[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"von-willebrand-diseases\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:von-willebrand-diseases":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,11,0,[8,49,82,111,135,145,171,196,236,262,285],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":21,"conditions":22,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100054158","athn-transcends-a-natural-history-study-of-non-neoplastic-hematologic-disorders-100054158",false,"NCT04398628","ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders","ATHN Transcends: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People With Non-Neoplastic Hematologic Disorders","Participants who meet the following inclusion criteria and none of the exclusion criteria are eligible for enrollment in one of the open disease-specific arms.\n\nInclusion Criteria:\n\n1. Any age\n2. Having a congenital or acquired blood disorder; or\n3. Having a bleeding phenotype as indicated by an age adjusted abnormal ISTH Bleeding Assessment Tool score with an unknown diagnosis; or\n4. Connective tissue disorder with bleeding tendency as indicated by an age adjusted abnormal ISTH Bleeding Assessment Tool score.\n5. Eligible for a currently active disease-specific arm.\n6. Concurrent enrollment in the ATHNdataset or current ATHNdataset participant.\n\nExclusion Criteria:\n\n1\\. Does not qualify for inclusion in a currently activedisease-specific arm; participants may be eligible to enroll as future cohorts and arms are activated; 2. Unable to give informed consent or assent 3. Unwilling to perform study procedures\n\nCohort Participant Selection\n\nEach participant is to be enrolled in the cohort for which they qualify as defined below.\n\nHemophilia Cohort\n\nInclusion Criteria:\n\nParticipants who meet any of the following inclusion criteria are eligible for enrollment into this cohort:\n\n1. Factor VIII or factor IX activity \\\u003C50%, without another explanation for low clotting factor other than congenital hemophilia or being a known carrier for congenital hemophilia; OR\n2. Carrier for congenital hemophilia with a factor VIII \\>=50% or factor IX activity \\>=50% with or without a bleeding phenotype as indicated by an ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years OR\n3. Known congenital hemophilia that have a factor level \\>50% after receiving vector, OR 4. Acquired hemophilia.\n\nExclusion Criteria:\n\nNone\n\nVon Willebrand Disease Cohort\n\nInclusion Criteria:\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into this cohort:\n\n1\\. Meeting the definition of VWD or low VWF per most recent international guidelines\n\nExclusion Criteria:\n\nNone\n\nCongenital Platelet Disorders Cohort\n\nInclusion Criteria:\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into this cohort:\n\n1. Abnormalities of platelet function a. Glanzmann thrombasthenia (GPIIb or GPIIIa) b. Bernard-Soulier syndrome (GPIbalpha, GPIbbeta, or GPIX)\n2. Abnormalities of platelet granules\n3. Abnormalities of platelet signal transduction\n4. Abnormalities of platelet secretion\n5. Collagen Receptor Defect\n6. ADP Receptor Defect\n7. Thromboxane Receptor Defect\n8. Giant Platelet Disorder\n9. Abnormalities in platelet aggregation testing due to another or unknown cause (not drug related)\n\nExclusion Criteria:\n\n1\\. Platelet disorders secondary to medications or other substances\n\nRare Disorders Cohort\n\nInclusion Criteria:\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into this cohort:\n\n1\\. Have an established Rare Coagulation Disorder (RCD) diagnosis of one of the following:\n\n1. PAI-1 deficiency\n2. Factor I, II, V, VII, X, XI, XIII deficiencies\n3. Combined FV and FVIII deficiency\n4. Plasminogen deficiency\n5. Decreased tissue plasminogen activator\n6. Afibrinogenemia\u002Fhypofibrinogenemia\u002Fdysfibrinogenemia\n7. Thrombotic Thrombocytopenia Purpura or Congenital Hemolytic Uremic Syndrome\n8. Wiskott-Aldrich\n9. Methylenetetrahydrofolate Reductase Deficiency\n\nExclusion Criteria:\n\nNone\n\nBleeding NOS Cohort\n\nInclusion Criteria:\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into this cohort:\n\n1. Have a bleeding phenotype as indicated by an ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years with an unknown diagnosis, OR\n2. Connective tissue disorder with bleeding tendency as indicated by an ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years.\n\nExclusion Criteria:\n\nNone\n\nThrombosis\u002FThrombophilia Cohort\n\nInclusion Criteria\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into this cohort:\n\n1\\. Have a prior history of arterial or venous thrombosis. 2. Participants with a known congenital or acquired thrombophilia with or without thrombosis.