[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hemophilia-a\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hemophilia-a":30},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,45,0,25,[9,52,83,106,137,158,180,202,226,245,266,290,310,329,355,375,395,423,446,483,508,533,580,603,635],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":4,"eligibilityCriteria":16,"healthyVolunteers":17,"sex":18,"minAge":19,"maxAge":20,"enrollmentInfo":21,"targetDuration":4,"studyType":24,"phases":25,"briefSummary":27,"conditions":28,"keywords":33,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":41,"startDateStruct":44,"completionDateStruct":46,"leadSponsor":48,"locationsCount":51},"100542946","phase-1-a-study-of-safety-tolerability-pharmacokinetics-and-pharmacodynamics-of-sr604-in-two-participants-groups-part-a-healthy-participants-and-part-b-participants-with-hemophilia-a-or-hemophilia-b-or-factor-vii-deficiency-100542946",false,"NCT06349473","A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)","A Phase 1 Single and Multiple Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Healthy Participants (Part A) and the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SR604 in Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency (Part B)","Key Inclusion Criteria:\n\nPart A:\n\n* Male participants aged 18 to 55 years, inclusive.\n* Body mass index between 18 and 30 kilograms per meter square (kg\u002Fm\\^2), inclusive, and weighs greater than or equal to (\\>=) 50 kilograms (kg), less than or equal to (\\\u003C=) 90 kg.\n* No clinically significant findings on medical examination, including physical examination, 12-lead electrocardiogram, and clinical laboratory tests.\n* Sexually active men must commit to use an effective method of birth control while taking the study intervention and for 90 days after the dose of study intervention.\n\nPart B:\n\n* Male and female participants (only female participants with congenital FVII deficiency) aged 18 to 60 years, inclusive.\n* Participants must have one of the following bleeding disorders: Severe hemophilia A (\\\u003C1% Factor VIII \\[FVIII\\]); or Severe and\u002For moderately severe Hemophilia B (≤ 2% Factor IX \\[FIX\\]); or Severe FVII deficiency (\\\u003C10% FVII activity). Participants with severe FVII deficiency must satisfy with either of following criteria:\n\n  1. Participants with history of \\>2 bleeding events in the last 12 months require on-demand treatment with recombinant factor VIIa (rFVIIa) or plasma-derived FVII concentrates (pd-FVII) or fresh frozen plasma (FFP) for bleeding control.\n  2. Participants on prophylaxis treatment with rFVIIa or pd-FVII or FFP regardless of bleeding history.\n* Participants with Hemophilia A or Hemophilia B must satisfy either of the following criteria:\n\n  1. Participants not on prophylaxis must have a documented ABR of 6 in 12 months before screening.\n  2. Participants on prophylaxis must have a documented ABR of ≥ 2 in 12 months before screening.\n  3. Intolerant to current treatment regimen.\n* Medical records documenting a minimum of 2 years of bleeding event history.\n* Willing to undergo a weaning period from prior treatment or prophylaxis for Hemophilia A or Hemophilia B or FVII deficiency.\n* Sexually active men must commit to use an effective method of birth control while taking the study intervention and for 90 days after the dose of SR604.\n* Women of childbearing potential must have a negative pregnancy test at the Screening Visit and agree to follow the contraception guidance during the intervention period and for at least 90 days after the last dose of SR604.\n\nKey Exclusion Criteria:\n\nPart A:\n\n* Participant has clinically significant history or evidence of cardiovascular, respiratory (including all chronic lung diseases), hepatic, renal, gastrointestinal, endocrine, neurological, immunological, bleeding, or psychiatric disorder(s).\n* Participant has a mean pulse less than (\\\u003C) 40 or greater than (\\>) 90 beats per minute (bpm), mean systolic blod pressure (BP) \\\u003C 90 millimeter of mercury (mmHg) or \\> 140 mmHg, or mean diastolic BP \\\u003C 50 mmHg or \\> 90 mmHg at the screening visit.\n* Participant has a mean corrected QT corrected for heart rate by Fridericia's formula (QTcF) of \\> 450 msec at the Screening Visit.\n* Participant has had injury, trauma, and\u002For major surgery within 3 months before Screening, or is planned to undergo surgery during the study.\n* Participant has received vaccination within 14 days before the dose of study intervention or has a vaccination planned during the study.\n* History of one or more of the following in participants and\u002For family members:\n\n  1. Factor V (FV) Leiden mutation.\n  2. Activated protein C (APC) resistant.\n  3. Protein C (PC) or protein S (PS) deficiency.\n  4. Prothrombin 20210 mutation;\n  5. Antithrombin III (ATIII) deficiency.\n* History of clinically significant intracranial hemorrhage, pneumonia, chronic liver disease, liver or kidney transplants, or malignant diseases.\n* Any medical condition (eg, diabetes, obesity.) which, in the Investigator's opinion, could compromise participant safety, interfere with study intervention metabolism, or put the study outcome at undue risk. Any condition for which, in the opinion of the Investigator, participation would not be in the best interest of the participant or could prevent, limit or confound protocol-specified assessments.\n* Participants with a history of all types of thrombosis, including any arterial and\u002For venous thrombosis, superficial thrombophlebitis, or embolism. Additionally, participants with a history of thrombotic microangiopathy, stroke, and transient ischemic attack (TIA), or abnormal findings in any prior laboratory thrombophilia evaluation will be excluded.\n\nPart B:\n\n* Participants with a history of all types of thrombosis, including any arterial and\u002For venous thrombosis, superficial thrombophlebitis, or embolism. Additionally, participants with a history of thrombotic microangiopathy, stroke, and TIA, or abnormal findings in any prior laboratory thrombophilia evaluation will be excluded.\n* History of one or more of the following in participants and\u002For family members:\n\n  1. FV Leiden mutation.\n  2. APC resistant.\n  3. PC or PS deficiency.\n  4. Prothrombin 20210 mutation.\n  5. ATIII deficiency.\n* Impaired cardiac function or clinically significant cardiac disease, including any of the following:\n\n  1. Clinically significant and\u002For uncontrolled heart disease such as congestive heart failure requiring treatment (New York Heart Association Grade \\>=2), left ventricular ejection fraction \\\u003C 50% as determined by multiple gated acquisition or echocardiogram, or clinically significant arrhythmia.\n  2. QTcF \\> 450 ms ECG or congenital Long QT Syndrome at the Screening Visit.\n  3. Acute myocardial infarction or unstable angina pectoris \\\u003C 3 months prior to study entry.\n* Uncontrolled hypertension (systolic BP \\> 150 mmHg and diastolic BP \\> 100 mmHg), a history of hypertension crisis, or a history of hypertensive encephalopathy.\n* Participant with the following laboratory abnormalities:\n\n  1. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> 1.5 × upper limit of normal (ULN);\n  2. Total bilirubin ˃3.0 × ULN and direct bilirubin ˃1.5 × ULN (unless due to Gilbert's syndrome).\n* Calculated creatinine clearance ˂ 60 mL\u002Fmin using the 2021 Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula at the Screening Visit.\n* Participant has positive test result for human immunodeficiency virus (HIV) antibody.\n\n  1. If participants test positive for hepatitis B core antibody (HBcAb), additional tests including hepatitis B surface antibody, hepatitis B surface antigen (HBsAg), and hepatitis B viral deoxyribonucleic acid (DNA) polymerase chain reaction (PCR) will be conducted to determine if there is an active infection. Participants with active infection will be excluded from the study.\n  2. Participants who test positive for hepatitis C virus antibody will be required to have a negative result for hepatitis C viral ribonucleic acid (RNA) PCR before enrollment. Individuals with positive results for hepatitis C PCR will be excluded from the study.\n* Chronic liver disease (Child-Pugh class C hepatic impairment), or history of liver or kidney transplants.\n* Injury, trauma, and\u002For major surgery (mediastinoscopy, insertion of a central venous access device, and insertion of a feeding tube are not considered major surgery), major dental procedures (extractions, etc.) within 4 weeks of the first dose of SR604 or planned surgery during the study.\n* Active infection requiring systemic antibiotic or antiviral therapy or in a sepsis condition within 14 days prior to the first dose of SR604.\n* Any medical condition (eg, diabetes, obesity) which, in the Investigator's opinion, could compromise participant safety, interfere with SR604 metabolism, or put the study outcome at undue risk.\n* Female participants who are pregnant or are currently breastfeeding or planning to become pregnant while enrolled in this study or within 90 days after the last dose of SR604.",true,"ALL","18 Years","60 Years",{"count":22,"type":23},31,"ESTIMATED","INTERVENTIONAL",[26],"PHASE1","The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604 in participants with Hemophilia A or Hemophilia B, or Factor VII (FVII) deficiency, with or without inhibitors (Part B).",[29,30,31,32],"Healthy Participants","Hemophilia A","Hemophilia B","Factor VII Deficiency",[34,35,36,37,38],"Single ascending dose","Multiple ascending dose","Factor IX","Factor VIII","Factor VII","RECRUITING","2026-06-30",{"date":42,"type":43},"2026-07-02","ACTUAL",{"date":45,"type":43},"2024-05-10",{"date":47,"type":23},"2026-09-28",{"name":49,"class":50},"Equilibra Bioscience LLC","INDUSTRY",10,{"id":53,"slug":54,"hasResults":12,"nctId":55,"briefTitle":56,"officialTitle":57,"acronym":4,"eligibilityCriteria":58,"healthyVolunteers":12,"sex":59,"minAge":60,"maxAge":61,"enrollmentInfo":62,"targetDuration":4,"studyType":24,"phases":64,"briefSummary":66,"conditions":67,"keywords":68,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":74,"startDateStruct":76,"completionDateStruct":78,"leadSponsor":80,"locationsCount":82},"100450410","phase-3-open-label-extension-study-of-marstacimab-in-hemophilia-participants-with-or-without-inhibitors-100450410","NCT05145127","Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors","AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF MARSTACIMAB PROPHYLAXIS IN SEVERE (COAGULATION FACTOR ACTIVITY \u003C1%) HEMOPHILIA A PARTICIPANTS WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY ≤2%) WITH OR WITHOUT INHIBITORS","Inclusion Criteria:\n\n* All participants will have a minimum body weight as defined by parent studies\n* Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.\n* Participants have successfully completed participation in parent studies, defined as did not require \"Early Termination\"\n\nExclusion Criteria:\n\n* Previous or current treatment for or history of coronary artery disease, venous or arterial thrombosis (CTCAE Grade \\>3), or ischemic disease (except catheter-associated thrombosis)\n* Abnormal renal function as defined by eGFR \\\u003C30 mL.min\u002F1.73 m(2)\n* Known planned surgical procedure during the planned study period\n* Unstable hepatic function as determined by the Investigator clinical assessment and review of the participant's most recent laboratory results, which would make the participant inappropriate for the study\n* For participants known to be HIV+, worsening disease status as determined by the Investigator clinical assessment and review of participant's most recent laboratory results, to include recent locally available CD4 count (if available), which would make the participant inappropriate for the study\n* Regular, concomitant therapy with immunomodulatory drugs (eg, IVIG, and routine systemic corticosteroids, rituximab)\n* Ongoing or planned use of immune tolerance induction or prophylaxis with FVIII or FIX replacement during the study\n* Participation in other study involving investigational drug(s) or investigational vaccine(s) within 30 days or 5 half-lives prior to or during study participation, with the exception of participation in parent studies\n* Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the Investigator, and their respective family members","MALE","1 Year","74 Years",{"count":63,"type":23},245,[65],"PHASE3","Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require \"Early Termination\" from the Phase 3 Study B7841005 and from the Phase 3 Study B7841008.\n\nStudy B7841005: approximately 145 adolescent and adult participants 12 to \\\u003C75 years of age with severe hemophilia A or moderately severe to severe hemophilia B (defined as FVIII activity \\\u003C1% or FIX activity ≤2%, respectively) with or without inhibitors are expected to be enrolled in Study B7841005 during which they will receive prophylaxis (defined as treatment by SC injection of marstacimab).