[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hemophilia-b\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hemophilia-b":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,24,0,[8,51,82,104,127,155,179,208,235,273,300,321,370,402,425,459,480,501,522,542,561,586,621,641],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":16,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":32,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":39,"lastUpdatePostDateStruct":40,"startDateStruct":43,"completionDateStruct":45,"leadSponsor":47,"locationsCount":50},"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":21,"type":22},31,"ESTIMATED","INTERVENTIONAL",[25],"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).",[28,29,30,31],"Healthy Participants","Hemophilia A","Hemophilia B","Factor VII Deficiency",[33,34,35,36,37],"Single ascending dose","Multiple ascending dose","Factor IX","Factor VIII","Factor VII","RECRUITING","2026-06-30",{"date":41,"type":42},"2026-07-02","ACTUAL",{"date":44,"type":42},"2024-05-10",{"date":46,"type":22},"2026-09-28",{"name":48,"class":49},"Equilibra Bioscience LLC","INDUSTRY",10,{"id":52,"slug":53,"hasResults":11,"nctId":54,"briefTitle":55,"officialTitle":56,"acronym":4,"eligibilityCriteria":57,"healthyVolunteers":11,"sex":58,"minAge":59,"maxAge":60,"enrollmentInfo":61,"targetDuration":4,"studyType":23,"phases":63,"briefSummary":65,"conditions":66,"keywords":67,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":39,"lastUpdatePostDateStruct":73,"startDateStruct":75,"completionDateStruct":77,"leadSponsor":79,"locationsCount":81},"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":62,"type":22},245,[64],"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.",[29,30],[68,69,70,71,72],"Factor VIII Inhibitor","Factor IX Inhibitor","PF-06741086","Marstacimab","Anti-TFPI",{"date":74,"type":42},"2026-07-01",{"date":76,"type":42},"2021-11-17",{"date":78,"type":22},"2030-07-31",{"name":80,"class":49},"Pfizer",75,{"id":83,"slug":84,"hasResults":11,"nctId":85,"briefTitle":86,"officialTitle":87,"acronym":4,"eligibilityCriteria":88,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":89,"targetDuration":4,"studyType":23,"phases":91,"briefSummary":92,"conditions":93,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":94,"lastUpdatePostDateStruct":95,"startDateStruct":97,"completionDateStruct":99,"leadSponsor":101,"locationsCount":103},"100516351","phase-3-efficacy-and-safety-of-csl222-etranacogene-dezaparvovec-gene-therapy-in-adults-with-hemophilia-b-with-pretreatment-adeno-associated-virus-serotype-5-aav5-neutralizing-antibodies-nabs-100516351","NCT06003387","Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)","Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies","Inclusion Criteria:\n\n* Considered legally an adult, as defined by country regulations.\n* Has congenital hemophilia B with known severe or moderately severe FIX deficiency (less than or equal to \\[\\\u003C=\\] 2% of normal circulating FIX) for which the participant is on continuous routine FIX prophylaxis.\n* Has 2 consecutive detectable AAV5 NAb titer results between Screening and Visit L-Final using a validated AAV5 NAb assay (based on central laboratory results).\n* Has greater than (\\>) 150 previous exposure days to FIX replacement therapy.\n* Has been on stable FIX prophylaxis for at least 2 months before Screening.\n* Has demonstrated capability to independently, accurately, and in a timely manner complete the eDiary during the Lead-in Period, as judged by the investigator.\n* Acceptance to adhere to contraception guidelines.\n* Able to provide informed consent after receipt of verbal and written information about the study.\n* Investigator believes that the participant (or the participant's legally acceptable representative\\[s\\]) understands the nature, scope, and possible consequences of the study and is able to adhere to the study procedures.\n\nExclusion Criteria:\n\n* • History of FIX inhibitors or positive FIX inhibitor test at Prescreening, Screening or Visit L-Final (based on central laboratory results).\n* • Screening or Visit L-Final laboratory values (based on central laboratory results) of total bilirubin \\> 2 × the upper limit of normal (ULN) (except if caused by Gilbert's syndrome).\n* • Screening or Visit L-Final laboratory values (based on central laboratory results) of any of the following laboratory abnormalities:\n* a) ALT \\> 2 × the ULN\n* b) AST \\> 2 × the ULN\n* c) Alkaline phosphatase \\> 2 × the ULN\n* d) Serum creatinine \\> 2 × the ULN\n* e) Hemoglobin less than (\\\u003C) 8 g\u002FdL\n* • Any condition other than hemophilia B resulting in an increased bleeding tendency.\n* • Thrombocytopenia, defined as a platelet count \\\u003C50 × 10\\^9\u002FL, at Screening or Visit L Final (based on central laboratory results).\n* • Any uncontrolled or untreated infection (human immunodeficiency virus \\[HIV\\], hepatitis B virus \\[HBV\\] and hepatitis C virus \\[HCV\\], or any other significant concurrent, uncontrolled medical condition including, but not limited to, renal, hepatic, cardiovascular, hematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease, alcoholism, drug dependency, or any other psychological disorder evaluated by the investigator to interfere with adherence to the clinical study protocol procedures or with the degree of tolerance to CSL222.\n* • Known history of allergy to corticosteroids or known medical condition that would require chronic administration of oral corticosteroids.\n* • Known uncontrolled allergic conditions or allergy \u002F hypersensitivity to any component of the CSL222 excipients (ie, sucrose, potassium chloride, potassium dihydrogen phosphate, sodium chloride, and disodium hydrogen phosphate).\n* • Previous AAV5 gene therapy treatment.