\n\na. Common congenital thrombophilias: i. Protein C deficiency ii. Protein S deficiency iii. Antithrombin deficiency iv. Factor V Leiden v. Prothrombin gene mutation b. Rare genetic factors i. Hyperhomocysteinemia c. Indeterminate genetic factors i. Elevated factor VIII ii. Elevated factor IX iii. Elevated factor XI iv. Elevated lipoprotein (a) d. Acquired thrombophilias i. Lupus anticoagulant ii. Anti-cardiolipin antibodies\u002FBeta2 glycoprotein antibodies iii. Antiphospholipid syndrome\n\nExclusion Criteria Acquired thrombophilia secondary to medications (birth control pills or hormone replacement therapy), overweight or obesity, smoking, cancer, pregnancy, surgery, injury, prolonged inactivity\u002Fbedrest, heart failure, inflammatory bowel disease, or kidney disease\n\nNon-Neoplastic Hematologic Conditions Cohort\n\nInclusion Criteria\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into this cohort:\n\n1\\. Having any congenital or acquired non-neoplastic hematologic disorder not included in any other cohort\n\nExclusion Criteria None\n\nArm\u002FModule Participant Selection\n\nPreviously Untreated Patients Arm\n\nInclusion Criteria:\n\n1. Diagnosis of congenital hemophilia A (FVIII \\\u003C40%) or hemophilia B (FIX \\\u003C40% or below lower limit for age)\n2. Age \\\u003C18 years at time of enrollment\n3. Parent or authorized guardian or legally authorized representative (LAR) can provide informed consent\n4. Care established at one of the ATHN Transcends participating HTCs\n5. Clotting Factor Concentrate (CFC) exposure, fresh frozen plasma (FFP), cryoprecipitate, and single donor platelets \\\u003C3 exposure days (ED)\n\nExclusion Criteria\n\n1. Concomitant diagnosis with another bleeding disorder\n2. History of a confirmed, positive inhibitor\n\nINHIBIT Module\n\nInclusion Criteria:\n\n1\\. Diagnosis of severe factor VIII deficiency with baseline factor VIII level \\\u003C1% 2. Initiating or plan to initiate prophylaxis with emicizumab or factor replacement 3. Factor concentrate exposure, Fresh Frozen Plasma (FFP), cryoprecipitate, and single donor platelets ≤3 EDs 4. ≤5 years of age\n\nExclusion Criteria\n\n1. Concomitant diagnosis with bleeding disorder other than hemophilia A\n2. Immune disorder\n3. Previous history or presence of factor VIII inhibitor. A confirmed, positive inhibitor is defined as two consecutive positive inhibitor titers (≥ 0.6 BU) that result in changes in treatment recommendations.\n\nEfanesoctocog alfa (ALTUVIIIO®) Module\n\nInclusion criteria:\n\n1. Ability of the potential participant's legally authorized representative (e.g., their parent or legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulation.\n2. People with severe HA with a baseline FVIII activity of less than 1%. (While inclusion for participation in ATHN Transcends lists \\\u003C5% FVIII activity, this proposed module will limit enrollment to people with FVIII activity levels of \\\u003C1%.) Other severities may be included per ATHN Transcends PI approval.\n3. \\\u003C18 years of age.\n4. No history of a confirmed, positive FVIII inhibitor.\n5. Sex assigned at birth of male, female, or intersex.\n6. Participants should have no more than three (3) exposure days of blood products (fresh frozen plasma, cryoprecipitate, or platelets), no more than three (3) doses of any FVIII concentrate other than efanesoctocog alfa, and up to three (3) doses of efanesoctocog alfa prior to enrollment.\n7. Site PI confirmed all inclusion criteria has been met.\n\nExclusion criteria:\n\n1. Not meeting all the inclusion criteria; confirmed by site PI.\n2. Any exposure to blood products or FVIII replacement products except as described in the inclusion criteria.\n3. History of positive inhibitor testing.\n4. History of hypersensitivity reactions associated with efanesoctocog alfa administration.\n5. Other coagulation disorder(s) in addition to Hemophilia A.\n6. Any concurrent clinically significant major disease such as cancer that, in the opinion of the investigator, would make the participant unsuitable for enrollment.\n7. Concurrent systemic treatment with chemotherapy and\u002For other immunosuppressant medications. Use of corticosteroids for the treatment of asthma or management of acute allergic or otherwise life-threatening episodes is allowed except for systemic corticosteroid treatment given to children daily or on an alternate day schedule at \\> 2 mg\u002Fkg\u002Fday of prednisone or its equivalent or \\> 20 mg\u002Fday if the duration is longer than 14 days.\n8. Enrollment in a concurrent clinical interventional drug study.\n9. Intake of an Investigational Medicinal Product within three (3) months prior to inclusion in this study.\n10. Inability to comply with study requirements.\n11. Other, unspecified reasons that, in the investigator's opinion, make the participant unsuitable for enrollment.\n\nHemophilia Natural History Arm\n\nInclusion Criteria\n\n1. Congenital or acquired hemophilia A or B of any severity with or without inhibitors receiving a current therapy, a non-factor product, or for whom use of a non-factor product is a possibility, OR\n2. Females of any age, with confirmed congenital hemophilia A or B carrier status with genetic mutational analysis and any factor level.\n\nExclusion Criteria\n\n1. Presence of any known bleeding disorder other than congenital hemophilia A or B\n2. Presence of concurrent hemophilia and a second hemostatic defect (low von Willebrand Factor (vWF) without vWD diagnosis is not excluded)\n3. Unable or unwilling to comply with the study arm protocol.