[{"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:":137},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,39,71,102],{"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":4,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":28,"startDateStruct":31,"completionDateStruct":33,"leadSponsor":35,"locationsCount":38},"100498916","a-real-world-effectiveness-study-of-pegcetacoplan-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-100498916",false,"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.","ALL","18 Years",{"count":20,"type":21},200,"ESTIMATED","OBSERVATIONAL","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.",[25],"Paroxysmal Nocturnal Hemoglobinuria","RECRUITING","2026-06-30",{"date":29,"type":30},"2026-07-01","ACTUAL",{"date":32,"type":30},"2023-06-26",{"date":34,"type":21},"2029-08-21",{"name":36,"class":37},"Swedish Orphan Biovitrum","INDUSTRY",87,{"id":40,"slug":41,"hasResults":11,"nctId":42,"briefTitle":43,"officialTitle":44,"acronym":4,"eligibilityCriteria":45,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":46,"targetDuration":4,"studyType":48,"phases":49,"briefSummary":51,"conditions":52,"keywords":54,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":63,"lastUpdatePostDateStruct":64,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":70},"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":47,"type":21},250,"INTERVENTIONAL",[50],"PHASE4","The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.",[53],"Hemophilia A",[53,55,56,57,58,59,60,61,62],"Joint Health","Prophylaxis","Factor VIII Deficiency","Blood coagulation disorder","Efanesoctogoc alfa","Coagulation protein disorder","ALTUVOCT","ALTUVIIIO","2026-06-26",{"date":27,"type":30},{"date":66,"type":30},"2025-05-21",{"date":68,"type":21},"2028-12",{"name":36,"class":37},29,{"id":72,"slug":73,"hasResults":11,"nctId":74,"briefTitle":75,"officialTitle":76,"acronym":77,"eligibilityCriteria":78,"healthyVolunteers":11,"sex":79,"minAge":80,"maxAge":4,"enrollmentInfo":81,"targetDuration":4,"studyType":48,"phases":83,"briefSummary":85,"conditions":86,"keywords":88,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":94,"lastUpdatePostDateStruct":95,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":101},"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":82,"type":21},104,[84],"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.",[87],"Haemophilia A (Moderate or Severe)",[89,58,90,91,60,92,93,87],"Haemophilia A","Factor VIII","FVIII","Severe Haemophilia A","Efanesoctocog alfa","2026-06-25",{"date":63,"type":30},{"date":97,"type":30},"2025-03-06",{"date":99,"type":21},"2027-03",{"name":36,"class":37},21,{"id":103,"slug":104,"hasResults":11,"nctId":105,"briefTitle":106,"officialTitle":107,"acronym":4,"eligibilityCriteria":108,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":109,"targetDuration":4,"studyType":48,"phases":111,"briefSummary":113,"conditions":114,"keywords":117,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":128,"lastUpdatePostDateStruct":129,"startDateStruct":131,"completionDateStruct":133,"leadSponsor":135,"locationsCount":136},"100576213","phase-2-a-study-to-assess-the-effectiveness-and-safety-of-pacritinib-in-patients-with-vexas-syndrome-paxis-100576213","NCT06782373","A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)","PAXIS: A Randomized, Double-blind, Placebo-controlled Dose-finding Phase 2 Study (Part 1) Followed by an Open-label Period (Part 2) to Assess the Efficacy and Safety of Pacritinib in Patients With VEXAS Syndrome","Key Inclusion Criteria:\n\n* Documented evidence of a pathogenic mutation at methionine-41 (M41) or neighboring splice site mutation (c.118-1, c.118-2) position in UBA1 mutation based on myeloid next-generation sequencing (NGS) droplet digital polymerase chain reaction (ddPCR), or Sanger sequencing in peripheral blood or bone marrow samples.\n* Current or documented evidence of past inflammatory involvement within 6 months prior to enrollment of at least one of the following organ systems by VEXAS syndrome: cutaneous (e.g., neutrophilic dermatosis, cutaneous vasculitis), vasculature (e.g., vasculitis), musculoskeletal (e.g., chondritis, arthritis), ocular (e.g., uveitis, scleritis), periorbital (e.g. periorbital edema), genitourinary (e.g., epididymitis), or pulmonary (e.g., alveolitis).\n* Receiving ongoing GC therapy (stable prednisone or prednisolone dose of 15-45 mg\u002Fday) leading up to enrollment.\n* Karnofsky Performance Status ≥50%\n* Adequate organ function, meeting all the following criteria within 30 days prior to enrollment:\n\n  1. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3 × upper limit of normal (ULN)\n  2. Total bilirubin ≤4 × ULN (≤8 × ULN in the setting of Gilbert's syndrome)\n  3. Creatinine clearance (CrCl) ≥30 mL\u002Fmin based on the Cockcroft-Gault formula\n  4. Absolute neutrophil count ≥500\u002FμL\n  5. Prothrombin time (PT) or international normalized ratio (INR) ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation)\n  6. Partial thromboplastic time (PTT) or activated PTT ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation)\n  7. Platelet count ≥25 × 10\\^9\u002FL (value must be obtained in the absence of platelet transfusion in the prior 7 days)\n  8. Peripheral blasts \\\u003C5%\n* QT corrected by the Fridericia method (QTcF) ≤450 msec in males or ≤470 msec in females. Participants with QRS prolongation \\>100 msec may enroll if their QTcF is ≤480 msec. If QTcF is thought to be prolonged due to a modifiable factor (e.g., medication \u002F electrolyte abnormality), QTcF may be reevaluated.