\n\nStudy B7841008: this is an ongoing Phase 3, open-label study in pediatric participants \\\u003C18 years of age with severe hemophilia A (FVIII Coagulation Factor Activity \\\u003C1%) or moderately severe to severe hemophilia B (FIX Coagulation Factor Activity ≤2%). A sequential approach will be used in enrolling at least 100 pediatric participants, at least 20 of which will be aged ≥12 to \\\u003C18 years and at least 80 participants will be aged ≥1 to \\\u003C12 years. At the start of study B7841008, the dosing and data available in adolescent and adult participants in Study B7841005 supported the initiation of B7841008 study in participants aged ≥12 to \\\u003C18 years. Subsequently, additional safety and efficacy data from adolescent participants in Study B7841005 became available for benefit\u002Frisk assessment in support of dosing participants aged ≥6 to \\\u003C12 years. Based on the positive benefit\u002Frisk assessment conducted by both internal Pfizer review and eDMC review, dosing of the ≥6 to \\\u003C12 years age group was initiated in June 2023 in B7841008 Study. Data from participants ≥6 years from B7841008 Study and Study B7841005 will support the dosing of participants aged ≥1 to \\\u003C6 years.\n\nAll participants will be provided the prefilled pen (PFP) for administration of marstacimab in the study. Use of the prefilled syringe (PFS) will be permitted at the investigator's discretion for those participants who have difficulty with administration of the PFP. Additionally, participants will be provided the PFS for use in this study in countries where the PFS is anticipated to be the only presentation available commercially. An optional, open-label, single arm, substudy using the PFP was completed in the first 23 participants rolled over from Study B7841005 who agreed to participate in the substudy.",[30,31],[69,70,71,72,73],"Factor VIII Inhibitor","Factor IX Inhibitor","PF-06741086","Marstacimab","Anti-TFPI",{"date":75,"type":43},"2026-07-01",{"date":77,"type":43},"2021-11-17",{"date":79,"type":23},"2030-07-31",{"name":81,"class":50},"Pfizer",75,{"id":84,"slug":85,"hasResults":12,"nctId":86,"briefTitle":87,"officialTitle":88,"acronym":89,"eligibilityCriteria":90,"healthyVolunteers":12,"sex":18,"minAge":91,"maxAge":92,"enrollmentInfo":93,"targetDuration":4,"studyType":95,"phases":4,"briefSummary":96,"conditions":97,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":98,"startDateStruct":99,"completionDateStruct":101,"leadSponsor":103,"locationsCount":51},"100440987","prevention-of-bleeding-in-patients-with-moderate-and-severe-hemophilia-a-playing-sports-a-comparison-between-factor-viii-and-emicizumab-prophylaxis-100440987","NCT05022459","Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis","Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis -STEP: SporTs Emicizumab Prophylaxis","STEP","Inclusion Criteria:\n\n1. Participant (if 18 years of age or older) or parent\u002FLAR is willing and able to provide written informed consent; minor participant is willing and able to provide assent, if applicable based on site and local regulations\n2. Males and females between 6 to ≤ 19 years of age at time of enrollment with moderate to severe Hemophilia A (FVIII activity ≤ 5%) without inhibitors are eligible for participation in this study\n3. Participants must be on Emicizumab or standard FVIII prophylaxis per institutional\u002Fprimary hematologist recommendations\n4. Participants must be engaging in or registered to start participating in one or more sports activities with moderate to high risk of bleeding as defined by the NHF- Playing it Safe guidelines (numerical rating \\>\u002F= 2).\n5. Participant must be willing to keep activity, bleed, and treatment logs for the duration of the study\n\nExclusion Criteria:\n\n1. Participant\u002Fparent\u002FLAR unwilling to provide informed consent\u002Fassent\n2. Unwilling to log or document bleeds and treatment information as per study guidelines\n3. Participants with any other bleeding disorders will be excluded\n4. Patients who are pregnant, planning to become pregnant, or breastfeeding should not be enrolled in the study\n5. Participants on concomittent FVIII replacement and emicizumab for sports participation","6 Years","19 Years",{"count":94,"type":23},72,"OBSERVATIONAL","Hemophilia A (HA) is a genetic bleeding disorder resulting from a deficiency or absence of factor VIII (FVIII), which is necessary in the clotting process. This disorder occurs mostly in males and in severe cases causes frequent bleeding episodes in joints and muscles which can lead to progressive damage that affects mobility and quality of life. Prophylactic FVIII administered intravenously every other day has been the standard of care treatment for HA for the past few decades.\n\nSports and physical activity are generally encouraged in patients with hemophilia on appropriate prophylactic treatment to increase strength, prevent or decrease obesity, accrue and maintain bone density and encourage normal socialization. To ensure safety with participation in sports in persons with hemophilia A (PWHA), timing of FVIII administration is often adjusted to maximize FVIII at the time of sports. The exact factor level that is needed to safely participate in sports and minimize bleeding risk is not yet known. Based on clinical practice, infusion of FVIII to near the lower limit of normal right before participation in sports generally works to prevent bleeding.\n\nThe study is looking at how well the newly approved medication Emicizumab works compared to Factor VIII to prevent bleeding in patients with Hemophilia A who play sports. The study will enroll children and adolescents who are already on Emicizumab or Factor VIII who are currently playing sports.",[30],{"date":42,"type":43},{"date":100,"type":43},"2023-08-16",{"date":102,"type":23},"2029-03",{"name":104,"class":105},"Wayne State University","OTHER",{"id":107,"slug":108,"hasResults":12,"nctId":109,"briefTitle":110,"officialTitle":111,"acronym":4,"eligibilityCriteria":112,"healthyVolunteers":12,"sex":18,"minAge":4,"maxAge":4,"enrollmentInfo":113,"targetDuration":4,"studyType":24,"phases":115,"briefSummary":117,"conditions":118,"keywords":119,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":128,"lastUpdatePostDateStruct":129,"startDateStruct":130,"completionDateStruct":132,"leadSponsor":134,"locationsCount":136},"100588394","phase-4-long-term-study-evaluating-joint-health-in-people-with-haemophilia-a-receiving-real-world-prophylactic-treatment-with-efanesoctocog-alfa-100588394","NCT06940830","Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa","A Low-interventional Study to Evaluate Long-term Effectiveness of Real-world Prophylactic Treatment With Efanesoctocog Alfa on Joint Health in People With Haemophilia A (ALTITUDE)","Inclusion Criteria:\n\n1. Male or female patient with a diagnosis of haemophilia A.\n2. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.\n3. Prescribed with efanesoctocog alfa prophylactic treatment within 6 months prior to study enrolment or at the latest at the enrolment visit, in accordance with local regulations.\n4. Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being prescribed with efanesoctocog alfa treatment.\n5. Have documented pre-study treatment data on haemophilia prophylaxis prescriptions and on treated bleeding episodes from the 12-months prior to being prescribed efanesoctocog alfa treatment and until enrolment into this study.\n6. Willing to adhere to the visit schedule and to undergo mandatory study assessment (HJHS, PROs).\n7. Willingness and the ability of the patient or their legally designated representative to document all bleeding episodes, including any treatments for those episodes.\n8. For HEAD-US subgroup: Accept to undergo annual ultrasound assessment on the index joints (ankles, elbows, knees).\n\nExclusion Criteria:\n\n1. Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A.\n2. Any positive FVIII inhibitor result (defined as inhibitor titre ≥0.6 Bethesda unit \\[BU\\]\u002FmL) from the medical records in connection to the switch to efanesoctocog alfa until the enrolment visit.\n3. Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), including for haemophilia prophylaxis, within 3 months prior to enrolment in this study.\n4. Patient not suitable for participation, whatever the reason, as judged by the Investigator, e.g., patient is not able or willing to perform the study assessments.",{"count":114,"type":23},250,[116],"PHASE4","The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.",[30],[30,120,121,122,123,124,125,126,127],"Joint Health","Prophylaxis","Factor VIII Deficiency","Blood coagulation disorder","Efanesoctogoc alfa","Coagulation protein disorder","ALTUVOCT","ALTUVIIIO","2026-06-26",{"date":40,"type":43},{"date":131,"type":43},"2025-05-21",{"date":133,"type":23},"2028-12",{"name":135,"class":50},"Swedish Orphan Biovitrum",29,{"id":138,"slug":139,"hasResults":12,"nctId":140,"briefTitle":141,"officialTitle":141,"acronym":142,"eligibilityCriteria":143,"healthyVolunteers":12,"sex":18,"minAge":144,"maxAge":145,"enrollmentInfo":146,"targetDuration":4,"studyType":95,"phases":4,"briefSummary":148,"conditions":149,"keywords":4,"overallStatus":150,"whyStopped":4,"lastUpdateSubmitDate":151,"lastUpdatePostDateStruct":152,"startDateStruct":153,"completionDateStruct":154,"leadSponsor":156,"locationsCount":4},"100599743","an-observational-study-to-learn-more-about-how-well-damoctocog-alfa-pegol-works-in-previously-treated-children-with-hemophilia-a-100599743","NCT07088458","An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A","HEM-POWR Kids","Inclusion Criteria:\n\n* Diagnosis of hemophilia A\n* Patients must be 7 to \\\u003C 12 years of age at enrollment\n* Patients previously treated for hemophilia A ((≥50 exposure days to FVIII)\n* No current evidence of FVIII inhibitor or clinical suspicion of FVIII inhibitor\n* Patients without a previous history of inhibitors or patients with a previous history of inhibitors on standard prophylaxis therapy for at least 1 year prior to study entry\n* Initiation of prescription or currently prescribed damoctocog alfa pegol for regular continuous prophylaxis (intent to treat for 52 weeks\u002Fyear)\n* Signed informed consent\u002Fassent.\n\nExclusion Criteria:\n\n* Presence of a FVIII inhibitor\n* Concurrent participation in an investigational program with interventions outside of routine clinical practice\n* Diagnosis of any other bleeding\u002Fcoagulation disorder other than hemophilia A\n* Use of another hemostatic agent as the primary method of bleeding prophylaxis during the observation period\n* Contra-indications according to the local marketing authorization\n* Current immune tolerance induction (ITI) treatment for a FVIII inhibitor","7 Years","12 Years",{"count":147,"type":23},40,"This is an observational study of children with mild, moderate, or severe hemophilia A who are receiving damoctocog alfa pegol, and are between 7 to \\\u003C12 years of age at the time of enrolment. Observational studies use data that are collected as part of routine medical care and participants do not receive any advice or any changes to healthcare as part of the study.\n\nIn this study, the data will be collected from participants who are receiving their usual treatment with damoctocog alfa pegol as prescribed by their doctor. These children have previously received damoctocog alfa pegol or other factor 8 (FVIII) products.\n\nHemophilia A is a genetic bleeding disorder. It is caused by the lack of a protein called clotting factor 8 (FVIII) that helps blood to clot properly. Lack of FVIII can result in excessive blood loss or bleeding inside the body after being injured or having surgery. At times, there is spontaneous bleeding into the joint spaces that leads to joint damage.\n\nThe drug observed in this study, damoctocog alfa pegol, is approved for doctors to prescribe to children who are at least 7 years old with hemophilia A. It is used to prevent or treat bleeding episodes and works by replacing missing FVIII in the body of people with hemophilia A.\n\nThe participants will receive damoctocog alfa pegol as prescribed independently by their own doctors during routine practice, not as a part of the study. Participants may choose to enroll in the study at any time after their doctor has prescribed damoctocog alfa pegol to prevent bleeding episodes.\n\nThe main purpose of this study is to learn more about how a treatment with damoctocog alfa pegol works to prevent bleeding episodes in routine medical practice. To answer this question, doctors will collect:\n\n* information about bleeding episodes including the type and the location of the bleed\n* information about the treatment with damoctocog alfa pegol and other FVIII products\n* the overall health status of the participants\n\nData will be collected from participants over two years after they enroll in the study or until they choose to leave the study or switch to another hemophilia A treatment. Historical data will come from the participants' medical records or by interviewing the patient and parent\u002F legal guardian. The children's parents\u002F guardians will be asked to maintain a health diary to record details of bleeding episodes and treatment with damoctocog alfa pegol. The children's parents\u002F guardians will also be asked to answer a questionnaire (Hemo QoL-SF and PedHAL) to assess the effect of hemophilia on their child's daily life.