[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"CSL Behring\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":460},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,17,0,[8,44,67,98,128,153,181,206,234,261,289,314,340,368,391,418,435],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":27,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"100618474","phase-2-efficacy-and-safety-of-vamifeport-in-adult-participants-with-homeostatic-iron-regulator-gene-hfe-related-hereditary-hemochromatosis-100618474",false,"NCT07332091","Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis","A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study of the Efficacy and Safety of Vamifeport in Adult Subjects With HFE-related Hereditary Hemochromatosis (FERROCLEAR Study)","Inclusion Criteria:\n\n* Adult (≥ 18 years) and has provided written informed consent.\n* Confirmed diagnosis of HFE-HH in medical history.\n* Evidence of iron overload as shown by:\n\n  * TSAT \\> 45% (confirmed at 2 visits, at least 14 days apart) at Screening; and\n  * Serum ferritin ≥ 200 nanogram per milliliter (ng\u002FmL) and \\\u003C 5000 ng\u002FmL (confirmed at 2 visits, at least 14 days apart) at Screening; and\n  * MRI-based LIC between 3 and 16 mg\u002Fg (53.7 and 286.5 millimol per kilogram \\[mmol\u002Fkg\\]) dry weight (dw) at Screening.\n* Body mass index between 18.5 and 32 kilograms per meter squared (kg\u002Fm\\^2).\n\nExclusion Criteria:\n\n* Clinically relevant laboratory abnormalities, 12-lead electrocardiogram (ECG) findings, or medical history.","ALL","18 Years",{"count":19,"type":20},84,"ESTIMATED","INTERVENTIONAL",[23],"PHASE2","This is a phase 2, multicenter, randomized, placebo-controlled, double-blind, parallel-group, proof-of-concept study to assess vamifeport in adult participants with homeostatic iron regulator gene-related hereditary hemochromatosis (HFE-HH). The primary objective of the study is to assess the effect of vamifeport treatment on magnetic resonance imaging (MRI)-based liver iron concentration (LIC) in adult participants with HFE-HH.",[26],"Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis",[28,29,30],"Small Molecule Ferroportin Inhibitor","Hereditary hemochromatosis","Iron overload","RECRUITING","2026-06-23",{"date":34,"type":35},"2026-06-26","ACTUAL",{"date":37,"type":35},"2026-01-22",{"date":39,"type":20},"2028-04-06",{"name":41,"class":42},"CSL Behring","INDUSTRY",98,{"id":45,"slug":46,"hasResults":11,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":4,"eligibilityCriteria":50,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":51,"targetDuration":4,"studyType":21,"phases":53,"briefSummary":55,"conditions":56,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":66},"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":52,"type":20},35,[54],"PHASE3","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.",[57],"Hemophilia B","2026-06-15",{"date":60,"type":35},"2026-06-16",{"date":62,"type":35},"2024-01-30",{"date":64,"type":20},"2032-04-02",{"name":41,"class":42},27,{"id":68,"slug":69,"hasResults":11,"nctId":70,"briefTitle":71,"officialTitle":72,"acronym":4,"eligibilityCriteria":73,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":74,"targetDuration":4,"studyType":21,"phases":76,"briefSummary":77,"conditions":78,"keywords":80,"overallStatus":90,"whyStopped":4,"lastUpdateSubmitDate":91,"lastUpdatePostDateStruct":92,"startDateStruct":93,"completionDateStruct":95,"leadSponsor":97,"locationsCount":4},"100642034","phase-3-single-arm-study-of-igpro20-in-adults-with-secondary-immune-deficiencies-due-to-hematologic-malignancies-treated-with-b-cell-targeting-chimeric-antigen-receptor-t-cell-and-t-cell-redirecting-therapies-100642034","NCT07648264","Single-arm Study of IgPro20 in Adults With Secondary Immune Deficiencies Due to Hematologic Malignancies Treated With B-cell Targeting Chimeric Antigen Receptor T-cell and T-cell Redirecting Therapies","A Phase 3, Prospective, Open-label, Multicenter, Single-arm Study to Investigate the Efficacy, Safety, and Pharmacokinetics of IgPro20 in Subjects With Secondary Immune Deficiency Due to Hematologic Malignancies Treated With B-cell Targeting Chimeric Antigen Receptor T-cell and T-cell Redirecting Therapies","Inclusion Criteria:\n\n* Participants greater than or equal to (≥) 18 years of age at the time of providing written informed consent.\n* Confirmed diagnosis of B-cell hematologic malignancy (ie, Multiple myeloma \\[MM\\], Chronic lymphocytic leukemia \\[CLL\\], Non-Hodgkin lymphoma \\[NHL\\], or BALL) according to applicable diagnostic criteria.\n* Participants treated with Chimeric antigen receptor T-cell (CAR T-cell) therapy or TCE BsAb and are:\n\n  1. At least 2 months after receipt of an approved CAR T-cell therapy for the B-cell hematologic malignancy at the time of Screening, or\n  2. At least 1 month after initiation of an approved TCE BsAb therapy for the B-cell hematologic malignancy at the time of Screening and expected to continue with the therapy.\n* Documented partial or complete response to CAR T-cell or TCE BsAb therapy based on applicable response criteria at the time of Screening:\n\n  1. CLL based on International Workshop on Chronic Lymphocytic Leukemia response criteria\n  2. MM based on International Myeloma Working Group response criteria\n  3. NHL based on Lugano Classification criteria\n  4. B-ALL based on National Comprehensive Cancer Network guidelines\n* IgG level (excluding paraprotein, if relevant) at Screening:\n\nIf participant has ongoing IgRT (intravenous immunoglobulin \\[IVIG\\] or subcutaneous immunoglobulin \\[SCIG\\]) for SID during Screening, then any IgG level at Screening is acceptable for enrollment. Participants with IgG less than (\\\u003C) 500 milligrams per deciliter (mg\u002FdL) are assigned to the Loading Cohort, participants with IgG ≥ 500 mg\u002FdL are assigned to the Maintenance-only Cohort.