\n\nNonacog beta pegol (Rebinyn®) Module\n\nInclusion Criteria:\n\n1. Has provided signed written consent for the nonacog beta pegol (Rebinyn®)Module before any study-related activities.\n2. Male participants, at any age with hemophilia B, naïve or minimally exposed (up to 3 EDs) to nonacog beta pegol treatment at time of study enrollment. Additional doses may be allowable per ATHN Transcends PI approval.\n3. Decision to initiate continuous prophylaxis treatment with commercially available nonacog beta pegol has been made by the participant(s)\u002FLegally Authorized Representative(s) (LAR(s)) and the treating physician before and independently from the decision to include the participant in this study.\n\nExclusion Criteria:\n\n1. Previous participation in this study. Participation is defined as having given informed consent in this study.\n2. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation, including a diagnosis or suspicion of attention deficit hyperactivity disorder (ADHD) or autism spectrum disorder (ASD) per the discretion of the Principal Investigator.\n3. Known or suspected hypersensitivity to nonacog beta pegol or related products.\n4. Clinical suspicion or presence of FIX inhibitor at time of inclusion.\n5. Inability or unwillingness to undergo neurological assessment\u002Fstructured developmental history.\n\nEmicizumab (Hemlibra®) Module\n\nInclusion Criteria:\n\n1. Participant currently treated with emicizumab (Hemlibra®)\n2. Currently enrolled in the Hemophilia Natural History Arm of ATHN Transcends\n\nExclusion Criteria:\n\n1\\. Unable or unwilling to comply with the protocol\n\nDistress Module\n\nInclusion Criteria:\n\n1. Congenital hemophilia A or B of any severity with or without inhibitors receiving a current therapy, a non-factor product, or for whom use of a non-factor product is a possibility\n2. Age 18 years of age or older\n3. English speaking\n\nExclusion Criteria:\n\n1. Presence of any known bleeding disorder other than congenital hemophilia A or B;\n2. Presence of concurrent hemophilia and a second hemostatic defect (low von Willebrand Factor (vWF) without vWD diagnosis is not excluded); and\n3. Unable or unwilling to comply with the study arm protocol\n\nHemophilia Gene Therapy Outcomes Arm\n\nInclusion Criteria\n\n1. Hemophilia A or B of any severity with or without inhibitors having received or will receive a hemophilia gene transfer product in the next 6 months.\n2. Age 18 years and older.\n3. Able to give informed consent.\n\nExclusion Criteria None\n\nEtranacogene dezaparvovec (HEMGENIX®) Module\n\nInclusion Criteria:\n\nEtranacogene dezaparvovec (HEMGENIX®) Cohort\n\n1. Age 18 years of age or older\n2. Treatment with commercial etranacogene dezaparvovec (HEMGENIX®)\n3. Have provided signed written informed consent within 3 months before or within 6 months after etranacogene dezaparvovec (HEMGENIX®) treatment, or within 6 months of when the study is initiated at the treating site.\n\nFIX Prophylaxis Cohort\n\n1. Age 18 years of age or older\n2. Treatment with FIX prophylaxis therapy\n3. Has provided signed written consent at any time for ATHN Transcends Study\n\nExclusion Criteria, both cohorts:\n\n1\\. Have been treated with etranacogene dezaparvovec in a clinical trial prior to commercial availability. These patients are still eligible for enrollment in the Gene Therapy Outcomes Arm, and their data may be collected for separate analysis.\n\nCongenital Platelet Disorders Arm\n\nInclusion Criteria\n\n1. Platelet adhesion defect\n\n   1. Bernard Soulier syndrome (Defective GPIb-IX-V receptor, impaired adhesion to vWF)\n   2. Velocardio-facial syndrome\u002FDiGeorge syndrome (Defective GPIb-IX-V receptor)\n   3. Platelet type vWD (Defective GPIb-IX-V, gain of function interaction between vWF-GP1bα)\n2. Platelet aggregation defect\n\n   1. Glanzmann thrombasthenia (Defective integrin αIIbβ3 (GPIIb\u002FIIIa)\n   2. Platelet aggregation defect, NOS\n3. Agonist receptor defects\n\n   1. Epinephrine\n   2. ADP\n   3. Collagen\n   4. Thromboxane A2\n4. Platelet signaling defects\n\n   1. Cyclooxygenase deficiency (PTGS1 mutation)\n   2. Phospholipase A2 deficiency\n   3. Thromboxane synthase deficiency (TBXAS1 mutation)\n   4. G protein activation defect (GNAS mutation)\n   5. Scott syndrome (defect in phosphatidyl serine translocation)\n5. Platelet Granule disorders\n\n   1. Dense granule storage pool disorder\n\n      * Hermansky Pudlak syndrome\n      * Chediak Higashi syndrome\n      * Griscelli syndrome\n   2. Alpha granule storage pool disorder\n\n      * Grey platelet syndrome\n      * Arthrogryposis-Renal Dysfunction-Cholestasis (ARC) syndrome\n      * Quebec platelet disorder\n      * Paris-Trousseau syndrome\n   3. Combined alpha delta granule deficiency\n6. Platelet cytoskeletal structure defects\n\n   1. Wiskott Aldrich syndrome\n   2. MYH9 associated disorders (myosin heavy chain)\n\n      * May Hegglin syndrome\n      * Fechtner syndrome\n      * Sebastian syndrome\n      * Epstein syndrome\n   3. Other mutations\n\n      * FLNA mutations (Filamin)\n      * DIAPH1 (Actin and microtubules)\n      * ACTN1 (alpha actinin)\n      * TPM4 (tropomyosin)\n      * TUBB1 (beta tubulin)\n7. Other Congenital thrombocytopenias\n\n   1. Familial platelet disorders and predisposition to AML (RUNX1)\n   2. X linked thrombocytopenia with dyserythropoiesis (GATA1)\n   3. Congenital amegakaryocytic thrombocytopenia (MPL)\n\nExclusion Criteria\n\n1. Diagnosis of von Willebrand Disease (Meeting the definition of vWD or low vWF per most recent international guidelines)\n2. Diagnosis of Hemophilia A or Hemophilia B (Factor VIII or IX ≤ 40%)\n\nGlanzmann Thrombasthenia (GT) Module\n\nInclusion Criteria\n\n1. Participant has signed the informed consent\u002Fassent form\n2. Participant has flow cytometry or aggregometry or genetics confirmed GT\n3. Participant is willing to perform study procedures, including daily bleed tracking for 3 months and further if requested\n4. Participants are 2 years or older at time of consent\n\nExclusion Criteria None","ALL",{"count":18,"type":19},3000,"ESTIMATED","OBSERVATIONAL","In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have not yet demonstrated long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.2,3,4,5\n\nIn 2019 alone, the FDA has issued approvals for 24 new therapies for congenital and acquired hematologic conditions.6 In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.7\n\nWith this increase in potential new therapies possible, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.8",[23,24,25,26,27,28,29,30,31,32,33,34,35],"Hematologic Disorder","Bleeding Disorder","Connective Tissue Disorder","Hemophilia","Thrombosis","Von Willebrand Diseases","Thrombophilia","Rare Bleeding Disorder","Platelet Disorder","Factor IX Deficiency","Factor VIII Deficiency","Thalassemia","Sickle Cell Disease","RECRUITING","2026-07-10",{"date":39,"type":40},"2026-07-13","ACTUAL",{"date":42,"type":40},"2020-09-30",{"date":44,"type":19},"2035-12",{"name":46,"class":47},"American Thrombosis and Hemostasis Network","NETWORK",71,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":55,"eligibilityCriteria":56,"healthyVolunteers":11,"sex":16,"minAge":57,"maxAge":4,"enrollmentInfo":58,"targetDuration":4,"studyType":60,"phases":61,"briefSummary":63,"conditions":64,"keywords":66,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":71,"lastUpdatePostDateStruct":72,"startDateStruct":74,"completionDateStruct":76,"leadSponsor":78,"locationsCount":81},"100371709","videomicroscopy-for-the-prediction-of-bleeding-in-constitutional-haemorrhagic-diseases-100371709","NCT04119908","Videomicroscopy for the Prediction of Bleeding in Constitutional Haemorrhagic Diseases","Interest of Sublingual Videomicroscopy for the Prediction of Bleeding in Von Willebrand Disease and Other Constitutional Haemorrhagic Diseases","VIDEO-BLEED","Inclusion Criteria:\n\n* Adult patient with a significant form of von Willebrand disease (according to the inclusion criteria of the Willebrand Disease French Reference Center), a Glanzmann Thrombasthenia, a moderate to severe Haemophilia A or a woman carrying the hemophilia gene\n* Social insured patient\n\nExclusion Criteria:\n\n* Minor patient\n* Refusal of consent\n* Person benefiting from a system of legal protection\n* Pregnant patient","18 Years",{"count":59,"type":19},400,"INTERVENTIONAL",[62],"NA","In Willebrand disease, there is currently no test available to identify non-invasively patients with a high risk of bleeding from angiodysplasias The study propose to use a sublingual capillary bed analysis by video-microscopy, a sensitive, reproducible and non-invasive technique, to assess whether sublingual capillary density is predictive of hemorrhagic risk for patients with von Willebrand disease.",[28,65],"Glanzmann Thrombasthenia",[67,68,69,70],"constitutional haemorrhagic diseases, Videomicroscopy, prediction, bleeding","Videomicroscopy","prediction","bleeding","2026-05-19",{"date":73,"type":40},"2026-05-20",{"date":75,"type":40},"2023-05-24",{"date":77,"type":19},"2028-05-24",{"name":79,"class":80},"University Hospital, Lille","OTHER",1,{"id":83,"slug":84,"hasResults":11,"nctId":85,"briefTitle":86,"officialTitle":87,"acronym":4,"eligibilityCriteria":88,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":89,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":91,"conditions":92,"keywords":96,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":103,"lastUpdatePostDateStruct":104,"startDateStruct":106,"completionDateStruct":108,"leadSponsor":110,"locationsCount":81},"100579147","athndataset-registry-100579147","NCT06820515","ATHNdataset Registry","American Thrombosis and Hemostasis Network ATHNdataset Registry","Inclusion Criteria:\n\n* Any participant evaluated for or the potential to have a blood disorder who has an encounter with an ATHN Affiliate.\n* Participants of any age.\n* Participant is able to provide consent or assent; a Legally Authorized Representative (LAR) may provide consent on a participant's behalf if a participant is unable to provide self-consent\n\nExclusion Criteria:\n\n* Any participant unable to provide consent or assent to participate in the ATHNdataset",{"count":90,"type":19},200000,"The Hemophilia Treatment Center (HTC) where you receive care is working with The American Thrombosis and Hemostasis Network (ATHN) to look at the quality of life of people with blood disorders and problems.