\n\nIn this study, only available data from routine care will be collected. No additional visits or tests are required as part of this study.",[30],"NOT_YET_RECRUITING","2026-06-25",{"date":128,"type":43},{"date":40,"type":23},{"date":155,"type":23},"2030-03-01",{"name":157,"class":50},"Bayer",{"id":159,"slug":160,"hasResults":12,"nctId":161,"briefTitle":162,"officialTitle":163,"acronym":4,"eligibilityCriteria":164,"healthyVolunteers":12,"sex":59,"minAge":19,"maxAge":4,"enrollmentInfo":165,"targetDuration":4,"studyType":24,"phases":167,"briefSummary":169,"conditions":170,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":171,"lastUpdatePostDateStruct":172,"startDateStruct":174,"completionDateStruct":175,"leadSponsor":177,"locationsCount":179},"100610332","phase-1-a-gene-therapy-study-of-spk-8011qq-in-adults-with-severe-or-moderately-severe-hemophilia-a-100610332","NCT07226206","A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A","A Phase 2b, Single-Arm, Open-Label, Multicenter Study of the Safety of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A","Inclusion Criteria:\n\n* Signed Informed Consent Form (ICF)\n* ≥18 years of age at the time of signing the ICF\n* Male sex assigned at birth\n* Severe or moderately severe hemophilia A, defined as endogenous FVIII:C activity levels ≤3%, as documented (historically or during the Screening Period) by a certified laboratory and where the FVIII:C level is measured more than 96 hours after the prior dose of an extended half-life FVIII replacement product or more than 72 hours after the prior dose of a standard half-life FVIII replacement product\n* Have documented treatment for a minimum of 6 months prior to screening with either of the following: plasma coagulation factor VIII (FVIII) prophylaxis, defined as receiving a prescribed dose and frequency of FVIII infusions with the intent to treat continuously for 52 weeks per year; or FVIII on demand, with a history of ≥ 5 breakthrough bleeds in the 6 months prior to screening\n* No prior history of hypersensitivity or anaphylaxis associated with the administration of any FVIII product\n* Have ≥150 exposure days to a FVIII protein product such as recombinant, plasma-derived, or extended half-life FVIII product\n* Negative screening test for inhibitor against FVIII (i.e., \\\u003C0.6 BU)\n* Candidates with prior FVIII inhibitors who are tolerized having completed successful ITI at least 5 years before screening are eligible provided they have had no evidence of inhibitor recurrence (permanent or temporary) within 5 years prior to screening as may be indicated by detection of an inhibitor, FVIII half-life \\\u003C6 hours, or FVIII recovery \\\u003C66% since completing ITI\n* Confirmed negative anti-Spark200 antibodies as documented through central laboratory testing of a serum sample\n* Acceptable hepatobiliary function according to all of the following criteria: ALT, AST, and ALP ≤2×ULN and INR \\\u003C1.4 at the time of screening; No evidence of cirrhosis or advanced liver disease on screening liver ultrasound; Otherwise no laboratory or clinical evidence of liver disease or cirrhosis, per the Investigator's judgement\n* Adequate renal function, defined as creatinine clearance ≥30 mL\u002Fmin\u002F1.73 m2 by Chronic Kidney Disease Epidemiology Collaboration formula; patients on dialysis are not eligible for the study\n* Platelet count ≥50,0000 cells\u002FµL\n* Negative HIV test at screening, with the following exception: Individuals with a positive HIV test at screening are eligible provided they are stable on an antiretroviral treatment regimen, have a cluster of differentiation (CD4) count \\>200\u002Fmm3, and undetectable viral load (\\\u003C50 gc\u002FmL)\n* Negative hepatitis B surface antigen (HBsAg) at screening\n* Positive hepatitis surface antibody (HBsAb) at screening, or a negative HBsAb at screening accompanied by either of the following: Negative hepatitis B core antibody (HBcAb); Positive HBcAb and negative hepatitis B virus (HBV) DNA test\n* Negative hepatitis C virus (HCV) antibody test at screening, or positive HCV antibody test at screening accompanied by negative HCV RNA test\n* Otherwise appropriate medical history and physical and laboratory evaluation that are acceptable for inclusion in this clinical trial\n* Are able and willing to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including the completion of applicable patient-reported outcome questionnaires\n* Agreement to adhere to the contraception requirements described in the protocol\n\nExclusion Criteria:\n\n* Are currently undergoing antiviral therapy for chronic hepatitis B or chronic hepatitis C\n* Have an inherited or acquired bleeding disorder other than hemophilia A\n* Have known inherited or acquired thrombophilia, have signs of thromboembolic disease in the Investigator's judgement, or are on current treatment for thromboembolic disease. A history of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing is not considered an exclusion criterion\n* Have had prior treatment with a vector or gene transfer agent. Nucleic acid-based vaccines, such as the vaccine for coronavirus disease 2019 (COVID-19), are not considered gene transfer agents\n* Are receiving an investigational drug concurrently or have received an investigational drug within 30 days or 5 half-lives of the last investigational drug administration, whichever is longer\n* Have a major surgical procedure planned in the 15-month period following SPK-8011QQ infusion\n* Are unable (or unwilling) to receive blood or blood products (or any standard-of-care treatment for a life-threatening condition)\n* Have concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study or that would, in the opinion of the Investigator preclude the candidate's safe participation in and completion of the study, or the interpretation of the study results\n* History of malignancy within 5 years prior to screening and up to investigational study drug administration (Day 1) with the following exceptions: Participants with curatively treated basal or squamous cell carcinoma of the skin at any time prior to investigational study drug administration (Day 1) are eligible",{"count":166,"type":23},5,[26,168],"PHASE2","This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.",[30],"2026-06-19",{"date":173,"type":43},"2026-06-23",{"date":40,"type":23},{"date":176,"type":23},"2031-07-31",{"name":178,"class":50},"Hoffmann-La Roche",2,{"id":181,"slug":182,"hasResults":12,"nctId":183,"briefTitle":184,"officialTitle":185,"acronym":4,"eligibilityCriteria":186,"healthyVolunteers":12,"sex":18,"minAge":145,"maxAge":4,"enrollmentInfo":187,"targetDuration":4,"studyType":95,"phases":4,"briefSummary":189,"conditions":190,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":193,"lastUpdatePostDateStruct":194,"startDateStruct":196,"completionDateStruct":198,"leadSponsor":200,"locationsCount":201},"100533198","a-study-to-learn-more-about-the-safety-of-damoctocog-alfa-pegol-when-used-in-routine-medical-care-in-korean-participants-with-hemophilia-a-100533198","NCT06222697","A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A","Post Marketing Surveillance Study for Jivi (Damoctocog Alfa Pegol) in Korean Patients With Hemophilia A","Inclusion Criteria:\n\n* ≥12 years of age with hemophilia A\n* Previously treated with FVIII concentrate(s) (plasma derived or recombinant)\n* Patients who have been treated with Jivi (damoctocog alfa pegol) and those for whom the decision to initiate treatment with Jivi was made as per physician's routine treatment practice with any kind of treatment modality (on-demand, prophylaxis, etc.)\n* Written informed consent from subject or legal representative; assent from subject when appropriate\n\nExclusion Criteria:\n\n* Contraindication according to the local authorized indication (including known hypersensitivity to the drug substance or any of its components (e.g., mouse or hamster protein))\n* Patients participating in an investigational program with interventions outside of routine clinical practice\n* Patients with any other diagnosis of bleeding\u002Fcoagulation disorder other than hemophilia A\n* Patients on immune tolerance induction treatment at the time of enrollment",{"count":188,"type":23},20,"In this study, researchers will observe and study the data from participants with hemophilia A who receive damoctocog alfa pegol as prescribed by their doctors. Participants will not receive any advice or changes to their healthcare during the study.\n\nHemophilia A is a genetic bleeding disorder. It is caused by the lack of a protein called clotting factor 8 (FVIII) that helps blood to clot properly. Lack of FVIII can result in excessive blood loss or bleeding inside the body after being injured or having surgery.\n\nThe study drug, damoctocog alfa pegol, can be used to prevent or treat bleeding episodes by replacing missing FVIII in the body of people with hemophilia A. It is already approved for people with hemophilia A who are at least 12 years old and have previously used other hemophilia A treatments.\n\nThrough this study, researchers want to learn more about its safety in a real-world setting.\n\nThe participants will receive damoctocog alfa pegol as prescribed by their doctors during routine practice according to the approved product information.\n\nThe main purpose of this study is to learn more about how safe damoctocog alfa pegol is in Korean participants with hemophilia A who previously used other hemophilia A treatments. To do this, researchers will collect information about any medical problems participants have during their treatment.\n\nData will be collected from December 2023 to March 2026 and cover a period of about 8 months for each participant. Data will come from participants' health records and information collected during their routine clinic visits.\n\nIn this study, only available data from routine care will be collected. No visits or tests are required as part of this study.",[30,191,192],"Prophylaxis of Bleeding","Treatment of Bleeding","2026-06-18",{"date":195,"type":43},"2026-06-22",{"date":197,"type":43},"2024-01-24",{"date":199,"type":23},"2028-03-31",{"name":157,"class":50},1,{"id":203,"slug":204,"hasResults":12,"nctId":205,"briefTitle":206,"officialTitle":207,"acronym":4,"eligibilityCriteria":208,"healthyVolunteers":12,"sex":18,"minAge":209,"maxAge":210,"enrollmentInfo":211,"targetDuration":4,"studyType":24,"phases":212,"briefSummary":213,"conditions":214,"keywords":215,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":193,"lastUpdatePostDateStruct":219,"startDateStruct":220,"completionDateStruct":222,"leadSponsor":224,"locationsCount":201},"100302430","phase-1-lentiviral-fviii-gene-therapy-100302430","NCT03217032","Lentiviral FVIII Gene Therapy","Lentiviral FVIII Gene Therapy for Hemophilia A","Inclusion Criteria:\n\n1. Able to provide informed consent and comply with requirements of the study.\n2. Males ≥2 years with confirmed diagnosis of hemophilia A (endogenous factor VIII ≤2 IU\u002FdL or ≤2% of normal).\n3. A minimum average of 4 bleeding events per year requiring episodic treatment of factor VIII infusions or prophylactic factor VIII infusions.\n4. No measurable factor VIII inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor VIII protein.\n5. Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences.\n\nExclusion Criteria:\n\n1. Significant liver dysfunction as defined by abnormal alanine transaminase, bilirubin and alkaline phosphatase.\n2. History of inhibitor against factor VIII.\n3. Evidence of active hepatitis B or C and currently on antiviral therapy.\n4. Have serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200\u002Fmm3 (subjects who are HIV+ and stable with CD4 count \\>200\u002Fmm3 and undetectable viral load are eligible to enroll).\n5. Any evidence of active infection or any immunosuppressive disorder.\n6. Participated in a gene transfer trial within the last 6 months or in a clinical trial with an investigational drug within the last 12 weeks.\n7. Unable or unwilling to comply with study assessments.","2 Years","65 Years",{"count":51,"type":23},[26],"This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.",[30],[30,216,217,218],"factor VIII","Gene therapy","lentiviral vector",{"date":173,"type":43},{"date":221,"type":43},"2025-06-01",{"date":223,"type":23},"2028-06-01",{"name":225,"class":105},"Shenzhen Geno-Immune Medical Institute",{"id":227,"slug":228,"hasResults":12,"nctId":229,"briefTitle":230,"officialTitle":231,"acronym":4,"eligibilityCriteria":232,"healthyVolunteers":12,"sex":59,"minAge":19,"maxAge":4,"enrollmentInfo":233,"targetDuration":4,"studyType":95,"phases":4,"briefSummary":235,"conditions":236,"keywords":4,"overallStatus":150,"whyStopped":4,"lastUpdateSubmitDate":237,"lastUpdatePostDateStruct":238,"startDateStruct":239,"completionDateStruct":241,"leadSponsor":243,"locationsCount":244},"100627236","post-approval-observational-study-to-learn-more-about-how-safe-octocog-alfa-is-and-how-well-it-works-in-patients-with-severe-hemophilia-a-in-india-100627236","NCT07446010","Post Approval Observational Study to Learn More About How Safe Octocog Alfa is and How Well it Works in Patients With Severe Hemophilia A in India","A Prospective, Multicenter, Open-label, Phase IV Post-authorization Safety Study Conducted in India to Assess the Safety and Treatment Outcomes of Octocog Alfa in Real-world Practice for On-demand Treatment of Acute Bleeds in Previously Treated Severe Hemophilia A Patients in India","Inclusion Criteria:\n\n* Male patients aged 18 years or older with a documented diagnosis of severe Hemophilia A, defined by a baseline Factor VIII (FVIII) activity level of less than 1% (\\\u003C0.01 IU\u002FmL) in accordance with the Hemophilia Severity Classification\n* Previously treated with FVIII concentrate(s) (plasma derived or recombinant, including Octocog alfa) either on-demand or prophylactically for at least 100 Exposure Days (EDs).