[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"von-willebrand-disease-vwd\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:von-willebrand-disease-vwd":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,12,0,[8,52,80,102,125,155,181,212,232,269,297,320],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":32,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":41,"startDateStruct":44,"completionDateStruct":46,"leadSponsor":48,"locationsCount":51},"100574097","phase-1-a-study-assessing-hmb-002-in-participants-with-von-willebrand-disease-100574097",false,"NCT06754852","A Study Assessing HMB-002 in Participants With Von Willebrand Disease","A Phase 1\u002F2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-002 in Participants With Von Willebrand Disease (Velora Pioneer)","Key Inclusion Criteria:\n\n1. Weight 50 to 120 kg, inclusive.\n2. Documented diagnosis of Congenital VWD, confirmed by laboratory testing consistent with ISTH\u002FASH) diagnostic guidelines).\n3. Vital signs are within normal ranges at Screening.\n4. Participants must meet the following baseline organ function, indicated by laboratory criteria as Screening:\n\n   1. Renal: Estimated glomerular filtration rate (eGFR) of ≥45 mL\u002Fmin\u002F1.73m\\^2.\n   2. Hepatic: Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and total bilirubin ≤1.5 upper limit of normal (ULN) at Screening. For participants with a history of Gilbert's Syndrome, total bilirubin ≤2 × ULN.\n   3. Hematology \\>85 g\u002FL and platelet count \\>120 x 10\\^9\u002FL.\n\n   Part A Only:\n5. Age: ≥18 and \\\u003C70 years of age at the time of informed consent.\n6. VWD Subtype Eligibility:\n\n   * Cohorts A1 and A2: Participants with Type 1 VWD, only.\n   * Cohorts A3 and A4: Participants with Type 1 VWD (including Type 1C) and Type 2A VWD\n7. Residual VWF activity of ≤ 50 IU\u002FdL and FVIII activity ≤ 70 IU\u002FdL during screening.\n\n   Part B Only:\n8. Age: ≥16 and \\\u003C70 years of age at the time of informed consent.\n9. VWD Subtype Eligibility: Participants with Type 1 VWD (including Type 1C) and Type 2A.\n10. Residual VWF activity of ≤50 IU\u002FdL and FVIII activity ≤70 IU\u002FdL during screening.\n11. Symptomatic Disease: Participants must be symptomatic, typically reporting bleeding events on a monthly basis.\n12. Bleeding History (must meet one of the following):\n\n    1. Prior Observational Study Participation:\n\n       The participant must have participated in the observational study HMB-002-101\\_SCR (VELORA Discover), have a minimum annualized treated bleeding event (ATBR) of 3; OR\n    2. Medical Record-Documented Bleeding History:\n\n    The Investigator confirms that ≥3 treated bleeding events have been documented in the participant's medical record within the preceding 12 months.\n\n    Part C Only:\n13. Age: ≥18 and \\\u003C70 years of age at the time of informed consent.\n14. Participants with Type 3 VWD or Type 1 VWD with low residual VWF and FVIII activity levels (VWF activity \\\u003C5 IU\u002FdL and FVIII activity \\\u003C10 IU\u002FdL).\n15. Receives regular VWF concentrate (at least 1\u002Fweek) as part of their routine care (usual dose ≤50 IU\u002Fkg).\n\nKey Exclusion Criteria:\n\n1. Personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial venous thrombosis.\n2. High risk thrombophilia: Homozygous Factor V Leiden (FVL), compound heterozygous FVL\u002FProthrombin gene mutation, Antithrombin deficiency with activity \\\u003C50%. Congenital Protein C and Protein S deficiency with levels \\\u003C50%.\n3. Body mass index (BMI) \\>35 kg\u002Fm\\^2 (obese, adjusted for ethnicity).\n4. Presence of other conditions that substantially increase risk of thrombosis either individually (for participants \\>65 years of age) or in combination (for participants ≤65 years of age), at the discretion of the Investigator or Medical Monitor.\n5. Clinically significant cardiovascular disease.\n6. Other known severe bleeding disorder(s) other than VWD.\n7. Requirement for concomitant medications that affect hemostasis (including, but not limited to anticoagulation, antiplatelet agents, certain non-steroidal anti-inflammatory drugs) and cannot refrain from use for 14 days prior to the first dose of study drug and throughout the study.\n\n   Exclusion Criteria for Part A and Part B Only\n8. Requirement for ongoing hemostatic treatment to prevent bleeding (bleed prophylaxis). Prophylaxis administered intermittently for procedures or surgery to reduce bleeding risk is permitted.","ALL","16 Years","69 Years",{"count":20,"type":21},108,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","This is a first-in-human (FIH), Phase 1\u002F2, 3-part open-label, dose escalation, safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and efficacy study evaluating HMB-002 in participants with VWD. Part A of the study involves a single ascending dose (SAD) regimen design to establish safety, tolerability, PK, and PD effect. In Part B of the study, the safety and tolerability of repeat dosing will be established prior to cohort expansion to explore efficacy. Part C will evaluate the safety, PK, and PD of a single concomitant dose of HMB-002 and factor concentrate with Type 3 VWD or Type 1 VWD with low residual VWF and FVIII who use factor concentrate as prophylaxis.",[28,29,30,31],"Von Willebrand Disease (VWD)","Von Willebrand Disease (VWD), Type 1","Von Willebrand Disease (VWD), Type 2","Von Willebrand