[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hemophilia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hemophilia":27},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,28,0,25,[9,50,79,108,131,154,184,212,261,285,310,335,355,378,401,432,460,470,493,524,549,572,591,616,637],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":4,"eligibilityCriteria":16,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":44,"leadSponsor":46,"locationsCount":49},"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":19,"type":20},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",[24,25,26,27,28,29,30,31,32,33,34,35,36],"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":40,"type":41},"2026-07-13","ACTUAL",{"date":43,"type":41},"2020-09-30",{"date":45,"type":20},"2035-12",{"name":47,"class":48},"American Thrombosis and Hemostasis Network","NETWORK",71,{"id":51,"slug":52,"hasResults":12,"nctId":53,"briefTitle":54,"officialTitle":55,"acronym":56,"eligibilityCriteria":57,"healthyVolunteers":12,"sex":58,"minAge":59,"maxAge":60,"enrollmentInfo":61,"targetDuration":4,"studyType":63,"phases":64,"briefSummary":66,"conditions":67,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":68,"lastUpdatePostDateStruct":69,"startDateStruct":71,"completionDateStruct":73,"leadSponsor":75,"locationsCount":78},"100614889","phase-3-a-study-to-investigate-the-efficacy-and-safety-of-fitusiran-prophylaxis-in-male-participants-aged-1-to-less-than-12-years-with-hemophilia-a-or-b-100614889","NCT07285460","A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B","An Open-label, Parallel, Phase 3, Two-arm Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B With or Without Inhibitory Antibodies to Factors VIII or IX","ATLAS-KIDS","Inclusion Criteria:\n\nParticipants not previously exposed to fitusiran are eligible to be included in the study only if all of the following criteria apply:\n\n* Participant must be 1 to \\\u003C12 years of age at the time of enrollment.\n* Participants must have severe hemophilia A or B (FVIII \\\u003C1% or FIX ≤2%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.\n* Participants must meet inhibitor or non-inhibitor status as defined below:\n\nInhibitor:\n\nRequiring use of BPA for prophylaxis or BPA as on-demand therapy for any bleeding episodes for at least the last 3 months prior to screening, and meet one of the following Nijmegen-modified Bethesda assay results criteria:\n\n* Inhibitor titer of ≥0.6 BU\u002FmL at screening, OR\n* Inhibitor titer of \\\u003C0.6 BU\u002FmL at screening with medical record evidence of 2 consecutive titers ≥0.6 BU\u002FmL, OR\n* Inhibitor titer of \\\u003C0.6 BU\u002FmL at screening with medical record evidence of 1 inhibitor titer ≥0.6 BU\u002FmL and a history of anamnestic response, or severe allergic reaction (eg, anaphylaxis) or nephrotic syndrome\n\nNon-inhibitor:\n\nRequiring use of clotting factor concentrates (CFCs) for prophylaxis or CFCs as on-demand therapy for any bleeding episodes for at least the last 3 months prior to screening, and meet each of the following criterion:\n\n* Nijmegen-modified Bethesda assay inhibitor titer of \\\u003C0.6 BU\u002FmL at screening, AND\n* No use of BPA to treat bleeding episodes for at least the last 3 months prior to screening\n\n  * Participants must have adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol.\n  * Male: There are no contraceptive requirements for this study except where required by local regulations.\n  * Capable of giving signed informed consent\u002Fassent. A signed written informed consent must be obtained from parent(s)\u002Flegal guardian (hereafter referred to as the \"parent\"), as well as a written or oral assent obtained from participant, per local and national requirements.\n\nExclusion Criteria:\n\nParticipants not previously exposed to fitusiran are excluded from the study if any of the following criteria apply:\n\n* Known co-existing bleeding disorders other than hemophilia A or B.\n* Presence of clinically significant liver disease.\n* History of antiphospholipid antibody syndrome.\n* History of arterial or venous thromboembolism, unrelated to an indwelling venous access\n* Any condition (eg, medical concern), which in the opinion of the Investigator, would make the participant unsuitable for dosing or which could interfere with the study compliance, the participant's safety and\u002For the participant's participation in the completion of the treatment period of the study.\n* History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.\n* Subjects with a central or peripheral indwelling catheter, with a history of venous access complications (such as infections, thrombosis) leading to hospitalization and\u002For systemic anticoagulation therapy in the last 12 months.\n* At screening, anticipated need of surgery during the study or planned surgery scheduled to occur during the study.\n* Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional BPA infusion for postoperative hemostasis.\n* History of intolerance to SC injection(s).\n* Current participation in ITI therapy.\n* The use of emicizumab (Hemlibra®) or any non-factor bleed management treatment within 6 months prior to screening\n* Prior gene therapy\n* Current or future participation in another clinical study, scheduled to occur during this study, involving an investigational product other than fitusiran or an investigational device.\n* AT activity \\\u003C60% at screening, as determined by central laboratory analysis.\n* Co-existing thrombophilic disorder.\n* Presence of an active Hepatitis C virus infection\n* Presence of acute hepatitis A or Hepatitis E virus infection.\n* Presence of acute or chronic hepatitis B virus infection.\n* Platelet count ≤100 000\u002FμL.\n* Presence of acute infection at screening.\n* Human immunodeficiency virus (HIV) positive with a CD4 count of \\\u003C400 cells\u002FμL.\n* Estimated glomerular filtration rate ≤45 mL\u002Fmin\u002F1.73 m2 (using the Schwartz formula).\n\nThe above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.","MALE","1 Year","11 Years",{"count":62,"type":20},85,"INTERVENTIONAL",[65],"PHASE3","This is a parallel, Phase 3, two-arm, open-label study to evaluate the efficacy and safety of treatment with fitusiran prophylaxis administered to male pediatric participants (aged 1 to \\\u003C12 years) who have severe hemophilia A or B, with or without inhibitory antibodies to FVIII or FIX.\n\nNumber of participants:\n\nApproximately 85 participants will be enrolled into the study:\n\n* Approximately 60 fitusiran-naïve participants with severe hemophilia A or B, with or without inhibitors (fitusiran-naïve arm), and\n* Approximately 25 participants with severe hemophilia A or B with inhibitors rolling over from the EFC15467\\* dose confirmation study (roll-over arm).\n\n  * Fitusiran has been investigated in the pediatric population in study EFC15467, which enrolled male participants aged 1 to \\\u003C12 years with hemophilia A or B with inhibitors to examine the safety and tolerability of fitusiran in the pediatric population.\n\nParticipants will be enrolled into 1 of 2 arms:\n\n* Fitusiran-naïve: these participants have not previously received fitusiran, and they will undergo screening and study eligibility assessments. Once enrolled, they will go through a 24-week standard of care (SOC) period before starting fitusiran prophylaxis.\n* Roll-over participants from the EFC15467 study: only participants who are still on active treatment in study EFC15467 and consenting to study EFC17905 will be eligible to roll over. They will not need to undergo screening or further eligibility assessments. They will directly enroll into the fitusiran treatment period and continue treatment on their current fitusiran dose.\n\nThe duration of fitusiran treatment will be up to 160 weeks for the fitusiran-naïve arm and up to 60 weeks for the roll-over arm.",[27],"2026-06-24",{"date":70,"type":41},"2026-06-25",{"date":72,"type":41},"2025-12-18",{"date":74,"type":20},"2031-12-30",{"name":76,"class":77},"Sanofi","INDUSTRY",30,{"id":80,"slug":81,"hasResults":12,"nctId":82,"briefTitle":83,"officialTitle":84,"acronym":4,"eligibilityCriteria":85,"healthyVolunteers":12,"sex":17,"minAge":86,"maxAge":87,"enrollmentInfo":88,"targetDuration":4,"studyType":63,"phases":90,"briefSummary":92,"conditions":93,"keywords":95,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":98,"lastUpdatePostDateStruct":99,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":104,"locationsCount":107},"100640335","effects-of-photobiomodulation-in-hemophilia-patients-100640335","NCT07579585","Effects of Photobiomodulation in Hemophilia Patients.","Effects of Photobiomodulation on Knee Hemarthosis in Patients With Hemophilia.","Inclusion Criteria:\n\n* Hemophilia patients with knee hemarthosis\n* Patients of age 9-14 yrs will be taken\n* Bothmaleand female population will be included\n\nExclusion Criteria:\n\n* Presence of open wounds at or near the treatment site.\n* Kneejoint subluxation.\n* Presence of congenital or acquired skeletal deformities.\n* Children diagnosed with cardiopulmonary dysfunctions.