[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100592829":3},{"organization":4,"outcomesModule":7,"designInfo":71,"detailedDescription":11,"studyPopulation":11,"armGroups":78,"interventions":102,"overallOfficials":125,"centralContacts":129,"locations":137,"responsibleParty":431,"collaborators":11,"id":433,"slug":434,"hasResults":435,"nctId":436,"briefTitle":437,"officialTitle":438,"acronym":439,"eligibilityCriteria":440,"healthyVolunteers":435,"sex":441,"minAge":442,"maxAge":11,"enrollmentInfo":443,"targetDuration":11,"studyType":446,"phases":447,"briefSummary":449,"conditions":450,"keywords":11,"overallStatus":140,"whyStopped":11,"lastUpdateSubmitDate":452,"lastUpdatePostDateStruct":453,"startDateStruct":456,"completionDateStruct":458,"leadSponsor":460,"locationsCount":461},{"fullName":5,"class":6},"Hoffmann-La Roche","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":11},[9],{"measure":10,"description":11,"timeFrame":12},"Annualized Bleed Rate (ABR) for Treated Bleeds in the Randomized Arms",null,"From Baseline to at least 24 weeks",[14,16,18,20,22,24,26,28,31,33,35,37,39,41,43,46,49,52,54,56,58,60,63,65,69],{"measure":15,"description":11,"timeFrame":12},"ABR for All Bleeds in the Randomized Arms",{"measure":17,"description":11,"timeFrame":12},"ABR for Treated Spontaneous Bleeds in the Randomized Arms",{"measure":19,"description":11,"timeFrame":12},"ABR for Treated Joint Bleeds in the Randomized Arms",{"measure":21,"description":11,"timeFrame":12},"Intra-Participant Comparison of the ABR for Treated Bleeds with Prophylactic Emicizumab Versus Prophylactic SOC from the Preceeding Non-Interventional Study (NIS) WP45335",{"measure":23,"description":11,"timeFrame":12},"Intra-Participant Comparison of the ABR for All Bleeds with Prophylactic Emicizumab Versus Prophylactic SOC from the Preceeding NIS WP45335",{"measure":25,"description":11,"timeFrame":12},"Intra-Participant Comparison of the ABR for Treated Spontaneous Bleeds with Prophylactic Emicizumab Versus Prophylactic SOC from the Preceeding NIS WP45335",{"measure":27,"description":11,"timeFrame":12},"Intra-Participant Comparison of the ABR for Treated Joint Bleeds with Prophylactic Emicizumab Versus Prophylactic SOC from the Preceeding NIS WP45335",{"measure":29,"description":11,"timeFrame":30},"Incidence and Severity of Adverse Events, with Severity Determined According to the World Health Organization (WHO) Toxicity Grading Scale","From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months)",{"measure":32,"description":11,"timeFrame":30},"Incidence and Severity of Thromboembolic Events",{"measure":34,"description":11,"timeFrame":30},"Incidence and Severity of Thrombotic Microangiopathy Events",{"measure":36,"description":11,"timeFrame":30},"Incidence and Severity of Injection-Site Reactions",{"measure":38,"description":11,"timeFrame":30},"Incidence of Adverse Events Leading to Drug Discontinuation",{"measure":40,"description":11,"timeFrame":30},"Incidence of Severe Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions",{"measure":42,"description":11,"timeFrame":30},"Incidence of Clinical Laboratory Abnormalities",{"measure":44,"description":11,"timeFrame":45},"Trough Plasma Concentration of Emicizumab at Prespecified Timepoints During the Treatment Period","Predose and at prespecified timepoints from first dose of emicizumab until study completion (up to 3 years, 11 months)",{"measure":47,"description":11,"timeFrame":48},"Percentage of Participants with Anti-Drug Antibodies (ADAs) to Emicizumab at Baseline and with ADAs to Emicizumab During the Treatment Period","Baseline and at prespecified timepoints from first dose of emicizumab until study completion (up to 3 years, 11 months)",{"measure":50,"description":11,"timeFrame":51},"Change from Baseline in Respiratory Rate Over Time","Baseline, Weeks 1, 2, 25, and every 12 weeks thereafter (weeks after switch to emicizumab for Arm B only) until study completion (up to 3 years, 11 months)",{"measure":53,"description":11,"timeFrame":51},"Change from Baseline in Pulse Rate Over Time",{"measure":55,"description":11,"timeFrame":51},"Change from