Disease (VWD), Type 3",[33,34,35,36,37,38],"Von Willebrand Disease (VMD)","Type 1 VWD","Type 1 with low residual VWF and FVIII who use factor concentrate as prophylaxis","Type 2 VWD","Type 3 VWD","Von Willebrand Factor (VWF)","RECRUITING","2026-06-26",{"date":42,"type":43},"2026-06-30","ACTUAL",{"date":45,"type":43},"2025-02-06",{"date":47,"type":21},"2027-07",{"name":49,"class":50},"Hemab ApS","INDUSTRY",25,{"id":53,"slug":54,"hasResults":11,"nctId":55,"briefTitle":56,"officialTitle":57,"acronym":58,"eligibilityCriteria":59,"healthyVolunteers":11,"sex":60,"minAge":17,"maxAge":61,"enrollmentInfo":62,"targetDuration":4,"studyType":22,"phases":64,"briefSummary":65,"conditions":66,"keywords":68,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":71,"lastUpdatePostDateStruct":72,"startDateStruct":74,"completionDateStruct":75,"leadSponsor":77,"locationsCount":79},"100643166","phase-2-hmbeacon-a-phase-2-study-to-evaluate-aln-6400-in-adult-and-adolescent-female-patients-with-vwd-and-hmb-100643166","NCT07575308","HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB","HMBeacon: A Phase 2, Randomized, Double-blind Study of the Safety, Tolerability, Efficacy, and Pharmacodynamics of Multiple Dose ALN-6400 in Adult and Adolescent Female Patients With Von Willebrand Disease (VWD) and Heavy Menstrual Bleeding (HMB)","HMBeacon","Inclusion Criteria -\n\n* Is an adolescent or adult patient with a diagnosis of VWD (including Type 1, Type 2, Type 3, and platelet-type VWD) and HMB for 2 cycles during screening\n\nExclusion Criteria -\n\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 2 times the upper limit of normal (ULN)\n* Has total bilirubin greater than 1.5×ULN (except for patients with Gilbert's syndrome)\n* Has an estimated glomerular filtration (eGFR) less than 30 mL\u002Fmin\u002F1.73m\\^2 at screening\n* Is not willing to comply with the contraceptive requirements during the study period\n* Used routine factor prophylaxis (greater than or equal to 1 infusion of factor per week over 12 weeks) within 4 weeks of screening or plans use for routine factor prophylaxis during the study\n* New placement of any hormonal or nonhormonal intrauterine device (IUD) or hormonal implants within 24 weeks of screening\n* Known current gynecological conditions causing abnormal uterine bleeding (including infection, fibroids, endometriosis, polycystic ovary syndrome, or dysplasia)\n\nNote: other protocol defined inclusion \u002F exclusion criteria apply","FEMALE","45 Years",{"count":63,"type":21},24,[25],"The purpose of this study is to evaluate the safety, tolerability, efficacy, and pharmacodynamics (PD) of multiple doses of ALN-6400 in adult and adolescent patients with VWD and HMB",[28,67],"Heavy Menstrual Bleeding (HMB)",[69],"siRNA, RNAi therapeutic, Plasminogen, PLG, VWD, HMB","NOT_YET_RECRUITING","2026-06-11",{"date":73,"type":43},"2026-06-15",{"date":42,"type":21},{"date":76,"type":21},"2028-11-29",{"name":78,"class":50},"Alnylam Pharmaceuticals",1,{"id":81,"slug":82,"hasResults":11,"nctId":83,"briefTitle":84,"officialTitle":85,"acronym":4,"eligibilityCriteria":86,"healthyVolunteers":11,"sex":16,"minAge":87,"maxAge":88,"enrollmentInfo":89,"targetDuration":4,"studyType":22,"phases":90,"briefSummary":91,"conditions":92,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":95,"completionDateStruct":97,"leadSponsor":99,"locationsCount":101},"100643086","phase-2-a-phase-ii-clinical-trial-to-evaluate-the-efficacy-safety-and-pharmacokinetics-of-sr604-injection-in-patients-with-von-willebrand-disease-100643086","NCT07640893","A Phase II Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of SR604 Injection in Patients With Von Willebrand Disease","A Multi-Dose, Randomized, Multicenter Phase II Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetic Profile of SR604 Injection in Patients With Von Willebrand Disease","Inclusion Criteria:\n\n* Patients must meet ALL of the following inclusion criteria to be enrolled:\n\n  1. Age \\>= 18 years and \\\u003C= 65 years at the time of signing informed consent, regardless of sex;\n  2. At screening, patients with a confirmed diagnosis of von Willebrand disease (VWD) with documented evidence and a defined VWD subtype;\n  3. At least 4 new bleeding episodes within 6 months prior to screening;\n  4. No active bleeding symptoms prior to the first dose;\n  5. The subject or impartial witness fully understands and is able to comply with the protocol requirements, is willing to complete the study as planned, and voluntarily agrees to provide biological samples for testing as required by the protocol; is able to understand the procedures and methods of this clinical trial, provides voluntary participation after full informed consent, and personally signs the informed consent form.\n\nExclusion Criteria:\n\n* Patients meeting ANY of the following exclusion criteria will not be enrolled:\n\n  1. Known history of hypersensitivity to the investigational drug formulation or any of its components;\n  2. Intolerance to subcutaneous injection or presence of other local skin abnormalities or dermatological conditions that may affect drug administration and safety assessment;\n  3. Meeting any of the following criteria at screening:\n\n     1. Hemoglobin \\\u003C 60 g\u002FL;\n     2. Platelet count \\\u003C 80 x 10\\^9\u002FL;\n     3. Hepatic or renal dysfunction: Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>= 2.5 x upper limit of normal (ULN), or total bilirubin \\>= 1.5 x ULN; or serum creatinine (Cr) \\>= 1.5 x ULN;\n  4. Positive for anti-human immunodeficiency virus (HIV) antibody;\n  5. Presence of any bleeding disorder other than von Willebrand disease \\[hemophilia A or B, congenital coagulation factor VII deficiency, acquired von Willebrand disease (AVWS), platelet-type VWD, inherited platelet disorders, etc.