\n\n* IgG level (excluding paraprotein, if relevant) at Screening:\n\nIf participant does not have ongoing IgRT (IVIG for \\> 8 weeks or SCIG for \\> 2 weeks) for SID during Screening and are not expected to receive IgRT during Screening, then IgG \\\u003C 500 mg\u002FdL is required for enrollment (participant is assigned to the Loading Cohort)\n\nExclusion Criteria:\n\n* Documented history of diseases for which IgRT may be indicated: primary immune deficiency, chronic inflammatory demyelinating polyneuropathy, Guillain-Barré syndrome, immune thrombocytopenia, Kawasaki disease, Lambert-Eaton myasthenic syndrome, multifocal motor neuropathy, myasthenia gravis, stiff person syndrome, solid organ transplant, and rejection prior to Screening.\n* History of thromboembolic event (TEE) within 6 months before Screening.\n* Eastern Cooperative Oncology Group performance status \\> 1.\n* Presence of any systemic active infection at Screening.\n* Participants on any prohibited therapies, including anti-infective treatments.\n* Absolute neutrophil count \\\u003C 1 × 10\\*9\u002FL (Common Terminology Criteria for Adverse Events \\[CTCAE\\] Grade 3 or worse), unless proven to be due to the underlying disease and raised above the limit by granulocyte colony-stimulating factor.\n* Concurrent participation in other interventional clinical studies. Note: a participant may be enrolled if their participation in the other study will not jeopardize their safety and \u002F or the scientific validity of this study (eg, an observational study, a long-term safety follow-up of an interventional study, diagnostic device studies, phase 4 studies with medicines used within their approved indication); the investigator may consult with the medical monitor.",{"count":75,"type":20},63,[54],"This is a prospective, multicenter, open-label, single-arm study to assess the efficacy, safety, and pharmacokinetics (PK) of IgPro20 in adults with hematologic malignancies treated with B-cell targeting Chimeric antigen receptor T-cell (CAR T-cell) and T-cell redirecting therapies (such as T-cell engager bispecific antibody \\[TCE BsAb\\] therapy). The primary objective is to demonstrate that true annualized rate of serious bacterial infection (SBIs) is less than (\\\u003C) 1.0.\n\nThis study includes two cohorts:\n\n1. Loading Cohort: Participants with serum immunoglobulin G (IgG) \\\u003C 500 milligrams per deciliter (mg\u002FdL) at Screening, with or without ongoing immunoglobulin replacement therapy (IgRT) during Screening, who must have received five doses of IgPro20 during the Initial Treatment Period.\n2. Maintenance-only Cohort: Participants with serum IgG greater than or equal to (≥) 500 mg\u002FdL and ongoing IgRT at Screening, who must have received one dose of IgPro20 during the Initial Treatment Period.",[79],"Secondary Immune Deficiency",[81,82,83,84,85,86,87,88,89],"Hematologic Malignancies","B-cell acute lymphoblastic leukemia (B-ALL)","Multiple myeloma (MM)","Chronic lymphocytic leukemia (CLL)","Small lymphocytic lymphoma (SLL)","Non-Hodgkin lymphomas (NHL)","T-cell engager bispecific antibody (TCE BsAb) therapy","Hypogammaglobulinemia","Lowered serum immunoglobulin levels","NOT_YET_RECRUITING","2026-06-10",{"date":58,"type":35},{"date":94,"type":20},"2026-07-25",{"date":96,"type":20},"2029-02-16",{"name":41,"class":42},{"id":99,"slug":100,"hasResults":11,"nctId":101,"briefTitle":102,"officialTitle":103,"acronym":104,"eligibilityCriteria":105,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":106,"targetDuration":4,"studyType":21,"phases":108,"briefSummary":109,"conditions":110,"keywords":112,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":119,"lastUpdatePostDateStruct":120,"startDateStruct":122,"completionDateStruct":124,"leadSponsor":126,"locationsCount":127},"100476594","phase-2-combined-dose-finding-and-cv-outcomes-study-with-csl300-clazakizumab-in-adult-subjects-with-eskd-undergoing-dialysis-posibil6eskd-100476594","NCT05485961","Combined Dose-Finding and CV Outcomes Study With CSL300 (Clazakizumab) in Adult Subjects With ESKD Undergoing Dialysis (POSIBIL6ESKD)","A Phase 2b \u002F 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Combined Dose-Finding and Cardiovascular Outcome Study to Investigate the Efficacy and Safety of CSL300 (Clazakizumab) in Subjects With End Stage Kidney Disease Undergoing Dialysis","POSIBIL6ESKD","Inclusion Criteria:\n\n* Male or female at least 18 years of age.\n* A diagnosis of ESKD undergoing maintenance dialysis for at least 12 weeks.\n* Serum hs-CRP ≥ 2.0 mg\u002FL.\n* A diagnosis of diabetes mellitus OR ASCVD.\n\nExclusion Criteria:\n\n* Subjects who participated in Part 1 (phase 2b) are not eligible to participate in Part 2 (phase 3).\n* Concomitant use of systemic immunosuppressant drugs.\n* Abnormal LFTs.\n* Any life-threatening disease expected to result in death within 12 months.\n* A history of GI perforation, inflammatory bowel disease (except fully excised. ulcerative colitis), or peptic ulcer disease.\n* Clinically significant active infection or history of opportunistic or invasive fungal infection.",{"count":107,"type":20},3110,[23,54],"This is a two-part, phase 2b and phase 3 combined prospective, interventional, multicenter, randomized, double-blind, placebo-controlled study.