\n* • Receipt of an experimental agent or device within 60 days before Screening until the end of the study.",{"count":90,"type":22},35,[64],"The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.",[30],"2026-06-15",{"date":96,"type":42},"2026-06-16",{"date":98,"type":42},"2024-01-30",{"date":100,"type":22},"2032-04-02",{"name":102,"class":49},"CSL Behring",27,{"id":105,"slug":106,"hasResults":11,"nctId":107,"briefTitle":108,"officialTitle":108,"acronym":4,"eligibilityCriteria":109,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":110,"enrollmentInfo":111,"targetDuration":4,"studyType":23,"phases":113,"briefSummary":115,"conditions":116,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":117,"lastUpdatePostDateStruct":118,"startDateStruct":120,"completionDateStruct":122,"leadSponsor":124,"locationsCount":126},"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.","65 Years",{"count":112,"type":22},76,[25,114],"PHASE2","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).",[29,30,31],"2026-06-08",{"date":119,"type":42},"2026-06-12",{"date":121,"type":42},"2024-05-31",{"date":123,"type":22},"2026-12-31",{"name":125,"class":49},"Shanghai RAAS Blood Products Co., Ltd.",9,{"id":128,"slug":129,"hasResults":11,"nctId":130,"briefTitle":131,"officialTitle":131,"acronym":4,"eligibilityCriteria":132,"healthyVolunteers":11,"sex":58,"minAge":18,"maxAge":133,"enrollmentInfo":134,"targetDuration":4,"studyType":136,"phases":4,"briefSummary":137,"conditions":138,"keywords":139,"overallStatus":144,"whyStopped":4,"lastUpdateSubmitDate":145,"lastUpdatePostDateStruct":146,"startDateStruct":147,"completionDateStruct":149,"leadSponsor":151,"locationsCount":154},"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":135,"type":22},8,"OBSERVATIONAL","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.",[29,30],[140,141,142,29,30,143],"AAV-mediated factor VIII (FVIII) gene transfer","AAV-mediated factor IX (FIX) gene transfer","Gene Therapy","Transjugular liver biopsy","NOT_YET_RECRUITING","2026-06-05",{"date":117,"type":42},{"date":148,"type":22},"2026-07",{"date":150,"type":22},"2030-01",{"name":152,"class":153},"St. Jude Children's Research Hospital","OTHER",1,{"id":156,"slug":157,"hasResults":11,"nctId":158,"briefTitle":159,"officialTitle":160,"acronym":161,"eligibilityCriteria":162,"healthyVolunteers":11,"sex":58,"minAge":163,"maxAge":164,"enrollmentInfo":165,"targetDuration":4,"studyType":23,"phases":167,"briefSummary":168,"conditions":169,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":170,"lastUpdatePostDateStruct":171,"startDateStruct":173,"completionDateStruct":175,"leadSponsor":177,"locationsCount":178},"100599162","phase-3-phase-3-open-label-single-dose-study-of-csl222-in-adolescent-male-subjects--12-to--18-years-of-age-with-severe-or-moderately-severe-hemophilia-b-100599162","NCT07080905","Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to \u003C 18 Years of Age) With Severe or Moderately Severe Hemophilia B","Phase 3, Open-label, Single-dose, Multicenter Study Investigating Efficacy, Safety, and Tolerability of CSL222 (Etranacogene Dezaparvovec) Administered to Adolescent Male Subjects (≥ 12 to \u003C 18 Years of Age) With Severe or Moderately Severe Hemophilia B","IX-TEND 3004","Inclusion Criteria:\n\n* Key Inclusion Criteria for the Lead-in Period:\n\nAssigned male sex at birth\n\n* Aged ≥ 138 months (11 years and 6 months) to less than (\\\u003C) 206 months (17 years and 2 months) at the time of informed consent \u002F assent.\n* Congenital hemophilia B with known severe or moderately severe FIX deficiency (less than or equal to \\[≤\\] 2% of normal circulating FIX) for which the participant has been on continuous FIX prophylaxis.\n* On stable continuous FIX prophylaxis for at least 2 months before Screening.\n* Minimum of 75 previous exposure days of treatment with FIX protein before Screening.\n* Additional Key Inclusion Criteria for the Treatment Period:\n\nCompleted the Lead-in Period: minimum of 6 months (26 weeks) of lead-in data collected and eligibility has been confirmed.\n\n* Aged ≥ 12 to \\\u003C 18 years at the time of CSL222 treatment.\n\nExclusion Criteria:\n\n* Key Exclusion Criteria for the Lead-in Period:\n\nHistory of FIX inhibitors or positive FIX inhibitor test at Screening (based on central laboratory results).\n\n* Screening laboratory values (based on central laboratory results):\n\n  * Total bilirubin \\> 2 × the upper limit of normal (ULN).\n  * Alanine aminotransferase (ALT) \\> 2 × the ULN.\n  * Aspartate aminotransferase (AST) \\> 2 × the ULN.\n  * Alkaline phosphatase (ALP) \\> 2 × the ULN.\n  * Serum creatinine \\> 2 × the ULN.\n  * Hemoglobin \\\u003C 8 g\u002FdL.\n* Any condition other than hemophilia B resulting in an increased bleeding tendency.\n* Thrombocytopenia, defined as a platelet count below 50 × 10\\^9\u002FL, at screening (based on central laboratory results).\n* Any uncontrolled or untreated infection (human immunodeficiency virus, hepatitis C, etc) or any other significant concurrent, uncontrolled medical condition, as evaluated by the investigator, including, but not limited to renal, hepatic, cardiovascular, hematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral, or psychiatric disease, alcoholism, drug dependency, or any other psychological disorder evaluated by the investigator to interfere with adherence to the Clinical Study Protocol procedures or with the degree of tolerance to CSL222.\n* Additional Key Exclusion Criteria for the Treatment Period:\n\nPositive FIX inhibitor test at Visit L-Final (based on central laboratory results)\n\n* AAV5 NAb titer \\> 1:900 as assessed at Visit LX (last visit before Visit L-Final).\n* Visit L-Final laboratory values (based on central laboratory results) of:\n\n  * Total bilirubin \\> 2 × the ULN\n  * ALT \\> 2 × the ULN.\n  * AST \\> 2 × the ULN.\n  * ALP \\> 2 × the ULN.\n  * Serum creatinine \\> 2 × the ULN.\n  * Hemoglobin \\\u003C 8 g\u002FdL.\n* Thrombocytopenia, defined as a platelet count below 50 × 10\\^9\u002FL, at Visit L-Final (based on central laboratory results).","138 Months","206 Months",{"count":166,"type":22},20,[64],"This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately severe hemophilia B.",[30],"2026-06-03",{"date":172,"type":42},"2026-06-04",{"date":174,"type":42},"2025-07-28",{"date":176,"type":22},"2033-10-24",{"name":102,"class":49},2,{"id":180,"slug":181,"hasResults":11,"nctId":182,"briefTitle":183,"officialTitle":184,"acronym":4,"eligibilityCriteria":185,"healthyVolunteers":11,"sex":58,"minAge":18,"maxAge":4,"enrollmentInfo":186,"targetDuration":4,"studyType":23,"phases":188,"briefSummary":189,"conditions":190,"keywords":191,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":199,"lastUpdatePostDateStruct":200,"startDateStruct":202,"completionDateStruct":204,"leadSponsor":206,"locationsCount":207},"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":187,"type":22},173,[64],"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.",[29,30],[192,193,194,195,196,197,198],"bleeding","adeno-associated virus based vector","gene therapy","giroctocogene fitelparvovec","fidanacogene elaparvovec","factor VIII","factor