\\]; or significantly abnormal coagulation parameters due to diseases other than von Willebrand disease (e.g., platelet disorders, vitamin K deficiency, etc.);\n  6. Presence of protein C deficiency or protein S deficiency;\n  7. History of thrombosis or family history of thrombosis prior to signing informed consent or currently, or history of thrombophilia;\n  8. Severe bleeding due to VWD within 2 years prior to screening, such as intracranial hemorrhage, esophageal variceal bleeding, etc.;\n  9. Severe cardiac disease, such as unstable angina, congestive heart failure (New York Heart Association class \\>= III), severe arrhythmia (QTc interval \\> 500 ms, corrected by Fridericia formula), uncontrolled hypertension (systolic blood pressure \\>= 160 mmHg or diastolic blood pressure \\>= 100 mmHg), etc.;\n  10. Female patients with menstrual abnormalities due to organic gynecological diseases (e.g., uterine fibroids, endometriosis, adenomyosis, etc.);\n  11. Previous or current life-threatening malignant neoplasms or end-stage liver disease;\n  12. Use of DDAVP or plasma-derived VWF-containing factor VIII concentrate, plasma-derived\u002Frecombinant VWF preparations, or antifibrinolytic therapy within 1 week prior to the first dose;\n  13. Use of antithrombotic agents within 1 week prior to the first dose;\n  14. Receipt of fresh blood\u002Fplasma or cryoprecipitate therapy within 2 weeks prior to the first dose;\n  15. Receipt of vaccination within 1 month prior to the first dose or planned vaccination during the study period;\n  16. Major surgery (major surgery defined as Grade III and IV surgeries) within 1 month prior to the first dose, or planned surgery during the study period;\n  17. Enrollment in other clinical trials within 1 month prior to the first dose;\n  18. History of drug abuse or alcohol dependence (alcohol dependence criteria: long-term drinking history exceeding 5 years, equivalent ethanol intake \\>= 40 g\u002Fday, or heavy drinking within 2 weeks, equivalent ethanol intake \\> 80 g\u002Fday. Ethanol amount (g) conversion formula = alcohol consumption (mL) x alcohol content (%) x 0.8);\n  19. Presence of psychiatric disease or significant mental disorder, or other reasons resulting in incapacity or lack of cognitive ability;\n  20. Plans for procreation or sperm donation throughout the study period up to 3 months after the last dose, or unwillingness to use effective physical contraceptive measures (e.g., condoms);\n  21. Presence of clinically significant disease or other reasons rendering the patient unsuitable for clinical trial participation in the investigator's opinion (e.g., patient unlikely to benefit from the clinical trial);\n  22. Patients whom the investigator considers to have poor compliance, rendering efficacy evaluation difficult or likelihood of completing the planned treatment course and follow-up low.","18 Years","65 Years",{"count":63,"type":21},[25],"The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in patients with von Willebrand disease.",[28],"2026-06-05",{"date":71,"type":43},{"date":96,"type":43},"2025-11-26",{"date":98,"type":21},"2027-12-31",{"name":100,"class":50},"Shanghai RAAS Blood Products Co., Ltd.",9,{"id":103,"slug":104,"hasResults":11,"nctId":105,"briefTitle":106,"officialTitle":107,"acronym":4,"eligibilityCriteria":108,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":109,"enrollmentInfo":110,"targetDuration":4,"studyType":22,"phases":111,"briefSummary":113,"conditions":114,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":115,"lastUpdatePostDateStruct":116,"startDateStruct":118,"completionDateStruct":120,"leadSponsor":122,"locationsCount":124},"100484050","phase-3-a-study-of-vonicog-alfa-rvwf-in-children-with-severe-von-willebrand-disease-vwd-100484050","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.","17 Years",{"count":63,"type":21},[112],"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.",[28],"2026-05-22",{"date":117,"type":43},"2026-05-27",{"date":119,"type":43},"2024-11-06",{"date":121,"type":21},"2030-04-11",{"name":123,"class":50},"Takeda",21,{"id":126,"slug":127,"hasResults":11,"nctId":128,"briefTitle":129,"officialTitle":130,"acronym":131,"eligibilityCriteria":132,"healthyVolunteers":11,"sex":16,"minAge":87,"maxAge":4,"enrollmentInfo":133,"targetDuration":4,"studyType":22,"phases":135,"briefSummary":137,"conditions":138,"keywords":139,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":144,"lastUpdatePostDateStruct":145,"startDateStruct":147,"completionDateStruct":149,"leadSponsor":151,"locationsCount":154},"100566136","algorithm-based-management-to-reduce-the-recurrence-of-gi-bleeding-and-severe-epistaxis-in-von-willebrand-disease-100566136","NCT06651255","Algorithm-based Management to Reduce the Recurrence of GI Bleeding and Severe Epistaxis in Von Willebrand Disease","Algorithm-based Management to Reduce the Recurrence of Gastrointestinal Bleeding and Severe Epistaxis in Von Willebrand Disease: WILL-MANAGE Trial","WILL MANAGE","Inclusion Criteria:\n\n* Males and females