\n\nPart 1: Phase 2b is a dose-finding study for CSL300 vs placebo. Part 2: Phase 3 aims to assess the efficacy of CSL300 vs placebo on cardiovascular (CV) outcomes and safety in subjects with systemic inflammation and either atherosclerotic cardiovascular disease (ASCVD) or diabetes with end stage kidney disease (ESKD) undergoing maintenance dialysis.",[111],"Atherosclerotic Cardiovascular Disease in Patients With ESKD",[113,114,115,116,117,118],"End Stage Kidney Disease (ESKD)","Myocardial infarction (MI)","Atherosclerotic cardiovascular disease (ASCVD)","Coronary artery disease","Peripheral artery disease","Diabetes","2026-06-05",{"date":121,"type":35},"2026-06-08",{"date":123,"type":35},"2022-10-21",{"date":125,"type":20},"2029-05-22",{"name":41,"class":42},547,{"id":129,"slug":130,"hasResults":11,"nctId":131,"briefTitle":132,"officialTitle":133,"acronym":134,"eligibilityCriteria":135,"healthyVolunteers":11,"sex":136,"minAge":137,"maxAge":138,"enrollmentInfo":139,"targetDuration":4,"studyType":21,"phases":141,"briefSummary":142,"conditions":143,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":144,"lastUpdatePostDateStruct":145,"startDateStruct":147,"completionDateStruct":149,"leadSponsor":151,"locationsCount":152},"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).","MALE","138 Months","206 Months",{"count":140,"type":20},20,[54],"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.",[57],"2026-06-03",{"date":146,"type":35},"2026-06-04",{"date":148,"type":35},"2025-07-28",{"date":150,"type":20},"2033-10-24",{"name":41,"class":42},2,{"id":154,"slug":155,"hasResults":11,"nctId":156,"briefTitle":157,"officialTitle":158,"acronym":159,"eligibilityCriteria":160,"healthyVolunteers":11,"sex":16,"minAge":161,"maxAge":4,"enrollmentInfo":162,"targetDuration":4,"studyType":164,"phases":4,"briefSummary":165,"conditions":166,"keywords":168,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":173,"lastUpdatePostDateStruct":174,"startDateStruct":175,"completionDateStruct":177,"leadSponsor":179,"locationsCount":180},"100593041","a-study-investigating-the-effectiveness-and-safety-of-garadacimab-for-treating-patients-with-hereditary-angioedema-hae-100593041","NCT07001280","A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)","Garadacimab Real-world Treatment Outcomes of Effectiveness, Safety, and Quality-of-Life in Patients With HAE (GREAT Study)","GREAT","Inclusion Criteria:\n\n* 1\\. Participants aged greater than or equal to (\\>=) 12 years at enrollment.\n* 2\\. Participants with clinical and\u002For laboratory confirmed diagnosis of HAE.\n* 3\\. Participants newly initiating garadacimab, as prescribed according to the decision of the treating physician per routine clinical practice and in accordance with the indication per the approved local label, independent of and prior to enrollment in the study.\n* 4\\. Willing and able to provide written informed consent and\u002For assent by parent or legal guardian for children less than (\\\u003C) 18 years of age (or legal age of consent in the respective countries).\n* 5\\. Ability to use an electronic device such as a smartphone or a computer for data collection in the study.\n\nExclusion Criteria:\n\n* 1\\. Participants with a concomitant diagnosis of another form of angioedema such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria (histaminergic angioedema).\n* 2\\. Participants participating in any ongoing interventional clinical study, including interventional studies with garadacimab. Participants in this study who later chose to enroll in any interventional clinical study (including garadacimab) will be discontinued from this study.","12 Years",{"count":163,"type":20},200,"OBSERVATIONAL","This is a multinational, multicenter, prospective, observational cohort study of patients with HAE in the real-world setting. The study will include patients newly initiating garadacimab in routine clinical practice. Each participant will be followed for 48 months after index date (date of the first administration of garadacimab).\n\nPatient data will be collected from the HAE eDiary, patient medical records (MRs) and\u002For during a routine clinical visit and will be entered into the electronic case report form (eCRF) via an electronic data capture (EDC) system. Data pertaining to HAE attacks, prior HAE treatments, retrospective focused safety data collection, and healthcare resource utilization (HCRU) over a look-back period of 12 months prior to the enrollment will be extracted from the MR, and patients will also record retrospective HAE attack related data over a look-back period of 3 months prior to enrollment in the HAE eDiary.\n\nThe primary aim of this study is to investigate the real-world effectiveness of garadacimab as measured by HAE attack rate before and after garadacimab initiation in patients with HAE over 24 months of follow-up. The study will aim to complement the data available from the clinical development program on the efficacy, safety, and health-related quality-of-life (HRQoL) in patients with HAE taking garadacimab.",[167],"Hereditary Angioedemas",[169,170,171,172],"Angioedema","Genetic disorder","Long term prophylaxis","Health-related quality of life","2026-06-02",{"date":144,"type":35},{"date":176,"type":35},"2025-07-21",{"date":178,"type":20},"2030-08-31",{"name":41,"class":42},22,{"id":182,"slug":183,"hasResults":11,"nctId":184,"briefTitle":185,"officialTitle":186,"acronym":4,"eligibilityCriteria":187,"healthyVolunteers":11,"sex":16,"minAge":188,"maxAge":189,"enrollmentInfo":190,"targetDuration":4,"studyType":21,"phases":192,"briefSummary":194,"conditions":195,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":198,"startDateStruct":200,"completionDateStruct":202,"leadSponsor":204,"locationsCount":205},"100338251","phase-4-two-dose-levels-of-privigen-in-pediatric-cidp-100338251","NCT03684018","Two Dose Levels of Privigen in Pediatric CIDP","Randomized Study of Two Dose Levels of Privigen in Pediatric CIDP","Inclusion Criteria:\n\n* \\- Male or female subjects 2 to ≤ 17 years of age with confirmed or possible CIDP.