\n\nDoctors, scientists, policymakers, and other health care providers need a large amount of information from a lot of people to answer scientific, public health, and policy questions about better ways to treat blood disorders. They will use the information from the ATHNdataset to answer these questions.",[26,27,93,94,35,65,24,95,28],"Hemophilia A","Hemophilia B","Blood Disorder",[97,98,99,100,101,102],"bleed event","bleed treatments","adverse events","joint bleed","bleeding disorder","bleeding symptoms","2026-04-16",{"date":105,"type":40},"2026-04-21",{"date":107,"type":40},"2024-10-25",{"date":109,"type":19},"2055-10-31",{"name":46,"class":47},{"id":112,"slug":113,"hasResults":11,"nctId":114,"briefTitle":115,"officialTitle":115,"acronym":116,"eligibilityCriteria":117,"healthyVolunteers":118,"sex":16,"minAge":57,"maxAge":4,"enrollmentInfo":119,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":121,"conditions":122,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":125,"lastUpdatePostDateStruct":126,"startDateStruct":128,"completionDateStruct":130,"leadSponsor":132,"locationsCount":134},"100345089","development-of-a-device-for-evaluating-primary-hemostasis-under-whole-blood-flow-conditions-100345089","NCT03773159","Development of a Device for Evaluating Primary Hemostasis Under Whole Blood Flow Conditions","INDONESIA","Inclusion Criteria:\n\n* person who has given oral consent\n* adult\n* blood donor at EFS Bourgogne Franche-Comté\n* or patient with von Willebrand disease or major constitutional thrombopathy followed by the Resource and Competence Centre (CRC) - Constitutional Hemorrhagic Diseases of Dijon or Besançon\n* or patient on antiplatelet drugs consulting for thrombosis at the Dijon Bourgogne or Besançon Hospital\n\nExclusion Criteria:\n\n* a person who is not affiliated to or not a beneficiary of national health insurance\n* person subject to court-ordered protection (curatorship, guardianship)\n* pregnant, parturient or breastfeeding woman\n* a person who is unable to consent\n* person on anti-inflammatory treatment and serotonin reuptake inhibitor antidepressants (platelet function disorders)",true,{"count":120,"type":19},200,"Currently, the exploration of primary hemostasis (a physiological phenomenon used to stop bleeding) is imperfect because it is based on targeted tests for platelets or von Willebrand factor, without taking into account blood flow and other blood cells (red and white blood cells). Tests for whole blood and flow conditions exist, but there is currently no test that comes close to actual physiological conditions.\n\nAn exploration of whole blood haemostasis in a device that is similar to a blood vessel and at different flow conditions (venous and arterial) could help to better identify the risk of bleeding in predisposed patients (von Willebrand factor deficiency, antiplatelet therapy).\n\nThe objective of the study is to evaluate the performance of this new hemostasis test in whole blood and under flow conditions.\n\nParticipation in the study is ad hoc and is limited to adding a maximum of 4 citrated tubes (20 mL or the equivalent of 4 teaspoons) and 1 EDTA tube (5mL) to a routine blood sample (for patients) or during blood donation (for controls).",[28,123,124],"Major Constitutional Thrombopathy","Patient on Antiplatelet Drugs","2026-04-01",{"date":127,"type":40},"2026-04-02",{"date":129,"type":40},"2019-05-06",{"date":131,"type":19},"2028-05",{"name":133,"class":80},"Centre Hospitalier Universitaire Dijon",2,{"id":136,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":137,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":21,"conditions":138,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":139,"lastUpdatePostDateStruct":140,"startDateStruct":142,"completionDateStruct":143,"leadSponsor":144,"locationsCount":48},"100393100",{"count":18,"type":19},[23,24,25,26,27,28,29,30,31,32,33,34,35],"2026-01-09",{"date":141,"type":40},"2026-01-12",{"date":42,"type":40},{"date":44,"type":19},{"name":46,"class":47},{"id":146,"slug":147,"hasResults":11,"nctId":148,"briefTitle":149,"officialTitle":150,"acronym":151,"eligibilityCriteria":152,"healthyVolunteers":11,"sex":153,"minAge":57,"maxAge":4,"enrollmentInfo":154,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":156,"conditions":157,"keywords":158,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":162,"lastUpdatePostDateStruct":163,"startDateStruct":165,"completionDateStruct":167,"leadSponsor":169,"locationsCount":5},"100373740","von-willebrand-factor-in-pregnancy-vip-study-100373740","NCT04146376","Von Willebrand Factor in Pregnancy (VIP) Study","Von Willebrand Factor in Pregnancy (VIP) Study: A Multicenter Study of Wilate Use in Von Willebrand Disease for Childbirth","VIP","Inclusion Criteria:\n\n* von Willebrand Disease (VWD) patients defined prepartum as Type 1 per National Heart, Lung, and Blood Institute (NHLBI) criterion of von Willebrand Factor (VWF) level less than 30 percent, or Type 2, or Type 3 VWD\n\nor\n\n* A diagnosis of VWD and VWF and Factor VIII (FVIII) levels obtained in gestational weeks 34-38 that determine enrollment in the non-corrector group:\n* Patients with gestational week 34-38 VWF:Ag, VWF:Act (or VWF:RCo), or FVIII:C less than 100 percent will be enrolled in the non-corrector group. In patients with an isolated VWF:CB type 2 defect, VWF:CB less than 100 percent can also be determined as a non-corrector\n* Patients with all VWF parameter levels greater than or equal to 100 percent self-corrected at gestational weeks 34-38 will be enrolled in the corrector group\n\nWritten informed consent from the patient prepartum, before gestational week 39\n\nExclusion Criteria:\n\n* Presence of a clinical contraindication to receive wilate or tranexamic acid, as determined by the health care provider, such as a prior drug reaction\n* Presence of other concurrent disorder of hemostasis, platelet dysfunction, or collagen disorders\n* Presence of liver disease or renal disease, clinical suspicion or diagnosis of preeclampsia or eclampsia, HELLP syndrome, TTP, DIC, or other acquired vasculopathy or coagulopathy\n* Age less than 18 years\n* Inability of the local laboratory to monitor the VWF laboratory tests needed during the course of treatment to determine Wilate dosing adjustments","FEMALE",{"count":155,"type":19},110,"In pregnant women with von Willebrand disease (VWD) who by the third trimester do not have von Willebrand factor (VWF) or factor VIII (FVIII) levels greater than 50-100%, specific guidance is lacking for delivery planning in terms of how high of a VWF level should be achieved to reduce bleeding.\n\nThis is a prospective, open-label, cohort study in women with VWD using Wilate VWF replacement therapy to maintain trough or minimum VWF levels of 100-150% for delivery and the immediate postpartum period, followed by levels of 50-100% for 5-10 days after delivery, depending upon the route of delivery. The primary objective is to document the rate of primary postpartum hemorrhage (PPH). The secondary objective is to document further effectiveness outcomes and safety.",[28],[159,160,161],"von Willebrand Disease","von Willebrand Factor","postpartum hemorrhage","2025-12-10",{"date":164,"type":40},"2025-12-18",{"date":166,"type":40},"2019-10-12",{"date":168,"type":19},"2026-12",{"name":170,"class":80},"University of Washington",{"id":172,"slug":173,"hasResults":11,"nctId":174,"briefTitle":175,"officialTitle":175,"acronym":176,"eligibilityCriteria":177,"healthyVolunteers":11,"sex":153,"minAge":178,"maxAge":179,"enrollmentInfo":180,"targetDuration":182,"studyType":20,"phases":4,"briefSummary":183,"conditions":184,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":186,"lastUpdatePostDateStruct":187,"startDateStruct":189,"completionDateStruct":191,"leadSponsor":193,"locationsCount":195},"100509672","heavy-menstrual-bleeding-progestin-treatment-in-bleeding-disorders-study-100509672","NCT05916469","Heavy Menstrual Bleeding Progestin Treatment in Bleeding Disorders Study","HMB-BD","Inclusion Criteria:\n\n* Post-menarcheal\n* Ages 10-24\n* Decision to initiate a trial of progestin therapy with either LNG-IUD or NETA\n* Able to provide assent and written informed consent by one parent (ages \\\u003C18) or written informed consent (age 18+)\n* Must meet trial criteria for heavy menstrual bleeding\n* Must meet trial criteria for a bleeding disorder or have minimum workup to rule out bleeding disorder\n\nExclusion Criteria:\n\n* Pregnant or seeking pregnancy","10 Years","24 Years",{"count":181,"type":19},300,"5 Years","The goal of this multicenter prospective observational study and registry of U.S. adolescents and young adults with heritable bleeding disorders is to determine the bleeding outcomes, satisfaction, hemostatic parameter changes, and patient reported quality of life after 6 months of use of either of two commonly used hormonal treatments for menstrual suppression - levonorgestrel intrauterine device (LNG-IUD) and norethindrone acetate (NETA). Under this application the study will compare the two treatments and compare outcomes after LNG-IUD treatment results to a control group without a bleeding disorder, with the goal of determining the benefits and expected outcomes of these treatment options for this population.",[185,24,28],"Heavy Menstrual Bleeding","2025-12-09",{"date":188,"type":40},"2025-12-17",{"date":190,"type":40},"2025-09-24",{"date":192,"type":19},"2029-06",{"name":194,"class":80},"Oregon Health and Science University",9,{"id":197,"slug":198,"hasResults":11,"nctId":199,"briefTitle":200,"officialTitle":201,"acronym":4,"eligibilityCriteria":202,"healthyVolunteers":118,"sex":16,"minAge":203,"maxAge":204,"enrollmentInfo":205,"targetDuration":4,"studyType":60,"phases":207,"briefSummary":210,"conditions":211,"keywords":212,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":225,"lastUpdatePostDateStruct":226,"startDateStruct":228,"completionDateStruct":230,"leadSponsor":232,"locationsCount":235},"100498885","phase-1-study-of-vga039-in-healthy-volunteers-and-patients-with-von-willebrand-disease-vivid-100498885","NCT05776069","Study of VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease (VIVID)","A Multi-Modular Trial to Evaluate VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease and Other Bleeding Disorders (VIVID)","Key Inclusion Criteria (All Subjects)\n\n* Subjects, 18 to 60 years of age, inclusive for Parts 1 and 2\n* Subjects, 12 to 60 years of age, inclusive for Parts 3 and 5\n* No clinically significant laboratory, ECG, or vital signs results.