\n* Women of child-bearing potential (WOCBP) must have a negative serum pregnancy test within 30 days prior to enrollment and a negative urine pregnancy test on Day 1 prior to randomization and dosing.\n* WOCBP and male participants must agree to use a highly effective method of contraception starting at the first dose of trial therapy through 30 days after the last dose of trial therapy.\n\nKey Exclusion Criteria\n\n* Prior allogenic hematopoietic stem cell transplant (allo-HSCT) or solid organ transplant (other than corneal).\n* Current use of systemic GCs for conditions other than VEXAS syndrome, which, in the opinion of the Investigator, would interfere with adherence to a GC taper regimen and\u002For assessment of efficacy.\n* More than one prior admission to an intensive care unit due to a VEXAS Syndrome flare within the prior 6 months.\n* Received ≥9 units of intensive red blood cell (RBC) transfusions in the 90 days prior to enrollment.\n* Known concurrent myelodysplastic syndrome (MDS) requiring antineoplastic treatment, or allo-HSCT, or known high-risk or very high-risk MDS based on the Revised International Prognostic Scoring System (IPSS-R). Participants with MDS who do not meet these criteria may enroll.\n* Malignancy within 1 year prior to enrollment with the exception of MDS (per exclusion criterion), curatively treated non-melanoma skin cancer, or curatively treated carcinoma in situ. Participants with pre-malignant hematologic conditions (e.g., monoclonal gammopathy of unknown significance \\[MGUS\\], clonal cytopenia of unknown significance) may enroll.\n* Exposure to hypomethylating agents (HMA) within 6 months prior to enrollment, or exposure to more than 6 cycles of HMAs at any time.\n* Exposure to non-GC anti-inflammatory therapy or hematologic support therapy within protocol defined timeframes prior to enrollment\n* Exposure to anti-platelet therapy with the exception of low-dose aspirin (≤100 mg daily) within 28 days prior to enrollment.\n* Known concomitant multiple myeloma, or serum M-protein ≥3 g\u002FdL, involved-to uninvolved free light chain (FLC) ratio ≥100, or involved FLC level ≥100 mg\u002FdL. Participants with MGUS may enroll.\n* Systemic treatment with a strong cytochrome P450 3A4 (CYP3A4) inhibitor or inducer within 5 half-lives prior to enrollment.\n* Significant recent bleeding history defined as National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) grade ≥2 within 3 months prior to enrollment, unless precipitated by an inciting event.\n* History of clinically significant cardiovascular disease, or clinically significant abnormalities in rhythm or conduction during Screening ECG, including:\n\n  1. QT corrected by the Fridericia method (QTcF) \\> 480 msec within 30 days prior to enrollment; if QTcF is thought to be prolonged due to a modifiable factor (e.g., medication \u002F electrolyte abnormality), QTcF may be re-evaluated\n  2. Severe cardiac event (CTCAE grade ≥3) within 3 months prior to enrollment\n  3. Heart failure resulting in limitations during ordinary activity.\n* Arterial or venous thrombotic or embolic events, including deep vein thrombosis, pulmonary embolism, and cerebrovascular accident (including transient ischemic attacks), within 60 days prior to enrollment.\n* Moderate or severe hepatic impairment that meets criteria for Child-Pugh Class B or C, or active viral hepatitis.\n* Uncontrolled human immunodeficiency virus (HIV) off antiretrovirals, or on antiretrovirals with detectable viral load.\n* Positive Quantiferon (or other interferon gamma release assay) during Screening.\n* Known history of disseminated mycobacterial infection.\n* Concurrent enrollment in another interventional trial, or treatment with an experimental therapy within 28 days or five half-lives prior to enrollment, whichever is longer.\n* Pregnant, intending to become pregnant during the trial, or currently breastfeeding\u002Flactating.\n* Participants with any acute, active infection requiring systemic antimicrobial treatment at the time of enrollment. Exceptions are made for prophylactic antibiotics or chronic antibiotic therapy for non-acute conditions.\n* Known hypersensitivity to pacritinib or any of the following inactive ingredients: microcrystalline cellulose, polyethylene glycol, and magnesium stearate.",{"count":110,"type":21},78,[112],"PHASE2","This trial is to assess the effectiveness and safety of pacritinib in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzyme, X-linked, autoinflammatory manifestations, and somatic) syndrome. 78 participants will be enrolled, randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. Randomization will be stratified by prescribed GC dose on the day of randomization.",[115,116],"VEXAS","VEXAS Syndrome",[118,119,120,121,122,123,124,125,126,127],"Vacuoles","E1 Ubiquitin-activating enzyme","X-linked","Autoinflammatory","Somatic syndrome","UBA1","Hematologic neoplasms","Myelodysplastic Syndromes","Pacritinib","Myeloid progenitors","2026-04-23",{"date":130,"type":30},"2026-04-24",{"date":132,"type":30},"2025-05-28",{"date":134,"type":21},"2028-05-22",{"name":36,"class":37},39,""]