR",[45],{"name":46,"role":47,"phone":48,"phoneExt":49,"email":50},"Takeda Contact","CONTACT","+1-877-825-3327",null,"medinfoUS@takeda.com",[52,74,90,106,122,138,153,169,185,201,216,232,249,264,281,293,302,320,336,352,367],{"facility":53,"status":54,"city":55,"state":56,"zip":57,"country":58,"countryCode":59,"cosmosGeoPoint":60,"geoPoint":65,"contacts":66},"University of Alabama at Birmingham","RECRUITING","Birmingham","Alabama","35223","United States","US",{"type":61,"coordinates":62},"Point",[63,64],-86.80249,33.52066,{"lat":64,"lon":63},[67,71],{"name":68,"role":47,"phone":69,"phoneExt":49,"email":70},"Site Contact","205-638-9285","cbstolz@uabmc.edu",{"name":72,"role":73,"phone":49,"phoneExt":49,"email":49},"Christina Bemrich-Stolz","PRINCIPAL_INVESTIGATOR",{"facility":75,"status":54,"city":76,"state":77,"zip":78,"country":58,"countryCode":59,"cosmosGeoPoint":79,"geoPoint":83,"contacts":84},"Bleeding and Clotting Disorders Institute","Peoria","Illinois","61614",{"type":61,"coordinates":80},[81,82],-89.58899,40.69365,{"lat":82,"lon":81},[85,88],{"name":68,"role":47,"phone":86,"phoneExt":49,"email":87},"309-692-5337","jroberts@ilbcdi.org",{"name":89,"role":73,"phone":49,"phoneExt":49,"email":49},"Jonathan Roberts",{"facility":91,"status":54,"city":92,"state":93,"zip":94,"country":58,"countryCode":59,"cosmosGeoPoint":95,"geoPoint":99,"contacts":100},"Riley Hospital for Children Indiana University Health","Indianapolis","Indiana","46202",{"type":61,"coordinates":96},[97,98],-86.15804,39.76838,{"lat":98,"lon":97},[101,104],{"name":68,"role":47,"phone":102,"phoneExt":49,"email":103},"317-944-2143","kmfitzpa@iu.edu",{"name":105,"role":73,"phone":49,"phoneExt":49,"email":49},"Kerry Hege",{"facility":107,"status":54,"city":108,"state":109,"zip":110,"country":58,"countryCode":59,"cosmosGeoPoint":111,"geoPoint":115,"contacts":116},"University of Iowa Hospitals & Clinics PARENT","Iowa City","Iowa","52242",{"type":61,"coordinates":112},[113,114],-91.53017,41.66113,{"lat":114,"lon":113},[117,120],{"name":68,"role":47,"phone":118,"phoneExt":49,"email":119},"(319) 335-6688","Janice-staber@uiowa.edu",{"name":121,"role":73,"phone":49,"phoneExt":49,"email":49},"Janice Staber",{"facility":123,"status":54,"city":124,"state":125,"zip":126,"country":58,"countryCode":59,"cosmosGeoPoint":127,"geoPoint":131,"contacts":132},"Childrens Hospital of Michigan","Detroit","Michigan","48201",{"type":61,"coordinates":128},[129,130],-83.04575,42.33143,{"lat":130,"lon":129},[133,136],{"name":68,"role":47,"phone":134,"phoneExt":49,"email":135},"313-745-5515","Rajpu1ma@cmich.edu",{"name":137,"role":73,"phone":49,"phoneExt":49,"email":49},"Madhvi Rajpurkar",{"facility":139,"status":54,"city":140,"state":125,"zip":141,"country":58,"countryCode":59,"cosmosGeoPoint":142,"geoPoint":146,"contacts":147},"Michigan State University Center for Bleeding Disorders & Clotting Disorders","East Lansing","48824",{"type":61,"coordinates":143},[144,145],-84.48387,42.73698,{"lat":145,"lon":144},[148,151],{"name":68,"role":47,"phone":149,"phoneExt":49,"email":150},"517-353-9385","soodaari@msu.edu",{"name":152,"role":73,"phone":49,"phoneExt":49,"email":49},"David Cervi",{"facility":154,"status":54,"city":155,"state":156,"zip":157,"country":58,"countryCode":59,"cosmosGeoPoint":158,"geoPoint":162,"contacts":163},"Children's Health Care d\u002Fb\u002Fa Children's Minnesota","Minneapolis","Minnesota","55404",{"type":61,"coordinates":159},[160,161],-93.26384,44.97997,{"lat":161,"lon":160},[164,167],{"name":68,"role":47,"phone":165,"phoneExt":49,"email":166},"612-813-5940","Susan.kuldanek@childrensmn.org",{"name":168,"role":73,"phone":49,"phoneExt":49,"email":49},"Susan Kuldanek",{"facility":170,"status":54,"city":171,"state":172,"zip":173,"country":58,"countryCode":59,"cosmosGeoPoint":174,"geoPoint":178,"contacts":179},"Cure 4 the Kids","Las