[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hemophilia-a-severe\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hemophilia-a-severe":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,60,86,117],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":17,"sex":18,"minAge":4,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":23,"conditions":24,"keywords":38,"overallStatus":47,"whyStopped":4,"lastUpdateSubmitDate":48,"lastUpdatePostDateStruct":49,"startDateStruct":52,"completionDateStruct":54,"leadSponsor":56,"locationsCount":59},"100624813","hemophilia-a-research-program-100624813",false,"NCT07414511","Hemophilia A Research Program","Hemophilia A Research Program (HARP): An Observational Intergenerational Cohort Study of Hemophilia A and Factor VIII Immunogenicity","HARP","Maternal Inclusion Criteria:\n\nPregnant individuals who meet the following criteria are eligible for enrollment as study participants:\n\n* Currently pregnant and prior to 37 weeks gestation\n* Known to have or at-risk of having a severe hemophilia A genotype\n* Pregnant with at least one fetus at-risk of inheriting severe hemophilia A\n* Ability to understand and willingness to provide informed consent\n* 18 years of age or older\n\nBefore the 38th week of pregnancy, enrolled participants must meet all the following criteria to continue to remain in the study:\n\n* The pregnant mother has a severe hemophilia A genotype.\n* A fetus is determined to have a \\>\u002F= 25% risk of inheriting severe hemophilia A, or prenatal testing indicates a fetus is affected by severe hemophilia A.\n* No other discontinuation criteria have been identified.\n\nPediatric Continuation \u002F Inclusion Criteria:\n\nEligibility of the child to continue is assessed by age 8 weeks. Mother-child pairs in which a child meets the following criteria will remain in the study:\n\n* Severe hemophilia A defined by a baseline FVIII:C \\\u003C 0.01 IU\u002FmL (or FVIII:C \\\u003C 1%) or a genotype predicted to cause severe hemophilia A\n* Born to a mother participating in the study\n\nThereafter, mothers and their children will continue in the study as long as no new discontinuation criteria occur.\n\nInclusion Criteria for Blood Relatives:\n\nBlood relatives of the child may be offered participation if one of the following criteria are met:\n\n* First-degree blood relatives (e.g., father, sibling) of the child\n* Second-degree blood relatives (e.g., aunt, uncle, grandparent, half-sibling) of the child\n* Any more distant male or female blood relative whose data or samples may be informative for the planned genetic studies of hemophilia and inhibitors\n\nExclusion\u002FDiscontinuation Criteria:\n\nMaternal: For the pregnant person, exclusion or discontinuation criteria are as follows:\n\n* Genetic testing is negative for a severe hemophilia A genotype\n* Prenatal clinical diagnostic testing that indicates there is no fetus affected with severe hemophilia A\n* Presence of another clinically significant bleeding disorder\n* Participation in another study for which any blood collection total would exceed safety limits defined in this study\n* Will deliver outside the United States or plans for regular pediatric care for the child to be delivered outside the United States\n* Is a prisoner\n* Any other reason that, in the opinion of the investigator, would render the individual unsuitable for participation in the study\n* Inability for study team to obtain translated study documents in time for participation if participant is not fluent in English\n\nPediatric: For the child, discontinuation criteria are as follows:\n\n* Infant does not have severe hemophilia A defined by a baseline FVIII:C \\\u003C 0.01 IU\u002FmL (or FVIII:C \\\u003C 1%) or does not have a genotype predicted to cause severe hemophilia A\n* Mother or child did not have minimal required study samples or data collected before birth, around the time of delivery, or in the neonatal period\n* Child