\n\nAdditional Key Inclusion Criteria (for Subjects in Part 1 Only) • Body mass index of 18-32 kg\u002Fm2\n\nAdditional Key Inclusion Criteria (for Subjects in Part 2 Only)\n\n* Subjects with VWD who are symptomatic, defined as having a history of bleeding or bruising.\n* Hemoglobin level ≥ 8 g\u002FdL and platelet count ≥ 150 × 109\u002FL at Screening.\n\nExclusion Key Criteria (All Subjects)\n\n* Use of hormonal contraceptives within 56 days prior to administration of the study drug.\n* Subjects with detection of FV Leiden or Prothrombin G20210A mutation, protein C or S deficiency, antithrombin deficiency, or antiphospholipid antibody syndrome at Screening.\n* Subjects with other known pro-thrombotic disorders or abnormal findings in any prior laboratory thrombophilia evaluation.\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\nAdditional Key Exclusion Criterion (Subjects in Part 1 Only)\n\n• Baseline FVIII activity \\> 150 IU\u002FdL.\n\nAdditional Key Exclusion Criteria (Subjects in Parts 2, 3, 4 and 5 Only)\n\n* Baseline FVIII activity \\> 50 IU\u002FdL.\n* Any acute, clinically significant bleeding event requiring surgical or procedural intervention within 7 days prior to receiving study drug.","12 Years","60 Years",{"count":206,"type":19},116,[208,209],"PHASE1","PHASE2","The VIVID study is structured in a master protocol format comprised of multiple parts that evaluate intravenous (IV) and subcutaneous (SC) VGA039 in healthy volunteers and subjects with von Willebrand Disease (VWD) and other bleeding disorders.",[28],[213,214,215,216,217,218,219,220,221,222,223,224],"Von Willebrand Disorder","VWD","Star Therapeutics","Vega Therapeutics","VIVID","VIVID-1","VIVID-2","VIVID-3","VIVID-5","Bleeding disorders","VGA039","Blood coagulation disorders","2025-08-19",{"date":227,"type":40},"2025-08-26",{"date":229,"type":40},"2023-03-16",{"date":231,"type":19},"2029-12",{"name":233,"class":234},"Vega Therapeutics, Inc","INDUSTRY",25,{"id":237,"slug":238,"hasResults":11,"nctId":239,"briefTitle":240,"officialTitle":240,"acronym":241,"eligibilityCriteria":242,"healthyVolunteers":11,"sex":16,"minAge":57,"maxAge":4,"enrollmentInfo":243,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":245,"conditions":246,"keywords":247,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":252,"lastUpdatePostDateStruct":253,"startDateStruct":255,"completionDateStruct":257,"leadSponsor":259,"locationsCount":261},"100498698","cardiovascular-and-venous-thromboembolism-disease-in-patients-with-von-willebrand-disease-in-the-french-west-100498698","NCT05773638","Cardiovascular and Venous Thromboembolism Disease in Patients with Von Willebrand Disease in the French West","TWIGO","Inclusion Criteria:\n\n* Von Willebrand disease\n* Adult\n* Followed-up in a reference center of West of France (Caen, Brest, Nantes, Rennes, Le Mans, Angers)\n* Consented to be included in BERHLINGO database\n* Have presented a thrombotic and\u002For embolic event or cardiovascular disease including deep vein thrombosis, pulmonary embolism, ischemic stroke, transient ischemic attack, acute coronary syndrome, acute limb ischemia or angina or obliterating arteriopathy of the lower limbs, atrial fibrillation.\n* Have not expressed their opposition\n\nExclusion Criteria:\n\n* \\- Expression of opposition\n* Acquired von Willebrand syndrome\n* Patients under legal protection\n* Minor",{"count":244,"type":19},100,"Due to increasing of life expectancy, patients with von Willebrand disease are exposed to age-related pathologies such as cancer or cardiovascular diseases. Management of thrombotic events is challenging given the inherent bleeding risk in von Willebrand disease. Few data are currently available in the literature.\n\nThe aim of the study is to describe the frequency and nature of arterial and venous thromboembolic events and atrial fibrillation in patients with von Willebrand disease in the West of France.