[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"von-willebrand-disease-vwd-type-2\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:von-willebrand-disease-vwd-type-2":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,52,79],{"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":4,"eligibilityCriteria":58,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":59,"targetDuration":4,"studyType":61,"phases":4,"briefSummary":62,"conditions":63,"keywords":67,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":70,"lastUpdatePostDateStruct":71,"startDateStruct":73,"completionDateStruct":75,"leadSponsor":77,"locationsCount":78},"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":60,"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)",[28,29,30,31,64,65,66],"Von Willebrand Disease, Type 2A","Von Willebrand Disease, Type 2M","Von Willebrand Disease, Type 2N",[28,68,34,36,37,69,38],"Prospective Study","Prophylaxis","2026-04-16",{"date":72,"type":43},"2026-04-21",{"date":74,"type":43},"2024-08-30",{"date":76,"type":21},"2026-12",{"name":49,"class":50},17,{"id":80,"slug":81,"hasResults":11,"nctId":82,"briefTitle":83,"officialTitle":84,"acronym":85,"eligibilityCriteria":86,"healthyVolunteers":11,"sex":16,"minAge":87,"maxAge":4,"enrollmentInfo":88,"targetDuration":4,"studyType":22,"phases":90,"briefSummary":91,"conditions":92,"keywords":93,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":98,"lastUpdatePostDateStruct":99,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":105,"locationsCount":108},"100613986","phase-2-efficacy-and-safety-of-bt200-rondaptivon-pegol-in-patients-with-type-2b-von-willebrand-disease-100613986","NCT07273721","Efficacy and Safety of BT200 (Rondaptivon Pegol) in Patients With Type 2B Von Willebrand Disease","Efficacy and Safety of BT200 (Rondoraptivon Pegol) in Patients With Type 2B Von Willebrand Disease","BT200-VWD2B","Inclusion Criteria:\n\n1. ≥18 years old\n2. Type 2B VWD with thrombocytopenia and a recent bleeding history (e.g. recurrent haematomas)\n3. Able to comprehend and to give informed consent\n4. Able to cooperate with the Investigator, to comply with the requirements of the study, and to complete the full sequence of protocol-related procedures\n\nExclusion Criteria:\n\n1. Clinically significant medical history or ongoing chronic illness that would jeopardise the safety of the patient or compromise the quality of the data derived from his\u002Fher participation in this study\n2. History of significant drug allergy or anaphylactic reactions\n3. Substance abuse, mental illness, or any reason that makes it unlikely in the judgment of the Investigator for the patient to be able to comply fully with study procedures\n4. Use of medication during 2 weeks before the start of the study, which in the judgment of the Investigator may adversely affect the patient's welfare or the integrity of the study's results\n5. Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 elimination half-lives (whichever is longer) prior to treatment start","18 Years",{"count":89,"type":21},6,[25],"This randomized clinical trial with a cross-over design is being conducted at the Department of Clinical Pharmacology at the Medical University of Vienna, and a total of 4-6 patients with type 2B von Willebrand disease (VWD) will participate.\n\nThe main purpose of this clinical trial is to investigate the efficacy and safety of BT200, a new drug for thrombocytopenic patients with type 2B von Willebrand disease (VWD). Based on previous studies, we expect that this drug will inhibit the breakdown of von Willebrand factor (VWF) in small doses, leading to an increase in von Willebrand factor (VWF), platelet count, and factor VIII. This should also lead to a reduced tendency to bleed.\n\nThis study will begin with an observation phase and will then proceed in two periods of approximately 64 days each:\n\nPlacebo or BT200 will be administered subcutaneously at a dose of 12 mg on the first day of the study. After that, patients will self-administer the drug at a dose of 6 mg (0.4 mL) or placebo once a week for another 4 weeks starting the following week (a total of 4 times over a period of 4 weeks). During this time, they will be asked to come to our clinic for a follow-up visit.\n\nAfter a \"washout phase\" lasting several weeks, during which patients do not receive the study drug\u002Fplacebo but are asked to record any bleeding events, the second period begins on day 64:\n\nBT200 or placebo is administered again, depending on what the patients received in the first period. Patients therefore receive the study drug for 4 weeks and placebo for 4 weeks; which is administered when is randomized; a follow-up examination also takes place during this period.\n\nAt the end of the second period, there is another \"washout phase\" lasting several weeks. On day 127, the final examination takes place at the clinic, after which patients have the opportunity to participate in an extension study (to be amended).",[30],[94,95,96,97],"von Willebrand's disease","thrombocytopenia","bleeding","placebo","2025-11-28",{"date":100,"type":43},"2025-12-09",{"date":102,"type":43},"2025-08-14",{"date":104,"type":21},"2026-07-31",{"name":106,"class":107},"Medical University of Vienna","OTHER",1]