[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hemophilia-a-patient\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hemophilia-a-patient":24},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":25,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100656777","efanesoctocog-prophylaxis-or-the-optimization-of-treatment-real-world-experience-of-taylorizing-clotting-therapy-in-hemophilia-a-100656777",false,"NCT07830693","Efanesoctocog Prophylaxis Or The Optimization Of Treatment: Real-World Experience Of Taylorizing Clotting Therapy In Hemophilia A","Historical Prospective Study Evaluating the Impact of the Marketing of ALTUVOCT® (Efanesoctocog Alfa) on the Prophylactic Outpatient Management of Severe and Oderate Hemophilia A (FVIII:C \u003C 3 IU\u002FdL)","E-PROTECT","Inclusion Criteria:\n\n* Conduct of research: patients followed up at one of the eight investigator centers of the BERHLINGO network\n* Pathology: severe HA (FVIII:C \\\u003C1 IU\u002FdL) or moderate HA (1 IU\u002FdL \\\u003C FVIII:C \\\u003C 3 IU\u002FdL), without inhibitor \u002F with history of inhibitor (inhibitor rate \\\u003C 0.6 Bethesda unit per milliliter (BU\u002FmL))\n* Prophylactic Treatment with FVIII or emicizumab since at least March 1, 2025 -\n* Sex: male or female\n* Age: patients of all age\n* Consent: patient who did not object to their participation in the E-PROTECT study\n\nExclusion Criteria:\n\n* Patients under guardianship","ALL",{"count":19,"type":20},553,"ESTIMATED","OBSERVATIONAL","Prophylactic management of severe Hemophilia A (HA) without inhibitors has historically relied on frequent intravenous infusions of standard or EHL FVIII concentrates or on subcutaneous emicizumab administered weekly to monthly intervals , yet both approaches maintain a notable treatment burden and variable factor utilization.\n\nEfanesoctocog alfa (ALTUVOCT®, Swedish Orphan Biovitrum SOBI) was engineered as a fusion of FVIII, von Willebrand factor (VWF) binding domains, and XTEN polypeptides to decouple FVIII from endogenous VWF and extend its circulatory half-life beyond that of existing EHL concentrates.\n\nIn the pivotal XTEND-1 trial (NCT04161495) of 159 patients aged ≥ 12 years, once-weekly prophylaxis (50 IU\u002Fkg) yielded a mean ABR of 0.70 episodes per patient-year versus approximately 3.0 episodes on prior regimens. The study ended with superior bleeding protection, with FVIII activity above 40 IU\u002FdL for the majority of each week and of 15 IU\u002FdL at day 7. In children under 12, XTEND-Kids phase 3 data (n = 74) demonstrated comparable bleeding control; the average ABR for patients per year was 0.70. The XTEND-ed long-term extension (3-year data) assessed maintaining low ABRs, stable FVIII levels, and consistent tolerability. Efanesoctocog alfa was also well-tolerated in all trials, with adverse events similar to those of other FVIII products and no inhibitor development reported as a result of treatment. The analysis of pharmacokinetics indicated an extended half-life that supports once-weekly dosing and maintains FVIII activity in the normal to near-normal range (\\> 40 IU\u002FdL) for the majority of each dosing interval.\n\nWhile regulatory and health-technology assessments recognize its favorable pharmacokinetics and hemostatic efficacy, they underscore the non-randomized, intra-patient design of these studies, the paucity of robust head-to-head comparisons with standard FVIII or emicizumab, and the absence of real-world QoL and consumption data. Despite the promising results, critical questions remain unanswered in routine clinical practice: efanesoctocog alfa is designed for once-weekly dosing but how does switching to efanesoctocog alfa alter real-world FVIII consumption, what clinical or psychosocial factors drive clinicians and patients to transition, and how do patients perceive efficacy, convenience, and treatment burden post-switch?\n\nThis study is designed to bridge this knowledge gap by quantifying pre- and post-switch FVIII utilization, systematically capturing switch-motivations through patient interviews, and applying validated patient-reported outcome measures to assess satisfaction and QoL impacts. Addressing these dimensions will not only inform personalized prophylaxis regimens and clarify resource utilization but also enrich pharmaco-economic models and guide future therapeutic innovations in HA management.\n\nIndividual profit - The switched patient must benefit from a lower injection frequency for the same clinical outcome (change of prescription); this could also favorably impact his quality of life. The possibility of changing the replacement therapy with FVIII or emicizumab to efanesoctocog alfa will be proposed to the patient during a routine consultation.\n\nThe Patient-Reported Outcome Measures (PROMs) will make it possible to properly appraise the patient's view. QoL questionnaires (PERQOLATEUR), Hemophilia Functional Ability Scoring Tool questionnaires (Hemo-FAST©) and treatment evaluation questionnaires will be presented (See Appendice 8), particularly during the shared decision-making consultation on the switch and the following routine consultation.\n\nGroup profit - This study will allow a better understanding of the use of health resources (quantification of FVIII consumption before and after switching to efanesoctocog alfa), to better understand the motivations of a person with HA to change treatment, and to evaluate in a real situation the impact on the patient's QoL of a new substitutive treatment.\n\nThe research has no risks and constraints because only questionnaires (part of routine care or without health data) are distributed to patients.",[24],"Hemophilia A Patient",[26,27,28,29],"Hemophilia A","factor VIII (FVIII) deficiency","efanesoctocog alfa","ALTUVOCT®","NOT_YET_RECRUITING","2026-09-22",{"date":33,"type":34},"2026-09-25","ACTUAL",{"date":36,"type":20},"2027-01-01",{"date":38,"type":20},"2028-12-31",{"name":40,"class":41},"Nantes University Hospital","OTHER",8]