adults aged \\> 18 years\n* With a constitutional von Willebrand Disease (according to the phenotypic criteria of the French Reference Center of von Willebrand Disease)\n* With a GI bleeding \\[either overt (any hematemesis, any melena or hematochezia with at least a 2g\u002FdL drop in hemoglobin) or occult (iron deficiency anemia with at least 2g\u002FdL drop in hemoglobin)\\] with the presence of angiodysplasia or a negative finding on digestive conventional endoscopy OR with a severe epistaxis (requiring red blood cells transfusion or treatment with VWF concentrates)\n* Be affiliated to a social security scheme\n* Written informed consent obtained\n\nExclusion Criteria:\n\n* Acquired von Willebrand Disease\n* Presence of an inhibitor to VWF or a contra indication to VWF concentrates\n* Contra-indication to videocapsule endoscopy\n* Refusal of the procedures part of the study\n* Pregnant women or breastfeeding\n* Short-life expectancy\n* Liver cirrhosis Child-Pugh C or diagnosed portal hypertension\n* Cancer currently undergoing chemotherapy\n* Inability to provide informed consent\n* Patient under justice protection",{"count":134,"type":21},66,[136],"NA","WILL MANAGE is a prospective multicenter controlled open label randomized trial comparing an algorithm-based multidisciplinary management to a standard of care to reduce the incidence of bleeding (GI bleeding or severe epistaxis) recurrence in von Willebrand disease.",[28],[140,141,142,143],"Von Willebrand disease","gastrointestinal bleeding","angiodysplasia","epistaxis","2026-05-13",{"date":146,"type":43},"2026-05-18",{"date":148,"type":43},"2026-03-10",{"date":150,"type":21},"2032-03",{"name":152,"class":153},"University Hospital, Lille","OTHER",17,{"id":156,"slug":157,"hasResults":11,"nctId":158,"briefTitle":159,"officialTitle":160,"acronym":4,"eligibilityCriteria":161,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":162,"targetDuration":4,"studyType":164,"phases":4,"briefSummary":165,"conditions":166,"keywords":170,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":173,"lastUpdatePostDateStruct":174,"startDateStruct":176,"completionDateStruct":178,"leadSponsor":180,"locationsCount":154},"100562979","a-study-of-bleeding-and-treatment-in-participants-with-von-willebrand-disease-100562979","NCT06610201","A Study of Bleeding and Treatment in Participants With Von Willebrand Disease","Velora Discover: A Prospective, Screening Study of Bleeding and Treatment in Participants With Von Willebrand Disease","Inclusion Criteria:\n\n1. Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations.\n2. Has an understanding, ability, and willingness to comply with Study procedures and restrictions.\n3. Is 16 years and \\\u003C 70 years at the time of screening.\n4. Weight 50 to 120 kg (±10%) at Screening and body mass index (BMI) \\\u003C38.5 kg\u002Fm\\*2.\n5. Has Von Willebrand Disease: Type 1 VWD (including Type 1C VWD) or Type 2A VWD. All participants must have: Documented lab results confirming their diagnosis consistent with ISTH\u002FASH diagnostic guidelines; VWF Activity ≤30 IU\u002FdL and FVIII activity ≤70 IU\u002FdL during Screening.\n6. Has symptomatic disease as defined by a history of bruising or bleeding events, with an expected minimum of 3 bleeding episodes (including heavy menstrual bleeding) per year that require treatment to control bleeding symptoms, and\u002For has recurrent and ongoing episodes of heavy menstrual bleeding at the time of enrollment.\n\nExclusion Criteria:\n\n1. Has a history of clinically significant hypersensitivity associated with monoclonal antibody therapies.\n2. Has a personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial vein thrombosis events.\n3. Has a high-risk thrombophilia: Homozygous Factor V Leiden (FVL), compound heterozygous FVL\u002Fprothrombin gene mutation, antithrombin \\\u003C50%, congenital protein C and protein S deficiency with levels \\\u003C50%.\n4. Requires ongoing hemostatic (bleed-prophylaxis) treatment to prevent bleeding\n5. Has other known severe bleeding disorder(s) other than VWD.\n6. Planned major surgery during the study period.\n7. Has other conditions that substantially increase the risk of thrombosis either individually or in combination, at the discretion of the Investigator, including but not limited to: significant family history; BMI \\>30 and ≤38.5 kg\u002Fm² (moderately obese, adjusted for ethnicity and increased central adiposity); reduced mobility; active malignancy; major surgery within 6 weeks preceding Screening; or postpartum within 12 weeks preceding Screening.\n8. Is pregnant or plans to become pregnant within the next 6 months following informed consent sign off.\n9. Has clinically significant cardiovascular disease including, but not limited to: NYHA Class III or IV heart failure, coronary artery disease, uncontrolled arrythmia, moderate to severe valvular heart disease, peripheral vascular disease, and ischemic stroke.\n10. Has other combinations of conditions that substantially increase the risk of cardiovascular events at the discretion of the Investigator including, but not limited to, smoking, uncontrolled hyperlipidemia, and uncontrolled hypertension.\n11. Has any concurrent disease, treatment, medication (including but not limited to ongoing anticoagulation, antiplatelet therapy, or non-steroidal anti-inflammatory drugs or other drugs that affect hemostasis), condition, medication, or abnormality in clinical laboratory tests which may impact on the participant's bleeding symptoms or affect their ability to complete the study, in the Investigator's opinion.