has another clinically significant bleeding disorder\n* Child has a clinically severe immune disorder\n* Participation in another study for which any blood collection total would exceed safety limits defined in this study\n* Any other reason that, in the opinion of the investigator, would render the individual unsuitable for participation in the study",true,"ALL",{"count":20,"type":21},500,"ESTIMATED","OBSERVATIONAL","This study longitudinally observes the intergenerational (mother-child) continuum in hemophilia A from pregnancy through early childhood. Because the study follows mother-child pairs, the study includes both a maternal cohort and a pediatric cohort. Each cohort has a primary goal: for the mother with a severe hemophilia genotype, the overarching primary goal is to understand the risks for pregnancy-associated bleeding and postpartum hemorrhage (PPH); for the child, the overarching primary goal is to understand the risks, timing, and circumstances of development of anti-FVIII antibodies. From a longitudinal perspective, risks for both bleeding in the mother and anti-FVIII antibody development in the child are expected to be influenced over time by genetic and environmental factors that begin early in (or before) pregnancy. Enrollment of blood relatives is offered to improve power to better understand inherited contributions to bleeding and inhibitor development in the mother-baby pairs.",[25,26,27,28,29,30,31,32,33,34,35,36,37],"Hemophilia A","Hemophilia A, Severe","Factor VIII (FVIII)","FVIII Deficiency","Carrier of Hemophilia A","Inhibitors","Pregnancy","Maternal Blood Loss","Pregnancy Complications","Bleeding Disorder","Hemorrhage, Postpartum","Alloimmunization","X-Linked",[31,39,40,34,41,42,25,43,44,45,46],"Inhibitor","Bleeding","Maternal Child Health","Hemophilia","Hemophilia A Carrier","Hemophilia A Symptomatic Carrier","Coagulation","Factor VIII","RECRUITING","2026-02-09",{"date":50,"type":51},"2026-02-17","ACTUAL",{"date":53,"type":51},"2024-07-31",{"date":55,"type":21},"2029-08",{"name":57,"class":58},"University of Washington","OTHER",1,{"id":61,"slug":62,"hasResults":11,"nctId":63,"briefTitle":64,"officialTitle":64,"acronym":4,"eligibilityCriteria":65,"healthyVolunteers":11,"sex":66,"minAge":67,"maxAge":68,"enrollmentInfo":69,"targetDuration":4,"studyType":71,"phases":72,"briefSummary":74,"conditions":75,"keywords":4,"overallStatus":47,"whyStopped":4,"lastUpdateSubmitDate":77,"lastUpdatePostDateStruct":78,"startDateStruct":80,"completionDateStruct":82,"leadSponsor":84,"locationsCount":59},"100425248","liver-biopsy-in-haemophilia-gene-therapy-100425248","NCT04817462","Liver Biopsy In Haemophilia Gene Therapy","Inclusion criteria:\n\n1. Male and aged 18 to 80 years old\n2. Patients who were enrolled and treated in one of the following clinical trials at Royal Free Hospital:\n\n   * AGT4HB (EudraCT No 2005-005711-17) - FIX AAV gene therapy trial (Sponsor: St Jude Children's Research Hospital)\n   * GO-8 (EudraCT No 2016-000925-38) - FVIII AAV gene therapy trial (Sponsor: UCL)\n   * FLT180a-01 (EudraCT: 2017-000852-24) - FIX AAV gene therapy trial ((Sponsor: UCL) \\[now enrolled in long term follow up study FLT180a-04 (EudraCT No 2017-005080-40) (Sponsor: Freeline Therapeutics Ltd)\n3. Patients with endogenous FVIII:C\u002FFIX:C expression at \\>1% any time after gene transfer, associated with normal prothrombin (PT) and thrombin times (TT) as determined in a coagulation assay.\n\nExclusion Criteria:\n\n1. Patients with a platelet count measured at \\\u003C140 x109\u002FL\n2. Any condition that, in the opinion of the investigator or Sponsor would prevent the patient from fully complying with the requirements of the study and\u002For would influence or interfere with evaluation and interpretation of subject safety or efficacy result.