IX","2026-05-25",{"date":201,"type":42},"2026-05-27",{"date":203,"type":42},"2022-12-28",{"date":205,"type":22},"2040-02-25",{"name":80,"class":49},23,{"id":209,"slug":210,"hasResults":11,"nctId":211,"briefTitle":212,"officialTitle":213,"acronym":214,"eligibilityCriteria":215,"healthyVolunteers":11,"sex":58,"minAge":216,"maxAge":4,"enrollmentInfo":217,"targetDuration":4,"studyType":23,"phases":219,"briefSummary":220,"conditions":221,"keywords":222,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":225,"lastUpdatePostDateStruct":226,"startDateStruct":228,"completionDateStruct":230,"leadSponsor":232,"locationsCount":234},"100545276","phase-1-a-study-to-investigate-the-safety-and-effectiveness-of-a-coagulation-factor-ix-gene-insertion-therapy-regv131-lnp1265-in-pediatric-adolescent-and-adult-participants-with-hemophilia-b-100545276","NCT06379789","A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B","A Two-Part Open-Label Study of REGV131-LNP1265, A CRISPR\u002FCas9 Based Coagulation Factor IX Gene Insertion Therapy in Participants With Hemophilia B","BEYOND-9","Key Inclusion Criteria:\n\n1. Confirmed diagnosis of severe or moderately severe hemophilia B with medical history of FIX functional activity (≤2% or \\\u003C0.02 IU\u002FmL) or documented genotype known to produce severe hemophilia B\n2. Currently taking FIX prophylaxis and previous experience with FIX therapy, as defined in the protocol\n3. Participation in the lead-in period of this interventional study OR a separate lead-in study (R0000-HEMB-2187 \\[NCT05568459\\]) for at least 6 months for ABR data while taking FIX prophylaxis, as defined in the protocol\n\nKey Exclusion Criteria:\n\n1. History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions\n2. Bethesda inhibitor titer greater than the Upper Limit of Normal (ULN) at screening\n3. Detectable pre-existing antibodies to the AAV8 capsid; as measured by Enzyme-Linked ImmunoSorbent Assay (ELISA) at prescreening (or final lead-in visit, if applicable)\n4. Any significant underlying liver disease such as: cholestatic liver disease, liver cirrhosis, portal hypertension, splenomegaly, hepatic encephalopathy\n5. Evidence of advanced liver fibrosis or significant fatty liver, as defined in the protocol\n6. Evidence of cirrhosis and\u002For portal hypertension as assessed by abdominal ultrasound at screening or measured within 6 months prior to the screening visit\n7. History of arterial or venous thrombo-embolic events, as defined in the protocol\n8. History of hypersensitivity to corticosteroids or known medical condition that requires chronic administration of corticosteroids\n9. Previously received any AAV gene-based therapy or intends to receive approved or investigational AAV-based gene therapy other than REGV131-LNP1265 during the study period\n\nNOTE: Other Inclusion\u002FExclusion Protocol Defined Criteria Apply","2 Years",{"count":218,"type":22},130,[25,114],"Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B. This study is researching an experimental gene insertion therapy (the adding of a gene into your DNA) called REGV131-LNP1265, also called the \"study drug\". Gene insertion therapy aims to teach the body how to produce clotting factor long-term, without the need for factor replacement therapy.\n\nThe main aim of this study is to find a safe and well-tolerated dose of the study drug by checking the side effects that may happen from taking it, both in the near term and over time.\n\nThe study is looking at several other research questions including:\n\n* How much study drug is in the blood at different times\n* Whether the body makes antibodies against parts of the study drug, which could make the drug less effective or could lead to side effects. Antibodies are proteins produced by the body's immune system in response to a foreign substance\n* Whether the body makes antibodies against the clotting factor replacement therapy\n* How often factor replacement therapy is needed, both on a regular basis for prevention of bleeding, and as needed to treat bleeding events (and it if changes after taking study drug)\n* Whether there is a difference in 2 different methods for measuring Factor 9 activity in the blood",[30],[223,224],"Severe and moderately severe congenital hemophilia B","FIX functional activity","2026-05-18",{"date":227,"type":42},"2026-05-19",{"date":229,"type":42},"2024-09-11",{"date":231,"type":22},"2047-08-14",{"name":233,"class":49},"Regeneron Pharmaceuticals",42,{"id":236,"slug":237,"hasResults":11,"nctId":238,"briefTitle":239,"officialTitle":240,"acronym":241,"eligibilityCriteria":242,"healthyVolunteers":11,"sex":58,"minAge":59,"maxAge":243,"enrollmentInfo":244,"targetDuration":4,"studyType":23,"phases":246,"briefSummary":247,"conditions":248,"keywords":249,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":264,"lastUpdatePostDateStruct":265,"startDateStruct":267,"completionDateStruct":269,"leadSponsor":271,"locationsCount":272},"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":245,"type":22},100,[64],"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.",[29,30],[68,69,70,71,72,36,35,250,251,252,253,254,241,255,256,257,258,259,260,261,262,263],"Inhibitors","Anti-Tissue Factor Pathway Inhibitor (TFPI)","Subcutaneous (sc)","Prophylaxis","On-Demand","Inhibitor","SC","Subcutaneous","Injection","On demand","aTFPI","Severe hemophilia","Severe bleeding","Hemophilia","2026-05-05",{"date":266,"type":42},"2026-05-07",{"date":268,"type":42},"2022-12-09",{"date":270,"type":22},"2028-09-10",{"name":80,"class":49},34,{"id":274,"slug":275,"hasResults":11,"nctId":276,"briefTitle":277,"officialTitle":278,"acronym":279,"eligibilityCriteria":280,"healthyVolunteers":11,"sex":58,"minAge":281,"maxAge":4,"enrollmentInfo":282,"targetDuration":4,"studyType":136,"phases":4,"briefSummary":284,"conditions":285,"keywords":287,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":292,"lastUpdatePostDateStruct":293,"startDateStruct":294,"completionDateStruct":296,"leadSponsor":298,"locationsCount":154},"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.","12 Years",{"count":283,"type":22},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).",[286,29,30],"Haemophilia",[288,289,290,291],"Subclinical synovial proliferation","Ultrasound","MRI","Elastography","2026-04-28",{"date":264,"type":42},{"date":295,"type":42},"2025-06-01",{"date":297,"type":22},"2028-04-01",{"name":299,"class":153},"Van Creveldkliniek",{"id":301,"slug":302,"hasResults":11,"nctId":303,"briefTitle":304,"officialTitle":305,"acronym":4,"eligibilityCriteria":306,"healthyVolunteers":11,"sex":17,"minAge":216,"maxAge":110,"enrollmentInfo":307,"targetDuration":4,"studyType":23,"phases":308,"briefSummary":309,"conditions":310,"keywords":311,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":313,"lastUpdatePostDateStruct":314,"startDateStruct":316,"completionDateStruct":317,"leadSponsor":319,"locationsCount":154},"100359528","phase-1-lentiviral-fix-gene-therapy-100359528","NCT03961243","Lentiviral FIX Gene Therapy","Lentiviral FIX Gene Therapy for Hemophilia B","Inclusion Criteria:\n\n* 1\\. Able to provide informed consent and comply with requirements of the study. 