\n12. Has received any investigational product within 30 days prior to Screening. If the participant was enrolled and dosed in Velora Pioneer (study HMB-002-102; NCT06754852), they must have completed their End of Study Visit.",{"count":163,"type":21},200,"OBSERVATIONAL","The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD). Data from this study will be used to establish baseline bleeding and treatment rates in a population of participants with VWD and act as comparator data for future clinical study outcomes.(e.g. Velora Pioneer)",[28,29,30,31,167,168,169],"Von Willebrand Disease, Type 2A","Von Willebrand Disease, Type 2M","Von Willebrand Disease, Type 2N",[28,171,34,36,37,172,38],"Prospective Study","Prophylaxis","2026-04-16",{"date":175,"type":43},"2026-04-21",{"date":177,"type":43},"2024-08-30",{"date":179,"type":21},"2026-12",{"name":49,"class":50},{"id":182,"slug":183,"hasResults":11,"nctId":184,"briefTitle":185,"officialTitle":186,"acronym":187,"eligibilityCriteria":188,"healthyVolunteers":11,"sex":16,"minAge":189,"maxAge":190,"enrollmentInfo":191,"targetDuration":4,"studyType":22,"phases":193,"briefSummary":194,"conditions":195,"keywords":196,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":202,"lastUpdatePostDateStruct":203,"startDateStruct":205,"completionDateStruct":207,"leadSponsor":209,"locationsCount":211},"100601785","phase-3-study-to-evaluate-subcutaneous-sc-vga039-in-patients-with-von-willebrand-disease-vwd-100601785","NCT07115004","Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)","A Prospective, Multicenter, Open-label, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of Prophylactic VGA039 in Adolescent and Adult Patients With Von Willebrand Disease (VIVID-6)","VIVID-6","Key Inclusion Criteria\n\n* 12 to 75 years of age, inclusive\n* No clinically significant laboratory, ECG, or vital signs results\n* Documented diagnosis consistent with VWD of any type\n* Historical annualized bleeding rate (ABR; excluding menstrual bleeds and bleeds under the skin) of both untreated and treated bleeds ≥12 per year\n* Patients with VWD who are judged by the investigator to be suitable candidates for routine prophylaxis to reduce the frequency of bleeding episodes\n* Hemoglobin level ≥ 8 g\u002FdL and platelet count ≥ 100 x 109\u002FL at Screening\n\nKey Exclusion Criteria\n\n* Use of routine prophylaxis of VWF-containing concentrates defined as at least 1 VWF-containing concentrate infusion to prevent or reduce bleeding per week during the previous 6 months prior to screening\n* Planning to initiate routine prophylaxis with VWF-containing concentrates or any other hemostatic treatment during the study\n* Patients with pro-thrombotic disorders or abnormal findings on laboratory thrombophilia evaluation performed at screening or previously documented\n* History of arterial or venous thrombosis, including superficial thrombophlebitis, or embolism\n* Evidence of renal, hepatic, central nervous system, respiratory, cardiovascular disease, cerebrovascular disease, peripheral vascular disease, or metabolic dysfunction\n* Baseline FVIII activity \\> lower limit of normal (LLN)","12 Years","75 Years",{"count":192,"type":21},60,[112],"This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)",[28],[197,198,187,199,200,201],"VWD","von Willebrand Disease","Vega Therapeutics","Star Therapeutics","VGA039","2026-04-02",{"date":204,"type":43},"2026-04-03",{"date":206,"type":43},"2025-10-15",{"date":208,"type":21},"2028-10",{"name":210,"class":50},"Vega Therapeutics, Inc",14,{"id":213,"slug":214,"hasResults":11,"nctId":215,"briefTitle":216,"officialTitle":217,"acronym":4,"eligibilityCriteria":218,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":109,"enrollmentInfo":219,"targetDuration":4,"studyType":164,"phases":4,"briefSummary":221,"conditions":222,"keywords":224,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":225,"lastUpdatePostDateStruct":226,"startDateStruct":228,"completionDateStruct":229,"leadSponsor":231,"locationsCount":79},"100624054","an-observational-study-of-vonicog-alfa-rvwf-in-pediatric-participants-with-von-willebrand-disease-vwd-100624054","NCT07404644","An Observational Study of Vonicog Alfa (rVWF) in Pediatric Participants With Von Willebrand Disease (vWD)","Vonvendi Intravenous Specified Drug Use-results Survey \"Pediatric Administration\"","Inclusion Criteria :\n\n* Under 18 years old with vWD.\n* Participants who are treated with rVWF for the purpose of hemostatic treatment and management during bleeding episodes or perioperative periods.\n* Participants who have prescription or administration after the approval date of rVWF for pediatric use in Japan.\n\nExclusion Criteria :\n\n\\- Patients who are participating in clinical trials of rVWF.",{"count":220,"type":21},13,"This study is conducted in Japan of vonicog alfa (rVWF) used to treat pediatric participants with Von Willebrand Disease (vWD).\n\nThe main aim of the study is to evaluate adverse drug reaction and effectiveness of vonicog alfa (rVWF).\n\nDuring the study, pediatric participants with vWD will be administered with rVWF under routine normal practice. The investigators will evaluate adverse events due to rVWF for 1 year from the start of drug administration.