\n3. Patients with abnormal kidney function (estimated GFR \\\u003C50ml\u002Fmin)\n4. Patients with a known allergy to iodine-based intravenous contrast agents\n5. Patients with a known allergy to local or general anaesthetic\n6. Patients with a known reaction to FVIII\u002FFIX concentrate infusions\n7. Presence of FVIII or FIX inhibitor (done within 14 weeks of biopsy)\n8. Evidence of any bleeding disorder not related to haemophilia A or B\n9. Patients unable and unwilling to provide and sign an informed consent.","MALE","18 Years","80 Years",{"count":70,"type":21},10,"INTERVENTIONAL",[73],"NA","To perform a liver biopsy in haemophilia A and B patients with endogenous FVIII:C\u002FFIX:C expression at \\>1% any time after gene transfer following AAV mediated gene transfer. This is to obtain tissue for analysis, to understand if FIX\u002FFVIII transgenic protein expression is mediated by AAV proviral DNA that is integrated into the host cell DNA or if stable expression in humans is mediated by episomal maintained AAV genome.",[76,26],"Hemophilia B, Severe","2024-12-16",{"date":79,"type":51},"2024-12-20",{"date":81,"type":51},"2022-08-05",{"date":83,"type":21},"2025-07-01",{"name":85,"class":58},"University College, London",{"id":87,"slug":88,"hasResults":11,"nctId":89,"briefTitle":90,"officialTitle":91,"acronym":4,"eligibilityCriteria":92,"healthyVolunteers":11,"sex":66,"minAge":93,"maxAge":94,"enrollmentInfo":95,"targetDuration":4,"studyType":71,"phases":97,"briefSummary":99,"conditions":100,"keywords":101,"overallStatus":106,"whyStopped":4,"lastUpdateSubmitDate":107,"lastUpdatePostDateStruct":108,"startDateStruct":110,"completionDateStruct":112,"leadSponsor":114,"locationsCount":4},"100572871","phase-3-an-open-label-study-evaluating-the-efficacy-safety-pharmacokinetics-and-immunogenicity-of-skp-0141-for-the-treatment-and-prophylaxis-in-severe-hemophilia-a-patients-100572871","NCT06738901","An Open-label Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of SKP-0141 for the Treatment and Prophylaxis in Severe Hemophilia a Patients","A Phase 1\u002F3, Open-label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of Human Plasma-derived Factor VIII (SKP-0141) for the Treatment and Prophylaxis in Male Patients with Severe Hemophilia a","Inclusion Criteria:\n\n* A patient or parent\u002Flegal guardian who is capable of giving signed informed consent\n* Patients assigned male at birth and must be 12 to 65 years old at the time of Screening\n* Diagnosis of severe congenital hemophilia A, defined as an FVIII level of \\\u003C1% as documented in the patient's medical records at the time of Screening\n* Patients who have received or are currently receiving plasma-derived and\u002For recombinant FVIII products and have had at least 150 EDs with a FVIII product\n* Patients who can produce viable sperm and have a partner of childbearing potential must agree to take appropriate contraceptive measures consistently during the study, starting at Screening and until 30 days after the end of study\n\nExclusion Criteria:\n\n* Any history of or current FVIII inhibitors or any first order family history of FVIII inhibitors in terms of detectable FVIII inhibitors (ie, ≥0.6 Bethesda Units \\[BU\\]) using the Nijmegen-modification of the Bethesda assay\n* Any known congenital or acquired coagulation disorder other than the congenital hemophilia A\n* Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism, myocardial infarction, and arterial embolus within 3 months prior to Visit 1\n* Experienced life-threatening bleeding episode or had major surgery or an orthopedic surgical procedure during the 3 months prior to Visit 1\n* Has been tested positive for HIV with a CD4+ count ≤200\u002FμL at Screening (if available, hepatitis B surface antigen, or hepatitis C virus antibodies, and\u002For positive hepatitis B virus deoxyribonucleic acid\u002FHCV ribonucleic acid at Screening\n* Platelet count \\\u003C100 000\u002FμL at Screening\n* Patients with serum aspartate aminotransferase or serum alanine aminotransferase values \\>5 × the upper limit of normal or serum creatinine values \\>2 × ULN at Screening\n* Patients who are currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment within 3 months prior to Visit 1\n* Use of any other investigational medicinal product, cryoprecipitate, whole blood, or plasma within 30 days or 5 half-lives prior to Visit 1\n* Known or suspected hypersensitivity to any FVIII product or their excipients\n* Has a physical, medical, or psychological condition, that in the opinion of the PI, may interfere with the evaluation of the study.