\n* Patients for whom the decision to initiate on-demand treatment with Octocog alfa for acute bleeding was made as per the investigator's routine treatment practice. This will include patients who are already on on-demand treatment with Octocog alfa as well.\n* Written informed consent from the patient or legal representative\n\nExclusion Criteria:\n\n* Known contraindication according to the local prescriber information\n* Patients who are participating in an investigational program with interventions outside of routine clinical practice.\n* Patients with any other diagnosis of bleeding\u002Fcoagulation disorder other than Hemophilia A.\n* Patients who are on ongoing prophylactic treatment with any FVIII concentrate or non-factor treatments like emicizumab.\n* Patients exhibiting any of the following laboratory abnormalities at screening:\n* Known platelet count \\\u003C100,000 mm3\n* Known Serum Creatinine \\>2 folds upper the normal limit\n* Known Hepatic AST or ALT \\>5 folds upper the normal limit\n* Patients who have received an on-demand infusion with any other FVIII (different to Octocog alfa), FVII or Activated prothrombin complex concentrate product (aPCC\u002FFEIBA) 72 hours before the enrollment.\n* History or presence of FVIII inhibitor with a titer ≥ 0.6 with Nijmegen modified Bethesda Assay (NBA), or a clinical history suggestive of an inhibitor necessitating changes to treatment\n* Patient concerns or other barriers precluding adequate understanding or cooperation.",{"count":234,"type":23},33,"Hemophilia A is a genetic condition that makes it hard for blood to clot properly. This happens because the body does not have enough of a protein called Factor VIII, which helps stop bleeding. The main goal of treating someone with hemophilia is to stop and prevent bleeding by giving them the missing Factor VIII. This treatment can be given when a person starts bleeding (called on-demand treatment), or it can be given regularly to prevent bleeding (called prophylactic therapy). In India, most people with hemophilia A get treatment only when they have a bleeding episode, and only a few receive regular preventive treatment. Octocog alfa (also known as BAY 81-8973) is a modern, laboratory-made version of Factor VIII. It is made without using any human or animal materials and has special features that help it work better in the body. In India, Octocog alfa is approved for use in adults and children with hemophilia A to:\n\n* Treat and control bleeding episodes when they happen\n* Manage bleeding during surgery\n* Prevent bleeding by giving regular treatment The safety and effectiveness of Octocog alfa have been shown in several global studies. This new study is required by Indian health authorities to collect information about how safe Octocog alfa is and how well it works in people with hemophilia A who have already received treatment. The study will look at how Octocog alfa is used in real-life medical practice in India, including how doctors prescribe it, how patients use it, and what treatment results they have.",[30],"2026-06-17",{"date":193,"type":43},{"date":240,"type":23},"2026-09-01",{"date":242,"type":23},"2027-09-30",{"name":157,"class":50},6,{"id":246,"slug":247,"hasResults":12,"nctId":248,"briefTitle":249,"officialTitle":249,"acronym":4,"eligibilityCriteria":250,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":210,"enrollmentInfo":251,"targetDuration":4,"studyType":24,"phases":253,"briefSummary":254,"conditions":255,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":256,"lastUpdatePostDateStruct":257,"startDateStruct":259,"completionDateStruct":261,"leadSponsor":263,"locationsCount":265},"100641949","phase-1-an-open-label-multicenter-phase-iii-clinical-trial-to-evaluate-the-safety-tolerability-efficacy-and-pharmacokineticpharmacodynamic-pkpd-characteristics-of-sr604-injection-in-patients-with-hemophilia-ab-and-congenital-factor-vii-deficiency-100641949","NCT07644832","An Open-label, Multicenter Phase I\u002FII Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic\u002FPharmacodynamic (PK\u002FPD) Characteristics of SR604 Injection in Patients With Hemophilia A\u002FB and Congenital Factor VII Deficiency","Inclusion Criteria:\n\n1. Age ≥18 years and ≤65 years at the time of signing informed consent, regardless of sex;\n2. Clinically diagnosed with Hemophilia A or B or congenital coagulation Factor VII deficiency, and must meet the following criteria:\n\n   1. Hemophilia A or B patients with historical or screening FVIII activity level \\\u003C1% or FIX activity level ≤2%; Note: Hemophilia A or B patients with or without inhibitors may be enrolled. For patients without inhibitors (inhibitor titer \\\u003C0.6 BU\u002FmL), they must have previously received coagulation factor treatment with exposure days (EDs) \\>50 days.\n   2. Congenital coagulation Factor VII deficiency patients with historical or screening FVII activity \\\u003C10%;\n3. Part A only: Received on-demand treatment with FVIII, FIX, recombinant human coagulation Factor VIIa (rFVIIa), or PCC for bleeding events within 1 month prior to screening;\n4. Part B\u002FPart C only: Accessible bleeding and treatment records (factor replacement or bypassing agent therapy) for at least 3 months prior to enrollment. Hemophilia A or B patients must have received on-demand treatment with ≥3 treated de novo bleeding episodes within 3 months prior to enrollment. Congenital coagulation Factor VII deficiency patients must have ≥2 treated de novo bleeding episodes within 3 months prior to enrollment;\n5. No active bleeding symptoms prior to first dosing;\n6. The subject or a legally acceptable representative has a full understanding of and can comply with the protocol requirements, has the willingness to complete the study as planned, and voluntarily agrees to provide biological samples for testing as required by the protocol;\n7. The subject is able to understand the procedures and methods of this clinical trial, has been fully informed, and voluntarily participates in the trial by personally signing the informed consent form.\n\nExclusion Criteria:\n\n1. Subjects with a known history of hypersensitivity to the investigational medicinal product or any of its components;\n2. Intolerance to subcutaneous injection or presence of other local skin abnormalities or dermatological conditions that may affect administration and safety assessment;\n3. Subjects meeting any of the following criteria at screening:\n\n   1. Hemoglobin \\\u003C60 g\u002FL;\n   2. Platelet count \\\u003C100 × 10\\^9\u002FL;\n   3. Hepatic or renal impairment: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥2.5 × upper limit of normal (ULN), or total bilirubin ≥1.5 × ULN; or serum creatinine (Cr) ≥1.5 × ULN;\n4. Positive result(s) for hepatitis B virus surface antigen (HBsAg), anti-human immunodeficiency virus (HIV) antibody, and\u002For Treponema pallidum-specific antibody;\n5. Clinically diagnosed with active hepatitis C;\n6. Any other bleeding disorder or any other disease causing significant coagulation abnormalities (e.g., platelet disorders, vitamin K deficiency, etc.) other than Hemophilia A or B and congenital coagulation Factor VII deficiency;\n7. Protein C deficiency or protein S deficiency;\n8. History of or current thrombosis, family history of thrombosis, or history of thrombophilia prior to signing informed consent;\n9. Intracranial hemorrhage due to Hemophilia A or B or congenital coagulation Factor VII deficiency within 2 years prior to screening;\n10. Severe cardiac disease, such as unstable angina, congestive heart failure (New York Heart Association Class ≥III), severe arrhythmia (QTc interval \\>450 ms, corrected by Fridericia's formula), or uncontrolled hypertension (systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥95 mmHg);\n11. Received recombinant human coagulation Factor VIIa (rFVIIa) within 48 hours prior to first dosing; received any FVIII-containing product within 72 hours prior to first dosing; received any FIX-containing product within 96 hours prior to first dosing; long-acting products of the above have not completed a washout of 5 half-lives;\n12. Used or requires use of any anticoagulant, antifibrinolytic agent, or chemical drug, biological product, or traditional Chinese medicine affecting platelet function, including nonsteroidal anti-inflammatory drugs (NSAIDs) such as aspirin, within 1 week prior to first dosing or during the trial;\n13. Received whole blood or plasma therapy within 2 weeks prior to first dosing;\n14. Received emicizumab treatment within 6 months prior to first dosing;\n15. Received or planned to receive vaccination within 4 weeks prior to first dosing or during the trial;\n16. Underwent major surgery (e.g., orthopedic surgery, abdominal surgery) within 1 month prior to first dosing, or planned to undergo surgery during the study;\n17. Enrolled in another clinical trial within 1 month prior to first dosing;\n18. History of drug abuse or alcoholism (alcoholism criteria: long-term drinking history exceeding 5 years, equivalent to ethanol intake ≥40 g\u002Fday, or heavy drinking within 2 weeks, equivalent to ethanol intake \\>80 g\u002Fday. Ethanol amount (g) conversion formula = alcohol volume (mL) × ethanol content (%) × 0.8);\n19. Psychiatric illness or significant mental impairment, or incapacity or lack of cognitive ability due to other reasons;\n20. Plans to have children or donate sperm during the entire trial period up to 6 months after the last dose, or unwilling to use effective physical contraceptive measures (e.g., condoms);\n21. Subjects with clinically significant disease or other conditions that the investigator considers unsuitable for participation in the clinical trial (e.g., the patient cannot benefit from the clinical trial);\n22. Subjects deemed by the investigator to have poor compliance, rendering efficacy evaluation impossible or with low likelihood of completing the planned treatment course and follow-up.",{"count":252,"type":23},76,[26,168],"The purpose of this study is to evaluate the safety, tolerability, immunogenicity , PK, and PD of a single dose of SR604 in participants with Hemophilia A or Hemophilia B, with or without inhibitors （Part A）and to evaluate the safety, PK, PD, and efficacy of multiple doses of SR604 in participants with Hemophilia A or Hemophilia B, or Factor VII (FVII) deficiency, with or without inhibitors (Part B and Part C).",[30,31,32],"2026-06-08",{"date":258,"type":43},"2026-06-12",{"date":260,"type":43},"2024-05-31",{"date":262,"type":23},"2026-12-31",{"name":264,"class":50},"Shanghai RAAS Blood Products Co., Ltd.",9,{"id":267,"slug":268,"hasResults":12,"nctId":269,"briefTitle":270,"officialTitle":270,"acronym":4,"eligibilityCriteria":271,"healthyVolunteers":12,"sex":59,"minAge":19,"maxAge":272,"enrollmentInfo":273,"targetDuration":4,"studyType":95,"phases":4,"briefSummary":275,"conditions":276,"keywords":277,"overallStatus":150,"whyStopped":4,"lastUpdateSubmitDate":282,"lastUpdatePostDateStruct":283,"startDateStruct":284,"completionDateStruct":286,"leadSponsor":288,"locationsCount":201},"100510936","liver-biopsy-following-gene-therapy-for-hemophilia-100510936","NCT05932914","Liver Biopsy Following Gene Therapy For Hemophilia","Inclusion Criteria:\n\n* Age ≥18 to 80 years\n* Patients, who were enrolled and treated in one of the following clinical trials:\n\n  * AGT4HB (EudraCT number: 2005-005711-17; NCT00979238) - FIX AAV gene therapy trial (sponsor: St. Jude Children's Research Hospital)\n  * GO8 (EudraCT number:2014-003880-38; NCT02576795) - FVIII AAV gene therapy trial (sponsor: University College, London)\n* Able to give informed consent\n* Able to comply with study requirements\n\nExclusion Criteria (Do not apply to participants who will not undergo liver biopsy, and have leftover liver tissue from a previous biopsy procedure, because all exclusion criteria only cover the safety considerations for the biopsy procedure.):\n\n* Any condition that, in the opinion of the investigator or sponsor of the ongoing clinical trial in which the patient is participating in, would prevent the patient from fully complying with the requirements of the clinical trial and\u002For would influence or interfere with evaluation and interpretation of subject safety or efficacy result of that ongoing clinical trial\n* Platelet count \\\u003C140x10\\^9\u002FL\n* INR \\>1.5\n* Abnormal kidney function with estimated GFR \\\u003C50 mL\u002Fmin (calculated using the CKD-EPI equation)\n* Known allergy to iodine-based intravenous contrast agents\n* Known allergy to local or general anesthetics\n* Known allergic reaction to FVIII\u002FFIX concentrate infusions\n* Presence of FVIII inhibitor or FIX inhibitor (historical result can be used if done within 14 weeks of this liver biopsy)\n* Evidence of any bleeding disorder other than hemophilia A or B","80 Years",{"count":274,"type":23},8,"This observational study will obtain liver biopsy samples and evaluate the long-term effect of adeno-associated virus (AAV)-mediated gene therapy on the liver tissue in adult patients with hemophilia A or hemophilia B who have previously