Baseline in Systolic Blood Pressure Over Time",{"measure":57,"description":11,"timeFrame":51},"Change from Baseline in Diastolic Blood Pressure Over Time",{"measure":59,"description":11,"timeFrame":51},"Change from Baseline in Body Temperature Over Time",{"measure":61,"description":11,"timeFrame":62},"Change from Baseline in Electrocardiogram (ECG) Parameters Over Time: QT, QTcF, RR, PR, and QRS Intervals","Baseline and study completion (up to 3 years, 11 months)",{"measure":64,"description":11,"timeFrame":62},"Change from Baseline in Heart Rate Over Time, as Measured by Electrocardiogram (ECG)",{"measure":66,"description":67,"timeFrame":68},"Change from Baseline in the PROMIS-29 Questionnaire Pain Interference Domain Score Over Time","PROMIS-29 stands for Patient-Reported Outcomes Measurement Information System-29","Baseline and at prespecified timepoints until study completion (up to 3 years, 11 months)",{"measure":70,"description":67,"timeFrame":68},"Change from Baseline in the PROMIS-29 Questionnaire Fatigue Domain Score Over Time",{"allocation":72,"interventionModel":73,"interventionModelDescription":74,"primaryPurpose":75,"observationalModel":11,"timePerspective":11,"maskingInfo":76},"RANDOMIZED","PARALLEL","Participants on prior SOC on-demand therapy will be randomized into Arms A and B. Participants on prior SOC prophylactic therapy, who participated in the NIS WP45335 (NCT06883240) and meet the eligibility criteria for this study, will be enrolled into Arm C.","TREATMENT",{"masking":77,"maskingDescription":11,"whoMasked":11},"NONE",[79,85,94,98],{"label":80,"type":81,"description":82,"interventionNames":83},"Arm A (Prior On-Demand SOC): Emicizumab Prophylaxis for 24 Weeks","EXPERIMENTAL","Participants who are randomized to Arm A, taking on-demand standard of care (SOC) treatment at the time of study entry (and for at least 24 weeks prior to enrollment), will receive emicizumab SC prophylaxis.",[84],"Drug: Emicizumab",{"label":86,"type":87,"description":88,"interventionNames":89},"Arm B (Prior On-Demand SOC): On-Demand SOC for 24 Weeks","ACTIVE_COMPARATOR","Participants who are randomized to Arm B, taking on-demand standard of care (SOC) treatment at the time of study entry (and for at least 24 weeks prior to enrollment), will continue to receive their current SOC on-demand treatment until Week 24.",[90,91,92,93],"Drug: von Willebrand Factor (VWF) Concentrates","Drug: Factor VIII (FVIII) Concentrates","Drug: von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates","Drug: Bypassing Agents",{"label":95,"type":81,"description":96,"interventionNames":97},"Arm C (Prior Prophylaxis SOC): Emicizumab Prophylaxis for 24 Weeks","Participants who enroll in Arm C, taking SOC prophylactic treatment at the time of study entry and for at least 24 weeks of observation during the preceding NIS WP45335, will receive emicizumab SC prophylaxis.",[84],{"label":99,"type":81,"description":100,"interventionNames":101},"Treatment Extension Period for All Arms: Emicizumab Prophylaxis","Participants in Arms A and C who have completed 24 weeks of emicizumab prophylaxis and who derive benefit from emicizumab will have the opportunity to continue to receive emicizumab prophylaxis in the extension period. Participants in Arm B who have completed 24 weeks of SOC on-demand treatment will have the opportunity to receive emicizumab prophylaxis in the extension period.",[84],[103,112,116,119,122],{"type":104,"name":105,"description":106,"armGroupLabels":107,"otherNames":108},"DRUG","Emicizumab","Participants will receive emicizumab 3 milligrams per kilogram (mg\u002Fkg) subcutaneous (SC) injections every week (QW) for the first 4 weeks as loading doses, followed by maintenance doses of emicizumab 3 mg\u002Fkg SC once every 2 weeks (Q2W).