\n\nThe study sponsor will not be involved in how the participants are administered but will be recorded what happens during the study.",[223],"Von Willebrand Disease (vWD)",[223],"2026-02-19",{"date":227,"type":43},"2026-02-23",{"date":225,"type":43},{"date":230,"type":21},"2031-05-30",{"name":123,"class":50},{"id":233,"slug":234,"hasResults":11,"nctId":235,"briefTitle":236,"officialTitle":237,"acronym":238,"eligibilityCriteria":239,"healthyVolunteers":11,"sex":16,"minAge":240,"maxAge":87,"enrollmentInfo":241,"targetDuration":4,"studyType":164,"phases":4,"briefSummary":242,"conditions":243,"keywords":246,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":261,"lastUpdatePostDateStruct":262,"startDateStruct":264,"completionDateStruct":266,"leadSponsor":267,"locationsCount":79},"100624476","clinical-spectrum-and-management-of-von-willebrand-disease-among-children-in-assiut-governorate-100624476","NCT07410130","Clinical Spectrum and Management of Von Willebrand Disease Among Children in Assiut Governorate","Clinical Spectrum of Von Willebrand Disease Among Children: Frequency, Management, and Outcomes in Assiut Governorate","VWD-ASSIUT","Inclusion Criteria:\n\n1. Age 0-18 years.\n2. Residents of Assiut Governorate or receiving care at Assiut University Children's Hospital.\n3. Suspected or confirmed von Willebrand disease (VWD) based on clinical bleeding symptoms or referral for evaluation.\n4. Patients diagnosed with VWD using standard laboratory tests, including:\n5. VWF antigen (VWF:Ag).\n6. VWF ristocetin cofactor activity (VWF:RCo).\n7. Factor VIII activity.\n\nExclusion Criteria:\n\n1. Other inherited bleeding disorders, such as:\n2. Hemophilia A or B.\n3. Rare coagulation factor deficiencies (e.g., factors I, V, VII, X, XI deficiency).\n4. Platelet function disorders.\n5. Acquired bleeding disorders, including:\n\n   * Liver disease.\n   * Renal insufficiency.\n   * Vitamin K deficiency.\n   * Disseminated intravascular coagulation (DIC).\n   * Use of medications that may interfere with coagulation testing (e.g., anticoagulants, antiplatelet drugs).\n   * Incomplete clinical or laboratory data (for retrospective cases).\n   * Refusal of consent for participation (for prospective cases).","0 Years",{"count":51,"type":21},"Von Willebrand disease (VWD) is the most common inherited bleeding disorder in children. It occurs due to a deficiency or dysfunction of von Willebrand factor, a protein that plays an essential role in blood clotting. Children with VWD may experience frequent nosebleeds, easy bruising, prolonged bleeding after injuries or surgeries, and, in adolescent girls, heavy menstrual bleeding. The severity of symptoms varies widely depending on the type of the disease and the level of the clotting factor.\n\nDespite its clinical importance, data about the frequency, clinical presentation, and treatment outcomes of von Willebrand disease among children in Upper Egypt are limited. Early recognition and appropriate management are crucial to prevent complications, reduce hospital visits, and improve quality of life.\n\nThis observational study aims to assess the frequency of von Willebrand disease among children attending Assiut University Children's Hospital, describe the different disease subtypes, and evaluate the clinical bleeding patterns and management strategies used in routine practice. The study will include children aged 0-18 years with suspected or confirmed VWD.\n\nInformation will be collected from medical records and clinical evaluations, including bleeding symptoms, laboratory test results, disease classification, and treatment approaches. The results of this study are expected to improve understanding of von Willebrand disease in children in this region and support better diagnostic and therapeutic planning for affected patients.",[28,244,245],"Inherited Bleeding Disorders in Children","Pediatric Hemostatic Disorders",[247,197,248,249,250,251,252,253,254,255,256,257,258,259,260],"Von Willebrand Disease","Pediatric bleeding disorders","Inherited coagulation disorders","Von willebrand factor","Mucocutaneous bleeding","Epistaxis in children","Menorrhagia","Bleeding assessment tool","VWD antigen","VWD activity","Hemostasis","Pediatric hematology","Factor VIII","Observational study","2026-02-07",{"date":263,"type":43},"2026-02-13",{"date":265,"type":21},"2026-03",{"date":179,"type":21},{"name":268,"class":153},"Assiut University",{"id":270,"slug":271,"hasResults":11,"nctId":272,"briefTitle":273,"officialTitle":274,"acronym":275,"eligibilityCriteria":276,"healthyVolunteers":277,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":278,"targetDuration":4,"studyType":164,"phases":4,"briefSummary":280,"conditions":281,"keywords":283,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":288,"lastUpdatePostDateStruct":289,"startDateStruct":291,"completionDateStruct":293,"leadSponsor":295,"locationsCount":79},"100620468","endothelial-colony-forming-cells-in-patients-with-vwd-avws-and-healthy-subjects-100620468","NCT07358013","Endothelial Colony-Forming Cells in Patients With VWD, AVWS and Healthy Subjects","Isolation and Characterization of Endothelial Colony Forming Cells (ECFCs) in Patients Diagnosed With Von Willebrand Disease, Acquired Von Willebrand Syndrome and Healthy Subjects","ECFCs\u002F2022","Inclusion Criteria for patients:\n\nPatients with von Willebrand disease (VWD) or acquired von Willebrand syndrome (AVWS)\n\nAge ≥ 16 years.