[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Swedish Orphan Biovitrum\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":160},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,43,77,99,129],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":23,"conditions":24,"keywords":26,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":4},"100652455","pacritinib-effectiveness-in-real-world-settings-100652455",false,"NCT07774455","Pacritinib Effectiveness in Real-world Settings","Pacritinib Effectiveness in Real-world Settings (PACER)","PACER","Inclusion Criteria:\n\n* Adult patients must be ≥18 years of age at the index date\n* Patients diagnosed with MF with platelet counts ≥50 x 109\u002FL at the time of treatment initiation with pacritinib. If multiple values are available within 30 days prior to initiating treatment with pacritinib will use the value closest to index date\n* Patients will be required to have ≥1 month of treatment with pacritinib and ≥6 months of observation from the start of pacritinib unless the patient died within 6 months of starting pacritinib\n* If peripheral blasts were evaluated prior to index, they must be \\\u003C10%. If not evaluated, patients will be included unless a healthcare provider has indicated in the medical record that there is a concern that the patient has transitioned to accelerated\u002Fblast phase disease at or prior to index\n* According to local regulations, waivers of consent will be sought for study patients from the appropriate regulatory authorities and\u002For the independent ethics committee (IEC)\u002Finstitutional review board (IRB). For patients not covered by waivers of consent, signed and dated informed consent provided by the patient, or the patient's legally authorized representative(s) for patients under the legal age (with patient assent, as applicable), should be obtained before any study-related activities are undertaken.\n\nExclusion Criteria:\n\n* Diagnosis of acute myeloid leukemia prior to index\n* Physician-concern that the patient has transitioned to accelerated\u002Fblast phase disease if peripheral blasts were not evaluated\n* Treated with 2 or more JAK inhibitors prior to initiating treatment with pacritinib\n* Treated with pacritinib in a clinical trial setting","ALL","18 Years",{"count":20,"type":21},60,"ESTIMATED","OBSERVATIONAL","This study aims to evaluate real-world treatment patterns and effectiveness of pacritinib, including hematologic and clinical outcomes, and survival through a site-based retrospective chart review of medical records of patients with MF.",[25],"Myelofibrosis (MF)",[27,28,29,30,15],"Myelofibrosis","Pacritinib","Sobi","Sobi.PACRIT-RWE-101","NOT_YET_RECRUITING","2026-08-18",{"date":34,"type":35},"2026-08-19","ACTUAL",{"date":37,"type":21},"2026-08-01",{"date":39,"type":21},"2027-01-27",{"name":41,"class":42},"Swedish Orphan Biovitrum","INDUSTRY",{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":50,"targetDuration":4,"studyType":52,"phases":53,"briefSummary":55,"conditions":56,"keywords":59,"overallStatus":67,"whyStopped":4,"lastUpdateSubmitDate":68,"lastUpdatePostDateStruct":69,"startDateStruct":71,"completionDateStruct":73,"leadSponsor":75,"locationsCount":76},"100650424","phase-4-real-world-effectiveness-and-safety-of-pegcetacoplan-in-patients-with-c3g-or-ic-mpg-a-multi-country-study-100650424","NCT07746895","Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country Study","Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy (C3G) or Primary Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN): A Multi-Country Study","Inclusion Criteria:\n\n* Have received or plan to receive pegcetacoplan for the treatment of C3G or primary IC-MPGN.\n* Provided signed and dated informed consent. For participants under the legal age signed and dated informed consent is provided by the participant's legally authorised representative. Assent will also be obtained from paediatric participants as required by local regulations.\n\nExclusion Criteria:\n\n* Receiving an investigational treatment for C3G or primary IC-MPGN at the time of pegcetacoplan initiation.