\n\nExclusion Criteria:\n\n* \\- Absence of CIDP symptoms\n* -History or family history of inherited neuropathy\n* -Diagnosed developmental delay or regression\n* -History of thrombotic episode\n* -Known or suspected hypersensitivity to Privigen\n* -Known allergic or other severe reactions to blood products\n* -Female subject of childbearing potential either not using or not willing to use a medically reliable method of contraception or not sexually abstinent during the study\n* -Pregnant or breastfeeding mother\"","2 Years","17 Years",{"count":191,"type":20},30,[193],"PHASE4","A randomized, open-label, prospective, multicenter study designed to investigate 2 dose levels in pediatric subjects 2 to ≤ 17 years of age with confirmed or possible CIDP, either previously exposed to IVIG treatment or unexposed to IVIG treatment",[196],"Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)","2026-05-28",{"date":199,"type":35},"2026-06-01",{"date":201,"type":35},"2019-02-28",{"date":203,"type":20},"2029-12-20",{"name":41,"class":42},9,{"id":207,"slug":208,"hasResults":11,"nctId":209,"briefTitle":210,"officialTitle":211,"acronym":4,"eligibilityCriteria":212,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":213,"targetDuration":4,"studyType":21,"phases":215,"briefSummary":217,"conditions":218,"keywords":219,"overallStatus":90,"whyStopped":4,"lastUpdateSubmitDate":227,"lastUpdatePostDateStruct":228,"startDateStruct":229,"completionDateStruct":231,"leadSponsor":233,"locationsCount":4},"100637925","phase-1-a-phase-1b-study-to-evaluate-the-pk-of-csl300-clazakizumab-in-chinese-subjects-with-end-stage-kidney-disease-eskd-100637925","NCT07619820","A Phase 1b Study to Evaluate the PK of CSL300 (Clazakizumab) in Chinese Subjects With End Stage Kidney Disease (ESKD)","A Phase 1b, Randomized, Multicenter, Placebo-controlled Study to Evaluate the Pharmacokinetics and Safety of CSL300 (Clazakizumab) in Chinese Subjects With End Stage Kidney Disease Undergoing Dialysis","Inclusion Criteria:\n\n* Participants has provided written informed consent and is willing and able to adhere to all protocol requirements.\n* Aged 18 or older, inclusive, at the time of providing written informed consent.\n* Diagnosis of ESKD undergoing maintenance dialysis for at least 12 weeks before Screening.\n\nExclusion Criteria:\n\n* Exclusion related to risk of infection: concomitant use of systemic immunosuppressant agents, primary immunodeficiency, positive test for active tuberculosis (TB), history of latent TB without completion of full course of prophylactic treatment, evidence of human immunodeficiency virus infection during Screening, seropositivity for hepatitis B surface antigen or positive hepatitis B virus (HBV) DNA during Screening, seropositivity for hepatitis C virus ribonucleic acid during Screening, diagnosis of clinically significant active infection, history of \u002F OR current invasive fungal infection OR other opportunistic infection OR recurrent cellulitis (defined as 2 or more episodes in the year prior to screening), administration of a live vaccine within 6 weeks of start of Screening, presence of urinary catheter, or evidence of wet gangrene or nonhealing ulcers.\n* Exclusion related to laboratory abnormalities: abnormal liver function tests, neutropenia, thrombocytopenia, or significant anemia.\n* Exclusion related to medical history: any life-threatening disease expected to result in death within 12 months (other than cardiovascular disease), evidence of active hepatic disease and \u002F or moderate or severe hepatic impairment, recent unplanned hospitalization (\\\u003C 30 days) prior to Screening, recent (\\\u003C 3 months) major surgery or planned major surgery known at the time of Screening, poorly controlled hypertension, a present or previous (\\\u003C 5 years) malignancy except for basal cell carcinoma, fully excised squamous cell carcinoma of the skin, or nonrecurrent (\\\u003C 5 years of Screening) cervical carcinoma in situ, active or recent (\\\u003C 30 days of Screening) clinically severe bleeding, a scheduled kidney transplant within 6 months of Screening, a history of anaphylaxis or hypersensitivity to CSL300 or any constituents of the product, or a history of demyelinating disorders.\n* Exclusion related to risk of gastrointestinal perforation: a history of GI perforation, inflammatory bowel disease (except fully excised ulcerative colitis), or peptic ulcer disease (\\\u003C 12 months before Screening), a history of diverticular disease or diverticulitis (except if disease has been fully excised). An incidental finding of diverticulosis (presence of small diverticula) and no history of symptoms, complications, or any episodes requiring treatment may be eligible for the study based on investigator's judgment. However, any history of complications, inflammation, or infection suggestive of diverticulitis or diverticular disease is exclusionary, inflammatory bowel disease (ie, Crohn's disease, ulcerative colitis except if fully excised, or prior gastric bypass surgery.\n* Exclusion related to treatment compliance: evidence of inadequate dialysis, unwillingness or inability to comply with study procedures, a history of noncompliance with medical treatments, or ongoing alcohol or illicit substance abuse.\n* Current or recent participation in research study involving an experimental agent \\\u003C 3 months of Screening.\n* Pregnant, breastfeeding, or unwillingness to practice adequate contraception during the study and for 5 months after the last dose of investigational product.\n* The presence of any condition that in the opinion of the Investigator would (1) compromise the safety of the participant in case of participation in the study, (2) compromise the quality of the data, and \u002F or (3) limit the life expectancy of the participant to \\\u003C 1 year.