Vegas","Nevada","89135",{"type":61,"coordinates":175},[176,177],-115.13722,36.17497,{"lat":177,"lon":176},[180,183],{"name":68,"role":47,"phone":181,"phoneExt":49,"email":182},"702-732-1493","afoord@cure4thekids.org",{"name":184,"role":73,"phone":49,"phoneExt":49,"email":49},"Aimee Foord",{"facility":186,"status":54,"city":187,"state":188,"zip":189,"country":58,"countryCode":59,"cosmosGeoPoint":190,"geoPoint":194,"contacts":195},"Rutgers - Robert Wood Johnson Medical School","New Brunswick","New Jersey","08901",{"type":61,"coordinates":191},[192,193],-74.45182,40.48622,{"lat":193,"lon":192},[196,199],{"name":68,"role":47,"phone":197,"phoneExt":49,"email":198},"732-235-8864","murphysu@cinj.rutgers.edu",{"name":200,"role":73,"phone":49,"phoneExt":49,"email":49},"Susan Murphy",{"facility":202,"status":54,"city":203,"state":203,"zip":204,"country":58,"countryCode":59,"cosmosGeoPoint":205,"geoPoint":209,"contacts":210},"New York - Presbyterian\u002FWeill Cornell Medical Center","New York","10021",{"type":61,"coordinates":206},[207,208],-74.00597,40.71427,{"lat":208,"lon":207},[211,214],{"name":68,"role":47,"phone":212,"phoneExt":49,"email":213},"(212) 746-3978","cam9061@med.cornell.edu",{"name":215,"role":73,"phone":49,"phoneExt":49,"email":49},"McGuinn Catherine",{"facility":217,"status":54,"city":218,"state":219,"zip":220,"country":58,"countryCode":59,"cosmosGeoPoint":221,"geoPoint":225,"contacts":226},"Medical University of South Carolina (MUSC)","Charleston","South Carolina","29425",{"type":61,"coordinates":222},[223,224],-79.93275,32.77632,{"lat":224,"lon":223},[227,230],{"name":68,"role":47,"phone":228,"phoneExt":49,"email":229},"843-792-2957","bergmans@musc.edu",{"name":231,"role":73,"phone":49,"phoneExt":49,"email":49},"Shayla Bergmann",{"facility":233,"status":54,"city":234,"state":49,"zip":235,"country":236,"countryCode":237,"cosmosGeoPoint":238,"geoPoint":242,"contacts":243},"Hemostase Clinique - Institut Cœur-Poumons (4eme étage aile est) Bureau 419","Lille","59037","France","FR",{"type":61,"coordinates":239},[240,241],3.05512,50.63391,{"lat":241,"lon":240},[244,247],{"name":68,"role":47,"phone":245,"phoneExt":49,"email":246},"+33 3 20 44 48 45","sophie.susen@chu-lille.fr",{"name":248,"role":73,"phone":49,"phoneExt":49,"email":49},"Sophie Susen",{"facility":250,"status":54,"city":251,"state":49,"zip":252,"country":236,"countryCode":237,"cosmosGeoPoint":253,"geoPoint":257,"contacts":258},"Hopital Edouard Herriot - CHU Lyon","Lyon","69677",{"type":61,"coordinates":254},[255,256],4.84789,45.74906,{"lat":256,"lon":255},[259,262],{"name":68,"role":47,"phone":260,"phoneExt":49,"email":261},"04 72 11 88 10","sandrine.meunier@chu-lyon.fr",{"name":263,"role":73,"phone":49,"phoneExt":49,"email":49},"Sandrine Meunier",{"facility":265,"status":54,"city":266,"state":49,"zip":267,"country":268,"countryCode":269,"cosmosGeoPoint":270,"geoPoint":274,"contacts":275},"Children's Health Ireland","Dublin","D12N512","Ireland","IE",{"type":61,"coordinates":271},[272,273],-6.24889,53.33306,{"lat":273,"lon":272},[276,279],{"name":68,"role":47,"phone":277,"phoneExt":49,"email":278},"(353) 01 409 6913","beatrice.nolan@childrenshealthireland.ie",{"name":280,"role":73,"phone":49,"phoneExt":49,"email":49},"Beatrice Nolan",{"facility":282,"status":283,"city":284,"state":49,"zip":285,"country":286,"countryCode":287,"cosmosGeoPoint":288,"geoPoint":292,"contacts":49},"Azienda Ospedaliera Pediatrica Santobono Pausillipon","ACTIVE_NOT_RECRUITING","Naples","80123","Italy","IT",{"type":61,"coordinates":289},[290,291],14.26811,40.85216,{"lat":291,"lon":290},{"facility":294,"status":283,"city":295,"state":49,"zip":296,"country":286,"countryCode":287,"cosmosGeoPoint":297,"geoPoint":301,"contacts":49},"Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino","Turin (Torino)","10126",{"type":61,"coordinates":298},[299,300],7.68682,45.07049,{"lat":300,"lon":299},{"facility":303,"status":54,"city":304,"state":305,"zip":306,"country":307,"countryCode":308,"cosmosGeoPoint":309,"geoPoint":313,"contacts":314},"Japanese Red Cross Narita