2. Males ≥2 years with confirmed diagnosis of hemophilia B (endogenous factor IX ≤2 IU\u002FdL or ≤2% of normal).\n\n  3\\. A minimum average of 4 bleeding events per year requiring episodic treatment of factor IX infusions or prophylactic factor IX infusions.\n\n  4\\. No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein.\n\n  5\\. Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences.\n\nExclusion Criteria:\n\n* 1\\. Significant liver dysfunction as defined by abnormal alanine transaminase, bilirubin and alkaline phosphatase.\n\n  2\\. History of inhibitor against factor IX. 3. Evidence of active hepatitis B or C and currently on antiviral therapy. 4. 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).\n\n  5\\. Any evidence of active infection or any immunosuppressive disorder. 6. 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.\n\n  7\\. Unable or unwilling to comply with study assessments.",{"count":50,"type":22},[25],"This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor IX into patients with hemophilia B, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.",[30],[30,198,312],"Gene therapylentiviral vector","2026-04-21",{"date":315,"type":42},"2026-04-24",{"date":295,"type":42},{"date":318,"type":22},"2028-06-01",{"name":320,"class":153},"Shenzhen Geno-Immune Medical Institute",{"id":322,"slug":323,"hasResults":11,"nctId":324,"briefTitle":325,"officialTitle":326,"acronym":327,"eligibilityCriteria":328,"healthyVolunteers":11,"sex":329,"minAge":18,"maxAge":4,"enrollmentInfo":330,"targetDuration":4,"studyType":136,"phases":4,"briefSummary":332,"conditions":333,"keywords":347,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":361,"lastUpdatePostDateStruct":362,"startDateStruct":364,"completionDateStruct":366,"leadSponsor":368,"locationsCount":154},"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","FEMALE",{"count":331,"type":22},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.",[334,335,336,337,338,339,340,341,342,343,29,30,344,345,346],"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",[348,349,350,351,352,353,354,355,356,357,358,359,360],"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":363,"type":42},"2026-04-23",{"date":365,"type":42},"2024-10-23",{"date":367,"type":22},"2027-05",{"name":369,"class":153},"Assistance Publique - Hôpitaux de Paris",{"id":371,"slug":372,"hasResults":11,"nctId":373,"briefTitle":374,"officialTitle":375,"acronym":4,"eligibilityCriteria":376,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":377,"targetDuration":4,"studyType":136,"phases":4,"briefSummary":379,"conditions":380,"keywords":386,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":393,"lastUpdatePostDateStruct":394,"startDateStruct":395,"completionDateStruct":397,"leadSponsor":399,"locationsCount":154},"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":378,"type":22},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.",[263,381,29,30,335,382,383,384,385],"Thrombosis","Glanzmann Thrombasthenia","Bleeding Disorder","Blood Disorder","Von Willebrand Diseases",[387,388,389,390,391,392],"bleed event","bleed treatments","adverse events","joint bleed","bleeding disorder","bleeding symptoms","2026-04-16",{"date":313,"type":42},{"date":396,"type":42},"2024-10-25",{"date":398,"type":22},"2055-10-31",{"name":400,"class":401},"American Thrombosis and Hemostasis Network","NETWORK",{"id":403,"slug":404,"hasResults":11,"nctId":405,"briefTitle":406,"officialTitle":407,"acronym":408,"eligibilityCriteria":409,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":410,"targetDuration":4,"studyType":136,"phases":4,"briefSummary":412,"conditions":413,"keywords":414,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":416,"lastUpdatePostDateStruct":417,"startDateStruct":419,"completionDateStruct":421,"leadSponsor":423,"locationsCount":424},"100516777","an-observational-cohort-study-to-characterize-the-effectiveness-and-safety-of-hemgenix-in-patients-with-hemophilia-b-100516777","NCT06008938","An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B","An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B","IX-TEND 4001","Inclusion Criteria:\n\n* HEMGENIX Cohort:\n* \\- Treatment with commercial HEMGENIX.\n* \\- Have provided signed written informed consent within 3 months before or within 6 months after HEMGENIX treatment, or within 6 months of when the study is initiated at the participating site.\n* FIX Prophylaxis Cohort:\n* \\- Adult patients (≥ 18 years) with hemophilia B who have consented and enrolled in ATHN Transcends Hemophilia Cohort (or a similar registry) and are receiving FIX prophylaxis therapy.\n\nExclusion Criteria:\n\n* HEMGENIX Cohort:\n* \\- The patient population that will be observed in this study must not have been treated with etranacogene dezaparvovec in a clinical trial.",{"count":411,"type":22},500,"This observational, post-authorization, long-term follow-up study aims to investigate the short and long-term effectiveness and safety of HEMGENIX in patients with hemophilia B. The study will also include a cohort of patients with hemophilia B treated with FIX prophylaxis to enable interpretation of relevant efficacy and safety findings of HEMGENIX.",[30],[30,415],"HEMGENIX","2026-02-18",{"date":418,"type":42},"2026-02-19",{"date":420,"type":42},"2023-06-15",{"date":422,"type":22},"2043-08-01",{"name":102,"class":49},12,{"id":426,"slug":427,"hasResults":11,"nctId":428,"briefTitle":429,"officialTitle":430,"acronym":431,"eligibilityCriteria":432,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":433,"targetDuration":4,"studyType":136,"phases":4,"briefSummary":435,"conditions":436,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":450,"lastUpdatePostDateStruct":451,"startDateStruct":453,"completionDateStruct":455,"leadSponsor":457,"locationsCount":154},"100412026","national-longitudinal-cohort-of-hematological-diseases-100412026","NCT04645199","National Longitudinal Cohort of Hematological Diseases","National Longitudinal Cohort of Hematological Diseases (NICHE)","NICHE","Inclusion Criteria:\n\n* Patients who were diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders or received bone marrow transplantation in the investigating hospitals from January 1, 2020.