\n* The Sponsor determines that the participant is no longer needed for participation in study.",{"count":214,"type":20},24,[216],"PHASE1","This is a phase 1b, partial-blind (Sponsor unblinded), randomized, multicenter, placebo-controlled study. The primary objective of this study is to evaluate the pharmacokinetics (PK) of CSL300 after single and multiple doses in Chinese participants with end stage kidney disease (ESKD) undergoing dialysis.",[113],[220,221,222,223,224,225,226],"High-sensitivity C-reactive protein","High-density lipoprotein cholesterol","Interleukin-6","Humanized Monoclonal antibody","Atherosclerotic cardiovascular disease","Diabetes mellitus","Systemic inflammation","2026-05-26",{"date":173,"type":35},{"date":230,"type":20},"2026-06-30",{"date":232,"type":20},"2027-11-30",{"name":41,"class":42},{"id":235,"slug":236,"hasResults":11,"nctId":237,"briefTitle":238,"officialTitle":239,"acronym":4,"eligibilityCriteria":240,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":241,"enrollmentInfo":242,"targetDuration":4,"studyType":21,"phases":244,"briefSummary":245,"conditions":246,"keywords":250,"overallStatus":90,"whyStopped":4,"lastUpdateSubmitDate":253,"lastUpdatePostDateStruct":254,"startDateStruct":256,"completionDateStruct":258,"leadSponsor":260,"locationsCount":4},"100618051","phase-4-phase-4-double-blind-study-evaluating-the-response-on-computed-tomography-ct-lung-density-decline-rates-of-respreeza--zemaira-weekly-for-3-years-in-adults-with-alpha1-antitrypsin-deficiency-aatd-100618051","NCT07326592","Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza \u002F Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD)","A Phase 4, Multicenter, Double-blind, Study to Investigate the Efficacy, Safety, and Tolerability of 3 Active Doses of Respreeza® \u002F Zemaira® Weekly Intravenous Infusions Administered Over 3 Years as Longterm Maintenance Therapy in Adult Subjects With Emphysema Related to Alpha1 Antitrypsin Deficiency","Inclusion Criteria:\n\n* • Age greater than or equal to (\\>=) 18 and less than or equal to (\\\u003C=) 65 years at the time of providing written informed consent.\n* • Confirmed diagnosis of emphysema related to AATD with either the PiZZ, PiZ(null), or Pi(null\u002Fnull) genotype with documented serum AAT levels less than (\\\u003C) 11 micrometer (μM) (or \\\u003C 50 mg\u002FdL \\[milligram\u002Fdeciliter\\]) at any time before the first administration of CE1226 on Day 1 (Baseline).\n\nExclusion Criteria:\n\n* • Participants should not have acute illness or pulmonary exacerbation within 6 weeks before the first administration of CE1226 on Day 1 (Baseline).\n* • Participants should not have previously received gene therapy for AATD at any point.\n* • Participants with liver disease secondary to AATD.","65 Years",{"count":243,"type":20},270,[193],"This is a multicenter, parallel-group, double-blind, randomized phase 4 study designed to identify the optimal dose of CE1226 (2 active doses) to slow disease progression as assessed by reduced rates of annual lung density decline in alpha-1 antitrypsin (AAT) deficient participants over 3 years as compared with the marketed dose 60 milligrams per kilogram (mg\u002Fkg).",[247,248,249],"Alpha1 Antitrypsin Deficiency","Alpha1-Proteinase Inhibitor Deficiency","Emphysema",[251,252],"Progressive Emphysema","Chronic Pulmonary Disease","2026-05-14",{"date":255,"type":35},"2026-05-15",{"date":257,"type":20},"2026-07-20",{"date":259,"type":20},"2033-09-15",{"name":41,"class":42},{"id":262,"slug":263,"hasResults":11,"nctId":264,"briefTitle":265,"officialTitle":266,"acronym":4,"eligibilityCriteria":267,"healthyVolunteers":11,"sex":16,"minAge":161,"maxAge":4,"enrollmentInfo":268,"targetDuration":4,"studyType":21,"phases":270,"briefSummary":271,"conditions":272,"keywords":274,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":281,"lastUpdatePostDateStruct":282,"startDateStruct":283,"completionDateStruct":285,"leadSponsor":287,"locationsCount":288},"100569869","phase-2-safety-efficacy-and-pharmacokinetics-of-csl889-in-adults-and-adolescents-with-sickle-cell-disease-during-vaso-occlusive-crisis-100569869","NCT06699849","Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis","A Phase 2, Multicenter, Randomized, Multiple-Dose, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis","Inclusion Criteria:\n\n* At the time of informed consent:\n\n  * 18 years of age (adults); or\n  * 12 to less than (\\\u003C) 18 years of age (adolescents, where approved and when enrollment for adolescents has been opened by the sponsor, with the endorsement of the Independent Data Monitoring Committee \\[IDMC\\])\n* Diagnosed with SCD (any genotype).\n* Presented at the study site with a new acute VOC necessitating treatment with parenteral opioids.\n\nExclusion Criteria:\n\n* VOC pain onset greater than (\\>) 72 hours before administration of first parenteral opioid.\n* Must not have a history of \\> 5 VOCs requiring hospital admission in the past 6 months; or signs and \u002F or symptoms of ACS; or new neurological symptoms suggestive of acute stroke or transient ischemic attack; or any stage (acute kidney injury) AKI; or been discharged from inpatient hospital admission for VOC or other vaso-occlusive event within 14 days before the current presentation.\n* Serum hemoglobin \\\u003C 6 g\u002FdL, serum ferritin ≥ 2000 ng\u002FmL, receiving an approved medication for SCD that has not been on a stable, well-tolerated regimen, currently taking methadone or buprenorphine.",{"count":269,"type":20},70,[23],"This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to evaluate the safety and tolerability of CSL889 in study participants, and to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD.",[273],"Sickle Cell Disease Vaso-occlusive Crisis",[275,276,277,278,279,280],"Sickle cell disease","Acute kidney injury","Pharmacokinetics","Acute chest syndrome","Vaso-occlusive crisis","Hemopexin","2026-05-13",{"date":253,"type":35},{"date":284,"type":35},"2025-08-07",{"date":286,"type":20},"2027-10-22",{"name":41,"class":42},19,{"id":290,"slug":291,"hasResults":11,"nctId":292,"briefTitle":293,"officialTitle":294,"acronym":4,"eligibilityCriteria":295,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":296,"targetDuration":4,"studyType":21,"phases":297,"briefSummary":298,"conditions":299,"keywords":301,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":305,"lastUpdatePostDateStruct":306,"startDateStruct":308,"completionDateStruct":310,"leadSponsor":312,"locationsCount":313},"100610190","phase-2-safety-of-anumigilimab-csl324-in-adults-with-sickle-cell-disease-scd-100610190","NCT07224360","Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)","Phase 2a, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease","Inclusion Criteria:\n\n* • Adults aged greater than or equal to (\\>=) 18 years on the day of signing the informed consent form.