been treated with a factor VIII or factor IX gene-containing AAV-vector for liver-targeted gene transfer. Participants are from a cohort of patients treated with AAV-mediated gene transfer and at least 6 months after vector infusion.",[30,31],[278,279,280,30,31,281],"AAV-mediated factor VIII (FVIII) gene transfer","AAV-mediated factor IX (FIX) gene transfer","Gene Therapy","Transjugular liver biopsy","2026-06-05",{"date":256,"type":43},{"date":285,"type":23},"2026-07",{"date":287,"type":23},"2030-01",{"name":289,"class":105},"St. Jude Children's Research Hospital",{"id":291,"slug":292,"hasResults":12,"nctId":293,"briefTitle":294,"officialTitle":295,"acronym":296,"eligibilityCriteria":297,"healthyVolunteers":12,"sex":18,"minAge":145,"maxAge":4,"enrollmentInfo":298,"targetDuration":4,"studyType":24,"phases":300,"briefSummary":301,"conditions":302,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":303,"lastUpdatePostDateStruct":304,"startDateStruct":305,"completionDateStruct":307,"leadSponsor":309,"locationsCount":274},"100624974","phase-3-a-clinical-study-to-evaluate-the-effects-of-nxt007-compared-to-emicizumab-prophylaxis-in-people-with-hemophilia-a-100624974","NCT07416604","A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A","A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Emicizumab Prophylaxis in People With Hemophilia A","ZEBRHA 2","Inclusion Criteria:\n\n* Diagnosis of severe (FVIII:C \\\u003C1 International Unit per decilitre \\[IU\u002FdL\\]) or moderate (FVIII:C between ≥1 IU\u002FdL and ≤5 IU\u002FdL) congenital hemophilia A with or without inhibitors against FVIII\n* Diagnosis of mild (FVIII:C between \\>5 IU\u002FdL and \\\u003C40 IU\u002FdL) congenital hemophilia A with chronic FVIII inhibitors, defined as documented FVIII inhibitor ( ≥0.6 BU\u002FmL or ≥1.0 BU\u002FmL only for laboratories with a historical sensitivity cutoff for inhibitor detection of 1.0 BU\u002FmL) and chronic reduction of endogenous baseline FVIII:C to \\\u003C5 IU\u002FdL for ≥12 months\n* Documented historical FVIII inhibitor assay results within the 12 months prior to enrollment\n* Documentation of the details of prophylactic and episodic FVIII treatment, bypassing agent (BPA) treatment, emicizumab prophylaxis treatment, and the number and type of bleeding episodes for at least the last 6 months prior to screening\n* For potential participants taking on-demand treatments prior to study entry: agreement to move to a prophylaxis treatment with either emicizumab or NXT007, according to assigned randomization\n\nExclusion Criteria:\n\n* Sensitivity to any of the study investigations, or components thereof, or drug or other allergy that, in the opinion of the investigator, contraindicates participation in the study\n* Use of systemic immunomodulators (e.g., interferon or rituximab) at the time of enrollment or planned use during the study, except for antiretroviral therapy to treat HIV\n* Refusal to accept plasma-derived and\u002For blood product transfusion support in an emergency scenario\n* Planned surgery (excluding minor procedures, such as non-molar tooth extraction or incision and drainage) during the study\n* History of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease (e.g., severe left ventricular systolic dysfunction, left ventricular hypertrophy), coronary heart disease (symptomatic or with ischemia demonstrated by diagnostic testing)\n* History or presence of an abnormal ECG that is deemed clinically significant, (e.g., complete left bundle branch block, second- or third-degree atrioventricular heart block) or evidence or clinical history of prior myocardial infarction",{"count":299,"type":23},360,[65],"The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with emicizumab prophylaxis in people age 12 years and older with severe or moderate congenital hemophilia A without factor VIII (FVIII) inhibitors or with hemophilia A of any severity (severe, moderate, and mild) with FVIII inhibitors.",[30],"2026-06-04",{"date":256,"type":43},{"date":306,"type":43},"2026-04-27",{"date":308,"type":23},"2032-01-29",{"name":178,"class":50},{"id":311,"slug":312,"hasResults":12,"nctId":313,"briefTitle":314,"officialTitle":315,"acronym":316,"eligibilityCriteria":317,"healthyVolunteers":12,"sex":18,"minAge":145,"maxAge":4,"enrollmentInfo":318,"targetDuration":4,"studyType":24,"phases":320,"briefSummary":321,"conditions":322,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":303,"lastUpdatePostDateStruct":323,"startDateStruct":324,"completionDateStruct":326,"leadSponsor":328,"locationsCount":179},"100624968","phase-3-a-clinical-study-to-evaluate-the-effects-of-nxt007-compared-to-factor-viii-prophylaxis-in-participants-with-hemophilia-a-100624968","NCT07416526","A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A","A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Factor VIII Prophylaxis in People With Hemophilia A Without Inhibitors","ZEBRHA 1","Inclusion Criteria:\n\n* Diagnosis of severe (FVIII:C \\\u003C1 IU\u002FdL \\[International Unit per decilitre\\]) or moderate (FVIII:C between ≥1 IU\u002FdL and ≤5 IU\u002FdL) congenital hemophilia A without inhibitors against FVIII\n* No documented inhibitor (i.e., \\\u003C0.6 BU\u002FmL \\[Bethesda unit per millilitre\\]), FVIII half-life ≥6 hours, or FVIII recovery \\>66% in the last 3 years prior to screening\n* Documented historical negative test for FVIII inhibitor (i.e., \\\u003C0.6 BU\u002FmL) within 12 months prior to enrollment\n* Documentation of the details of prophylactic and episodic FVIII treatment and of the number and type of bleeding episodes for at least the last 6 months prior to screening\n* Agreement to adhere to the contraception requirements (for potential participants with childbearing potential)\n\nExclusion Criteria:\n\n* Sensitivity to any of the study investigations, or components thereof, or drug or other allergy that, in the opinion of the investigator, contraindicates participation in the study\n* Use of systemic immunomodulators (e.g., interferon or rituximab) at the time of enrollment or planned use during the study, except for anti-retroviral therapy to treat HIV\n* Planned surgery (excluding minor procedures such as non-molar tooth extraction, incision and drainage) during the study\n* History or presence of an abnormal ECG that is deemed clinically significant, (e.g., complete left bundle branch block, second- or third- degree atrioventricular heart block) or ECG evidence or clinical history of prior myocardial infarction\n* Refusal to accept plasma-derived and\u002For blood product transfusion support in an emergency scenario\n* History of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease (e.g., severe left ventricular systolic dysfunction, left ventricular hypertrophy), coronary heart disease (symptomatic or with ischemia demonstrated by diagnostic testing)",{"count":319,"type":23},126,[65],"The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis compared with Factor VIII (FVIII) prophylaxis in participants with severe or moderate congenital hemophilia A without inhibitors. The study will include people aged ≥12 years old with severe or moderate congenital hemophilia A without inhibitors on previous FVIII prophylaxis treatment.",[30],{"date":256,"type":43},{"date":325,"type":43},"2026-05-15",{"date":327,"type":23},"2031-09-23",{"name":178,"class":50},{"id":330,"slug":331,"hasResults":12,"nctId":332,"briefTitle":333,"officialTitle":334,"acronym":4,"eligibilityCriteria":335,"healthyVolunteers":12,"sex":59,"minAge":336,"maxAge":337,"enrollmentInfo":338,"targetDuration":4,"studyType":95,"phases":4,"briefSummary":340,"conditions":341,"keywords":343,"overallStatus":150,"whyStopped":4,"lastUpdateSubmitDate":348,"lastUpdatePostDateStruct":349,"startDateStruct":350,"completionDateStruct":351,"leadSponsor":353,"locationsCount":4},"100640043","a-prospective-assessment-of-bone-health-in-patients-with-severe-hemophilia-a-on-factor-viii-vs-factor-mimetic-prophylaxis-efa-emi-bone-health-study-100640043","NCT07582276","A Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)","A Multi-institution Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)","Inclusion Criteria:\n\n1. The participant or legally authorized representative is willing and able to provide written informed consent.\n2. Diagnosis of severe hemophilia A (factor VIII activity \\\u003C 1%).\n3. Male sex.\n4. Age between 30 and 50 years (inclusive).\n5. BMI between 18.5 and 40 kg\u002Fm2\n6. The participant must have been on prophylaxis with Efanesoctocog alfa or Emicizumab for at least 3 months prior to enrollment and intend to remain on the current regimen for the next 5 years.\n7. Willingness to undergo all research procedures, including DEXA scans and the collection of blood samples.\n8. Willingness to complete all standard-of-care bleeding and treatment logs.\n\nExclusion Criteria:\n\n1. Unwillingness of the participant, parent, or legally authorized representative to provide informed consent.\n2. Diagnosis of a bleeding disorder other than or in addition to severe hemophilia A.\n3. Active Factor VIII inhibitors at the time of enrollment\n4. History of a disease known to influence bone metabolism unrelated to a bleeding disorder. (Examples: Paget's disease, osteogenesis imperfecta, Ehlers Danlos syndrome, Hyperparathyroidism)\n5. Past or present treatment with any anti-osteoporotic medication, excluding oral vitamin D or oral calcium supplements.\n6. Documented HIV infection or HCV infection (whether in progress or cured) at the cirrhotic stage.\n7. Presence of a non-removable metal device that would interfere with research procedures.\n8. Inability to tolerate a DEXA scan due to limited range of motion or body habitus.\n9. History of bone fractures or surgical repair within 8 weeks prior to enrollment.\n10. Participants with weight \\>300 pounds, due to limitations of DEXA scanner","30 Years","50 Years",{"count":339,"type":23},50,"This study will compare bone mineral density in patients with severe hemophilia A receiving prophylaxis with emicizumab or efanesoctocog alfa. Participants will undergo assessments of bone mineral density, bone remodeling biomarkers, thrombin generation, plasmin generation, and joint health over a five-year period. The study aims to evaluate whether differences in prophylactic therapy are associated with differences in bone health outcomes.",[342],"Hemophilia a",[344,345,346,347],"Bone Health","Emicizumab","Efanesoctocog alfa","hemophilia","2026-06-03",{"date":303,"type":43},{"date":75,"type":23},{"date":352,"type":23},"2032-08-01",{"name":354,"class":105},"Arkansas Children's Hospital Research Institute",{"id":356,"slug":357,"hasResults":12,"nctId":358,"briefTitle":359,"officialTitle":360,"acronym":4,"eligibilityCriteria":361,"healthyVolunteers":12,"sex":59,"minAge":209,"maxAge":362,"enrollmentInfo":363,"targetDuration":4,"studyType":24,"phases":365,"briefSummary":366,"conditions":367,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":348,"lastUpdatePostDateStruct":368,"startDateStruct":369,"completionDateStruct":371,"leadSponsor":373,"locationsCount":374},"100515126","phase-1-a-study-to-evaluate-the-safety-tolerability-pharmacokinetics-pharmacodynamics-and-efficacy-of-nxt007-in-persons-with-severe-or-moderate-hemophilia-a-100515126","NCT05987449","A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A","A Phase I\u002FII Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A","Inclusion Criteria:\n\n* Diagnosis of severe (Factor VIII \\[FVIII\\] coagulant activity \\\u003C1 IU\u002FdL) or moderate (FVIII coagulant activity ≥1 IU\u002FdL and ≤5 IU\u002FdL) congenital hemophilia A with or without inhibitors against FVIII\n* Participants with FVIII inhibitors: participants using recombinant activated factor VII (rFVIIa) or willing to switch to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds, trauma, or procedures\n* Historic local FVIII inhibitor test results being available during screening to confirm any previous inhibitor history and current status\n* Participants who previously successfully completed immune tolerance induction (ITI) must have done so at least 5 years before screening and must have no evidence of inhibitor recurrence (permanent or temporary) since. FVIII tolerance defined as \\\u003C0.6 Bethesda unit (BU)\u002FmL (\\\u003C1.0 BU\u002FmL only for laboratories with an historical sensitivity cutoff for inhibitor detection of 1.0 BU\u002FmL) and in vivo recovery \\>66%\n* Documentation of number and type of bleeding episodes in the last 24 weeks prior to enrollment\n* Adequate hematologic function, defined as platelet count ≥100,000 cells\u002FμL and hemoglobin ≥11 g\u002FdL at the time of screening\n* Adequate hepatic function defined as total bilirubin ≤1.5× age-adapted upper limit of normal (ULN) (excluding Gilbert syndrome) and both aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3× age-adapted ULN at the time of screening, and no clinical signs or known laboratory\u002Fradiographic evidence consistent with cirrhosis. For patients with Gilbert syndrome, bilirubin should be \\\u003C4 mg\u002FdL or 68.4 umol\u002FL at the time of screening.\n* For Part 1 only: Adequate renal function, defined as serum creatinine ≤2.5× age-adapted ULN and calculated creatinine clearance ≥30 mL\u002Fmin by Cockroft-Gault formula\n* For Part 2 only: Adequate renal function, defined as serum creatinine ≤1.5× age-adapted ULN. When the serum creatinine is ≥1.5× ULN, creatinine clearance by Bedside Schwartz formula must be \\>70 mL\u002Fmin\u002F1.73m\\^2.