\n\nExclusion Criteria:\n\n* Long-term follow-up information for patients is not available for any reason, such as not being available or having a serious concomitant disease.\n* Alcohol and drug addictions affect their ability to comply with study requirements.\n* According to the investigator, there are conditions that may endanger the patient's safety or affect his\u002Fher compliance.",{"count":434,"type":22},2300,"Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in hematology. The purpose of this study is to analyze the incidence and risk factors of major blood diseases, the treatment methods, prognosis and medical expenses of these patients in China.\n\nMethod The study will include patients diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders, autoimmune hemolytic anemia, large granular lymphocyte leukemia, essential thrombocythemia, blood infection or received bone marrow transplantation in the investigating hospitals from January 1, 2020, and collect basic information, diagnostic and treatment information, prognosis information, as well as medical expense information from medical records. In its current form, the NICHE registry incorporates historical data (collected from 2000) and is systematically collecting prospective data in two phases with broadening reach, and prospectively follow-up to collect the prognosis information.",[437,438,263,29,30,439,440,441,442,443,383,444,445,446,447,448,449],"Multiple Myeloma","Acute Myeloid Leukemia","Myelodysplastic Syndrome","MDS","Lymphoma","Leukemia","Aplastic Anemia","Bone Marrow Transplantation","Blood Disease Infection","Autoimmune Hemolytic Anemia, AIHA","Essential Thrombocythemia, ET","Large Granular Lymphocyte Leukemia, LGLL","Paroxysmal Nocturnal Hemoglobinuria, PNH","2026-02-09",{"date":452,"type":42},"2026-02-12",{"date":454,"type":42},"2020-12-01",{"date":456,"type":22},"2030-12-01",{"name":458,"class":153},"Institute of Hematology & Blood Diseases Hospital, China",{"id":460,"slug":461,"hasResults":11,"nctId":462,"briefTitle":463,"officialTitle":463,"acronym":464,"eligibilityCriteria":465,"healthyVolunteers":11,"sex":58,"minAge":18,"maxAge":4,"enrollmentInfo":466,"targetDuration":4,"studyType":23,"phases":467,"briefSummary":468,"conditions":469,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":470,"lastUpdatePostDateStruct":471,"startDateStruct":473,"completionDateStruct":475,"leadSponsor":477,"locationsCount":479},"100569888","phase-3-an-open-label-comparative-study-of-the-efficacy-safety-and-pharmacodynamics-of-single-dose-of-anb-002-in-patients-with-hemophilia-b-100569888","NCT06700096","An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B","MAGNOLIA","Inclusion Criteria:\n\n* Men diagnosed with hemophilia B aged 18 or older\n* FIX activity ≤2%\n* Absense of FIX inhibitor\n* ≥150 previous exposure days of treatment with FIX concentrates\n\nExclusion Criteria:\n\n* Any diseases of blood and hematopoietic organs other than hemophilia B\n* A history of any gene therapy, including ANB-002\n* Diagnosed HIV-infection, not controlled with anti-viral therapy\n* Active HBV or HCV infection\n* Anti-AAV5 antibodies\n* Any active systemic infections or recurrent infections requiring systemic therapy\n* Any other disorders associated with severe immunodeficiency\n* Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder\n* Malignancies with less than 5 years of remission",{"count":5,"type":22},[64],"The aim of the study is to demonstrate non-inferiority of ANB-002 compared with preventive use of coagulation factor IX (FIX) in adult subjects with hemophilia B with FIX activity ≤2% and without FIX inhibitor. The study will have an open-label single-arm design.",[30],"2025-11-17",{"date":472,"type":42},"2025-11-18",{"date":474,"type":42},"2024-11-07",{"date":476,"type":22},"2032-03",{"name":478,"class":49},"Biocad",14,{"id":481,"slug":482,"hasResults":11,"nctId":483,"briefTitle":484,"officialTitle":485,"acronym":4,"eligibilityCriteria":486,"healthyVolunteers":11,"sex":58,"minAge":18,"maxAge":4,"enrollmentInfo":487,"targetDuration":4,"studyType":23,"phases":489,"briefSummary":490,"conditions":491,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":492,"lastUpdatePostDateStruct":493,"startDateStruct":495,"completionDateStruct":497,"leadSponsor":499,"locationsCount":500},"100525349","phase-1-study-of-the-safety-pharmacodynamics-and-efficacy-of-anb-002-in-patients-with-hemophilia-b-safran-100525349","NCT06120582","Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)","An Open-label, Non-comparative, Single Dose-Escalation Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B","Inclusion Criteria:\n\n1. Male with hemophilia B.\n2. Age ≥18 years.\n3. FIX activity at screening ≤2% without FIX inhibitor.\n4. ≥150 previous exposure days of treatment with FIX concentrates.\n\nExclusion Criteria:\n\n1. Previous gene therapy.\n2. Other blood or hematopoietic disorders.\n3. Positive Anti-AAV5 antibodies (for Cohorts 1-3).\n4. Diagnosed HIV-infection, not controlled with anti-viral therapy.\n5. Hepatitis B (for Cohorts 1-3), acute or chronic hepatitis C.\n6. Any active systemic infections or recurrent infections requiring systemic therapy at screening.\n7. Any other disorders associated with severe immunodeficiency.\n8. Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc).\n9. Malignancies with remission \\\u003C5 years.",{"count":488,"type":22},28,[25,114],"The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-002 in subjects with hemophilia В. The study will have a dose-escalation design with elements of phase I\u002FII seamless adaptive design.",[30],"2025-06-10",{"date":494,"type":42},"2025-06-13",{"date":496,"type":42},"2023-05-02",{"date":498,"type":22},"2029-11",{"name":478,"class":49},17,{"id":502,"slug":503,"hasResults":11,"nctId":504,"briefTitle":505,"officialTitle":506,"acronym":4,"eligibilityCriteria":507,"healthyVolunteers":11,"sex":58,"minAge":281,"maxAge":18,"enrollmentInfo":508,"targetDuration":4,"studyType":23,"phases":509,"briefSummary":510,"conditions":511,"keywords":512,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":514,"lastUpdatePostDateStruct":515,"startDateStruct":517,"completionDateStruct":519,"leadSponsor":521,"locationsCount":154},"100493756","phase-1-gene-therapy-for-hemophilia-b-patients-aged-12-18-years-old-100493756","NCT05709288","Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old","A Pilot Study Evaluating the Safety, Tolerability and Efficacy of Gene Therapy With BBM-H901 in Hemophilia B Patients Aged 12-18 Years Old","Inclusion Criteria:\n\n1. Subjects and statutory guardian must be able to understand the purpose and risks of the study and provide signed and dated informed consent;\n2. Be male and 12≤ age \\\u003C18 years of age, body wight ≥ 50kg;\n3. Have hemophilia B with ≤2 IU\u002FdL (≤2 %) endogenous FIX activity levels as documented by a certified clinical laboratory at the time of screening. If the screening result is \\>2% due to insufficient washout from FIX protein product, then the severity of hemophilia B may be confirmed by documented historical evidence from a certified clinical laboratory demonstrating ≤2% FIX coagulant activity (FIX:C) ;\n4. Had had ≥75 prior exposure days (EDs) to any recombinant and\u002For plasma-derived FIX protein products based on historical data from the subject's record\u002Fhistory;\n5. With ≤ 1:4 neutralizing antibodies and ≤1:200 binding antibodies against BBM-H901 capsid;\n6. Subjects with bleeding episode and\u002F or FIX agents infusion events within 12 weeks prior to screening;\n7. Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV immunoglobulin administration;\n8. Have no measurable FIX inhibitor as assessed by laboratory; or documented no prior history of FIX inhibitor (family history of inhibitors will not exclude the subject) and no clinical signs or symptoms of decreased response to FIX administration;\n9. Have acceptable laboratory values:\n\n   1. Hemoglobin ≥11 g\u002FdL ;\n   2. Platelets ≥100,000 cells\u002FμL;\n   3. AST, ALT ≤1.5x upper limit of normal at the testing laboratory;\n   4. Bilirubin ≤1.5x ULN ;\n   5. glomerular filtration rate eGFR ≥ 60ml\u002Fmin.\n10. For those subjects with sexual maturity, subject and statutory guardian must know that subjects must agree to use reliable barrier contraception until 52 weeks;\n11. with good compliance to the schedule of visit and fill in the subject diary.\n\nExclusion Criteria:\n\n1. Hepatitis B surface antigen antibody (HBSAg-Ab) or HBV-DNA positive; hepatitis C antibody or HCV-RNA positive;\n2. Currently on antiviral therapy for hepatitis B or C;\n3. With coagulation disorders other than hemophilia B;\n4. Had immunosuppressive therapy other than steroid and other suggested IST agents within 30 days prior to screening;\n5. Had vaccine 30 days prior to screening or have scheduled vaccination plan during the study (up to 52 weeks);\n6. Have significant underlying liver disease, as defined by a preexisting diagnosis of portal hypertension, splenomegaly, encephalopathy, etc; other liver conditions unsuitable to gene therapy judged by investigator;\n7. Have surgery plan within 52 weeks after gene therapy;\n8. Have history of chronic infection or high rish of infection that the Investigator considers to constitute an unacceptable risk;\n9. Had participated in a previous gene therapy research trial within the last 52 weeks or in a clinical study with an investigational drug within the last 12 weeks;\n10. Had any herb that may affect the liver function within 4 weeks prior to screening;\n11. Have history of fatal bleeding episode, eg intracranial hemorrhage, etc;\n12. Any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study;",{"count":126,"type":22},[25],"This is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU\u002Fdl residual FIX levels and aged 12-18 years old. BBM-H901 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.",[30],[194,513],"AAV","2025-02-20",{"date":516,"type":42},"2025-02-21",{"date":518,"type":42},"2023-03-23",{"date":520,"type":22},"2035-11",{"name":458,"class":153},{"id":523,"slug":524,"hasResults":11,"nctId":525,"briefTitle":526,"officialTitle":527,"acronym":4,"eligibilityCriteria":528,"healthyVolunteers":11,"sex":58,"minAge":18,"maxAge":110,"enrollmentInfo":529,"targetDuration":4,"studyType":23,"phases":531,"briefSummary":533,"conditions":534,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":514,"lastUpdatePostDateStruct":535,"startDateStruct":537,"completionDateStruct":539,"leadSponsor":541,"locationsCount":154},"100487712","the-efficacy-and-safety-of-zs801-in-chinese-hemophilia-b-patients-100487712","NCT05630651","The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.","A Non-randomized, Open-label Study to Evaluate the Safety, Kinetics and Efficacy of a Single Intravenous Infusion of ZS801 in Hemophilia B Subjects With Endogenous FIX ≤2%.","Inclusion Criteria:\n\n1. Male ≥18 years and ≤65years of age;\n2. Confirmed diagnosis of hemophilia B, and endogenous FIX ≤2%:\n3. Have had ≥100 prior exposure days (EDs) to any recombinant and\u002For plasma-derived FIX protein products;\n4. The subject had at least 3 or more bleeding events and\u002For chronic hemophilia arthritis in one or more joints in the previous 1 year requiring treatment with FIX agents;\n5. Agree to use reliable barrier contraception and prohibition of sperm donation until 52 weeks after the administration of ZS801.\n6. Subjects voluntarily participate and are fully informed, fully understand the research and can comply with the requirements of the research protocol, are willing to complete the research as planned, and voluntarily cooperate with the provision of biological samples for testing.\n\nExclusion Criteria:\n\n1. Hypersensitivity to any component of the study drug (including immunosuppressants) or a condition that can not use;\n2. Inability to tolerate immunosuppressants or steroid drugs;\n3. Have FIX inhibitor as assessed by laboratory; or documented history of FIX inhibitor;\n4. Who have a history or are currently suffering from any of the following serious clinical diseases:\n\n   1. History of malignancy or current presence of any malignancy;\n   2. Have active autoimmune disease;\n   3. Severe heart disease, including angina pectoris, myocardial infarction, heart failure, clinically significant congenital heart disease, heart valve disease, arrhythmia and atrioventricular block, etc.;\n   4. Have underlying liver disease or history of liver disease (such as portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy or hepatic fibrosis);\n   5. Have HBsAg positive or HCV-Ab positive, or are currently receiving hepatitis B or hepatitis C antiviral therapy;\n   6. Diabetes mellitus that is poorly controlled after drug treatment;\n   7. Uncontrolled hypertension or hypotension;\n5. laboratory values:\n\n   1. Hemoglobin\\\u003C110g\u002FL;\n   2. Platelets\\\u003C100×10\\^9\u002FL;\n   3. aspartate aminotransferase, Alanine transaminase, alkaline phosphatase\\>2×ULN;\n   4. Total bilirubin\\>1.5×ULN;\n   5. Creatinine\\>ULN;\n   6. Albumin\\\u003CLLN;\n   7. HIV antibody positive or Treponema pallidum antibody positive.