\n* Children with neurological deficits","9 Years","14 Years",{"count":89,"type":20},24,[91],"NA","Hemophilia is a genetic bleeding disorder that commonly leads to knee hemarthrosis, causing pain, swelling, and reduced joint mobility in children. While standard treatments include clotting factor replacement and physiotherapy, additional non-invasive approaches are being explored. This study aims to evaluate the effects of photobiomodulation on knee hemarthrosis in male hemophilia patients aged 9-14 years. It focuses on determining whether this therapy can reduce pain and swelling and improve joint range of motion when used alongside",[94,27],"Hemarthrosis, Knee",[96,97,27],"Photobiomodulation","Hemarthrosis","2026-05-05",{"date":100,"type":41},"2026-05-12",{"date":102,"type":41},"2026-04-05",{"date":38,"type":20},{"name":105,"class":106},"Riphah International University","OTHER",1,{"id":109,"slug":110,"hasResults":12,"nctId":111,"briefTitle":112,"officialTitle":112,"acronym":113,"eligibilityCriteria":114,"healthyVolunteers":12,"sex":17,"minAge":115,"maxAge":4,"enrollmentInfo":116,"targetDuration":118,"studyType":21,"phases":4,"briefSummary":119,"conditions":120,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":121,"lastUpdatePostDateStruct":122,"startDateStruct":124,"completionDateStruct":126,"leadSponsor":128,"locationsCount":130},"100592334","adult-and-adolescent-hemophilia-patients-treated-with-marstacimab-a-patient-experience-registry-100592334","NCT06992076","Adult and Adolescent Hemophilia Patients Treated With Marstacimab: a Patient Experience Registry","AMBER","Inclusion Criteria:\n\n\\- Participants with a diagnosis of hemophilia A or hemophilia B eligible to receive prophylaxis.\n\nClinical physicians prescribe marstacimab for routine prophylaxis treatment of hemophilia patients based on the actual conditions of the patients and in accordance with the approved indications of marstacimab.\n\nAdolescent patients(12 to less than 18 years) and adult (18 years and older) patients.\n\nSigned consent obtained before the study, participant or legally authorized representative, or participant's caregiver capable of giving signed informed consent.\n\nExclusion Criteria:\n\n\\- Those who are unable to complete at least one month follow-up based on the investigator's judgment.\n\nSevere impairment of speech, vision, memory or cognition that affects communication and ability to complete questionnaires and follow-up visits.\n\nWomen of childbearing age who plan to become pregnant within the next 2 months, as well as women who are pregnant or breastfeeding.\n\nPatients are participating in other clinical trials. There are other conditions that the investigator deems unsuitable for participation in this study.","12 Years",{"count":117,"type":20},100,"6 Months","Research Title：Adult and adolescent hemophilia patients treated with Marstacimab: a patient Experience Registry (AMBER) Protocol Number：94250855 Protocol Version Number：version 0.3 Date：15-November-2024 Leading site：Institution of Hematology \\& Blood Diseases Hospital, Chinese Academy of Medical Sciences Active Pharmaceutical Ingredient：a human IgG1 monoclonal antibody that targets the tissue factor pathway inhibitor (TFPI) Drug Name Generic name: marstacimab-hncq Purpose of the Study Primary Objectives:To quantify patient preferences for subcutaneous versus intravenous (IV) injection using the Marstacimab-Patient Preference Questionnaire (M-PPQ) after 1 month of marstacimab for routine prophylaxis treatment.\n\nTo assess the treatment burden using the Hemophilia Treatment Experience Measure (Hemo-TEM) in hemophilia patients after 6 months of marstacimab for routine prophylaxis treatment.\n\nExploratory Objectives:\n\nTo evaluate the annualized bleeding rate (ABR) of different types of bleeds after 6 months of subcutaneous marstacimab injections in hemophilia patients.\n\nTo assess changes in joint status scores between baseline and the final visit after 6 months of subcutaneous marstacimab injections in hemophilia patients, using the Hemophilia Early Arthropathy Detection with Ultrasound in China (HEAD-US-C).\n\nTo evaluate patient preferences regarding treatment experience via M-PPQ after 6 months",[27],"2026-04-24",{"date":123,"type":41},"2026-04-29",{"date":125,"type":41},"2026-02-15",{"date":127,"type":20},"2028-12-31",{"name":129,"class":106},"Institute of Hematology & Blood Diseases Hospital, China",2,{"id":132,"slug":133,"hasResults":12,"nctId":134,"briefTitle":135,"officialTitle":136,"acronym":4,"eligibilityCriteria":137,"healthyVolunteers":12,"sex":17,"minAge":115,"maxAge":138,"enrollmentInfo":139,"targetDuration":4,"studyType":63,"phases":141,"briefSummary":142,"conditions":143,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":144,"lastUpdatePostDateStruct":145,"startDateStruct":147,"completionDateStruct":149,"leadSponsor":151,"locationsCount":153},"100586950","phase-3-phase-iii-clinical-trial-of-stsp-0601-for-injection-in-hemophilia-patients-100586950","NCT06922045","Phase III Clinical Trial of STSP-0601 for Injection in Hemophilia Patients","A Phase III Clinical Study on the Efficacy and Safety of STSP-0601 for Injection in Patients With Hemophilia Associated With Inhibitors, With a Multicenter, Open Evaluation Approach","Inclusion Criteria:\n\n1. 12 ≤age≤70 years of age.\n2. Hemophilia A or B patients.\n3. Peak historical inhibitor titer ≥ 5 BU and a positive inhibitor test when enrolled.\n4. Establish proper venous access.\n5. There were at least 3 bleeding events that required treatment occurred in the past 6 months before screening.\n6. Agree to use adequate contraception to avoid pregnancy.\n7. Provide signed informed consent.\n\nExclusion Criteria:\n\n1. Have any coagulation disorder other than hemophilia.\n2. Plan to receive prophylactic treatment of coagulation factor during the trail.\n3. Patients plan to receive Emicizumab during the trial.\n4. Patients received anticoagulant or antifibrinolytic therapy 7 days before the first administration or plan to receive these drugs during the trial.\n5. Have a history of arterial and\u002For venous thrombotic events.\n6. Platelet \\\u003C100×109\u002FL.\n7. Hemoglobin\\\u003C90g\u002FL.\n8. Severe liver or kidney disease.\n9. Severe bleeding event occurred within 4 weeks before the first administration.\n10. Accepted major operation or blood transfusion within 4 weeks before the first administration.\n11. Have a known allergy to STSP-0601.\n12. Pregnant, lactating, or blood pregnancy test positive female subjects\n13. Participate in other clinical research within 4 weeks before enrollment (except for participating in prothrombin complex, FVII, FVIIa, FVIII, FIX trails).\n14. Within 1 day before the first administration, FVII, FVIIa, tranexamic acid, and aminocaproic acid were used. Within 3 days before the first administration, prothrombin complex, FVIII, and FIX were used. Within 4 weeks, treatment with Emicizumab was received.\n15. Patients not suitable for the trail according to the judgment of the investigators.","70 Years",{"count":140,"type":20},40,[65],"This study will assess the efficacy of multiple-dose of STSP-0601 for the treatment of bleeding episodes in hemophilia A or B patients with inhibitor",[27],"2026-04-22",{"date":146,"type":41},"2026-04-23",{"date":148,"type":41},"2025-03-26",{"date":150,"type":20},"2027-03",{"name":152,"class":77},"Jiangsu BioJeTay Biotechnology Co., Ltd.",18,{"id":155,"slug":156,"hasResults":12,"nctId":157,"briefTitle":158,"officialTitle":159,"acronym":4,"eligibilityCriteria":160,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":161,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":163,"conditions":164,"keywords":169,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":176,"lastUpdatePostDateStruct":177,"startDateStruct":179,"completionDateStruct":181,"leadSponsor":183,"locationsCount":107},"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":162,"type":20},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.",[27,28,165,166,36,167,25,168,29],"Hemophilia A","Hemophilia B","Glanzmann Thrombasthenia","Blood Disorder",[170,171,172,173,174,175],"bleed event","bleed treatments","adverse events","joint bleed","bleeding disorder","bleeding symptoms","2026-04-16",{"date":178,"type":41},"2026-04-21",{"date":180,"type":41},"2024-10-25",{"date":182,"type":20},"2055-10-31",{"name":47,"class":48},{"id":185,"slug":186,"hasResults":12,"nctId":187,"briefTitle":188,"officialTitle":189,"acronym":4,"eligibilityCriteria":190,"healthyVolunteers":12,"sex":58,"minAge":191,"maxAge":4,"enrollmentInfo":192,"targetDuration":194,"studyType":21,"phases":4,"briefSummary":195,"conditions":196,"keywords":197,"overallStatus":203,"whyStopped":4,"lastUpdateSubmitDate":204,"lastUpdatePostDateStruct":205,"startDateStruct":207,"completionDateStruct":208,"leadSponsor":210,"locationsCount":4},"100632672","central-pain-mechanisms-and-clinical-and-psychological-factors-associated-with-pain-interference-in-daily-life-in-adults-with-hemophilia-and-hemophilic-arthropathy-100632672","NCT07516730","Central Pain Mechanisms and Clinical and Psychological Factors Associated With Pain Interference in Daily Life in Adults