\n* Initiated treatment with pegcetacoplan in an interventional study.",{"count":51,"type":21},150,"INTERVENTIONAL",[54],"PHASE4","The purpose of this study is to evaluate the effectiveness and safety of Pegcetacoplan in patients with C3G and primary IC-MPGN in the real-world setting. This study will also assess biomarkers not routinely measured in clinical practice. Results will support the long-term evaluation of the benefit-risk profile of pegcetacoplan in a broad patient population, informing clinical decision-making.",[57,58],"C3 Glomerulopathy (C3G)","Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)",[60,61,62,63,64,65,66],"C3 Glomerulopathy","Primary Immune Complex Membranoproliferative","Glomerulonephritis","Pegcetacoplan","Glomerulopathy","Nephrology","Rare disease","RECRUITING","2026-07-30",{"date":70,"type":35},"2026-08-05",{"date":72,"type":21},"2026-07-09",{"date":74,"type":21},"2032-01-31",{"name":41,"class":42},101,{"id":78,"slug":79,"hasResults":11,"nctId":80,"briefTitle":81,"officialTitle":82,"acronym":83,"eligibilityCriteria":84,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":85,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":87,"conditions":88,"keywords":4,"overallStatus":67,"whyStopped":4,"lastUpdateSubmitDate":90,"lastUpdatePostDateStruct":91,"startDateStruct":93,"completionDateStruct":95,"leadSponsor":97,"locationsCount":98},"100498916","a-real-world-effectiveness-study-of-pegcetacoplan-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-100498916","NCT05776472","A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","COMPLETE","Inclusion Criteria:\n\n* Patients ≥18 years of age with a documented PNH diagnosis.\n* Patient started routine treatment with pegcetacoplan for PNH up to 12 months before enrollment or prescribed pegcetacoplan at enrollment. Decision to initiate treatment shall be made by the treating physician and independently from the decision to include the patient in the study.\n* Patient is willing and able to provide written informed consent to participate in the study in a manner approved by the Institutional Review Board\u002FIndependent Ethics Committee and local regulations.\n\nExclusion Criteria:\n\n* Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to the start of the current pegcetacoplan treatment.\n* Initiated current treatment with pegcetacoplan in an interventional study.",{"count":86,"type":21},200,"This is a 36-month, long-term, multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH. Patients meeting the eligibility criteria will be enrolled in the study and followed prospectively for approximately 36 months. Patient data will be collected from start of pegcetacoplan treatment to end of follow-up. Retrospective data on pegcetacoplan will be captured from the time of pegcetacoplan treatment initiation. Pegcetacoplan treatment data will be collected for a minimum of approximately 36 months and up to a maximum of approximately 72 months, including retrospective period depending on when the patient started pegcetacoplan treatment. After pegcetacoplan treatment discontinuation, patients will remain in the study for 8 weeks to capture any AEs. The scope of the study is to collect both retrospective and prospective data. Baseline is defined as start of pegcetacoplan treatment. The main part of the study will be prospective,collecting data on effectiveness, safety (all AEs), patient- and clinician-reported outcomes and health care resource use.",[89],"Paroxysmal Nocturnal Hemoglobinuria","2026-06-30",{"date":92,"type":35},"2026-07-01",{"date":94,"type":35},"2023-06-26",{"date":96,"type":21},"2029-08-21",{"name":41,"class":42},87,{"id":100,"slug":101,"hasResults":11,"nctId":102,"briefTitle":103,"officialTitle":104,"acronym":4,"eligibilityCriteria":105,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":106,"targetDuration":4,"studyType":52,"phases":108,"briefSummary":109,"conditions":110,"keywords":112,"overallStatus":67,"whyStopped":4,"lastUpdateSubmitDate":121,"lastUpdatePostDateStruct":122,"startDateStruct":123,"completionDateStruct":125,"leadSponsor":127,"locationsCount":128},"100588394","phase-4-long-term-study-evaluating-joint-health-in-people-with-haemophilia-a-receiving-real-world-prophylactic-treatment-with-efanesoctocog-alfa-100588394","NCT06940830","Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa","A Low-interventional Study to Evaluate Long-term Effectiveness of Real-world Prophylactic Treatment With Efanesoctocog Alfa on Joint Health in People With Haemophilia A (ALTITUDE)","Inclusion Criteria:\n\n1. Male or female patient with a diagnosis of haemophilia A.\n2. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.\n3. Prescribed with efanesoctocog alfa prophylactic treatment within 6 months prior to study enrolment or at the latest at the enrolment visit, in accordance with local regulations.\n4. Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being prescribed with efanesoctocog alfa treatment.\n5. Have documented pre-study treatment data on haemophilia prophylaxis prescriptions and on treated bleeding episodes from the 12-months prior to being prescribed efanesoctocog alfa treatment and until enrolment into this study.\n6. Willing to adhere to the visit schedule and to undergo mandatory study assessment (HJHS, PROs).