\n* • Confirmed diagnosis of SCD of any genotype.\n* • Experienced 1 to 12 VOCs requiring a visit to a medical facility and treatment with parenteral opioids or a parenteral nonsteroidal anti-inflammatory drug within the 12 months before Screening.\n* • HU Regimen:\n* a. On stable and well-tolerated Hydroxyurea (HU) regimen for at least 30 days before Screening.\n* or\n* b. HU was discontinued or refused (eg, due to concern of side effects or lack of effect).\n\nExclusion Criteria:\n\n* • Absolute neutrophil count less than (\\\u003C) 2.5 ×10\\^9 cells\u002FLitre at Screening or Baseline (Week 1 Day 1).\n* • If on SCD preventive medication, dose is not stable in the 30 days before Screening.",{"count":75,"type":20},[23],"This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD.\n\nThe primary aim of the study is to assess the safety of anumigilimab in participants with SCD. Participants will be treated for 64 weeks: for 12 weeks in the dose escalation period, where the dose will be escalated to each participant's individual MTD; and for 52 weeks at the MTD in the maintenance period.",[300],"Sickle Cell Disease",[302,303,304],"Anemia","Genetic Disorder","Vaso-occlusive Crises","2026-05-11",{"date":307,"type":35},"2026-05-12",{"date":309,"type":35},"2026-02-02",{"date":311,"type":20},"2028-06-29",{"name":41,"class":42},5,{"id":315,"slug":316,"hasResults":11,"nctId":317,"briefTitle":318,"officialTitle":319,"acronym":4,"eligibilityCriteria":320,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":321,"targetDuration":4,"studyType":21,"phases":322,"briefSummary":323,"conditions":324,"keywords":326,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":332,"lastUpdatePostDateStruct":333,"startDateStruct":334,"completionDateStruct":336,"leadSponsor":338,"locationsCount":339},"100600176","phase-3-efficacy-and-safety-of-4f-pcc-4-factor-prothrombin-complex-concentrate-in-adult-patients-undergoing-complex-cardiovascular-surgery-with-cardiopulmonary-bypass-cpb-100600176","NCT07094087","Efficacy and Safety of 4F-PCC (4-Factor Prothrombin Complex Concentrate) in Adult Patients Undergoing Complex Cardiovascular Surgery With Cardiopulmonary Bypass (CPB)","A Phase 3, Multicenter, Randomized, Open-label, Controlled Study to Investigate the Efficacy and Safety of 4-Factor Prothrombin Complex Concentrate in Adult Patients Undergoing Complex Cardiovascular Surgery With Cardiopulmonary Bypass","Inclusion Criteria:\n\n* Adult greater than or equal to (≥) 18 years and has provided written informed consent.\n* Undergoing elective complex cardiovascular surgery requiring CPB, including procedures of the thoracic aorta (with or without additional cardiac interventions), aortic valve replacement + coronary artery bypass graft (CABG), complex valve surgeries, mitral valve repair + CABG, and mitral valve replacement + CABG and reoperative CABG. Reoperative procedures are permitted. Excluded surgeries are as follows: heart transplantation, insertion or removal of ventricular assist devices (except for intra-aortic balloon pumps), and acute repair of thoracoabdominal aneurysms.\n* Coagulation factor replacement (ie, 4F-PCC or FFP) is ordered in the operating room for the management of bleeding, in accordance with accepted clinical standards. The following criteria must be met:\n* INR ≥ 1.6 (point-of-care INR testing by Hemochron ≥ 5 to 10 minutes after protamine infusion for heparin reversal). If a participant needs a second dose of protamine, a new INR measurement should be performed to confirm eligibility.\n* Significant microvascular hemorrhage (ie, not due to surgical complications), as defined by a BSS score of ≥ 2.\n\nExclusion Criteria:\n\n* Administration of any systemic hemostatic therapy, such as cryoprecipitate, platelets, FFP, PCC (eg, 4-factor \u002F 3-factor PCC \\[4F-PCC \u002F 3F-PCC\\]), Factor VIII (FVIII) inhibitor bypassing activity (FEIBA), recombinant activated Factor VIIa (rFVIIa), or other coagulation factor products, in the 24 hours before study surgery, except when FFP is added to the CPB circuit.",{"count":163,"type":20},[54],"This is a phase 3, multicenter, randomized, open-label, parallel-group, controlled study to assess the efficacy and safety of BE1116 compared with fresh frozen plasma (FFP) in adult participants undergoing complex cardiovascular surgery with CPB. The primary purpose of the study is to compare the efficacy of BE1116 and FFP in correcting coagulation factor deficiencies in bleeding participants undergoing complex cardiovascular surgery with CPB.",[325],"Complex Cardiovascular Surgery With Cardiopulmonary Bypass",[327,328,329,330,331],"Cardiopulmonary bypass","Coagulopathic Bleeding","Coagulation factor deficiency","Prothrombin complex concentrates","Fresh frozen plasma","2026-05-08",{"date":305,"type":35},{"date":335,"type":35},"2025-09-15",{"date":337,"type":20},"2026-12-17",{"name":41,"class":42},18,{"id":341,"slug":342,"hasResults":11,"nctId":343,"briefTitle":344,"officialTitle":345,"acronym":4,"eligibilityCriteria":346,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":347,"targetDuration":4,"studyType":21,"phases":349,"briefSummary":350,"conditions":351,"keywords":353,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":359,"lastUpdatePostDateStruct":360,"startDateStruct":362,"completionDateStruct":364,"leadSponsor":366,"locationsCount":367},"100563571","phase-3-phase-3-study-of-fibrinogen-concentrate-csl511-in-subjects-with-pseudomyxoma-peritonei-undergoing-cytoreductive-surgery-100563571","NCT06617897","Phase 3 Study of Fibrinogen Concentrate (CSL511) in Subjects With Pseudomyxoma Peritonei Undergoing Cytoreductive Surgery","A Phase 3, Single-center, Randomized, Controlled Clinical Study to Investigate the Efficacy of Fibrinogen Concentrate (CSL511) in Subjects With Pseudomyxoma Peritonei Undergoing Cytoreductive Surgery","Inclusion Criteria:\n\n* • Aged \\>= 18 years at the time of providing written informed consent.