\n\nDuring the extension period, participants may remain on maintenance dose of emicizumab 3 mg\u002Fkg Q2W, or change their emicizumab maintenance regimen to 1.5 mg\u002Fkg once every week (QW) or 6 mg\u002Fkg once every 4 weeks (Q4W), if they prefer and if agreed by the investigators.",[80,95,99],[109,110,111],"Hemlibra","RO5534262","RG6013",{"type":104,"name":113,"description":114,"armGroupLabels":115,"otherNames":11},"von Willebrand Factor (VWF) Concentrates","Used according to local labeling or local treatment guidelines.",[86],{"type":104,"name":117,"description":114,"armGroupLabels":118,"otherNames":11},"Factor VIII (FVIII) Concentrates",[86],{"type":104,"name":120,"description":114,"armGroupLabels":121,"otherNames":11},"von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates",[86],{"type":104,"name":123,"description":114,"armGroupLabels":124,"otherNames":11},"Bypassing Agents",[86],[126],{"name":127,"affiliation":5,"role":128},"Clinical Trials","STUDY_DIRECTOR",[130,135],{"name":131,"role":132,"phone":133,"phoneExt":11,"email":134},"Reference Study ID Number: WP45338 https:\u002F\u002Fforpatients.roche.com\u002F","CONTACT","888-662-6728 (U.S. Only)","global-roche-genentech-trials@gene.com",{"name":136,"role":132,"phone":11,"phoneExt":11,"email":11},"Fastest response: use the inquiry form. No email attachments. https:\u002F\u002Fwww.gene.com\u002Fcontact-us\u002Fsubmit-medical-inquiry",[138,152,162,172,182,192,202,213,225,235,245,256,265,276,285,289,301,311,321,332,341,352,363,374,385,394,405,416,422],{"facility":139,"status":140,"city":141,"state":142,"zip":143,"country":144,"countryCode":145,"cosmosGeoPoint":146,"geoPoint":151,"contacts":11},"UC Davis","RECRUITING","Sacramento","California","95817","United States","US",{"type":147,"coordinates":148},"Point",[149,150],-121.4944,38.58157,{"lat":150,"lon":149},{"facility":153,"status":140,"city":154,"state":155,"zip":156,"country":144,"countryCode":145,"cosmosGeoPoint":157,"geoPoint":161,"contacts":11},"University of Florida","Gainesville","Florida","32610",{"type":147,"coordinates":158},[159,160],-82.32483,29.65163,{"lat":160,"lon":159},{"facility":163,"status":140,"city":164,"state":165,"zip":166,"country":144,"countryCode":145,"cosmosGeoPoint":167,"geoPoint":171,"contacts":11},"University of Kentucky Children's Hospital","Lexington","Kentucky","40536",{"type":147,"coordinates":168},[169,170],-84.47772,37.98869,{"lat":170,"lon":169},{"facility":173,"status":140,"city":174,"state":175,"zip":176,"country":144,"countryCode":145,"cosmosGeoPoint":177,"geoPoint":181,"contacts":11},"University of Minnesota Medical Center","Minneapolis","Minnesota","55455",{"type":147,"coordinates":178},[179,180],-93.26384,44.97997,{"lat":180,"lon":179},{"facility":183,"status":140,"city":184,"state":185,"zip":186,"country":144,"countryCode":145,"cosmosGeoPoint":187,"geoPoint":191,"contacts":11},"Washington University School of Medicine","St Louis","Missouri","63110",{"type":147,"coordinates":188},[189,190],-90.19789,38.62727,{"lat":190,"lon":189},{"facility":193,"status":140,"city":194,"state":195,"zip":196,"country":144,"countryCode":145,"cosmosGeoPoint":197,"geoPoint":201,"contacts":11},"Virginia Commonwealth University","Richmond","Virginia","23298-0461",{"type":147,"coordinates":198},[199,200],-77.46026,37.55376,{"lat":200,"lon":199},{"facility":203,"status":140,"city":204,"state":11,"zip":205,"country":206,"countryCode":207,"cosmosGeoPoint":208,"geoPoint":212,"contacts":11},"UZ Leuven Gasthuisberg","Leuven","3000","Belgium","BE",{"type":147,"coordinates":209},[210,211],4.70093,50.87959,{"lat":211,"lon":210},{"facility":214,"status":140,"city":215,"state":216,"zip":217,"country":218,"countryCode":219,"cosmosGeoPoint":220,"geoPoint":224,"contacts":11},"The Hospital for Sick Children","Toronto","Ontario","M5G 1X8","Canada","CA",{"type":147,"coordinates":221},[222,223],-79.39864,43.70643,{"lat":223,"lon":222},{"facility":226,"status":140,"city":227,"state":228,"zip":229,"country":218,"countryCode":219,"cosmosGeoPoint":230,"geoPoint":234,"contacts":11},"McGill University Health Center","Montreal","Quebec","H4A 3J1",{"type":147,"coordinates":231},[232,233],-73.58781,45.50884,{"lat":233,"lon":232},{"facility":236,"status":140,"city":237,"state":11,"zip":11,"country":238,"countryCode":239,"cosmosGeoPoint":240,"geoPoint":244,"contacts":11},"IPS SURA Industriales