\n* Are study site personnel directly affiliated with this study and their immediate families","12 Years","65 Years",{"count":96,"type":21},55,[98],"PHASE3","This is a prospective, multicenter, open-label study to assess efficacy, safety, pharmacokinetics (PK), and immunogenicity of human plasma-derived Factor VIII (FVIII) in previously treated patients (PTPs) with severe hemophilia A. Overall, 55 male PTPs aged 12 to 65 years old with a FVIII level of \\\u003C 1% and at least 150 treatment exposure days (EDs) with a previous FVIII product will be enrolled. Patients will receive SKP-0141 at a dose of 25 to 50 IU\u002Fkg every second day or 3 times per week for at least 50 EDs and\u002For 6 months from the start of prophylactic treatment. Efficacy of SKP-0141 will be primarily evaluated in bleeding prophylaxis with annualized bleeding rate from start of treatment and until end of treatment (Visit 10).",[26],[102,103,104,105,25],"Congenital hemophilia A","Prophylaxis","On-demand","FVIII concentrate","NOT_YET_RECRUITING","2024-12-13",{"date":109,"type":51},"2024-12-18",{"date":111,"type":21},"2025-03-31",{"date":113,"type":21},"2026-08-31",{"name":115,"class":116},"SK Plasma Co., Ltd.","INDUSTRY",{"id":118,"slug":119,"hasResults":11,"nctId":120,"briefTitle":121,"officialTitle":122,"acronym":123,"eligibilityCriteria":124,"healthyVolunteers":11,"sex":66,"minAge":125,"maxAge":4,"enrollmentInfo":126,"targetDuration":4,"studyType":71,"phases":128,"briefSummary":130,"conditions":131,"keywords":137,"overallStatus":47,"whyStopped":4,"lastUpdateSubmitDate":142,"lastUpdatePostDateStruct":143,"startDateStruct":145,"completionDateStruct":147,"leadSponsor":149,"locationsCount":151},"100540729","phase-4-pharmacokinetic-guided-dosing-of-emicizumab-100540729","NCT06320626","Pharmacokinetic-guided Dosing of Emicizumab","Pharmacokinetic-guided Dosing of Emicizumab in Congenital Haemophilia A Patients - The DosEmi Study","DosEmi","Inclusion Criteria:\n\n* Confirmed diagnosis of congenital haemophilia A, with a baseline endogenous FVIII of \\\u003C6 IU\u002Fml\n* Aged \\> 1 year at inclusion (inclusion of children 1-16 years after favourable interim-analysis see protocol)\n* Receiving conventional dosing of emicizumab (6 mg\u002Fkg\u002F4 weeks with varying intervals) for a duration of at least 12 months prior to inclusion;\n* Having good bleeding control, defined as:\n\ni No spontaneous joint\u002Fmuscle bleeds in the previous 6 months AND ii A maximum of two treated (traumatic) bleeds in the previous 6 months.\n\n* Willing and able to provide written informed consent, either by the subject or its parents\u002Flegal guardian\n* Willing to provide bleeding assessment information\n* Willing to adhere to the medication regimen\n\nExclusion Criteria:\n\n* Acquired haemophilia A","1 Year",{"count":127,"type":21},95,[129],"PHASE4","The goal of this multicentre, prospective, open-label, cross-over clinical study is to determine whether individualized PK-guided dosing of emicizumab is non-inferior to conventional dosing of emicizumab in the prevention of bleeding in congenital haemophilia A patients.",[132,133,26,134,135,136],"Hemophilia A With Inhibitor","Hemophilia A Without Inhibitor","Adolescent","Child","Adult",[138,25,139,140,141],"Emicizumab","Monoclonal antibodies","PK-guided dosing","Hemorrhage","2024-03-12",{"date":144,"type":51},"2024-03-20",{"date":146,"type":51},"2022-09-08",{"date":148,"type":21},"2026-08",{"name":150,"class":58},"Kathelijn Fischer",8]