Hospital","Narita","Chiba","286-8523","Japan","JP",{"type":61,"coordinates":310},[311,312],140.31667,35.78333,{"lat":312,"lon":311},[315,318],{"name":68,"role":47,"phone":316,"phoneExt":49,"email":317},"+81-476-22-2311","nogunari@naritasekijyuji.jp",{"name":319,"role":73,"phone":49,"phoneExt":49,"email":49},"Yasushi Noguchi",{"facility":321,"status":54,"city":322,"state":323,"zip":324,"country":307,"countryCode":308,"cosmosGeoPoint":325,"geoPoint":329,"contacts":330},"Nara Medical University Hospital","Kashihara","Nara","634-8522",{"type":61,"coordinates":326},[327,328],135.78333,33.95,{"lat":328,"lon":327},[331,334],{"name":68,"role":47,"phone":332,"phoneExt":49,"email":333},"81-744-22-3051","roc-noga@naramed-u.ac.jp",{"name":335,"role":73,"phone":49,"phoneExt":49,"email":49},"Keiji Nogami",{"facility":337,"status":54,"city":338,"state":339,"zip":340,"country":307,"countryCode":308,"cosmosGeoPoint":341,"geoPoint":345,"contacts":346},"NHO Beppu Medical Center","Beppu","Oita Prefecture","874-0011",{"type":61,"coordinates":342},[343,344],131.49751,33.27945,{"lat":344,"lon":343},[347,350],{"name":68,"role":47,"phone":348,"phoneExt":49,"email":349},"+81(977)67-1111","koga.hiroshi.ab@mail.hosp.go.jp",{"name":351,"role":73,"phone":49,"phoneExt":49,"email":49},"Hiroshi Koga",{"facility":353,"status":54,"city":354,"state":49,"zip":355,"country":307,"countryCode":308,"cosmosGeoPoint":356,"geoPoint":360,"contacts":361},"Nagasaki University Hospital","Nagasaki","852-8501",{"type":61,"coordinates":357},[358,359],129.88333,32.75,{"lat":359,"lon":358},[362,365],{"name":68,"role":47,"phone":363,"phoneExt":49,"email":364},"+81958197200","funakoshi@nagasaki-u.ac.jp",{"name":366,"role":73,"phone":49,"phoneExt":49,"email":49},"Yasutomo Funakoshi",{"facility":368,"status":54,"city":369,"state":49,"zip":370,"country":371,"countryCode":372,"cosmosGeoPoint":373,"geoPoint":377,"contacts":378},"Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital for Children NHS Foundation Trust","London","WC1N 3HR","United Kingdom","UK",{"type":61,"coordinates":374},[375,376],-0.12574,51.50853,{"lat":376,"lon":375},[379,381],{"name":68,"role":47,"phone":49,"phoneExt":49,"email":380},"Mary.mathias@gosh.nhs.uk",{"name":382,"role":73,"phone":49,"phoneExt":49,"email":49},"Mary Mathias",{"type":384,"investigatorFullName":49,"investigatorTitle":49,"investigatorAffiliation":49,"oldNameTitle":49,"oldOrganization":49},"SPONSOR","100484050","phase-3-a-study-of-vonicog-alfa-rvwf-in-children-with-severe-von-willebrand-disease-vwd-100484050",false,"NCT05582993","A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)","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","1. The participant has a documented diagnosis of severe VWD (baseline von Willebrand factor ristocetin cofactor activity \\[VWF:RCo\\] \\\u003C20 international units per deciliter \\[IU\u002FdL\\]) with a history of replacement therapy with VWF concentrate required to control bleeding and a diagnosis of VWD type 1, type 2 (2A, 2B, 2M, 2N), or type 3. Diagnosis is confirmed, as applicable, by genetic testing and\u002For by multimer analysis, which may be documented in participant's history or at screening.\n2. The participant is \\\u003C18 years of age at the time of screening.\n3. Prescreening treatment requirements:\n\n   1. 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 menorrhagia\u002Fheavy menstrual bleeding \\[HMB\\], as applicable) in the last 12 months, and prophylactic treatment is recommended by the investigator (Prior OD participants); or\n   2. 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 recommended by the investigator (Switch participants).\n   3. For participants \\\u003C2 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 \\\u003C2 years of age should have experienced at least 1 VWF-treated bleeding event during the last 6 months based on medical records and be recommended to receive prophylactic treatment by the investigator.\n4. 