With Hemophilia and Hemophilic Arthropathy","Central Pain Mechanisms and Clinical and Psychological Factors Associated With Pain Interference in Daily Life in Adults With Hemophilia and Hemophilic Arthropathy: A Cross-Sectional Observational Study","Inclusion Criteria:\n\n* Diagnosis of hemophilia A or B\n* Age equal to or greater than 35 years\n* Clinical diagnosis of hemophilic arthropathy in at least one lower limb joint\n* Clinically relevant joint involvement, defined as a total score on the Hemophilia Joint Health Score (HJHS) greater than 4 points\n* Stable prophylactic treatment with FVIII\u002FFIX concentrates or monoclonal antibodies\n* Provision of written informed consent\n\nExclusion Criteria:\n\n* Presence of neurological or cognitive impairments that prevent understanding of the questionnaires or performance of the study tests\n* Episode of hemarthrosis in the lower limbs within the 3 months prior to assessment\n* Use of analgesics, nonsteroidal anti-inflammatory drugs, opioids, or other medications with potential effects on pain perception or modulation within the 72 hours prior to assessment\n* Receiving, at the time of the study, physiotherapy, infiltrative, or orthotic interventions aimed at pain or function of joints affected by hemophilic arthropathy","35 Years",{"count":193,"type":20},138,"1 Day","Introduction. Chronic pain is a frequent complication in adults with hemophilia and hemophilic arthropathy that affects functionality and quality of life. In addition to joint damage, central pain mechanisms and psychological factors may contribute to its functional impact, although evidence in this population is limited.\n\nObjective. To analyze the association between central pain mechanisms, pain intensity, and pain-related anxiety and pain interference in daily life in adults with hemophilia and hemophilic arthropathy, adjusting for relevant clinical variables.\n\nMethods. An analytical observational study with a cross-sectional design will be conducted in 138 adults with hemophilia and hemophilic arthropathy. The dependent variable will be pain interference in daily life (Brief Pain Inventory), the main predictor variables will be central sensitization (Central Sensitization Inventory), conditioned pain modulation (Conditioned Pain Modulation Index), global pain intensity (severity subscale of the Brief Pain Inventory), and pain-related anxiety (Pain Anxiety Symptoms Scale-20). As secondary predictor variables, sleep quality (Pittsburgh Sleep Quality Index) and pain self-efficacy (Pain Self-Efficacy Questionnaire) will be included. As confounding variables, joint damage, age, type of treatment, and history of inhibitor will be considered. The association between variables will be analyzed using multiple linear regression models adjusted for relevant clinical covariates.\n\nExpected results. It is expected to identify factors associated with pain interference in adults with hemophilia and hemophilic arthropathy, improving the understanding of its functional impact.",[27],[27,198,199,200,201,202],"Chronic pain","Pain measurement","Pain perception","Anxiety","Self-efficacy","NOT_YET_RECRUITING","2026-04-01",{"date":206,"type":41},"2026-04-08",{"date":144,"type":20},{"date":209,"type":20},"2026-07-11",{"name":211,"class":48},"Investigación en Hemofilia y Fisioterapia",{"id":213,"slug":214,"hasResults":12,"nctId":215,"briefTitle":216,"officialTitle":217,"acronym":218,"eligibilityCriteria":219,"healthyVolunteers":220,"sex":17,"minAge":221,"maxAge":222,"enrollmentInfo":223,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":225,"conditions":226,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":251,"lastUpdatePostDateStruct":252,"startDateStruct":254,"completionDateStruct":256,"leadSponsor":258,"locationsCount":260},"100383878","pharmacokinetics-pharmacodynamics-and-safety-profile-of-understudied-drugs-administered-to-children-per-standard-of-care-pops-100383878","NCT04278404","Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)","Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs","POPS or POP02","Inclusion Criteria:\n\n1. Participant is \\\u003C 21 years of age\n2. Parent\u002F Legal Guardian\u002F Adult Participant can understand the consent process and is willing to provide informed consent\u002FHIPAA:\n3. (a) Participant is receiving one or more of the study drugs of interest at the time of enrollment or (b) Participant is NOT receiving one or more of the study drugs of interest but is SARS-COV-2 positive within 60 days prior to enrollment\n\nExclusion Criteria:\n\n1. Participant has a known pregnancy\n\n   Below exclusion criteria apply only to:\n\n   Participants receiving one or more of the study drugs of interest at the time of enrollment, DOI administration or PK sampling: (Refer to DOI specific appendices for details on enrollment cohort specifications and additional eligibility criteria)\n2. Has had intermittent dialysis within previous 24 hours\n3. Has had a kidney transplant within previous 30 days\n4. Has had a liver transplant within previous 1 year\n5. Has had a stem cell transplant within previous 1 year\n6. Has had therapeutic hypothermia within previous 24 hours\n7. Has had plasmapheresis within the previous 24 hours\n8. Has a Ventricular Assist Device\n9. Has any condition which would make the participant, in the opinion of the investigator, unsuitable for the study",true,"0 Years","20 Years",{"count":224,"type":20},5000,"The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose for children. The primary objective of this study is to evaluate the PK of understudied drugs currently being administered to children per SOC as prescribed by their treating provider.",[227,228,229,230,231,232,233,234,235,236,27,237,238,239,240,241,242,243,244,245,246,247,248,249,250],"Coronavirus Infection (COVID-19)","Pulmonary Arterial Hypertension","Urinary Tract Infections in Children","Hypertension","Pain","Hyperphosphatemia","Primary Hyperaldosteronism","Edema","Hypokalemia","Heart Failure","Menorrhagia","Insomnia","Pneumonia","Skin Infection","Arrythmia","Asthma in Children","Bronchopulmonary Dysplasia","Adrenal Insufficiency","Fibrinolysis; Hemorrhage","Attention Deficit Hyperactivity Disorder","Multisystem Inflammatory Syndrome in Children (MIS-C)","Kawasaki Disease","Coagulation Disorder","Down Syndrome","2026-03-31",{"date":253,"type":41},"2026-04-06",{"date":255,"type":41},"2020-03-05",{"date":257,"type":20},"2027-07",{"name":259,"class":106},"Duke University",51,{"id":262,"slug":263,"hasResults":12,"nctId":264,"briefTitle":265,"officialTitle":266,"acronym":267,"eligibilityCriteria":268,"healthyVolunteers":12,"sex":17,"minAge":269,"maxAge":4,"enrollmentInfo":270,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":272,"conditions":273,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":276,"lastUpdatePostDateStruct":277,"startDateStruct":279,"completionDateStruct":281,"leadSponsor":283,"locationsCount":130},"100600779","monitoring-of-anti-tfpi-in-hemophilia-100600779","NCT07101926","Monitoring of Anti-TFPI in Hemophilia","Monitoring of Anti-TFPI in Hemophilia EUREKA","EUREKA","* Inclusion Criteria :\n\n  * \\- Male Patient\n  * 18 years old\n  * severe hemophilia patients A or B (FVIII \\\u003C 1%,FIX\\\u003C=2%)\n  * on prophylaxis with FVIII or IX concentrates after an adequate washout period of 48h for SHL FVIII molecules, at least 4 days for EHL-FVIII Fc and at least 10 days for EHL-FIX molecules.\n  * On prophylaxis with Marstacimab\n  * Willing to participate\n  * Capable of following protocol procedures under investigator appreciation\n* Exclusion Criteria :\n\n  * \\- patients refusing to provide 4 additional blood tubes for research\n  * Patient with an other coagulation disorder\n  * patients who received an injection of FVIII or FIX during the required washout period","18 Years",{"count":271,"type":20},11,"During the development of anti-TFPI antibodies, thrombin generation assay (TGA) was employed using both in vitro measurements (antibodies added to blood samples) and ex vivo approaches (blood samples from patients in phase II and III trials). While a significant improvement in thrombin generation was observed in all samples from patients with severe hemophilia, no correlation with clinical outcomes could be established. Notably, thrombin peak levels were consistently improved even in patients who experienced bleeding episodes. These measurements were conducted in platelet-poor plasma (PPP) with standard reagents, which may not adequately reflect the hemostatic efficacy of anti-TFPI antibodies given their mechanism of action. It is hypothesized that optimizing reagents and utilizing more appropriate biological materials could enhance TGA sensitivity, as previously demonstrated for monitoring emicizumab.