\n* Willingness and ability to comply with schedules visits, treatment plans, laboratory tests, and other study procedures\n\nExclusion Criteria:\n\n* Inherited or acquired bleeding disorders other than congenital hemophilia A\n* Ongoing or planned ITI therapy\n* Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease\n* At high risk for thrombotic microangiopathy (TMA), including past personal or family history of TMA, in the investigator's judgment\n* For Part 1 only: Personal history of ischemic heart disease, cerebrovascular disease, or diabetes mellitus\n* For Part 1 only: Strong family history of ischemic heart disease or cerebrovascular disease (i.e., first degree relatives such as parents, full siblings, or children): male relatives diagnosed under the age of 55 years and females under the age of 65 years\n* For Part 1 only: Previous or concomitant malignancies or leukemia\n* Other conditions (e.g., autoimmune conditions such as Systemic Lupus erythematosus and other systemic inflammatory disorders) that may currently increase the risk of bleeding or thrombosis\n* History of clinically significant allergies\n* Receipt of any of the following:\n\n  i) An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration or normalization of targeted parameters (e.g., anti-thrombin), whichever is longer; ii) A non-hemophilia-related investigational drug within last 30 days or 5 half-lives, whichever is shorter; iii) Any other investigational drug currently being administered or planned to be administered; iv) Prior gene therapy or gene therapy planned to be administered; v) Use of systemic immunomodulators (e.g., interferon or rituximab) at enrollment or planned use during the study, with the exception of anti-retroviral therapy to treat HIV.\n* Protein C activity, protein S free antigen, or anti-thrombin III activity levels below the lower limit of the reference range at screening\n* Known HIV infection with CD4 counts \\\u003C200 cells\u002FμL\n* History of severe allergic or anaphylactic reactions to monoclonal antibody therapy and to chimeric or humanized antibodies or fusion proteins\n* Known hypersensitivity to Chinese hamster ovary cell products or to excipient content\n* History or presence of an abnormal ECG that is deemed clinically significant, (e.g., complete left bundle branch block, second- or third -degree atrioventricular heart block), including atrial fibrillation or evidence of prior myocardial infarction\n* QT interval corrected through use of Fridericia's formula (QTcF) \\>450 ms demonstrated by at least two ECGs \\>30 minutes apart\n* History of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease (e.g., severe left ventricular systolic dysfunction, left ventricular hypertrophy), coronary heart disease (symptomatic or with ischemia demonstrated by diagnostic testing), clinically significant electrolyte abnormalities (e.g., hypokalemia, hypomagnesemia, hypocalcemia), or family history of sudden unexplained death or long QT syndrome\n* Current treatment with medications that are well known to prolong the QT interval","59 Years",{"count":364,"type":23},60,[26,168],"WP44714 is a Phase I\u002FII, open-label, non-randomized, global, multicenter trial consisting of two parts:\n\n* Part 1 is a multiple-ascending dose (MAD) study in adult and adolescent male participants with severe or moderate hemophilia A with or without factor VIII (FVIII) inhibitors.\n* Part 2 is a multiple-dose study in pediatric male participants with severe or moderate hemophilia A with or without FVIII inhibitors.\n\nThe overall aim of the study is to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy of NXT007.",[30],{"date":303,"type":43},{"date":370,"type":43},"2023-09-21",{"date":372,"type":23},"2030-06-16",{"name":178,"class":50},14,{"id":376,"slug":377,"hasResults":12,"nctId":378,"briefTitle":379,"officialTitle":380,"acronym":4,"eligibilityCriteria":381,"healthyVolunteers":12,"sex":59,"minAge":145,"maxAge":4,"enrollmentInfo":382,"targetDuration":4,"studyType":24,"phases":383,"briefSummary":384,"conditions":385,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":386,"lastUpdatePostDateStruct":387,"startDateStruct":389,"completionDateStruct":391,"leadSponsor":393,"locationsCount":179},"100527254","phase-4-a-study-to-test-a-medicine-fitusiran-for-preventing-bleeds-in-people-with-severe-hemophilia-who-previously-received-preventive-treatment-with-emicizumab-100527254","NCT06145373","A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab","An Open-label, Single-arm Treatment Study to Investigate the Safety and Tolerability of Switching From Emicizumab to Fitusiran Prophylaxis in Male Participants Aged ≥12 Years of Age With Severe Hemophilia A, With or Without Inhibitors","Inclusion Criteria:\n\n* Male participants must be ≥12 years of age inclusive, at the time of signing the informed consent\n* Diagnosis of severe congenital hemophilia A (FVIII \\\u003C 1%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.\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 anamnestic response.\n* Participants who are currently on the full labeled dose of emicizumab prophylaxis, irrespective of inhibitor\u002Fnon-inhibitor status.\n* Signed informed consent\u002Fassent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.\n\nExclusion Criteria:\n\nParticipants are excluded from the study if any of the following criteria apply:\n\n* Known coexisting bleeding disorders\n* History of antiphospholipid antibody syndrome.\n* History of arterial or venous thromboembolism, atrial fibrillation, significant valvular disease, myocardial infarction, angina, transient ischemic attack, or stroke. Participants who have experienced thrombosis associated with indwelling venous access may be enrolled.\n* Presence of clinically significant liver disease\n* Current or prior participation in a fitusiran trial\n* Current or prior participation in a gene therapy trial\n* AT activity \\\u003C60% at Screening, as determined by central laboratory measurement\n* Coexisting thrombophilic disorder - Hepatitis C virus antibody positive, except participants who have negative Hepatitis C viral load and no evidence of cirrhosis\n* Presence of acute hepatitis, ie, hepatitis A, hepatitis E.\n* Presence of acute or chronic hepatitis B infection\n* Known to be HIV positive with CD4 count \\\u003C200 cells\u002FμL.\n* Reduced renal function\n\nThe above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.",{"count":188,"type":23},[116],"This is an exploratory, single group, Phase 4, study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis.\n\nThis study aims to evaluate the safety and tolerability of switching to fitusiran after a transition period from the last dose of emicizumab. The study will be conducted in male participants with severe hemophilia A, with or without inhibitors, aged ≥12 years, who were previously receiving emicizumab prophylaxis.\n\nStudy details include:\n\n* The study duration will be up to approximately 28 months:\n\n  * There will be an approximately 2-month screening period.\n  * There will be an approximately 2-month period before fitusiran treatment starts (pre-fitusiran treatment period)\n  * The fitusiran treatment duration will be up to 18-months (fitusiran treatment period)\n  * The antithrombin (AT) follow-up (FU) period will be approximately 6 months after the last dose of fitusiran (during which the AT activity level will be monitored at approximately monthly intervals following the final fitusiran dose until AT activity levels return to at least 60%).\n* The study site visits are scheduled at monthly\u002F every 2 months intervals of 28 days (4 weeks) \u002F 56 days (8 weeks), respectively, during the fitusiran treatment period.",[30],"2026-06-01",{"date":388,"type":43},"2026-06-02",{"date":390,"type":43},"2024-03-01",{"date":392,"type":23},"2030-01-30",{"name":394,"class":50},"Sanofi",{"id":396,"slug":397,"hasResults":12,"nctId":398,"briefTitle":399,"officialTitle":400,"acronym":4,"eligibilityCriteria":401,"healthyVolunteers":12,"sex":59,"minAge":19,"maxAge":4,"enrollmentInfo":402,"targetDuration":4,"studyType":24,"phases":404,"briefSummary":405,"conditions":406,"keywords":407,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":414,"lastUpdatePostDateStruct":415,"startDateStruct":417,"completionDateStruct":419,"leadSponsor":421,"locationsCount":422},"100482952","phase-3-safety-and-effectiveness-of-giroctocogene-fitelparvovec-or-fidanacogene-elaparvovec-in-patients-with-hemophilia-a-or-b-respectively-100482952","NCT05568719","Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively","A PHASE 3, NON-INVESTIGATIONAL PRODUCT, MULTI COUNTRY COHORT STUDY TO DESCRIBE THE LONG-TERM SAFETY AND EFFECTIVENESS OF A PRIOR SINGLE-DOSE TREATMENT WITH INVESTIGATIVE GIROCTOCOGENE FITELPARVOVEC OR FIDANACOGENE ELAPARVOVEC IN PARTICIPANTS WITH HEMOPHILIA A OR HEMOPHILIA B, RESPECTIVELY","Inclusion Criteria:\n\n-Only participants who received investigational giroctocogene fitelparvovec or fidanacogene eleparvovec and were enrolled in a Pfizer-sponsored study (C0371002, C0371003, C0371005, C3731001, C3731003) are eligible.\n\nExclusion Criteria:\n\n-None",{"count":403,"type":23},173,[65],"A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior participation in a Pfizer-sponsored clinical trial. Data collection and participant visits will be based on standard of care.",[30,31],[408,409,410,411,412,216,413],"bleeding","adeno-associated virus based vector","gene therapy","giroctocogene fitelparvovec","fidanacogene elaparvovec","factor IX","2026-05-25",{"date":416,"type":43},"2026-05-27",{"date":418,"type":43},"2022-12-28",{"date":420,"type":23},"2040-02-25",{"name":81,"class":50},23,{"id":424,"slug":425,"hasResults":12,"nctId":426,"briefTitle":427,"officialTitle":428,"acronym":4,"eligibilityCriteria":429,"healthyVolunteers":12,"sex":430,"minAge":145,"maxAge":4,"enrollmentInfo":431,"targetDuration":4,"studyType":24,"phases":433,"briefSummary":434,"conditions":435,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":436,"lastUpdatePostDateStruct":437,"startDateStruct":439,"completionDateStruct":441,"leadSponsor":443,"locationsCount":445},"100511218","phase-4-nuwiq-dosing-and-outcomes-in-the-management-of-womengirls-with-haemophilia-a-needing-fviii-treatment-for-surgery-100511218","NCT05936580","Nuwiq Dosing and Outcomes In the ManagEment of Women\u002FGirls With Haemophilia A Needing FVIII Treatment for Surgery","Nuwiq Dosing and Outcomes In the ManagEment of Women\u002FGirls With Haemophilia A Needing FVIII Treatment for Surgery - an International, Open-label, Non-controlled Study (NuDIMENSION)","Inclusion Criteria:\n\n1. Women\u002Fgirls with haemophilia A (FVIII:C ≥1-\\\u003C40%) according to medical history. Additionally, women\u002Fgirls with documented FVIII activity levels between ≥ 40% and 50% may be included if there is a documented history of clinically significant bleeding episodes consistent with haemophilia A; and\u002For either documented prior treatment with FVIII concentrates or a clinical indication that FVIII treatment would have been appropriate (e.g., use of FVIII from a family member, or treatment with alternative haemostatic agents due to access limitations)\n2. At least 12 years of age\n3. Scheduled to undergo major surgery\\* requiring FVIII treatment, including elective and emergency procedures and caesarean section in pregnant women with haemophilia A\n4. Freely given written informed consent of the patient, or parent\u002Flegal representative where applicable, obtained in accordance with local regulations\n\nExclusion Criteria:\n\n1. Coagulation disorder other than haemophilia A\n2. Present or past FVIII inhibitor (≥0.6 Bethesda units \\[BU\\]\u002FmL)\n3. Severe liver or kidney disease (alanine aminotransferase \\[ALT\\] and\u002For aspartate aminotransferase \\[AST\\] levels \\>5 times the upper limit of normal; or creatinine \\>120 μmol\u002FL)\n4. Known hypersensitivity to Nuwiq's active substance or its excipients (sucrose, sodium chloride, calcium chloride dihydrate, arginine hydrochloride, sodium citrate dihydrate, poloxamer 188)\n5. Pregnancy, except in participants with a planned caesarean section\n6. Already had surgery in this study\n7. Current participation in another interventional clinical trial\n8. Treatment with any investigational medicinal product (IMP) within 30 days prior to screening visit","FEMALE",{"count":432,"type":23},28,[116],"Recombinant factor VIII for the prevention of bleeding in women\u002Fgirls with haemophilia A undergoing major surgery",[30],"2026-05-08",{"date":438,"type":43},"2026-05-12",{"date":440,"type":23},"2026-04",{"date":442,"type":23},"2027-02",{"name":444,"class":50},"Octapharma",16,{"id":447,"slug":448,"hasResults":12,"nctId":449,"briefTitle":450,"officialTitle":451,"acronym":452,"eligibilityCriteria":453,"healthyVolunteers":12,"sex":59,"minAge":60,"maxAge":454,"enrollmentInfo":455,"targetDuration":4,"studyType":24,"phases":457,"briefSummary":458,"conditions":459,"keywords":460,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":474,"lastUpdatePostDateStruct":475,"startDateStruct":477,"completionDateStruct":479,"leadSponsor":481,"locationsCount":482},"100486263","phase-3-a-clinical-trial-of-study-medicine-marstacimab-in-pediatric-patients-with-hemophilia-a-or-hemophilia-b-100486263","NCT05611801","A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B","AN OPEN-LABEL STUDY IN PEDIATRIC (\u003C18 YEARS OF AGE), SEVERE HEMOPHILIA A PARTICIPANTS (COAGULATION FACTOR ACTIVITY \u003C1%) WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY ≤2%) WITH OR WITHOUT INHIBITORS COMPARING 12 MONTHS OF HISTORICAL STANDARD TREATMENT TO MARSTACIMAB PROPHYLAXIS","BASIS KIDS","Inclusion Criteria:\n\n* Male participants of appropriate age and required minimum weight\n* Participants aged 12 to 17 years must be at least 25 kgs at time of consent.