\n\nPrevious diagnosis of von Willebrand disease or acquired von Willebrand syndrome, defined as one of the following:\n\nGroup A - Type 1 VWD:\n\nVWF levels ≤ 30 IU\u002FdL, regardless of bleeding history, or\n\nVWF levels ≤ 0.50 IU\u002FmL in the presence of abnormal bleeding.\n\nGroup B - Congenital or acquired VWD (VWD or AVWS):\n\nDiagnosis of congenital or acquired VWD, with or without gastrointestinal bleeding.\n\nGroup C - Subgroup study (Type 2A VWD):\n\nOne patient with type 2A VWD selected for a dedicated sub-study involving allele-specific siRNA silencing of the mutant allele.\n\nAbility and willingness to provide written informed consent.\n\nFor patients without prior molecular characterization: willingness to undergo VWF gene sequencing and to sign the related informed consent.\n\nInclusion criteria for healthy volunteers\n\n* No prior diagnosis of VWD, bleeding disorders, or thrombotic disorders.\n* Willingness to donate blood for study procedures.\n* Ability and willingness to provide written informed consent.\n* Age ≥ 18 years.\n\nExclusion criteria for both patients and healthy volunteers:\n\n* Pregnancy.\n* Anemia, as determined at screening or based on medical history.",true,{"count":279,"type":21},48,"The goal of this observational study is to learn how endothelial colony-forming cells (ECFCs) behave in people with von Willebrand disease (VWD), acquired von Willebrand syndrome (AVWS), and in healthy individuals.",[28,282],"Acquired Von Willebrand Disease",[284,285,286,287],"von Willebrand disease","von Willebrand factor","ex vivo","endothelial colony forming cells (ECFCs)","2026-01-13",{"date":290,"type":43},"2026-01-22",{"date":292,"type":43},"2023-11-11",{"date":294,"type":21},"2028-05-31",{"name":296,"class":153},"Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico",{"id":298,"slug":299,"hasResults":11,"nctId":300,"briefTitle":301,"officialTitle":302,"acronym":4,"eligibilityCriteria":303,"healthyVolunteers":11,"sex":16,"minAge":87,"maxAge":4,"enrollmentInfo":304,"targetDuration":4,"studyType":22,"phases":306,"briefSummary":307,"conditions":308,"keywords":309,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":311,"lastUpdatePostDateStruct":312,"startDateStruct":314,"completionDateStruct":316,"leadSponsor":318,"locationsCount":319},"100602886","phase-3-a-study-of-recombinant-von-willebrand-factor-rvwf-in-chinese-participants-with-von-willebrand-disease-vwd-100602886","NCT07129343","A Study of Recombinant Von Willebrand Factor (rVWF) in Chinese Participants With Von Willebrand Disease (vWD)","A Clinical Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in the Treatment of Bleeding Episodes in Chinese Subjects Diagnosed With Von Willebrand Disease","Inclusion Criteria\n\n1. Participant must voluntarily sign an institutional review board (IRB)\u002Findependent ethics committee-approved written informed consent form after all relevant aspects of the study have been explained and discussed with the participant.\n2. Participant has a documented diagnosis of severe VWD (baseline VWF:RCo less than \\[\\\u003C\\]20 international units \\[IU\\]deciliter \\[dL\\]) with a diagnosis of VWD type verified per the following recommended criteria:\n\n   * Type 1 (von Willebrand factor:Ristocetin cofactor activity \\[VWF:RCo\\] \\\u003C20 IU\u002FdL and by VWF activity\u002FVWF:antigen \\[Ag\\] ratio) or,\n   * Type 2A or type 2B (by VWF activity\u002FVWF:Ag ratio and multimer pattern, with genetics if necessary), type 2N (FVIII:C \\\u003C10% and genetics), type 2M (by VWF activity\u002FVWF:Ag ratio and multimer pattern) or\n   * Type 3 (VWF:Ag \\\u003C=3 IU\u002FdL). Diagnosis is confirmed, when applicable, by genetic testing and\u002For by multimer analysis.\n3. Participant is at least 18 years of age at screening.\n4. Participant is ethnic Chinese and lives in China, including those from Taiwan, Hong Kong, and Macao.\n5. If female of childbearing potential, participant presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study.\n6. Participant is willing and able to comply with the requirements of the protocol.\n7. Participant has had a minimum of 3 documented bleeds that indicated the need for VWF coagulation factor replacement therapies during the previous 12 months prior to enrollment.\n\nExclusion Criteria\n\n1. Participant has been diagnosed with pseudo VWD or another hereditary or acquired coagulation disorder other than VWD (example \\[eg\\], qualitative and quantitative platelet disorders or elevated prothrombin time \\[PT\\]\u002Finternational normalized ratio \\>1.4).\n2. Participant has a history or presence of a VWF inhibitor at screening.\n3. Participant has a documented history of a VWF:RCo half-life of \\\u003C6 hours.\n4. Participant has a history or presence of a FVIII inhibitor with a titer greater than or equal to \\[\\>=\\] 0.6 Bethesda units per milliliter \\[BU\u002FmL\\] (by Bethesda assay or Bethesda method with Nijmegen modification).\n5. Participant has a known hypersensitivity to any of the components of the study drugs, such as to mouse or hamster proteins.\n6. Participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis\u002Fconjunctivitis, mild asthma, food allergies or animal allergies.\n7. Participant has a medical history of a thromboembolic event.\n8. Participant is human immunodeficiency virus (HIV) positive with an absolute cluster of differentiation 4 (CD4) count \\\u003C200\u002Fcubic millimeter (mm\\^3).