\n\nThe absence of a laboratory assay to monitor anti-TFPI (tissue factor pathway inhibitor) antibodies poses a significant challenge for managing patients in surgical settings and treating acute severe bleeding. This study aims to develop a reliable assay to evaluate the hemostatic efficacy of anti-TFPI antibodies and their combined procoagulant effect with factor concentrates (FVIII or FIX) or bypassing agents.",[27,274,275],"Rebalancing Agents","Prophylaxis","2026-03-19",{"date":278,"type":41},"2026-03-23",{"date":280,"type":41},"2025-10-23",{"date":282,"type":20},"2026-08",{"name":284,"class":106},"Hospices Civils de Lyon",{"id":286,"slug":287,"hasResults":12,"nctId":288,"briefTitle":289,"officialTitle":290,"acronym":4,"eligibilityCriteria":291,"healthyVolunteers":12,"sex":17,"minAge":292,"maxAge":269,"enrollmentInfo":293,"targetDuration":4,"studyType":63,"phases":294,"briefSummary":295,"conditions":296,"keywords":297,"overallStatus":203,"whyStopped":4,"lastUpdateSubmitDate":302,"lastUpdatePostDateStruct":303,"startDateStruct":305,"completionDateStruct":306,"leadSponsor":308,"locationsCount":4},"100627741","3d-ultrasound-in-hemophilic-ankles-100627741","NCT07452575","3D Ultrasound in Hemophilic Ankles","Can Ultrasound \"See\" What Clinicians Cannot in Hemophilic Arthropathy? Point-of-Care Ultrasound and Physical Examination Perspectives","Inclusion Criteria:\n\n* Diagnosis of hemophilia A or B or other bleeding disorders\n* Cooperative patients\n* Previous study joint bleed (by history) or suspicion of an acute joint bleed\n\nExclusion Criteria:\n\n* Co-morbid chronic illnesses causing osteoarticular findings\n* MRI contraindications","5 Years",{"count":271,"type":20},[91],"The study examines whether a mechanical arm ultrasound system provides diagnostic accuracy comparable to expert-performed full ultrasound for detecting key joint components and whether it can reduce acquisition variability without compromising accuracy, as measured by false-positive and false-negative rates.",[27],[298,299,300,301],"hemophilia","ultrasound","mechanical arm","children","2026-03-02",{"date":304,"type":41},"2026-03-05",{"date":204,"type":20},{"date":307,"type":20},"2027-12-31",{"name":309,"class":106},"The Hospital for Sick Children",{"id":311,"slug":312,"hasResults":12,"nctId":313,"briefTitle":314,"officialTitle":315,"acronym":4,"eligibilityCriteria":316,"healthyVolunteers":12,"sex":58,"minAge":317,"maxAge":4,"enrollmentInfo":318,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":320,"conditions":321,"keywords":322,"overallStatus":203,"whyStopped":4,"lastUpdateSubmitDate":327,"lastUpdatePostDateStruct":328,"startDateStruct":330,"completionDateStruct":332,"leadSponsor":334,"locationsCount":107},"100625262","incidence-of-ultrasonographically-detected-articular-damage-in-initially-healthy-joints-of-patients-with-hemophilia-a-receiving-prophylactic-treatment-with-emicizumab-100625262","NCT07420348","Incidence of Ultrasonographically Detected Articular Damage in Initially Healthy Joints of Patients With Hemophilia A Receiving Prophylactic Treatment With Emicizumab","Incidence of Ultrasonographically Detected Articular Damage in Initially Healthy Joints of Patients With Hemophilia A Receiving Prophylactic Treatment With Emicizumab. A Prospective Observational Study","Inclusion Criteria:\n\n* Confirmed diagnosis of hemophilia A;\n* Receiving prophylactic treatment with emicizumab (Hemlibra®) according to routine clinical practice;\n* Age ≥10 years at the time of study enrollment;\n* Absence of ultrasonographic joint damage in at least one of the evaluated joints (elbows, knees, or ankles), defined as HEAD-US = 0 at baseline assessment;\n* No documented history of clinically evident hemarthrosis in the corresponding joints from initiation of emicizumab prophylaxis to the study baseline evaluation;\n* Ability to understand and complete study procedures (interviews, questionnaires, and clinical assessments), in accordance with the participant's age; and\n* Provision of written informed consent; for minors, written informed consent from parents or legal guardians and assent from the minor participant, in accordance with applicable regulations.\n\nExclusion Criteria:\n\n* Presence of ultrasonographic joint damage (HEAD-US ≥1) in all evaluated joints at the baseline visit;\n* Documented history of clinically evident hemarthrosis in the joints under study;\n* Prior major orthopedic surgery or arthroplasty in the evaluated joints;\n* Presence of concomitant musculoskeletal pathology unrelated to hemophilia that could interfere with joint assessment (e.g., inflammatory arthritis, recent severe trauma);\n* Inability to complete the planned 24-month follow-up or to undergo study assessments;\n* Concurrent participation in another interventional study that could interfere with the joint health variables under evaluation; and\n* Any clinical or social condition that, in the investigator's judgment, could compromise participant safety or the validity of the study data.","10 Years",{"count":319,"type":20},70,"Introduction: Prophylaxis with emicizumab has substantially improved hemorrhagic control in hemophilia A. However, the longitudinal incidence of ultrasonographically detected articular damage in initially healthy joints remains insufficiently characterized.\n\nObjective: To estimate the incidence of ultrasonographically detected articular damage in initially healthy joints among patients with hemophilia A receiving prophylaxis with emicizumab and to explore its association with relevant clinical variables.\n\nMethods: A prospective, longitudinal, observational study will be conducted in approximately 70 patients with hemophilia A receiving emicizumab, with an estimated recruitment of approximately 270 initially healthy joints. The study is purely observational and does not involve evaluation of the investigational product nor modification of the therapeutic regimen; dosing, administration intervals, and all clinical decisions regarding emicizumab will be determined exclusively at the discretion of the treating hematologist. Assessments will be performed at baseline and at 12 and 24 months. The unit of analysis will be the joint, including elbows, knees, and ankles without ultrasonographic evidence of articular damage and without a history of clinically evident hemarthrosis at study entry.\n\nThe primary endpoint will be the occurrence of incident ultrasonographically detected articular damage, assessed using the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) protocol. Secondary outcomes will include clinical joint health assessed by the Hemophilia Joint Health Score (HJHS), version 2.1; the frequency of joint hemarthroses measured by the annualized joint bleeding rate (AJBR); and habitual physical activity levels evaluated through age-specific validated questionnaires. Statistical analyses will account for intra-patient correlation among joints.\n\nExpected Results: A low to moderate incidence of ultrasonographically detected articular damage in initially healthy joints is anticipated during follow-up, providing clinically relevant information regarding structural joint preservation in patients with hemophilia A receiving emicizumab prophylaxis.",[27],[323,324,325,326],"Joint damage","emicizumab","prophylaxis","joint preservation","2026-02-18",{"date":329,"type":41},"2026-02-20",{"date":331,"type":20},"2026-02-27",{"date":333,"type":20},"2028-05-03",{"name":211,"class":48},{"id":336,"slug":337,"hasResults":12,"nctId":338,"briefTitle":339,"officialTitle":340,"acronym":4,"eligibilityCriteria":341,"healthyVolunteers":12,"sex":58,"minAge":269,"maxAge":4,"enrollmentInfo":342,"targetDuration":4,"studyType":63,"phases":343,"briefSummary":344,"conditions":345,"keywords":346,"overallStatus":203,"whyStopped":4,"lastUpdateSubmitDate":327,"lastUpdatePostDateStruct":349,"startDateStruct":350,"completionDateStruct":352,"leadSponsor":354,"locationsCount":107},"100625265","effectiveness-of-an-educational-program-for-pain-management-in-patients-with-hemophilic-arthropathy-100625265","NCT07420387","Effectiveness of an Educational Program for Pain Management in Patients With Hemophilic Arthropathy","Effectiveness of an Educational Program for Pain Management in Patients With Hemophilic Arthropathy: A Randomized Controlled Trial","Inclusion Criteria:\n\n* Adult patients with a confirmed diagnosis of hemophilia A or B.\n* Presence of hemophilic arthropathy.\n* Presence of chronic pain.\n\nExclusion Criteria:\n\n* Patients with neurological or cognitive impairments that preclude understanding of the educational sessions.\n* Patients who have undergone surgical intervention within the two months preceding the educational program",{"count":319,"type":20},[91],"Introduction: Hemophilic arthropathy is a common complication of hemophilia, characterized by chronic pain, functional limitation, and impaired quality of life. Cognitive-emotional factors such as catastrophizing and kinesiophobia significantly influence the pain experience, supporting the rationale for interventions grounded in the biopsychosocial model and pain neuroscience education.\n\nObjective: To evaluate the efficacy of an educational program based on pain neurobiology, emotional regulation, and cognitive-behavioral strategies on the pain experience in adult patients with hemophilic arthropathy.