\n\nThe investigators will perform a retrospective multicenter study conducted in the von Willebrand population of the French West. Von Willebrand adult patients followed in one of the French West medical centers participating in the study, who presented deep vein thrombosis, pulmonary embolism, ischemic stroke, transient ischemic attack, acute coronary syndrome, acute limb ischemia, atrial fibrillation, arteriopathy of the lower limbs, angina will be eligible.",[28],[248,249,250,251],"von Willebrand disease","venous thromboembolism disease","cardiovascular disease","atrial fibrillation","2025-02-04",{"date":254,"type":40},"2025-02-06",{"date":256,"type":40},"2021-12-26",{"date":258,"type":19},"2026-12-26",{"name":260,"class":80},"University Hospital, Brest",6,{"id":263,"slug":264,"hasResults":11,"nctId":265,"briefTitle":266,"officialTitle":267,"acronym":268,"eligibilityCriteria":269,"healthyVolunteers":11,"sex":153,"minAge":57,"maxAge":4,"enrollmentInfo":270,"targetDuration":4,"studyType":60,"phases":272,"briefSummary":274,"conditions":275,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":276,"lastUpdatePostDateStruct":277,"startDateStruct":279,"completionDateStruct":281,"leadSponsor":283,"locationsCount":81},"100531845","phase-3-a-pilot-crossover-trial-of-prophylactic-wilate-compared-to-placebo-for-heavy-menstrual-bleeding-in-patients-with-vwd-100531845","NCT06205095","A Pilot Crossover Trial of Prophylactic Wilate Compared to Placebo for Heavy Menstrual Bleeding in Patients with VWD","A Multi-cEnter, Pilot, Crossover Trial of Prophylactic Wilate CoMpared to PlacebO for Heavy Menstrual Bleeding in Patients with Von WillEbRand Disease","EMPOWER","Inclusion Criteria:\n\n1. Patient capable of providing informed consent;\n2. Female patients with HMB over the age of 18 years, for whom prophylactic treatment with Wilate® is deemed clinically appropriate according to the medical discretion (based on their expert opinion given consideration of the patient's bleeding history and responsiveness to treatment) of the treating hemostasis-focused physician practicing at a Hemophilia Treatment Center;\n3. Modified PBAC score \\> 100 at screening;\n4. Patients with a diagnosis of inherited von Willebrand disease (any type);\n5. Stable treatment for HMB and iron deficiency anemia for 3 cycles before entering the study and anticipated to remain unchanged for the duration of the study;\n6. Patients willing to have an infusion administered by a nurse over the course of the study period;\n7. Patients who agree to use only the feminine hygiene products supplied by the sponsor.\n\nExclusion Criteria:\n\n1. Diagnosed with any other known bleeding disorder;\n2. Pregnancy or plans to become pregnant within the duration of the study;\n3. Breastfeeding or plans to breastfeed within the duration of the study;\n4. Known hypersensitivity reactions to human plasma-derived products or any ingredient in the formulation;\n5. Known antibodies to VWF or FVIII;\n6. Severe liver disease;\n7. Anticipated initiation of the following: oral, transdermal, injectable, and vaginal ring hormonal contraceptives; GnRH analogues; or a hormonal intrauterine device (IUD) within the study period;\n8. Anticipated elective procedure that is expected to require intensive treatment with VWF or FVIII for \\>10 days during the study period;\n9. Patients with \\>2 risk factors for VTE (risk factors are determined at discretion of treating physician) or recent history of thrombosis (i.e. within the last year).\n10. Patient concurrently receiving desmopressin (desmopressin cannot be taken concurrently with Wilate®, except for in the context of escalation treatment for excessive bleeding).\n11. Anticipated initiation of any new therapies for the treatment of heavy menstrual bleeding 3 weeks prior to enrollment",{"count":271,"type":19},20,[273],"PHASE3","The EMPOWER trial is a pilot multi-center, placebo-controlled (normal saline), double-blind (patient and outcome assessor), crossover, 2-year randomized trial in female outpatients with von Willebrand disease (VWD) and heavy menstrual bleeding to determine trial feasibility and viability, and to explore assay sensitivity of the proposed efficacy clinical outcomes for a definitive randomized controlled trial",[28],"2024-12-19",{"date":278,"type":40},"2024-12-24",{"date":280,"type":40},"2024-10-21",{"date":282,"type":19},"2026-09",{"name":284,"class":80},"Unity Health Toronto",{"id":286,"slug":287,"hasResults":11,"nctId":288,"briefTitle":289,"officialTitle":289,"acronym":290,"eligibilityCriteria":291,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":292,"targetDuration":182,"studyType":20,"phases":4,"briefSummary":294,"conditions":295,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":296,"lastUpdatePostDateStruct":297,"startDateStruct":299,"completionDateStruct":301,"leadSponsor":303,"locationsCount":81},"100310930","world-bleeding-disorders-registry-100310930","NCT03327779","World Bleeding Disorders Registry","WBDR","Inclusion Criteria:\n\n* Patients of participating Hemophilia Treatment Centres with Hemophilia A or B, or von Willebrand Disease\n\nExclusion Criteria:\n\n* none",{"count":293,"type":19},20000,"The WBDR is an international observational disease registry of patients with hemophilia. It will provide a platform for a network of hemophilia treatment centres (HTCs) around the world to collect uniform and standardized patient data and guide clinical practice. With informed consent from the patient, the WBDR stores anonymous data about the person's disease, such as hemophilia type and severity, symptoms, and treatment.",[93,94,28],"2023-08-07",{"date":298,"type":40},"2023-08-14",{"date":300,"type":40},"2018-01-26",{"date":302,"type":19},"2028-01",{"name":304,"class":80},"World Federation of Hemophilia"]