Medellín","Medellín","Colombia","CO",{"type":147,"coordinates":241},[242,243],-75.57151,6.245,{"lat":243,"lon":242},{"facility":246,"status":140,"city":247,"state":11,"zip":248,"country":249,"countryCode":250,"cosmosGeoPoint":251,"geoPoint":255,"contacts":11},"Hopital Claude Huriez - CHU Lille","Lille","59037","France","FR",{"type":147,"coordinates":252},[253,254],3.05512,50.63391,{"lat":254,"lon":253},{"facility":257,"status":140,"city":258,"state":11,"zip":259,"country":249,"countryCode":250,"cosmosGeoPoint":260,"geoPoint":264,"contacts":11},"Groupe Hospitalier Necker Enfants Malades","Paris","75015",{"type":147,"coordinates":261},[262,263],2.3488,48.85341,{"lat":263,"lon":262},{"facility":266,"status":140,"city":267,"state":11,"zip":268,"country":269,"countryCode":270,"cosmosGeoPoint":271,"geoPoint":275,"contacts":11},"Universitätsklinikum Bonn","Bonn","53127","Germany","DE",{"type":147,"coordinates":272},[273,274],7.09549,50.73438,{"lat":274,"lon":273},{"facility":277,"status":140,"city":278,"state":11,"zip":279,"country":269,"countryCode":270,"cosmosGeoPoint":280,"geoPoint":284,"contacts":11},"Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz","Duisburg","47051",{"type":147,"coordinates":281},[282,283],6.76516,51.43247,{"lat":283,"lon":282},{"facility":286,"status":140,"city":287,"state":11,"zip":288,"country":269,"countryCode":270,"cosmosGeoPoint":11,"geoPoint":11,"contacts":11},"Hämophiliezentrum Med. Klinik III\u002FInstitut für Transfusionsmedizin","Frankfurt\u002FM.","60590",{"facility":290,"status":140,"city":291,"state":292,"zip":293,"country":294,"countryCode":295,"cosmosGeoPoint":296,"geoPoint":300,"contacts":11},"Universita' Degli Studi La Sapienza-Ist.Di Ematologia","Rome","Lazio","00161","Italy","IT",{"type":147,"coordinates":297},[298,299],12.51133,41.89193,{"lat":299,"lon":298},{"facility":302,"status":140,"city":303,"state":304,"zip":305,"country":294,"countryCode":295,"cosmosGeoPoint":306,"geoPoint":310,"contacts":11},"IRCCS Ca' Granda Ospedale Maggiore Policlinico","Milan","Lombardy","20122",{"type":147,"coordinates":307},[308,309],9.18951,45.46427,{"lat":309,"lon":308},{"facility":312,"status":140,"city":313,"state":314,"zip":315,"country":294,"countryCode":295,"cosmosGeoPoint":316,"geoPoint":320,"contacts":11},"AOU Careggi","Florence","Tuscany","50134",{"type":147,"coordinates":317},[318,319],11.24626,43.77925,{"lat":319,"lon":318},{"facility":322,"status":140,"city":323,"state":11,"zip":324,"country":325,"countryCode":326,"cosmosGeoPoint":327,"geoPoint":331,"contacts":11},"Kurume University Hospital","Fukuoka","830-0011","Japan","JP",{"type":147,"coordinates":328},[329,330],130.41667,33.6,{"lat":330,"lon":329},{"facility":333,"status":140,"city":334,"state":11,"zip":335,"country":325,"countryCode":326,"cosmosGeoPoint":336,"geoPoint":340,"contacts":11},"Nagoya University Hospital","Nagoya","466-8560",{"type":147,"coordinates":337},[338,339],136.90641,35.18147,{"lat":339,"lon":338},{"facility":342,"status":140,"city":343,"state":11,"zip":344,"country":345,"countryCode":346,"cosmosGeoPoint":347,"geoPoint":351,"contacts":11},"Erasmus MC","Rotterdam","3015 GD","Netherlands","NL",{"type":147,"coordinates":348},[349,350],4.47917,51.9225,{"lat":350,"lon":349},{"facility":353,"status":140,"city":354,"state":11,"zip":355,"country":356,"countryCode":357,"cosmosGeoPoint":358,"geoPoint":362,"contacts":11},"Instytut Hematologii i Transfuzjologii","Warsaw","02-776","Poland","PL",{"type":147,"coordinates":359},[360,361],21.01178,52.22977,{"lat":361,"lon":360},{"facility":364,"status":140,"city":365,"state":11,"zip":366,"country":367,"countryCode":368,"cosmosGeoPoint":369,"geoPoint":373,"contacts":11},"Charlotte Maxeke Johannesburg Academic Hospital","Johannesburg","2193","South Africa","ZA",{"type":147,"coordinates":370},[371,372],28.04363,-26.20227,{"lat":372,"lon":371},{"facility":375,"status":140,"city":376,"state":11,"zip":377,"country":378,"countryCode":379,"cosmosGeoPoint":380,"geoPoint":384,"contacts":11},"Hospital Universitario la Paz","Madrid","28046","Spain","ES",{"type":147,"coordinates":381},[382,383],-3.70256,40.4165,{"lat":383,"lon":382},{"facility":386,"status":140,"city":387,"state":11,"zip":388,"country":378,"countryCode":379,"cosmosGeoPoint":389,"geoPoint":393,"contacts":11},"Hospital