\n* • Diagnosis of PMP requiring CRS with HIPEC.\n* • Bleeding risk: Predicted intraoperative blood loss of \\>=2L, assessed within 60 and 100 mins after start of study surgery (assessment made before 2 L of blood is lost)\n\nExclusion Criteria:\n\n* • Confirmed or suspected congenital or acquired coagulation disorder or a prothrombotic disorder\n* • Myocardial infarction, acute coronary syndrome, or stroke within 2 months before study surgery.\n* • Known history of chronic hepatitis.\n* • Clopidogrel or ticagrelor administration within 5 days before study surgery.\n* • Prasugrel administration within 7 days before study surgery.\n* • Oral factor Xa inhibitor administration within 2 days before study surgery.\n* • Glycoprotein IIb \u002F IIIa antagonist administration within 24 hours before study surgery.\n* • Oral direct thrombin inhibitor administration within 3 days before study surgery.\n* • Vitamin K antagonists within 5 days before study surgery.",{"count":348,"type":20},90,[54],"This study is a phase 3, prospective, single center, randomized, open label, controlled, parallel arm, interventional study to investigate the efficacy and safety of CSL511, in participants undergoing cytoreductive surgery (CRS) with hyperthermic intraperitoneal chemotherapy (HIPEC) for pseudomyxoma peritonei (PMP) with predicted intraoperative blood loss of greater than or equal to (\\>=) 2 liter (L). Eligible participants will be randomized in a 1:1 ratio to 1 of 2 treatment arms, to receive CSL511 or cryoprecipitate.",[352],"Acquired Fibrinogen Deficiency",[354,355,356,357,358],"Fibrinogen deficiency","Pseudomyxoma peritonei","Cytoreductive surgery","Hyperthermic intraperitoneal chemotherapy","Blood coagulation disorder","2026-03-09",{"date":361,"type":35},"2026-03-11",{"date":363,"type":35},"2024-10-01",{"date":365,"type":20},"2027-10-29",{"name":41,"class":42},1,{"id":369,"slug":370,"hasResults":11,"nctId":371,"briefTitle":372,"officialTitle":373,"acronym":374,"eligibilityCriteria":375,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":376,"targetDuration":4,"studyType":164,"phases":4,"briefSummary":378,"conditions":379,"keywords":380,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":382,"lastUpdatePostDateStruct":383,"startDateStruct":385,"completionDateStruct":387,"leadSponsor":389,"locationsCount":390},"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":377,"type":20},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.",[57],[57,381],"HEMGENIX","2026-02-18",{"date":384,"type":35},"2026-02-19",{"date":386,"type":35},"2023-06-15",{"date":388,"type":20},"2043-08-01",{"name":41,"class":42},12,{"id":392,"slug":393,"hasResults":11,"nctId":394,"briefTitle":395,"officialTitle":396,"acronym":4,"eligibilityCriteria":397,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":398,"enrollmentInfo":399,"targetDuration":4,"studyType":21,"phases":401,"briefSummary":402,"conditions":403,"keywords":405,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":409,"lastUpdatePostDateStruct":410,"startDateStruct":412,"completionDateStruct":414,"leadSponsor":416,"locationsCount":417},"100596653","phase-2-dose-range-finding-efficacy-and-safety-study-of-nebulized-csl787-in-adults-with-non-cystic-fibrosis-bronchiectasis-ncfb-100596653","NCT07048262","Dose Range Finding, Efficacy, and Safety Study of Nebulized CSL787 in Adults With Non-cystic Fibrosis Bronchiectasis (NCFB)","A Phase 2b, Multicenter, Randomized, Double-blind, Parallel-group, Placebo-controlled, Dose Range Finding Study to Evaluate the Efficacy, Safety, and Tolerability of Nebulized CSL787 in Adults (18 to 85 Years) With Non-cystic Fibrosis Bronchiectasis","Inclusion Criteria:\n\n* Adult between the ages of 18 to 85 years\n* Primary diagnosis of NCFB confirmed by chest computed tomography (CT) scan, where bronchiectasis has been documented by a radiologist. Diagnosis in the medical records based on historical scans is acceptable if the chest CT scan confirming the participant's NCFB diagnosis was performed within 12 months before enrollment. Participants for whom no chest CT scan results are available within the previous 12 months will undergo a chest CT scan during the Screening Period\n* Exacerbation history within the previous 1 year defined as either 1 of the following:\n* \\>= 2 documented exacerbations requiring oral and\u002For intravenous (IV) antibiotic therapy to treat a pulmonary infection.\n\nOR\n\n* 1 documented exacerbation requiring oral and\u002For IV antibiotic therapy to treat a pulmonary infection and a St. George's Respiratory Questionnaire (SGRQ) Symptoms score of \\> 40 at Screening.\n* Note: Other medications to treat NCFB such as: oral macrolides, or dipeptidyl peptidase-1 (DPP-1) inhibitors are allowed, provided \\>= 1 historical exacerbation occurred while on the medication for \\>= 3 months at a stable dose.\n* Postbronchodilator percentage of the predicted normal forced expiratory volume in 1 second of expiration \\[FEV1% predicted\\] \\> 35% and forced expiratory volume in 1 second (FEV1) \\>= 1 liter (L) obtained in accordance with American Thoracic Society (ATS) \u002F European Respiratory Society (ERS) standards for spirometry during Screening and at Baseline.\n\nExclusion Criteria:\n\n* History of bronchospasm in response to inhaled therapies including inhaled antibiotics\n* Known or suspected hypersensitivity, or other severe reactions, to the investigational product (IP), to any excipients of the IP, or to other immunoglobulin.