\n9. Participant has been treated with an immunomodulatory drug, other than antiretroviral chemotherapy eg, α-interferon, or corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 milligram (mg)\u002Fday (excluding topical treatment \\[eg, ointments, nasal sprays\\]), within 30 days prior to signing the informed consent (or assent, if appropriate).\n10. Participant is pregnant or lactating at the time informed consent is obtained.\n11. Participant has participated in another clinical study involving an 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.\n12. Participant is diagnosed with progressive fatal disease and\u002For has a life expectancy of less than 15 months.\n13. Participant is member of the study team conducting this study or in a dependent relationship with one of the study team members. Dependent relationships include close relatives (that is, children, partner\u002Fspouse, siblings, parents) as well as employees.\n14. Participant has an acute illness (eg, influenza, flu-like syndrome, allergic rhinitis\u002Fconjunctivitis, nonseasonal asthma) at screening.\n15. Participant is diagnosed with significant liver disease, as evidenced by, but not limited to, any of the following: serum alanine aminotransferase (ALT) greater than 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (eg, presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child-Pugh class B or C.\n16. Participant has been diagnosed with renal disease, with a serum creatinine level \\>=2.5 milligram per deciliter (mg\u002FdL).\n17. Participant has a platelet count \\\u003C100,000\u002F milliliter (mL) at screening (except for participants with type 2B VWD, whose platelet count\\[s\\] at screening will be evaluated taking into consideration historical trends in platelet counts and the investigator's medical assessment of the participant's condition).\n18. Participant has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).\n19. In the judgment of the investigator, the participant has another clinically significant concomitant condition (eg, uncontrolled hypertension) that may pose additional risks for the participant.\n20. Participant is identified by the investigator as being unable or unwilling to cooperate with study procedures.",{"count":305,"type":21},20,[112],"The main aim of this study is to find out if VONVENDI is safe for adult Chinese participants with VWD. The study will also check how well VONVENDI helps control bleeding with or without product ADVATE in the participants who may need elective surgery or dental procedures. In addition, the study will also examine how VONVENDI is processed by the body (known as pharmacokinetic \\[PK\\]) and how the drug helps the body respond or improve a condition (pharmacodynamic \\[PD\\]).\n\nParticipants will receive an initial dose of VONVENDI of 40 to 80 international units per kilogram (IU\u002Fkg) of body weight. If a participant's baseline factor VIII (FVIII) level is not high enough to help stop bleeding, VONVENDI will be given along with 30 to 45 IU\u002Fkg of ADVATE rFVIII.\n\nParticipants will be in the study for approximately 14 months. During the study, participants will be followed up at clinics or over telephone calls.",[28],[310],"Drug Therapy","2026-01-07",{"date":313,"type":43},"2026-01-08",{"date":315,"type":43},"2025-10-13",{"date":317,"type":21},"2027-11-25",{"name":123,"class":50},7,{"id":321,"slug":322,"hasResults":11,"nctId":323,"briefTitle":324,"officialTitle":325,"acronym":4,"eligibilityCriteria":326,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":327,"phases":4,"briefSummary":328,"conditions":329,"keywords":330,"overallStatus":331,"whyStopped":4,"lastUpdateSubmitDate":332,"lastUpdatePostDateStruct":333,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":335,"locationsCount":4},"100529380","post-trial-access-program-of-tak-577-for-von-willebrand-disease-vwd-100529380","NCT06173024","Post Trial Access Program of TAK-577 for Von Willebrand Disease (VWD)","Post Trial Access for Study SHP677-304: Recombinant Von Willebrand Factor (rVWF) for Adult and Pediatric Subjects With Severe Von Willebrand Disease (VWD)","Inclusion Criteria:\n\n1. Participant has completed the treatment period of the SHP677-304 study (at least 12 months on study treatment).\n2. Participant had good clinical response to rVWF treatment.\n3. Participant does not have access to any comparable or satisfactory alternative replacement therapy available at country level.\n4. Participant will be\u002Fhas been negatively impacted by discontinuation of rVWF.\n5. Participant and\u002For a parent(s)\u002Flegal guardian is informed of the nature of the post-trial access program and can provide written informed consent for themselves or the child to participate (with assent from a child when appropriate) before treatment).\n\nExclusion Criteria:\n\n1\\. Participants with known hypersensitivity\u002Fintolerance to the study drug will not be eligible for this study","EXPANDED_ACCESS","The post-trial access program allows eligible participants to gain access to unlicensed treatment on compassionate grounds. Recombinant von Willebrand factor (rVWF) also known as TAK-577, is a medicine to help treat Von Willebrand Disease (VWD). This post-trial access program enables continued access to children and adults who are benefitting from treatment on study SHP677-304 (NCT03879135) study.",[28],[310],"AVAILABLE","2025-09-02",{"date":334,"type":43},"2025-09-03",{"name":123,"class":50}]