\n\nMethods: A randomized, controlled clinical trial with two parallel groups (intervention and control) and three assessment time points (pre-intervention, post-intervention, and 6-month follow-up) will be conducted. A total of 70 adult patients with hemophilia A or B and a diagnosis of hemophilic arthropathy with chronic pain will be enrolled and randomly assigned in a 1:1 ratio. The intervention group will receive a structured educational program consisting of three 60-minute sessions focused on pain neurobiology, emotional regulation, cognitive restructuring, coping strategies, and physiological downregulation techniques, including supervised physical activity as an analgesic strategy. The control group will continue with usual care without additional educational intervention. The primary outcome will be pain intensity and pain interference, assessed using the Brief Pain Inventory. Statistical analyses will be performed using repeated-measures ANOVA, with the Group × Time interaction considered the primary effect of interest, under the intention-to-treat principle.\n\nExpected Results: It is anticipated that the intervention group will demonstrate a statistically and clinically significant reduction in pain intensity and pain-related functional interference compared with the control group. A sustained clinical improvement at six months is also expected, supporting the utility of structured educational interventions as a safe and complementary strategy in the management of chronic pain in patients with hemophilic arthropathy.",[27],[27,347,198,348],"Hemophilic arthropathy","Pain neuroscience education",{"date":329,"type":41},{"date":351,"type":20},"2026-03-27",{"date":353,"type":20},"2027-09-11",{"name":211,"class":48},{"id":356,"slug":357,"hasResults":12,"nctId":358,"briefTitle":359,"officialTitle":360,"acronym":4,"eligibilityCriteria":361,"healthyVolunteers":220,"sex":58,"minAge":362,"maxAge":269,"enrollmentInfo":363,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":365,"conditions":366,"keywords":369,"overallStatus":203,"whyStopped":4,"lastUpdateSubmitDate":371,"lastUpdatePostDateStruct":372,"startDateStruct":373,"completionDateStruct":375,"leadSponsor":376,"locationsCount":107},"100625645","validity-and-reliability-of-the-two-minute-step-test-in-hemophilia-100625645","NCT07425327","Validity and Reliability of the Two-minute Step Test in Hemophilia","Investigating the Validity and Reliability of the Two-minute Step Test in Patients With Hemophilia","Inclusion Criteria:\n\n* Diagnosed with Hemophilia (A-B) by a physician,\n* Aged 6-18 years,\n* Receiving regular prophylactic treatment,\n* Inhibitor positive or negative,\n* No history of lower extremity joint bleeding within the last month,\n* Individuals who are willing to participate in the study,\n\nExclusion Criteria:\n\n* Patients who have undergone surgery on the lower extremities for any reason within the last 6 months,\n* Patients with neurological sequelae following a history of intracranial hemorrhage,\n* Patients experiencing cognitive impairment to the extent that they cannot understand the tests,\n* Patients who indicate they will be unable to participate in follow-up measurements.","6 Years",{"count":364,"type":20},35,"The aim of our study was to evaluate the psychometric properties of the 2-minute step test in hemophilia patients, assess its intra-rater and inter-rater reliability, and evaluate its convergent validity and construct validity supported by measurements of gait, balance, and functionality.",[27,367,368],"Step","Reliability and Validity",[165,166,370],"Two minute step test","2026-02-13",{"date":329,"type":41},{"date":374,"type":20},"2026-02-25",{"date":70,"type":20},{"name":377,"class":106},"Hasan Kalyoncu University",{"id":379,"slug":380,"hasResults":12,"nctId":381,"briefTitle":382,"officialTitle":383,"acronym":384,"eligibilityCriteria":385,"healthyVolunteers":12,"sex":17,"minAge":115,"maxAge":386,"enrollmentInfo":387,"targetDuration":4,"studyType":63,"phases":388,"briefSummary":390,"conditions":391,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":393,"lastUpdatePostDateStruct":394,"startDateStruct":396,"completionDateStruct":398,"leadSponsor":400,"locationsCount":107},"100624169","phase-4-pcc-treatment-for-hemophilia-patients-with-inhibitor2022pcc-a-100624169","NCT07406139","PCC Treatment for Hemophilia Patients With Inhibitor（2022PCC-A）","A Multicenter, Prospective, Single-arm Exploratory Study of Prothrombin Complex Concentrate in the Treatment of Bleeding Episodes in Patients With Hemophilia A With Inhibitors","2022PCC-A","Inclusion Criteria:\n\n1. Diagnosed with hemophilia A with inhibitors. For high-responding patients (those who have previously had an inhibitor titer \\>5 BU), the inhibitor titer at enrollment must be \\>0.6 BU;\n2. Age between 12 and 65 years;\n3. At least three joint bleeding episodes within the past six months;\n4. Signed informed consent form.\n\nExclusion Criteria:\n\n1. Presence of other congenital or acquired bleeding disorders;\n2. Liver function tests (ALT, AST) \\>2.5 times the upper limit of normal, or renal function tests (BUN, Cr) \\>1.5 times the upper limit of normal;\n3. Currently receiving immune tolerance induction (ITI) therapy with an inhibitor titer \\\u003C5 BU;\n4. History of thrombotic events;\n5. Known history of drug allergy, asthma, urticaria, or other allergic conditions;\n6. Deemed unsuitable for study participation by the investigator.","65 Years",{"count":78,"type":20},[389],"PHASE4","This study is a multicenter, prospective, single-arm exploratory clinical trial designed to evaluate the efficacy and safety of prothrombin complex concentrate (PCC) in the treatment of bleeding episodes in patients with hemophilia A with inhibitors. All participants received on-demand PCC therapy during bleeding episodes, with dosing adjusted by investigators according to the type of bleeding. The recommended dose was 50 IU\u002Fkg per infusion, administered every 8-12 hours, with a maximum total daily dose not exceeding 150 IU\u002Fkg. If no effective hemostasis was achieved within 24 hours, investigators could decide to add other hemostatic agents or switch to alternative treatments.",[27,392],"Inhibitors","2026-02-09",{"date":395,"type":41},"2026-02-12",{"date":397,"type":20},"2026-03-01",{"date":399,"type":20},"2026-12-20",{"name":129,"class":106},{"id":402,"slug":403,"hasResults":12,"nctId":404,"briefTitle":405,"officialTitle":406,"acronym":407,"eligibilityCriteria":408,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":409,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":411,"conditions":412,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":393,"lastUpdatePostDateStruct":426,"startDateStruct":427,"completionDateStruct":429,"leadSponsor":431,"locationsCount":107},"100412026","national-longitudinal-cohort-of-hematological-diseases-100412026","NCT04645199","National Longitudinal Cohort of Hematological Diseases","National Longitudinal Cohort of Hematological Diseases (NICHE)","NICHE","Inclusion Criteria:\n\n* Patients who were diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders or received bone marrow transplantation in the investigating hospitals from January 1, 2020.\n\nExclusion Criteria:\n\n* Long-term follow-up information for patients is not available for any reason, such as not being available or having a serious concomitant disease.\n* Alcohol and drug addictions affect their ability to comply with study requirements.\n* According to the investigator, there are conditions that may endanger the patient's safety or affect his\u002Fher compliance.",{"count":410,"type":20},2300,"Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in hematology. The purpose of this study is to analyze the incidence and risk factors of major blood diseases, the treatment methods, prognosis and medical expenses of these patients in China.\n\nMethod The study will include patients diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders, autoimmune hemolytic anemia, large granular lymphocyte leukemia, essential thrombocythemia, blood infection or received bone marrow transplantation in the investigating hospitals from January 1, 2020, and collect basic information, diagnostic and treatment information, prognosis information, as well as medical expense information from medical records. In its current form, the NICHE registry incorporates historical data (collected from 2000) and is systematically collecting prospective data in two phases with broadening reach, and prospectively follow-up to collect the prognosis information.",[413,414,27,165,166,415,416,417,418,419,25,420,421,422,423,424,425],"Multiple Myeloma","Acute Myeloid Leukemia","Myelodysplastic Syndrome","MDS","Lymphoma","Leukemia","Aplastic Anemia","Bone Marrow Transplantation","Blood Disease Infection","Autoimmune Hemolytic Anemia, AIHA","Essential Thrombocythemia, ET","Large Granular Lymphocyte Leukemia, LGLL","Paroxysmal Nocturnal