\n* Primary diagnosis of other pulmonary disorders, including chronic obstructive pulmonary disease (COPD) asthma or, diffuse panbronchiolitis (DPB), as determined by the investigator.\n* Pulmonary exacerbation requiring antibiotic therapy within the 4 weeks before Baseline.","85 Years",{"count":400,"type":20},450,[23],"This study is a phase 2b, multicenter, randomized, double-blind, placebo-controlled, parallel-group, dose range finding study designed to explore the efficacy, safety, and tolerability of 2 active treatment regimens of CSL787 (immunoglobulin G \\[IgG\\] inhalation solution) compared with placebo over a period of 6 to 12 months independent of the occurrence of pulmonary exacerbations.\n\nThe primary aim of the study is to characterize the overall effect of CSL787 as well as the dose response of 2 active treatment regimens of inhaled CSL787 administered to participants with NCFB toward prolonging the TTF exacerbation.",[404],"Non-cystic Fibrosis Bronchiectasis",[406,407,408],"Chronic respiratory disease","Inflammation","Chronic bacterial infection","2025-11-06",{"date":411,"type":35},"2025-11-10",{"date":413,"type":35},"2025-09-03",{"date":415,"type":20},"2028-03-28",{"name":41,"class":42},13,{"id":419,"slug":420,"hasResults":11,"nctId":421,"briefTitle":422,"officialTitle":423,"acronym":4,"eligibilityCriteria":424,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":425,"enrollmentInfo":4,"targetDuration":4,"studyType":426,"phases":4,"briefSummary":427,"conditions":428,"keywords":4,"overallStatus":430,"whyStopped":4,"lastUpdateSubmitDate":431,"lastUpdatePostDateStruct":432,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":434,"locationsCount":4},"100605203","post-study-access-of-csl312-garadacimab-for-pediatric-participants-with-hereditary-angioedema-who-have-completed-the-csl3123003-study-100605203","NCT07159464","Post Study Access of CSL312 (Garadacimab) for Pediatric Participants With Hereditary Angioedema Who Have Completed the CSL312_3003 Study","Post Study Access of CSL312 (Garadacimab) for the Routine Prevention of Hereditary Angioedema (HAE) Attacks in Pediatric Participants (2-11 Years Old) With HAE Who Have Completed the CSL312_3003 Study","Inclusion Criteria:\n\n* Completion of treatment period in study CSL312\\_3003 (NCT05819775)\n* The participant responded to CSL312 treatment with no or very limited number of HAE attacks during the CSL312\\_3003 study\n* The participant experienced no clinically significant adverse effects associated with CSL312 treatment\n* In the opinion of the treating physician, the participant continues to receive benefit from CSL312\n* There is no other suitable alternative prophylactic treatment available at the time of consenting into the Post Study Access program\n\nExclusion Criteria:\n\n* In the opinion of the treating physician, participant may not be compliant with the Protocol requirements\n* Participant is 12 years or older at the time of consent\n* In the opinion of the treating physician, other study medication of prophylaxis treatment of HAE may benefit the participant more than continuing treatment with CSL312\n* Participant who is pregnant, breastfeeding, or not willing to cease breastfeeding","11 Years","EXPANDED_ACCESS","This protocol for post study access allows pediatric participants (2-11 years old at the time of consent) with HAE who have completed study CSL312\\_3003 (NCT05819775) to continue treatment with CSL312 for routine prevention of HAE attacks. The continuing treatment with the study product will be administered under a Post Study Access program in accordance with the applicable laws and regulations, to be dictated by CSL Behring (Sponsor) and approved by the appropriate local\u002Fcentral Ethics Committees and all other competent authorities required by law, as applicable.",[429],"Hereditary Angioedema (HAE)","AVAILABLE","2025-08-28",{"date":433,"type":35},"2025-09-08",{"name":41,"class":42},{"id":436,"slug":437,"hasResults":11,"nctId":438,"briefTitle":439,"officialTitle":439,"acronym":440,"eligibilityCriteria":441,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":442,"targetDuration":4,"studyType":164,"phases":4,"briefSummary":444,"conditions":445,"keywords":447,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":452,"lastUpdatePostDateStruct":453,"startDateStruct":455,"completionDateStruct":457,"leadSponsor":459,"locationsCount":66},"100414139","hizentra-in-inflammatory-neuropathies---phenix-study-100414139","NCT04672733","Hizentra® in Inflammatory Neuropathies - pHeNIx Study","pHeNIx","Inclusion Criteria:\n\n* Adult patient (aged ≥18 years)\n* Patients suffering from CIDP according to EAN\u002FPNS 2021 criteria\n* Planned switch from IVIg to Hizentra®\n* Patient treated with at least 3 courses of IV immunoglobulin and deemed by the investigator to be dependent on immunoglobulins\n* Patient deemed to be stable, with no change in their treatment for the disease during the 3 months prior to inclusion\n* Patients who have a smartphone, a tablet or a computer\n* Patients who have been informed verbally and in writing of the purposes of the study\n\nExclusion Criteria:\n\n* Concomitant participation in an interventional clinical study",{"count":443,"type":20},100,"The pHeNIx study, a national multicentre prospective non-interventional study, should help to describe the conditions of use for Hizentra® and the methods for switching from the IV to SC route in everyday practice, together with the tolerability and efficacy of treatment, which is monitored using a patient application (PRO: Patient-Reported Outcomes).",[446],"Chronic Inflammatory Demyelinating Polyneuropathy",[448,449,450,451],"CIDP","IgPro20","Hizentra","Subcutaneous Immunoglobulins","2025-01-06",{"date":454,"type":35},"2025-01-07",{"date":456,"type":35},"2022-06-10",{"date":458,"type":20},"2027-12",{"name":41,"class":42},""]