\n* Participants aged 6 to 11 years must be at least 19 kgs at time of consent.\n* Minimum weight requirement for participants aged 1 to 5 years is to be determined.\n* Participants with a diagnosis of severe hemophilia A or moderately severe to severe hemophilia B\n* Participants must have at least 1 year of diary and\u002For medical records available in which exogenous FVIII or FIX replacement or bypass agent infusions and hemophilic bleeding episodes were consistently documented over the 12 months prior to the time of consent.\n\nParticipants who are enrolled into the Non-Inhibitor Cohort must also meet the following criteria:\n\n* No current detectable inhibitor and no documented history of inhibitors in the 5 years prior to consent\n* Must have at least 50 exposure days to FVIII\u002FFIX replacement products\n* Must be at least 80% compliant with a stable and effective routine prophylaxis regimen with FVIII\u002FFIX replacement products, for at least 12 months prior to consent\n\nParticipants who are enrolled into the Inhibitor Cohort must also meet the following criteria:\n\n* Documentation of current high titer inhibitor (≥5 BU\u002FmL); or current low titer inhibitor (\\\u003C5 BU\u002FmL) refractory to FVIII or FIX replacement and with FVIII or FIX recovery \\\u003C60% of expected within previous 12 months prior to the time of consent\n* Participants who have documented inhibitors while on factor-replacement therapy but who do not meet the high quantitative inhibitor criteria described in the prior bullet at the time of screening (eg, participant with a previously documented high-titer inhibitor ≥5 BU\u002FmL) and whose condition precludes re-challenge with FVIII or FIX replacement may be considered for eligibility on a case-by-case basis with discussion and agreement from the Pfizer medical monitor.\n* Hemophilia A participants with on-demand treatment regimen with ≥12 bleeding episodes or hemophilia B participants with on-demand treatment regimen with ≥8 bleeding episodes (spontaneous or traumatic) necessitating treatment with bypass factor in the 12 months prior to informed consent\n* Participants must be on an on-demand bypass treatment regimen during the 12 months prior to informed consent\n\nExclusion Criteria:\n\n* Known coronary artery, thrombotic, or ischemic disease, or current evidence of congenital or acquired thrombophilic disease such as Anti-thrombin III deficiency, Factor V Leiden mutation, prothrombin 20210 mutation, protein C deficiency, protein S deficiency and antiphospholipid syndrome.\n* Known planned surgical procedure during the planned study period\n* Known hemostatic defect other than hemophilia A or B\n* Abnormal hematology, renal or hepatic function laboratory results at screening\n* Other acute or chronic medical or psychiatric condition that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator\n* Individuals with known allergic reaction or hypersensitivity to hamster protein or other components of the study intervention\n* Current routine prophylaxis with bypassing agent, non-coagulation non-factor replacement therapy (eg, emicizumab), or any previous treatment with a gene therapy product for treatment of hemophilia\n* Participants with inhibitors who are being treated using a prophylaxis treatment regimen with a bypass agent, and, participants who have previously received non-factor-based hemophilia therapy (eg, fitusiran, concizumab, emicizumab) will be considered on a case-by-case basis, only after discussion and agreement between the investigator and the Pfizer medical monitor\n* Regular use of immunomodulatory medications (eg, IVIG, routine systemic corticosteroids, rituximab)\n* Use of systemic antifibrinolytics, medications that may increase the risk of bleeding, and certain non-steroidal anti-inflammatory drugs within 120 hours of first dose of study intervention and while on study\n* Ongoing or planned use of ITI, or prophylaxis with FVIII or FIX replacement at any time after initiation of treatment with study intervention\n* Participation in other studies involving investigational drug(s) or investigational vaccine(s) within 30 days (or as determined by local requirements) or 5 half-lives prior to study entry or during study participation\n* Previous exposure to marstacimab during participation in other marstacimab clinical studies\n* CD4 cell count ≤200\u002FuL if HIV-positive\n* Abnormal ECG of clinical relevance that may affect participant safety or interpretation of study results\n* Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, and sponsor and sponsor delegate employees directly involved in the conduct of the study and their family members","17 Years",{"count":456,"type":23},100,[65],"The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.\n\nThis study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll.\n\nThis study will enroll participants who:\n\n* have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without inhibitors)\n* have accurate historical records documenting all factor VIII, factor IX, or bypass agent infusions and hemophilia bleed events for at least 1 year prior to entering the study\n* if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor VIII or factor IX replacement products for at least 12 months prior to study entry\n* if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12 months prior to study entry\n\nAll participants in this study will receive marstacimab to use prophylactically. Marstacimab will be given once a week as a subcutaneous (under the skin) shot. The first dose of marstacimab will be given at the study site by the study site staff. During the 12-month treatment period, weekly doses of marstacimab can be given at home, or if preferred, the doses may be given by the study site staff.\n\nTo help us determine if the study medicine is safe and effective, we will compare participant experiences when they are taking the study medicine to a historical period when they were not. Researchers want to see if the study medicine works to prevent the bleeding episodes commonly experienced by patients with Hemophilia.\n\nParticipants will be in this study for about 14 months (approximately 1 month in a Screening period, 12 months receiving treatment, and 1 month in a follow-up period) during which they will visit the study site at least 10 times. If preferred, and if local regulations allow it, 2 of the study visits can be completed at the participant's home instead of at the study site. There will also be 6 scheduled telephone calls approximately every 2 months.",[30,31],[69,70,71,72,73,37,36,461,462,463,121,464,452,465,466,467,468,469,470,471,472,473],"Inhibitors","Anti-Tissue Factor Pathway Inhibitor (TFPI)","Subcutaneous (sc)","On-Demand","Inhibitor","SC","Subcutaneous","Injection","On demand","aTFPI","Severe hemophilia","Severe bleeding","Hemophilia","2026-05-05",{"date":476,"type":43},"2026-05-07",{"date":478,"type":43},"2022-12-09",{"date":480,"type":23},"2028-09-10",{"name":81,"class":50},34,{"id":484,"slug":485,"hasResults":12,"nctId":486,"briefTitle":487,"officialTitle":488,"acronym":489,"eligibilityCriteria":490,"healthyVolunteers":12,"sex":59,"minAge":145,"maxAge":4,"enrollmentInfo":491,"targetDuration":4,"studyType":95,"phases":4,"briefSummary":493,"conditions":494,"keywords":496,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":501,"lastUpdatePostDateStruct":502,"startDateStruct":503,"completionDateStruct":504,"leadSponsor":506,"locationsCount":201},"100578336","synovial-proliferation-on-routine-ultrasound-active-or-inactive-100578336","NCT06809972","Synovial Proliferation on Routine Ultrasound: Active or Inactive?","Synovial Proliferation on Routine Ultrasound: Active or Inactive? A Prospective Study","2BEGIN","Inclusion Criteria:\n\n* Gender: male\n* Patients with severe haemophilia A or B\n* Treated with registered prophylaxis medication including coagulation factors and by- passing agents.\n* Age ≥ 12 years\n* Subclinical synovial proliferation in ≥1 joint (ankle, knee and\u002For elbow), defined as the presence of hypertrophic synovium, score \\>0 according to the HEAD-US protocol, as confirmed during routine ultrasound screening.\n* Able to give written informed consent.\n\nExclusion Criteria:\n\n* A major bleed ≤ 3 months or a minor bleed ≤ 1 month prior to inclusion in the joint of interest.\n* On demand therapy.\n* Currently treated with any type of haemophilia prophylaxis medication.\n* Joints with prosthesis or treated with arthrodesis will not be included for physical examination and ultrasound analysis. However, participants may still be included in the study with their other joints.\n* Confirmed inflammatory joint diseases such as rheumatoid arthritis or psoriatic arthritis.\n* History of inhibitor development (≥ 5 Bethesda Units\\* (BU) at any time or 1-5 BU for\n\n  * 1 year prior to inclusion.\n* Contra-indication for treatment with NSAIDs, (allergy, severe liver failure, renal failure (GFR \\\u003C30ml\u002Fmin), congestive heart failure (NYHA II-IV), peripheral arterial disease and\u002For cerebrovascular disease.",{"count":492,"type":23},46,"There is cumulating evidence for the presence of non-observed or subclinical joint bleeding in patients with haemophilia. Early detection of active subclinical synovial proliferation would allow early intervention in order to prevent deterioration of joint health. Patients with subclinical (=non-observed) signs of synovial proliferation in knee(s), ankle(s) and\u002For elbow(s) will be invited to participate in this study to further characterize the synovial proliferation status (active or inactive) by means of physical examination, MRI, ultrasound and elastography. Synovial proliferation status will be monitored for a maximum period of 12 weeks, during which participants will also receive standard-of-care treatment, i.e. administration of optimized coagulation factor replacement therapy and prescription of the NSAID celecoxib (optional).",[495,30,31],"Haemophilia",[497,498,499,500],"Subclinical synovial proliferation","Ultrasound","MRI","Elastography","2026-04-28",{"date":474,"type":43},{"date":221,"type":43},{"date":505,"type":23},"2028-04-01",{"name":507,"class":105},"Van Creveldkliniek",{"id":509,"slug":510,"hasResults":12,"nctId":511,"briefTitle":512,"officialTitle":513,"acronym":514,"eligibilityCriteria":515,"healthyVolunteers":12,"sex":18,"minAge":91,"maxAge":4,"enrollmentInfo":516,"targetDuration":4,"studyType":24,"phases":517,"briefSummary":518,"conditions":519,"keywords":520,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":524,"lastUpdatePostDateStruct":525,"startDateStruct":527,"completionDateStruct":529,"leadSponsor":531,"locationsCount":179},"100405755","phase-3-safe-study-safety-of-apcc-following-emicizumab-prophylaxis-100405755","NCT04563520","SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis","aPCC and Emicizumab Safety Study in Congenital Hemophilia A Patients With Inhibitors (SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis)","SAFE","Inclusion Criteria:\n\n* Moderately severe hemophilia A, defined as FVIII level \\\u003C0.05 IU\u002FmL before development of an inhibitor\n* Age ≥6 years of age at time of informed consent\n* Documented on 2 occasions a high titer inhibitor (\\>5 BU\u002FmL) with a 72-hour washout within 2 years of enrollment\n* Parent\u002Fguardian (Legally Authorized Representative) or the patient has provided written informed consent\n* Adequate hematologic function (Hgb \\>8 g\u002FdL and platelet count \\>100,000 µL)\n* Adequate hepatic function (total bilirubin ≤1.5 x ULN and both AST\u002FALT ≤3x ULN at screening (excluding known Gilbert's)\n* Adequate renal function (≤2.5 x ULN and CrCl ≥30 mL\u002Fmin)\n\nExclusion Criteria:\n\n* Inherited or acquired bleeding disorder other than hemophilia A excluding low VWF (\\>30% VWF:RCo or VWF:GP1bm)\n* Had an active bleed requiring factor therapy at screening\n* Previous or current treatment for thromboembolic disease or signs of thromboembolic disease (excluding previously resolved line-associated thrombosis)\n* Had a surgical procedure 14 days before screening\n* Conditions that may increase the risk of bleeding or thrombosis\n* If the patient is treated with rFVIIa or aPCC seven days before screening\n* History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection\n* Had current use of any medication other than emicizumab that could affect the coagulation system.\n* Known HIV infection with CD4 count \\\u003C200 cells\u002FµL within 24 weeks before screening. Testing is not required if \\\u003C35 years of age.