Hemoglobinuria, PNH",{"date":395,"type":41},{"date":428,"type":41},"2020-12-01",{"date":430,"type":20},"2030-12-01",{"name":129,"class":106},{"id":433,"slug":434,"hasResults":12,"nctId":435,"briefTitle":436,"officialTitle":437,"acronym":4,"eligibilityCriteria":438,"healthyVolunteers":12,"sex":17,"minAge":439,"maxAge":269,"enrollmentInfo":440,"targetDuration":4,"studyType":63,"phases":441,"briefSummary":442,"conditions":443,"keywords":444,"overallStatus":203,"whyStopped":4,"lastUpdateSubmitDate":451,"lastUpdatePostDateStruct":452,"startDateStruct":454,"completionDateStruct":456,"leadSponsor":458,"locationsCount":107},"100605367","oral-health-status-and-oral-health-related-quality-of-life-in-a-group-pf-egyptian-hemophilic-children-100605367","NCT07161596","Oral Health Status and Oral Health Related Quality of Life in a Group pf Egyptian Hemophilic Children","Oral Health Status and Oral Health Related Quality of Life of a Group of Hemophilic Egyptian Children Before and After an Oral Health Educational Program: A Before and After Study","Inclusion Criteria:\n\n* both sexes having hemophilia A,B or C\n* no associated co morbid diseases\n* mild, moderate and severe forms of hemophilia\n* children whose parents agreed to participate in the study\n\nExclusion Criteria:\n\n* patients with psychiatric disorders\n* children whose parents refused to participate in the study\n* patients using drugs( as cyclosporine, phenytoin) inducing periodontal problems","8 Years",{"count":319,"type":20},[91],"aim of the study:\n\n1. to assess, investigate, and compare the oral health status and oral health related quality of life of a group of hemophilic Egyptian children before and after an oral health educational program\n2. evaluate the effectiveness of oral health educational programs on the oral health status of the Egyptian hemophilic children\n\nbenefits to patients: oral health awareness and change in the oral care levels with methods that match their health needs benefits to clinician: better understanding of the oral conditions associated with hemophilic patients, and their effects on oral health benefits to community: improvement of children's oral health, oral health awareness, promoting the importance of regular dental check ups for children with hemophilia",[27],[445,446,447,448,449,450],"oral hygiene index simplified","oral health related quality of life","oral health status","def","DMF","Hemophilic children","2026-01-21",{"date":453,"type":41},"2026-01-23",{"date":455,"type":20},"2026-04",{"date":457,"type":20},"2027-02",{"name":459,"class":106},"Cairo University",{"id":461,"slug":4,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":4,"eligibilityCriteria":16,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":462,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":22,"conditions":463,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":464,"lastUpdatePostDateStruct":465,"startDateStruct":467,"completionDateStruct":468,"leadSponsor":469,"locationsCount":49},"100393100",{"count":19,"type":20},[24,25,26,27,28,29,30,31,32,33,34,35,36],"2026-01-09",{"date":466,"type":41},"2026-01-12",{"date":43,"type":41},{"date":45,"type":20},{"name":47,"class":48},{"id":471,"slug":472,"hasResults":12,"nctId":473,"briefTitle":474,"officialTitle":475,"acronym":4,"eligibilityCriteria":476,"healthyVolunteers":12,"sex":17,"minAge":269,"maxAge":4,"enrollmentInfo":477,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":479,"conditions":480,"keywords":4,"overallStatus":203,"whyStopped":4,"lastUpdateSubmitDate":484,"lastUpdatePostDateStruct":485,"startDateStruct":487,"completionDateStruct":489,"leadSponsor":491,"locationsCount":4},"100610864","mri-role-in-knee-hemophilic-arthopathy-100610864","NCT07233122","MRI Role in Knee Hemophilic Arthopathy","MRI Vs Conventional Radiography in Assessing Knee Hemophilic Arthropathy : A Scoring Based Approach","Inclusion Criteria:\n\n* Adult patient ( more than 18 years old ) diagnosed as Haemophilia with history of bleeding in knee joint more than once\n\nExclusion Criteria:\n\n* \\*contraindications for MRI such as claustrophobia , metallic foriegn body carriers , cardiac pacemaker\n\n  * patient refused the exam\n  * Pregnant female patients",{"count":478,"type":20},95,"The aim of this study is to assess role of MRI in detecting synovial, cartilaginous , osseous abnormalities ، bleeding inside knee joint and to use a system for assessing HA as support for therapeutic regimes and for monitoring response to therapy .",[481,27,482,483],"MRI","Arthropathy Hemophilic","Arthropathy of Knee","2025-11-16",{"date":486,"type":41},"2025-11-18",{"date":488,"type":20},"2025-12-01",{"date":490,"type":20},"2027-12-01",{"name":492,"class":106},"Assiut University",{"id":494,"slug":495,"hasResults":12,"nctId":496,"briefTitle":497,"officialTitle":498,"acronym":499,"eligibilityCriteria":500,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":501,"targetDuration":503,"studyType":21,"phases":4,"briefSummary":504,"conditions":505,"keywords":513,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":515,"lastUpdatePostDateStruct":516,"startDateStruct":518,"completionDateStruct":520,"leadSponsor":522,"locationsCount":107},"100608743","high-altitude-hematology-observation-stem-cell-transplantation-halo-sct-100608743","NCT07205523","High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT)","High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT): A Prospective Real-World Cohort Study in the Qinghai-Tibet Plateau","HALO-SCT","Inclusion Criteria:\n\n1. Patients diagnosed with hematologic diseases who are admitted to the HSCT center of Qinghai University Affiliated Hospital on or after September 1, 2023.\n2. Planned or actual hematopoietic stem cell transplantation (HSCT).\n3. Provision of signed informed consent.\n\nExclusion Criteria:\n\n1. Inability to provide long-term follow-up data due to severe comorbidities or logistical reasons.\n2. Substance abuse compromising adherence.\n3. Any condition judged by investigators to jeopardize safety or compliance.",{"count":502,"type":20},1000,"100 Years","The High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT) study is the first prospective real-world cohort of hematologic diseases and transplantation in the Qinghai-Tibet Plateau. Patients undergoing hematopoietic stem cell transplantation (HSCT) at Qinghai University Affiliated Hospital, together with their donors, are systematically enrolled. The registry collects demographic, diagnostic, treatment, prognosis, and medical expense information, as well as biospecimens for future analyses. Historical data are incorporated, and prospective data collection is ongoing with long-term follow-up planned. The registry is designed as a sustainable research infrastructure to provide comprehensive data on disease incidence, treatment patterns, outcomes, and resource utilization in a high-altitude setting.",[506,507,508,509,27,415,416,417,418,419,510,420,413,511,512],"Hematopoietic Stem Cell Transplantation (HSCT)","Acute Myeloid Leukemia (AML)","Leukemias, Acute Myeloid","Myeloid Leukemias, Acute","Bleeding Disorders","Myeloma, Multiple","Immune Reconstitution",[514],"High-altitude Bone marrow transplantation Allogeneic HSCT Autologous HSCT Immune reconstitution Graft-versus-host disease Relapse Survival Quality of life","2025-09-25",{"date":517,"type":41},"2025-10-03",{"date":519,"type":41},"2023-09-01",{"date":521,"type":20},"2100-12-31",{"name":523,"class":106},"Yigeng Cao,MD,PhD",{"id":525,"slug":526,"hasResults":12,"nctId":527,"briefTitle":528,"officialTitle":529,"acronym":4,"eligibilityCriteria":530,"healthyVolunteers":12,"sex":58,"minAge":531,"maxAge":4,"enrollmentInfo":532,"targetDuration":534,"studyType":21,"phases":4,"briefSummary":535,"conditions":536,"keywords":537,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":540,"lastUpdatePostDateStruct":541,"startDateStruct":543,"completionDateStruct":545,"leadSponsor":547,"locationsCount":107},"100243915","global-haemostatic-methods-following-administration-of-bypassing-agents-to-patients-with-haemophilia-with-inhibitors-100243915","NCT02453542","Global Haemostatic Methods Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors","Global Haemostatic Methods to Measure the Treatment Effect Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors","Inclusion Criteria:\n\n* informed consent\n* meets the study population description\n\nExclusion Criteria:\n\n* no informed consent age\\\u003C7 years","7 Years",{"count":533,"type":20},20,"2 Years","Background\n\nThe treatment of haemophilia A and B has been revolutionized by the use of factor concentrate, both as prophylaxis and to treat bleeding episodes (on-demand treatment). However, despite its advantages, repeated treatment with factor concentrate can lead to development of inhibitors (antibodies) towards the coagulation factor in the concentrate. Another patient group in which the bleeding symptoms are difficult to treat because of inhibitors towards coagulation factors, most commonly FVIII, is patients with acquired haemophilia. Patients with high antibody titers exhibit a deficient or no response to factor concentrates and usually need treatment with bypassing agents, namely factor eight inhibitor bypassing agent (FEIBA®, Baxter) och recombinant activated factor VII (rFVIIa, Novo-Seven®, Novo Nordisk). The effect of the treatment cannot be accurately monitored by traditional coagulation tests.