\n6. Have AAV5 capsid neutralizing antibody titers \\>1:640;\n7. Those who have received clinical trials of gene therapy before screening, or have used FIX clinical trial drugs within 1 month, or participated in other drug\u002Fdevice clinical trials within 3 months, or plan to participate in other clinical trials during this study;\n8. Those who have planned surgery within 52 weeks after the infusion;\n9. Those who lost more than 400 mL of blood within 3 months before screening;\n10. Those with epilepsy, history of mental illness (such as schizophrenia, depression, mania or anxiety) or obvious mental disorder, incapacitated or incapacitated by other reasons;\n11. Patients with a history of drug abuse or alcoholism;\n12. Investigators believe that subjects have poor compliance or are expected to be less likely to complete follow-up;\n13. There are clinically significant diseases or other reasons that the researcher and\u002For collaborators consider unsuitable to participate in this researcher.",{"count":530,"type":22},6,[532],"NA","A non-randomized, open-label study to evaluate the safety, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.",[30],{"date":536,"type":42},"2025-02-24",{"date":538,"type":42},"2023-04-19",{"date":540,"type":22},"2028-12",{"name":458,"class":153},{"id":543,"slug":544,"hasResults":11,"nctId":545,"briefTitle":546,"officialTitle":547,"acronym":4,"eligibilityCriteria":548,"healthyVolunteers":11,"sex":58,"minAge":18,"maxAge":4,"enrollmentInfo":549,"targetDuration":4,"studyType":23,"phases":551,"briefSummary":552,"conditions":553,"keywords":554,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":514,"lastUpdatePostDateStruct":556,"startDateStruct":557,"completionDateStruct":559,"leadSponsor":560,"locationsCount":154},"100488553","phase-1-a-study-to-evaluate-the-safety-and-efficacy-of-zs801-in-adult-hemophilia-b-patients-100488553","NCT05641610","A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients","A Non-randomized, Open-label, Dose-escalation, Phase I\u002FII Study to Evaluate the Safety, Tolerability, Kinetics and Efficacy of a Single Intravenous Infusion of ZS801 in Hemophilia B Subjects With Endogenous FIX ≤2%.","Inclusion Criteria:\n\n1. Male ≥18 years of age;\n2. Confirmed diagnosis of hemophilia B, and endogenous FIX ≤2%;\n3. Have had ≥100 prior exposure days (EDs) to any recombinant and\u002For plasma-derived FIX protein products;\n4. The subject had at least 3 or more bleeding events and\u002For chronic hemophilia arthritis in one or more joints in the previous 1 year requiring treatment with FIX;\n5. Agree to use reliable barrier contraception and prohibition of sperm donation until 52 weeks after the administration of ZS801.\n6. Subjects voluntarily participate and are fully informed, fully understand the research and can comply with the requirements of the research protocol, are willing to complete the research as planned, and voluntarily cooperate with the provision of biological samples for testing.\n\nExclusion Criteria:\n\n1. Hypersensitivity to any component of the study drug (including immunosuppressants) or a condition that can not use;\n2. Inability to tolerate immunosuppressants or steroid drugs;\n3. Have FIX inhibitor as assessed by laboratory, or documented history of FIX inhibitor;\n4. Who have a history or are currently suffering from any of the following serious clinical diseases:\n\n   1. History of malignancy or current presence of any malignancy;\n   2. Have active autoimmune disease;\n   3. Severe heart disease, including angina pectoris, myocardial infarction, heart failure, clinically significant congenital heart disease, heart valve disease, arrhythmia and atrioventricular block, etc.;\n   4. Have underlying liver disease or history of liver disease (such as portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy or hepatic fibrosis);\n   5. Have active hepatitis B infection (HBsAg positive) or active hepatitis C infection (HCVAb positive), or are currently receiving hepatitis B or hepatitis C antiviral therapy;\n   6. Diabetes mellitus that is poorly controlled after drug treatment;\n   7. Uncontrolled hypertension or hypotension;\n5. laboratory values:\n\n   1. Hemoglobin\\\u003C110g\u002FL;\n   2. Platelets\\\u003C100×10\\^9\u002FL;\n   3. AST, ALT, alkaline phosphatase\\>2×ULN;\n   4. Total bilirubin\\>1.5×ULN;\n   5. Creatinine\\>ULN;\n   6. Albumin\\\u003CLLN;\n   7. HIV antibody positive or Treponema pallidum antibody positive.\n6. Have AAV5 capsid neutralizing antibody titers \\>1:5;\n7. Those who have received clinical trials of gene therapy before screening, or have used FIX clinical trial drugs within 1 month, or participated in other drug\u002Fdevice clinical trials within 3 months, or plan to participate in other clinical trials during this study;\n8. Those who have planned surgery within 52 weeks after the infusion;\n9. Those who lost more than 400 mL of blood within 3 months before screening;\n10. Those with epilepsy, history of mental illness (such as schizophrenia, depression, mania or anxiety) or obvious mental disorder, incapacitated or incapacitated by other reasons;\n11. Patients with a history of drug abuse or alcoholism;\n12. Investigators believe that subjects have poor compliance or are expected to be less likely to complete follow-up;\n13. There are clinically significant diseases or other reasons that the researcher and\u002For collaborators consider unsuitable to participate in this researcher.",{"count":550,"type":22},21,[25,114],"A non-randomized, open-label, dose-escalation, phase I\u002FII study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.",[30],[30,555],"Gene therapy",{"date":536,"type":42},{"date":558,"type":42},"2022-02-16",{"date":540,"type":22},{"name":458,"class":153},{"id":562,"slug":563,"hasResults":11,"nctId":564,"briefTitle":565,"officialTitle":566,"acronym":567,"eligibilityCriteria":568,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":569,"targetDuration":4,"studyType":23,"phases":571,"briefSummary":572,"conditions":573,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":577,"lastUpdatePostDateStruct":578,"startDateStruct":580,"completionDateStruct":582,"leadSponsor":584,"locationsCount":154},"100559542","measurement-properties-in-people-with-hemophilia-100559542","NCT06565481","Measurement Properties in People with Hemophilia","Measurement Properties (Reliability and Validity) of a Set of Assessments Evaluating Physical Function in People with Hemophilia","HEMOPRO","Inclusion Criteria:\n\n* Diagnosed Hemophilia Types A or B\n* Adult people ≥ 18 years\n* Patients on prophylactic factor or non-factor replacement treatment, or on demand factor replacement treatment.