Universitario Virgen del Rocio","Seville","41013",{"type":147,"coordinates":390},[391,392],-5.97317,37.38283,{"lat":392,"lon":391},{"facility":395,"status":140,"city":396,"state":11,"zip":397,"country":398,"countryCode":399,"cosmosGeoPoint":400,"geoPoint":404,"contacts":11},"Sahlgrenska Universitetssjukhuset","Gothenburg","S-413 45","Sweden","SE",{"type":147,"coordinates":401},[402,403],11.96679,57.70716,{"lat":403,"lon":402},{"facility":406,"status":140,"city":407,"state":11,"zip":408,"country":409,"countryCode":410,"cosmosGeoPoint":411,"geoPoint":415,"contacts":11},"St Thomas' Hospital","London","SE1 7EH","United Kingdom","UK",{"type":147,"coordinates":412},[413,414],-0.12574,51.50853,{"lat":414,"lon":413},{"facility":417,"status":140,"city":407,"state":11,"zip":418,"country":409,"countryCode":410,"cosmosGeoPoint":419,"geoPoint":421,"contacts":11},"Great Ormond Street Hospital","WC1N 3JH",{"type":147,"coordinates":420},[413,414],{"lat":414,"lon":413},{"facility":423,"status":140,"city":424,"state":11,"zip":425,"country":409,"countryCode":410,"cosmosGeoPoint":426,"geoPoint":430,"contacts":11},"Manchester Royal Infirmary","Manchester","M13 9WL",{"type":147,"coordinates":427},[428,429],-2.23743,53.48095,{"lat":429,"lon":428},{"type":432,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100592829","a-study-to-assess-the-efficacy-and-safety-of-emicizumab-in-participants-with-type-3-von-willebrand-disease-100592829",false,"NCT06998524","A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease","A Phase III, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Prophylaxis in Patients With Type 3 Von Willebrand Disease","WILL-EMI","Inclusion Criteria:\n\n* Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records\n* Preexisting medical record verifying the status of von Willebrand factor (VWF) inhibitor (positive or negative, including titer if available)\n* Adequate hematologic, hepatic, and renal function\n* For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements\n\nAdditional Inclusion Criteria for Arms A and B:\n\n* Age ≥1 month at the time of signing Informed Consent\u002FAssent Form\n* Documented previous use of on-demand therapy with intermittent (less than once a week) on-demand SOC therapy for VWD\n* Having ≥2 treated bleeds (except menstrual bleeds) with factor concentrate within 24 weeks prior to enrollment\n\nAdditional Inclusion Criteria for Arm C:\n\n* Age ≥2 years at the time of signing Informed Consent\u002FAssent Form\n* Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose) as described in the eligibility of Study WP45335\n* Have completed all study requirements as defined in the WP45335 protocol for at least 24 weeks\n\nExclusion Criteria:\n\n* Inherited or acquired bleeding disorder other than Congenital Type 3 VWD\n* History of gastrointestinal bleeding within 18 months prior to enrollment, or any previous diagnosis of angiodysplasia\n* History of intracranial hemorrhage\n* Previous or current treatment for thromboembolic disease or signs of thromboembolic disease\n* Other conditions (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis\n* History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection\n* Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy","ALL","1 Month",{"count":444,"type":445},75,"ESTIMATED","INTERVENTIONAL",[448],"PHASE3","This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been diagnosed with Type 3 von Willebrand disease (VWD). Participants on prior standard of care (SOC) on-demand therapy will be assessed via a randomized comparison (Arm A - emicizumab prophylaxis and Arm B - continuation of SOC on-demand therapy), while participants on prior SOC prophylactic therapy (Arm C - emicizumab prophylaxis) will be assessed via intra-participant analysis with data obtained from the preceding non-interventional study (NIS), WP45335 (NCT06883240).",[451],"Von Willebrand Disease, Type 3","2026-09-07",{"date":454,"type":455},"2026-09-09","ACTUAL",{"date":457,"type":455},"2025-06-27",{"date":459,"type":445},"2029-04-30",{"name":5,"class":6},29]