\n\nThe aim of the study is to evaluate the utility of the global haemostatic methods in patients with haemophilia with inhibitors. The objective is to improve the monitoring of the treatment effect and thus increase the safety of the patient and the effectiveness of the treatment.\n\nPatients and methods\n\nPatients\n\nThe primary cohort will consist of fifteen patients with inherited haemophilia with inhibitors as well as five adult patients with acquired haemophilia who are followed up at the Coagulation Department of the Karolinska University Hospital, Stockholm, Sweden.\n\nBlood samples will be collected from those patients at specific time points (see Design of the study) during the course of two years (for each patient). The treatment (type, dose, duration) will be determined by the treating physician.\n\nMethods (selection)\n\n* Thrombin generation (Calibrated Automated Thrombogram, CAT® and a commercial kit from Siemens®).\n* Overall haemostatic potential (OHP)\n\nDesign of the study\n\nTimeframe for blood sampling: i) baseline (inclusion in the study), and ii) prior and after administration of bypassing agents to either treat bleeding symptoms or before an invasive procedure or as prophylaxis.\n\nData analysis\n\nThe variations in coagulation markers measured as described above (Methods) will be associated to the clinical symptoms (bleeding), the level of coagulation factors (if measurable) and the titers of the inhibitors.",[27],[298,538,539],"inhibitors","thrombin","2025-08-25",{"date":542,"type":41},"2025-09-02",{"date":544,"type":41},"2015-03-01",{"date":546,"type":20},"2030-08",{"name":548,"class":106},"Karolinska Institutet",{"id":550,"slug":551,"hasResults":12,"nctId":552,"briefTitle":553,"officialTitle":553,"acronym":4,"eligibilityCriteria":554,"healthyVolunteers":12,"sex":17,"minAge":269,"maxAge":4,"enrollmentInfo":555,"targetDuration":4,"studyType":63,"phases":557,"briefSummary":559,"conditions":560,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":563,"lastUpdatePostDateStruct":564,"startDateStruct":566,"completionDateStruct":568,"leadSponsor":570,"locationsCount":107},"100542666","early-phase-1-topical-and-local-txa-in-facelifts---a-randomized-controlled-double-blinded-study-100542666","NCT06345833","Topical and Local TXA in Facelifts - A Randomized Controlled Double Blinded Study","Inclusion Criteria:\n\n* Eligible participants will consist of all regular clinic patients who elect and are deemed fit by the surgeon to undergo facelift surgery, including patients undergoing ancillary procedures\n* age 18 and older\n* English speaking.\n\nExclusion Criteria:\n\n* younger than 18\n* previously had an adverse reaction to tranexamic acid\n* non-English speaking\n* patients who elect not to participate or withdraw from the study.",{"count":556,"type":20},50,[558],"EARLY_PHASE1","Tranexamic acid (TXA) is a fibrinolytic inhibitor which prevents prolonged bleeding by interfering with fibrin clot breakdown by competitively binding to lysine receptors on plasminogen; this prevents the conversion of plasminogen to plasmin. TXA will be applied to a randomly assigned side of the face during facelift surgery. The intervention groups will include 1% TXA mixed with standard local consisting 1\u002F4% lidocaine with 1:100,000 epinephrine, 3% TXA on TXA-soaked pledgets applied for 10 minutes, and 1% TXA with local plus 3% TXA-soaked pledgets. Each treatment arm will be compared to saline in place of TXA on the contralateral side of the face.\n\nAlthough TXA has been widely used in surgical fields for decades and is officially recommended by agencies such as ACOG for use during maternal hemorrhage, its current FDA approval only pertains to oral TXA for heavy menstrual bleeding and IV use for patients with hemophilia to prevent or reduce hemorrhage (cite). The main concern with intravenous TXA is the increased risk for the potential formation of blood clots, mainly in patients with clotting disorders, such as Facor V Leiden, and patients on estrogen containing medication. A recent systemic review with metanalysis by Wang et.al contained a total of 2150 patients receiving IV TXA while undergoing plastic surgery concluded that use of IV TXA does not lead to increased adverse events.\\[12\\] Given the low rate of adverse events while using TXA systemically, this protocol's application of TXA topically and\u002For locally negates the risk for any potential systemic adverse effects. No systemic adverse effects have been reported in studies examining local TXA in facial plastic surgery to date.",[27,561,562],"Hemorrhage","Facelift Surgery","2025-08-18",{"date":565,"type":41},"2025-08-19",{"date":567,"type":41},"2024-07-01",{"date":569,"type":20},"2026-07-01",{"name":571,"class":106},"University of Minnesota",{"id":573,"slug":574,"hasResults":12,"nctId":575,"briefTitle":576,"officialTitle":577,"acronym":578,"eligibilityCriteria":579,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":580,"targetDuration":292,"studyType":21,"phases":4,"briefSummary":581,"conditions":582,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":563,"lastUpdatePostDateStruct":584,"startDateStruct":586,"completionDateStruct":588,"leadSponsor":589,"locationsCount":107},"100327954","patient-reported-outcomes-burdens-and-experiences---phase-3-100327954","NCT03549858","Patient Reported Outcomes Burdens and Experiences - Phase 3","Patient Reported Outcomes Burdens and Experiences (PROBE) - Phase 3 - Longitudinal Data Collection","PROBE-3","Inclusion Criteria:\n\n* PWH will be recruited through national hemophilia patient organizations utilizing their existing membership rosters, social media outlets and meetings \u002F events. The investigators are not proposing a pre-determined method of PWH recruitment. They will utilize the information acquired in the workshop and take-home project to inform best practice in recruitment methodology for the study. The investigators may consider requesting different countries test different PWH recruitment strategies to test reproducibility.\n\nIn the future, the questionnaire might also be administered to patients with other chronic conditions.\n\nExclusion Criteria:\n\n* Disease severity and Age bands or age limits (e.g. ≥ Age 18) may be utilized to narrow the study population.",{"count":502,"type":20},"The PROBE Phase-3 study will collect data on patient reported outcomes, burdens, and experiences in patients living with hemophilia. The investigators will perform comparisons among countries, within country over time, within country against national normative data.",[27,583],"Chronic Disease",{"date":585,"type":41},"2025-08-22",{"date":587,"type":41},"2018-01-01",{"date":307,"type":20},{"name":590,"class":106},"McMaster University",{"id":592,"slug":593,"hasResults":12,"nctId":594,"briefTitle":595,"officialTitle":596,"acronym":4,"eligibilityCriteria":597,"healthyVolunteers":12,"sex":58,"minAge":269,"maxAge":4,"enrollmentInfo":598,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":600,"conditions":601,"keywords":602,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":608,"lastUpdatePostDateStruct":609,"startDateStruct":611,"completionDateStruct":613,"leadSponsor":615,"locationsCount":107},"100600578","association-of-prophylactic-treatment-with-treatment-burden-and-psychosocial-variables-in-patients-with-hemophilia-100600578","NCT07099313","Association of Prophylactic Treatment With Treatment Burden and Psychosocial Variables in Patients With Hemophilia","Association of Prophylactic Treatment With Treatment Burden, Self-efficacy, Adherence, Sleep Quality, and Locus of Control in Patients With Hemophilia; an Ambispective Cohort Study","Inclusion Criteria:\n\n* Patients over 18 years of age.\n* Patients with a medical diagnosis of hemophilia A or B.\n* Patients who, regardless of disease phenotype (mild, moderate or severe), are receiving prophylactic treatment with recombinant or plasma clotting factor concentrates, either short half-life or extended half-life.\n* Patients must have maintained the same treatment regimen (SHL or EHL) continuously for at least six months prior to participation.\n\nExclusion Criteria:\n\n* Patients who have developed inhibitors or antibodies to FVIII or FIX concentrates.\n* People with a concomitant diagnosis of other serious or disabling chronic diseases (neurological, oncological, severe psychiatric or rheumatological) that may interfere with the perceived burden of treatment, sleep quality, functionality or perception of self-efficacy.\n* Patients with cognitive, linguistic or sensory impairments that prevent them from correctly understanding and completing the questionnaires.\n* Patients who are participating in clinical trials or intensive monitoring programmes that may alter their perception of the treatment and generate biases in the evaluation.",{"count":599,"type":20},114,"Introduction: Hemophilia is a congenital coagulopathy characterised by recurrent haemarthrosis, leading to chronic arthropathy and functional impairment. Prophylactic treatment with extended half-life (EHL) or short half-life (SHL) clotting factor concentrates is the most effective strategy for preventing these episodes. EHL products have demonstrated haemostatic efficacy, with a lower frequency of infusions, potentially reducing the treatment burden, although their psychosocial impact has not yet been sufficiently explored.