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 \\\u003C2 years of age, the participant has available records that reliably evaluate type, frequency, severity and treatment of BEs for at least 6 months preceding enrollment.\n5. If \\>=12 years old at the time of screening, the participant has a body mass index (BMI) \\>=15 but \\\u003C40 kilogram per square meter (kg\u002Fm\\^2). If \\>=2 to \\\u003C12 years old at the time of screening, the participant has a BMI of \\>=5th and \\\u003C95th percentile (per Centers for Disease Control and Prevention \\[CDC\\] clinical charts). For younger participants who are \\\u003C2 years old, the \"weight-for-age\" clinical charts (5th to 95th percentile) provided by the CDC should be utilized to ensure the participant has a body weight of \\>=5th and \\\u003C95th percentile based on gender (for clinical charts provided by CDC, refer to: https:\u002F\u002Fwww.cdc.gov\u002Fgrowthcharts\u002Fclinical\\_charts.htm).\n6. Female participants of childbearing potential (that is, had onset of menses\u002Freached puberty) must have a negative blood\u002Furine pregnancy test result at screening and agree to employ highly effective birth control measures for the duration of their participation in the study.\n7. The participant has voluntarily provided assent (if appropriate) and the legally authorized representative(s) has provided informed consent.\n8. The participant and\u002For 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.\n\nExclusion Criteria:\n\n1. 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\u002Finternational normalized ratio 1.4).\n2. The participant has a history or presence of a VWF inhibitor at screening.\n3. The participant has a history or presence of an factor VIII (FVIII) inhibitor with a titer \\>=0.6 Bethesda units per milliliter (\u002FmL).\n4. The participant has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins.\n5. The participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis\u002Fconjunctivitis, mild asthma, food allergies, or animal allergies.\n6. The participant has a medical history of a thromboembolic event.\n7. The participant is human immunodeficiency virus (HIV)-positive with an absolute helper T cell (CD4) count \\\u003C200 per cubic millimeter or microliter (\u002Fmm\\^3).\n8. 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.\n9. The participant has been diagnosed with renal disease, with a serum creatinine level \\>=2.5 milligram per deciliter (mg\u002FdL).\n10. The participant has a platelet count \\\u003C100,000\u002FmL 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).\n11. 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).\n12. The participant is pregnant or lactating at the time of enrollment.\n13. The participant has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).\n14. 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.\n15. The participant has not received OD or prophylactic treatment with a VWF product prior to this study.\n16. The participant has a progressive fatal disease and\u002For life expectancy of less than 15 months.\n17. The participant is unable to complete screening procedures and\u002For 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.\n18. The participant has a mental condition rendering him\u002Fher unable to understand the nature, scope, and possible consequences of the study and\u002For evidence of an uncooperative attitude.\n19. 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\u002Fspouse, siblings, and parents) as well as employees.","ALL","17 Years",{"count":395,"type":396},24,"ESTIMATED","INTERVENTIONAL",[399],"PHASE3","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.\n\nDuring the study, participants will visit the study clinic 5 times after treatment initiation.",[402],"Von Willebrand Disease (VWD)","2026-05-22",{"date":405,"type":406},"2026-05-27","ACTUAL",{"date":408,"type":406},"2024-11-06",{"date":410,"type":396},"2030-04-11",{"name":5,"class":6},21]