\n* Use of systemic immunomodulators at enrollment or planned use during the study\n* Participants who are at high risk for TMA (for example, have a previous medical\u002Ffamily history of TMA), in the investigator's judgment\n* Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose an additional risk, or would, in the opinion of the investigator, preclude the participant's safe participation in and completion of the study",{"count":166,"type":23},[65],"The purpose of the aPCC-emicizumab safety study is to investigate the hemostatic efficacy as measured by thrombin generation, of a low personalized dose of aPCC (FEIBA) in children and adults with hemophilia A and inhibitors on emicizumab prophylaxis.",[30],[521,522,523],"hemostatic efficacy","safety","prothrombin complex concentrate","2026-04-23",{"date":526,"type":43},"2026-04-29",{"date":528,"type":23},"2026-06",{"date":530,"type":23},"2027-03",{"name":532,"class":105},"Emory University",{"id":534,"slug":535,"hasResults":12,"nctId":536,"briefTitle":537,"officialTitle":538,"acronym":539,"eligibilityCriteria":540,"healthyVolunteers":12,"sex":430,"minAge":19,"maxAge":4,"enrollmentInfo":541,"targetDuration":4,"studyType":95,"phases":4,"briefSummary":543,"conditions":544,"keywords":558,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":572,"lastUpdatePostDateStruct":573,"startDateStruct":574,"completionDateStruct":576,"leadSponsor":578,"locationsCount":201},"100527411","development-of-non-invasive-prenatal-diagnosis-for-single-gene-disorders-100527411","NCT06147414","Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders","Evaluation of the Diagnostic Performance of Non-Invasive Prenatal Diagnosis for Single Gene Disorders","DANNIgene","Inclusion Criteria:\n\n* pregnant woman with 9 weeks of amenorrhea or more\n* singleton pregnancy\n* undergoing invasive PND in a context of family history of SGD involving the following genes : HBB, CFTR, FMR1, SMN1, DMPK, DMD, NF1, HTT, F8, F9, GCK, L1CAM, PKHD1, or undergoing prenatal counselling in a context of maternal history of diabetes MODY-GCK\n* germinal pathogenic paternal and\u002For maternal mutations previously identified\n* age 18 years old or over\n* signing an informed consent\n\nExclusion Criteria:\n\n* at risk of SGD involving a de novo pathogenic mutation in a previous child\n* woman under legal protection",{"count":542,"type":23},550,"Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma. Its presence in maternal plasma has allowed development of noninvasive prenatal diagnosis for single-gene disorders (SGD-NIPD). This can be performed from 9 weeks of amenorrhea and offers an early, safe and accurate definitive diagnosis without the miscarriage risk associated with invasive procedures. One of the major difficulties is distinguishing fetal genotype in the high background of maternal cfDNA, which leads to several technical and analytical challenges. Besides, unlike noninvasive prenatal testing for aneuploidy, NIPD for monogenic diseases represent a smaller market opportunity, and many cases must be provided on a bespoke, patient- or disease-specific basis. As a result, implementation of SGD-NIPD remained sparse, with most testing being delivered in a research setting.\n\nThe present project aims to take advantage of the unique French collaborative network to make SGD-NIPD possible for theoretically any monogenic disorder and any family.",[545,546,547,548,549,550,551,552,553,554,30,31,555,556,557],"Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including","Sickle Cell Disease","Cystic Fibrosis","Fragile X Syndrome","Proximal Spinal Muscular Atrophy","Myotonic Dystrophy","Muscular Dystrophy, Duchenne","Muscular Dystrophy, Becker","Neurofibromatosis-Noonan Syndrome","Huntington Disease","MODY2 Diabetes","X-Linked Hydrocephalus","Autosomal Recessive Polycystic Kidney Disease",[559,560,561,562,563,564,565,566,567,568,569,570,571],"Gene HBB","Gene CFTR","Gene FMR1","Gene SMN1","Gene DMPK","Gene DMD","Gene NF1","Gene HTT","Gene F8","Gene F9","Gene GCK","Gene L1CAM","Gene PKHD1","2026-04-20",{"date":524,"type":43},{"date":575,"type":43},"2024-10-23",{"date":577,"type":23},"2027-05",{"name":579,"class":105},"Assistance Publique - Hôpitaux de Paris",{"id":581,"slug":582,"hasResults":12,"nctId":583,"briefTitle":584,"officialTitle":585,"acronym":586,"eligibilityCriteria":587,"healthyVolunteers":12,"sex":18,"minAge":4,"maxAge":19,"enrollmentInfo":588,"targetDuration":590,"studyType":95,"phases":4,"briefSummary":591,"conditions":592,"keywords":594,"overallStatus":150,"whyStopped":4,"lastUpdateSubmitDate":596,"lastUpdatePostDateStruct":597,"startDateStruct":598,"completionDateStruct":600,"leadSponsor":601,"locationsCount":201},"100634416","searching-patterns-in-the-robustness-of-immunological-fviii-tolerance-100634416","NCT07539402","Searching Patterns In the Robustness of Immunological FVIII Tolerance","Searching Patterns In the Robustness of Immunological FVIII Tolerance (SPIRIT)","SPIRIT","Inclusion Criteria:\n\n* Congenital hemophilia A of all severities\n* Using NFT for prophylaxis\n* Aged under 18 years\n* Written informed consent\n\nExclusion Criteria:\n\n* Acquired hemophilia A\n* Any other bleeding disorder",{"count":589,"type":23},500,"5 Years","Children with hemophilia A lack clotting factor VIII (FVIII) due to a genetic mutation. It is well known that administration of FVIII concentrate leads to immunological tolerance for the FVIII protein in the majority of children. In 30% of these children tolerance is not achieved leading to the development of anti-FVIII antibodies (i.e. inhibitors). Our knowledge on the underlying immunological mechanisms leading to tolerance is limited. Recently, Non-Factor Therapy (NFT) has become available for prevention of bleeding in patients with hemophilia, i.e. prophylaxis. Currently, many children with severe hemophilia A use NFT as the subcutaneous administration of NFT is very convenient. In children on NFT prophylaxis, intravenous FVIII concentrate is exclusively used on-demand for treatment of bleeding. As NFT is very effective in the prevention of bleeds, patients may not be exposed to the deficient FVIII protein for periods up to a year or longer. It is currently not known how robust immunological tolerance is in the absence of exposure to a deficient antigen. The infrequent exposure to FVIII, enabled by NFT, provides an opportunity to study the immunological tolerance mechanisms for FVIII in children with hemophilia A.\n\nThe aim of SPIRIT is to investigate the mechanisms of the immunological tolerance to FVIII in patients with hemophilia A aged younger than 18 years using NFT for prophylaxis.\n\nIn this observational cohort study, children (aged \\\u003C18 years) with congenital hemophilia A, who are treated with non-factor therapy as prophylaxis, will be longitudinally followed. Participants will have blood drawn anually, during the regular clinic visits, and additionally following FVIII exposure. Feces samples will be collected and analyzed in children aged \\\u003C12 years, following the same scheme as blood sampling.\n\nThe main study endpoint are the immunological mechanisms underlying tolerance to FVIII, including presence, titers, subtypes and affinities of FVIII-specific (non-)neutralizing antibodies, FVIII-specific T and B cell responses and the role of gut microbiota.",[30,593],"Hemophilia A, Congenital",[30,37,595],"Tolerance","2026-04-16",{"date":572,"type":43},{"date":599,"type":23},"2026-05-01",{"date":102,"type":23},{"name":602,"class":105},"Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)",{"id":604,"slug":605,"hasResults":12,"nctId":606,"briefTitle":607,"officialTitle":608,"acronym":4,"eligibilityCriteria":609,"healthyVolunteers":12,"sex":18,"minAge":4,"maxAge":4,"enrollmentInfo":610,"targetDuration":4,"studyType":95,"phases":4,"briefSummary":612,"conditions":613,"keywords":619,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":596,"lastUpdatePostDateStruct":626,"startDateStruct":628,"completionDateStruct":630,"leadSponsor":632,"locationsCount":201},"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":611,"type":23},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.",[473,614,30,31,546,615,616,617,618],"Thrombosis","Glanzmann Thrombasthenia","Bleeding Disorder","Blood Disorder","Von Willebrand Diseases",[620,621,622,623,624,625],"bleed event","bleed treatments","adverse events","joint bleed","bleeding disorder","bleeding symptoms",{"date":627,"type":43},"2026-04-21",{"date":629,"type":43},"2024-10-25",{"date":631,"type":23},"2055-10-31",{"name":633,"class":634},"American Thrombosis and Hemostasis Network","NETWORK",{"id":636,"slug":637,"hasResults":12,"nctId":638,"briefTitle":639,"officialTitle":640,"acronym":4,"eligibilityCriteria":641,"healthyVolunteers":12,"sex":59,"minAge":19,"maxAge":210,"enrollmentInfo":642,"targetDuration":644,"studyType":95,"phases":4,"briefSummary":645,"conditions":646,"keywords":647,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":653,"lastUpdatePostDateStruct":654,"startDateStruct":656,"completionDateStruct":658,"leadSponsor":660,"locationsCount":201},"100633185","joint-health-balance-and-quality-of-life-in-adults-with-hemophilia-a-100633185","NCT07523399","Joint Health, Balance and Quality of Life in Adults With Hemophilia A","Quality of Life in Adults With Hemophilia A: Associations With Joint Health, Pain, Dynamic Balance, and Falls","Inclusion Criteria:\n\n* Diagnosis of severe hemophilia A\n* Age ≥18 years\n* A minimum single-joint score of 3 points for the knee or ankle according to the Hemophilia Joint Health Score (HJHS)\n\nExclusion Criteria:\n\n* Use of walking aids (e.g., crutches)\n* Presence of neurological or psychiatric disorders, chronic inflammatory disease, -Regular use of medications that could affect balance,\n* History of major lower extremity surgery within the previous six months.",{"count":643,"type":23},36,"2 Weeks","Hemophilia is a hereditary bleeding disorder characterized by recurrent bleeding episodes, particularly into joints and muscles, leading to chronic musculoskeletal complications. Repeated joint bleeding may result in hemophilic arthropathy, which is associated with progressive joint damage, chronic pain, reduced mobility, and functional limitations. Advances in prophylactic treatment have significantly improved life expectancy in individuals with hemophilia; however, long-term musculoskeletal complications continue to affect daily functioning and overall well-being. Therefore, the evaluation of health-related quality of life (QoL) has become increasingly important in adults with hemophilia, as it reflects the broader impact of the disease beyond clinical severity.\n\nJoint health is considered one of the major determinants of physical functioning in people with hemophilia. Hemophilic arthropathy may lead to chronic pain, muscle weakness, reduced range of motion, and impaired physical performance. In addition to structural joint changes, impairments in neuromuscular function, including reduced proprioception, muscle strength, and postural control, may also contribute to activity limitations. Balance impairments may increase the risk of falls, which may be particularly relevant for individuals with hemophilia because fall-related trauma can lead to bleeding episodes and further joint deterioration. Although previous studies have investigated the relationship between joint health, pain, and functional limitations, the combined contribution of joint health, pain severity, dynamic balance performance, and fall history to QoL in adults with hemophilia has not been sufficiently clarified.\n\nThis prospective cross-sectional study aims to evaluate QoL in adults with hemophilia and to investigate the associations of joint health, pain severity, dynamic balance performance, and fall history with QoL outcomes. The study includes 36 adults diagnosed with severe hemophilia A who are followed at the Adult Hematology Unit of Van Yuzuncu Yil University. Participants aged 18 years and older who met the inclusion criteria were recruited consecutively. Individuals using walking aids, having neurological or psychiatric disorders, chronic inflammatory diseases, recent lower extremity surgery, or regular medication use that could affect balance were excluded.\n\nJoint health is assessed using the Hemophilia Joint Health Score (HJHS), a clinician-administered tool evaluating joint impairment in the knees and ankles. Dynamic balance is assessed using a computerized stabilometric platform (Pro-kin Tecnobody Stabilometric Analysis System) with the Limits of Stability (LoS) test, which evaluates the ability to voluntarily control the center of pressure within the base of support. Pain intensity is measured using the Numeric Pain Rating Scale (NPRS), an 11-point scale ranging from 0 to 10. Fall history is determined based on the self-reported number of falls within the previous 12 months. QoL is assessed using the Turkish version of the Haemophilia-Specific Quality of Life Questionnaire for adults (Haem-A-QoL), a validated patient-reported outcome measure evaluating multiple domains of physical, emotional, and social well-being. Higher scores indicate poorer QoL.\n\nThe findings of this study are expected to improve the understanding of factors associated with QoL in adults with hemophilia and may help identify potentially modifiable clinical targets for rehabilitation interventions.",[30],[648,30,649,650,651,652],"Quality of life","Arthropathy","Postural balance","Falls","Musculoskeletal Pain","2026-04-04",{"date":655,"type":43},"2026-04-13",{"date":657,"type":43},"2026-02-21",{"date":659,"type":23},"2026-05-21",{"name":661,"class":105},"Yuzuncu Yil University"]