\n\nObjectives: To evaluate the association between perceived treatment burden and psychosocial variables such as self-efficacy, adherence, sleep quality and health locus of control, depending on the type of treatment received (EHL or SHL).\n\nMethods. Multicentre, ambispective cohort study. A total of 114 patients with haemophilia A or B undergoing EHL or SHL prophylactic treatment will be included. The primary variable will be treatment burden (Treatment Burden Questionnaire). Secondary variables will be perceived self-efficacy (General Self-Efficacy Scale), adherence (Torres scale), sleep quality (Pittsburgh Sleep Quality Index), treatment adherence (Torres Questionnaire) and health locus of control (Multidimensional Health Locus of Control). Potential confounding variables will include sociodemographic data (age, educational level, living arrangements) and clinical data (number of weekly infusions, type of hospital).\n\nExpected results: Patients treated with extended-half-life products are expected to report lower treatment burden, higher self-efficacy and better sleep quality, regardless of sociodemographic or clinical factors.",[27],[27,603,604,605,606,607],"Clotting factor concentrates","Treatment burden","Perceived self-efficacy","Adherence","Locus of control","2025-08-01",{"date":610,"type":41},"2025-08-05",{"date":612,"type":20},"2025-07-31",{"date":614,"type":20},"2025-10-15",{"name":211,"class":48},{"id":617,"slug":618,"hasResults":12,"nctId":619,"briefTitle":620,"officialTitle":621,"acronym":622,"eligibilityCriteria":623,"healthyVolunteers":12,"sex":17,"minAge":439,"maxAge":115,"enrollmentInfo":624,"targetDuration":4,"studyType":63,"phases":625,"briefSummary":626,"conditions":627,"keywords":4,"overallStatus":203,"whyStopped":4,"lastUpdateSubmitDate":628,"lastUpdatePostDateStruct":629,"startDateStruct":631,"completionDateStruct":633,"leadSponsor":635,"locationsCount":4},"100595640","isokinetic-strength-training-on-functional-performance-100595640","NCT07035080","Isokinetic Strength Training On Functional Performance","Efficacy Of Isokinetic Strength Training On Functional Performance In Children With Hemophilia","Isokinetic","Inclusion Criteria:\n\n* Patients of both groups (study and control groups ) were selected according to the following criteria:\n\n  1. Each group contained 15 male children. They were suffering from unilateral knee heamarthrothesis .\n  2. The joint problems (pain and bleeding ) ranges from mild to moderate according to the classification of hemophilia recommended by the Orthopedic advisory committee of the World Federation of hemophilia (appendix 1) (Holder and Cotta, 1989).\n  3. Patients were able to stand and walk independently.\n  4. They had no neurological or psychological problems.\n  5. All patients were clinically and medically stable.\n  6. They were able to understand the requirements of the study.\n  7. They were suffering from moderate hemophilia.\n  8. They were not suffering from acute joint and muscle bleeds during treatment time.\n  9. None of the children suffered from fixed deformities of the affected lower limb.\n  10. All the children received the same medical treatment to control bleeding.\n  11. They had no visual no hearing deficits B. Exclusion criteria\n\n  \u003C!-- -->\n\n  1. Patients with advanced radiographic changes including:\n\n     * Bone destruction.\n     * Bony ankylosis .\n     * Knee joint subluxation.\n     * Epiphyseal fracture.\n  2. Patients who had congenital or acquired skeletal deformities in both lower limbs.\n  3. Patients who had any neurological deficits such as convulsions involuntary movements or those receiving muscle relaxants.\n\n     Exclusion Criteria:",{"count":78,"type":20},[91],"Hemophilia is a congenital , recessive clotting disease featured by cerebra ( The most dangerous ) and musculoskeletal (the most common and disabling) hemorrhages . It is a gender -specific coagulopathy resulting from a factor VIII (F VIII) deficiency in hemophilia A and factor IX (F IX) deficiency in hemophilia B( Felip et al., 2011) Loss of muscle mass is typical of hemophilia arthropathy, There are two basic reasons that account for such muscle loss. The first reason is due to inactive joints as the result of long -term immobilization that hemophilic patients are obliged to do because of continuous lesions in muscles and joints, and the second reason is a decrease in physical exercise by hemophilic patients because of the risk of lesions that they face (Toca-Herrera et al., 2008).\n\nInterferential therapy is a type of transcutaneous electrical nerve stimulation (TENS). Two slightly different, medium frequency- alternating currents are simultaneously applied to the affected area through electrodes. Superpisition or interference between the currents causes the combined electrical current to rise and fall (Burch et al, 2008).\n\nLow amplitude modulated frequencies elicit a \"beating\" or \"tapping\" sensation and muscle twitch response, while higher amplitude modulated frequencies elicit \"buzzing\" or \"tingling sensation\" and titanic muscle contraction ( Defrin et al., 2007).\n\nSeveral methods are available for testing muscle strength. These include manual muscle testing and dynamometry includes the use of handheld dynamometers, handgrip dynamometers, and isokinetic dynamometers (Sisto and Dyson-hudson, 2007).\n\nIsokinetic dynamometers measure torque produced at the anatomic joint throughout the available range of motion (ROM). Isokinetic dynamometers. Such as the kinCom (Chatanooga Corp, Chatanooga, Tennessee). Biodex (Biodex Medical systems. Inc) and Lido Active isokinetic System (Loredan, Inc, Davis , California ) measure torque by controlling the velocity of the movement and measuring the force applied via a force transducer (Sisto and Dyson-hunson, 2007).\n\nIsokinetic test record the torque produced throughout the entire ROM and allow for the identification of regions of strength or weakness within the range ( Remauld et al., 2005).\n\nIsokinetic assessment has primarily been recommended for strength testing as maximal force is applied during all phase of the movement at a constant velocity . The isokinetic mode is also safe to use with children because there is minimal risk of the muscle and joint injuries that can results from efforts to control the load if using free weights in one repetition -maximum testing (Mark et al., 2003).",[27],"2025-06-16",{"date":630,"type":41},"2025-06-24",{"date":632,"type":20},"2025-06-20",{"date":634,"type":20},"2025-09-30",{"name":636,"class":106},"Kafrelsheikh University",{"id":638,"slug":639,"hasResults":12,"nctId":640,"briefTitle":641,"officialTitle":642,"acronym":4,"eligibilityCriteria":643,"healthyVolunteers":220,"sex":58,"minAge":269,"maxAge":386,"enrollmentInfo":644,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":646,"conditions":647,"keywords":648,"overallStatus":203,"whyStopped":4,"lastUpdateSubmitDate":653,"lastUpdatePostDateStruct":654,"startDateStruct":656,"completionDateStruct":658,"leadSponsor":659,"locationsCount":107},"100589906","predictors-of-physical-activity-in-adult-patients-with-hemophilic-arthropathy-100589906","NCT06960499","Predictors of Physical Activity in Adult Patients With Hemophilic Arthropathy","Predictors of Physical Activity in Adult Patients With Hemophilic Arthropathy. A Multicenter Cross-sectional Cohort Study","Inclusion Criteria:\n\n* Patients with hemophilia A or B\n* Over the age of majority\n* With a medical diagnosis of severe phenotype of the disease\n* With a medical diagnosis of hemophilic arthropathy in the lower limbs\n* Under on-demand or prophylactic drug treatment.\n\nExclusion Criteria:\n\n* Patients who require technical aids for walking\n* Patients who are dependent for activities of daily living\n* Patients over the age of 60\n* Patients with cognitive impairments that prevent them from understanding the questionnaires",{"count":645,"type":20},88,"Introduction. The development of hemophilic arthropathy causes degenerative joint damage that leads to functional impairment, limiting physical activity and causing disability in patients with hemophilia.\n\nObjectives. i) To assess the level of physical activity in patients with hemophilia; ii) To identify the best predictive model for physical activity in adult patients with hemophilic arthropathy.\n\nMaterial and method. Multicenter cross-sectional cohort study. Eighty-eight patients will be recruited. The dependent variable will be physical activity (International Physical Activity Questionnaire). Secondary variables will be kinesiophobia (Tampa Kinesiophobia Scale), functionality (Functional Independence Scale in Hemophilia), pain intensity, and clinical, anthropometric, and sociodemographic variables.",[27],[27,649,650,651,652],"Physical activity","Kinesiophobia","Functionality","Joint pain","2025-04-28",{"date":655,"type":41},"2025-05-07",{"date":657,"type":20},"2025-05-04",{"date":632,"type":20},{"name":660,"class":106},"Universidad Católica San Antonio de Murcia"]