\n7. Willingness and the ability of the patient or their legally designated representative to document all bleeding episodes, including any treatments for those episodes.\n8. For HEAD-US subgroup: Accept to undergo annual ultrasound assessment on the index joints (ankles, elbows, knees).\n\nExclusion Criteria:\n\n1. Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A.\n2. Any positive FVIII inhibitor result (defined as inhibitor titre ≥0.6 Bethesda unit \\[BU\\]\u002FmL) from the medical records in connection to the switch to efanesoctocog alfa until the enrolment visit.\n3. Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), including for haemophilia prophylaxis, within 3 months prior to enrolment in this study.\n4. Patient not suitable for participation, whatever the reason, as judged by the Investigator, e.g., patient is not able or willing to perform the study assessments.",{"count":107,"type":21},250,[54],"The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.",[111],"Hemophilia A",[111,113,114,115,116,117,118,119,120],"Joint Health","Prophylaxis","Factor VIII Deficiency","Blood coagulation disorder","Efanesoctogoc alfa","Coagulation protein disorder","ALTUVOCT","ALTUVIIIO","2026-06-26",{"date":90,"type":35},{"date":124,"type":35},"2025-05-21",{"date":126,"type":21},"2028-12",{"name":41,"class":42},29,{"id":130,"slug":131,"hasResults":11,"nctId":132,"briefTitle":133,"officialTitle":134,"acronym":135,"eligibilityCriteria":136,"healthyVolunteers":11,"sex":137,"minAge":138,"maxAge":4,"enrollmentInfo":139,"targetDuration":4,"studyType":52,"phases":141,"briefSummary":143,"conditions":144,"keywords":146,"overallStatus":67,"whyStopped":4,"lastUpdateSubmitDate":152,"lastUpdatePostDateStruct":153,"startDateStruct":154,"completionDateStruct":156,"leadSponsor":158,"locationsCount":159},"100571174","phase-3-study-to-provide-continued-access-to-treatment-for-patients-completing-a-previous-trial-with-efanesoctocog-alfa-100571174","NCT06716814","Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa","A Multinational, Prospective, Open-label, Roll-over Study (LIBERTY) to Provide Post-trial Access to Treatment for Patients With Haemophilia A Who Have Completed a Previous Trial With Efanesoctocog Alfa","LIBERTY","Inclusion Criteria:\n\n* Capable of giving signed informed consent. Parents or legally designated representatives' consent is required for patients who are below 18 years of age or unable to give consent. Patients who are below 18 years of age may provide assent in addition to the parents'\u002Flegally designated representatives' consent, if appropriate.\n* Must have completed one of the required parent studies: Sobi.BIVV001-001, Sobi.BIVV001-003, LTS16294, or Sobi.BIVV001-004, and be receiving a clinical benefit from the efanesoctocog alfa treatment, as judged by the Investigator.\n* Willingness and ability of patient or their parent or legally designated representative to complete training in the use of the study patient diary and to complete the diary throughout the study.\n\nExclusion Criteria:\n\n* Positive inhibitor result, defined as ≥0.6 Bethesda units (BU)\u002FmL, present at the Baseline Visit.\n* Ongoing or planned participation in any interventional clinical study at the Baseline Visit.\n* Patient not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.","MALE","6 Years",{"count":140,"type":21},104,[142],"PHASE3","This is a multinational, prospective, open-label, roll-over study in patients with haemophilia A, ≥6 years of age, who have completed participation in any of the parental studies with efanesoctocog alfa; XTEND-ed study (LTS16294), FREEDOM study (Sobi.BIVV001-001), PK comparison study (Sobi.BIVV001-003) or SHINE study (Sobi.BIVV001-004). The aim of the study is to provide patients with continuous benefit from efanesoctocog alfa treatment and to further continue clinical monitoring for safety and efficacy until efanesoctocog alfa is commercially available in each patient's respective country (or until March 2027, whichever comes first).\n\nThe study starts with the Baseline Visit, which will be done in connection to the end of treatment at the EoT\u002FEoS visit (or equivalent) in the respective parent study. Subsequent study visits (on site or phone call) will be done approximately every 13 weeks until End of Treatment. An End of Study safety phone call will be done 14 (+7) days after the End of Treatment Visit.",[145],"Haemophilia A (Moderate or Severe)",[147,116,148,149,118,150,151,145],"Haemophilia A","Factor VIII","FVIII","Severe Haemophilia A","Efanesoctocog alfa","2026-06-25",{"date":121,"type":35},{"date":155,"type":35},"2025-03-06",{"date":157,"type":21},"2027-03",{"name":41,"class":42},21,""]