\n* Able to walk for 20 meters independently, with or without walking aid\n* Signed informed consent to participate in the study\n\nExclusion Criteria:\n\n* Severe health conditions like severe cardiovascular, pulmonary, neurological or musculoskeletal diseases.\n* Unable to perform the assessments due to disability or language problems.",{"count":570,"type":22},50,[532],"The aim of this project is to determine the measurement properties (namely reliability and validity) of a set of clinical assessments, measuring different aspects of physical function.",[574,30,575,576],"Hemophilia a","Musculoskeletal Complication","Measurement Error","2025-01-10",{"date":579,"type":42},"2025-01-14",{"date":581,"type":42},"2024-10-31",{"date":583,"type":22},"2026-05-31",{"name":585,"class":153},"University of Zurich",{"id":587,"slug":588,"hasResults":11,"nctId":589,"briefTitle":590,"officialTitle":590,"acronym":4,"eligibilityCriteria":591,"healthyVolunteers":16,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":592,"targetDuration":594,"studyType":136,"phases":4,"briefSummary":595,"conditions":596,"keywords":609,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":611,"lastUpdatePostDateStruct":612,"startDateStruct":614,"completionDateStruct":616,"leadSponsor":618,"locationsCount":620},"100572008","longitudinal-cohort-of-thrombosis-and-hemostasis-diseases-100572008","NCT06727669","Longitudinal Cohort of Thrombosis and Hemostasis Diseases","Inclusion Criteria:\n\n* Patients who were diagnosed as thrombosis and hemostasis diseases.\n\nExclusion Criteria:\n\n* Long-term follow-up information for patients is not available for any reason, such as not being available or having a serious concomitant disease.\n* Patients with alcohol and drug addictions or mental illness affect their ability to comply with study requirements.\n* According to the investigator, there are conditions that may endanger the patient's safety or affect his\u002Fher compliance.",{"count":593,"type":22},3000,"5 Years","This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients. This study will collect basic information, diagnostic and treatment information, as well as medical expense information of patients from medical records.The incidence and risk factors of thrombosis and hemostasis diseases, the treatment methods, prognosis and medical expenses of these patients in China will be analyzed. The study will use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.",[597,598,599,600,601,602,603,604,605,29,30,606,607,608,383,381],"Immune Thrombocytopenia","Thrombotic Thrombocytopenic Purpura","Hemophilia A, Acquired","Disseminated Intravascular Coagulation","Thrombophilia","Deep Vein Thrombosis","Pulmonary Embolism","Thrombotic Microangiopathies","Coagulation Factor Deficiency","Hemophilia B, Acquired","Platelet Dysfunction","Arterial Thromboembolism",[381,610],"Hemostasis","2024-12-05",{"date":613,"type":42},"2024-12-11",{"date":615,"type":42},"2024-11-01",{"date":617,"type":22},"2030-12-31",{"name":619,"class":153},"Peking University People's Hospital",5,{"id":622,"slug":623,"hasResults":11,"nctId":624,"briefTitle":625,"officialTitle":625,"acronym":626,"eligibilityCriteria":627,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":628,"targetDuration":594,"studyType":136,"phases":4,"briefSummary":630,"conditions":631,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":632,"lastUpdatePostDateStruct":633,"startDateStruct":635,"completionDateStruct":637,"leadSponsor":639,"locationsCount":154},"100310930","world-bleeding-disorders-registry-100310930","NCT03327779","World Bleeding Disorders Registry","WBDR","Inclusion Criteria:\n\n* Patients of participating Hemophilia Treatment Centres with Hemophilia A or B, or von Willebrand Disease\n\nExclusion Criteria:\n\n* none",{"count":629,"type":22},20000,"The WBDR is an international observational disease registry of patients with hemophilia. It will provide a platform for a network of hemophilia treatment centres (HTCs) around the world to collect uniform and standardized patient data and guide clinical practice. With informed consent from the patient, the WBDR stores anonymous data about the person's disease, such as hemophilia type and severity, symptoms, and treatment.",[29,30,385],"2023-08-07",{"date":634,"type":42},"2023-08-14",{"date":636,"type":42},"2018-01-26",{"date":638,"type":22},"2028-01",{"name":640,"class":153},"World Federation of Hemophilia",{"id":642,"slug":643,"hasResults":11,"nctId":644,"briefTitle":645,"officialTitle":645,"acronym":646,"eligibilityCriteria":647,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":18,"enrollmentInfo":648,"targetDuration":650,"studyType":136,"phases":4,"briefSummary":651,"conditions":652,"keywords":4,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":655,"lastUpdatePostDateStruct":656,"startDateStruct":658,"completionDateStruct":660,"leadSponsor":662,"locationsCount":154},"100279037","german-pediatric-hemophilia-research-database-100279037","NCT02912143","German Pediatric Hemophilia Research Database","GEPHARD","Inclusion Criteria:\n\n* hemophilia A or B\n* FVIII\u002FFIX \\\u003C1% to 25%\n* informed consent\n\nExclusion Criteria:\n\n* no consent",{"count":649,"type":22},800,"10 Years","The German Pediatric Hemophilia Research Database will collect data on the prophylactic and therapeutic use of factor concentrates, complications, outcome measures (joint scores, QoL) and living circumstances in newly diagnosed children with hemophilia.",[29,30,653,654],"Children","Drug Specific Antibodies","2023-03-06",{"date":657,"type":42},"2023-03-08",{"date":659,"type":4},"2017-01-01",{"date":661,"type":22},"2028-12-31",{"name":663,"class":153},"Goethe University"]