Efanesoctocog alfa treatment in patients with congenital hemophilia A suffering from inflammation of joint lining
Key facts
- Where
- Berlin, Bonn, Dresden, Duisburg, Frankfurt am Main, Frankfurt Am Main, Giessen, Hamburg, Hanover, Homburg, Kiel, Leipzig és Regensburg, NémetországSee the 13 sites
- Phase
- 3. fázis
Where in Németország
- Vivantes Netzwerk fuer Gesundheit GmbHBerlin, NémetországTaking people
- Universitaetsklinikum Bonn AöRBonn, NémetországTaking people
- Technische Universitaet DresdenDresden, NémetországTaking people
- Gerinnungszentrum Rhein-Ruhr Aerztepartnerschaft Dr. med. Hannelore Rott Fachaerztin fuer Transfusionsmedizin Haemostaseologie Dr. med. Susan Halimeh Fachaerztin fuer Transfusionsmedizin Haemostaseologie Dr. med. Guenther Kappert Facharzt fuer Laboratoriumsmedizin HaemostaseologieDuisburg, NémetországTaking people
- HZRM Haemophilie-Zentrum Rhein Main GmbHFrankfurt am Main, NémetországTaking people
- Universitaetsklinikum Frankfurt AöRFrankfurt Am Main, NémetországTaking people
- Justus-Liebig-Universitaet GiessenGiessen, NémetországTaking people
- University Medical Center Hamburg-EppendorfHamburg, NémetországTaking people
- Medizinische Hochschule HannoverHanover, NémetországTaking people
- Universitaetsklinikum des Saarlandes AöRHomburg, NémetországTaking people
- Universitaetsklinikum Schleswig-Holstein AöRKiel, NémetországTaking people
- Universitaetsklinikum Leipzig AorLeipzig, NémetországTaking people
- Universitaetsklinikum Regensburg AöRRegensburg, NémetországTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- GWT-Tud GmbH
- Trial code
- EU 2025-523896-44-00
- Record updated
- 2026. október 27.
- Original description
The primary objective of the study is to assess the efficacy of Efanesoctocog alfa intensified replacement therapy (ERT) to reduce or resolve signs of synovitis in patients with congenital hemophilia A.Read full original descriptionShow less
Efanesoctocog alfa treatment in patients with synovitis in congenital hemophilia A: multicenter, randomized, open-label, phase 3 clinical trial
The primary objective of the study is to assess the efficacy of Efanesoctocog alfa intensified replacement therapy (ERT) to reduce or resolve signs of synovitis in patients with congenital hemophilia A.
Inclusion: Written informed consent must be obtained before any study-specific tests or procedures are performed; Male patients aged between 18 and 70 years at the first screening visit; Patients must be capable of giving informed consent and have the ability to understand and follow study-related instructions; Patients with severe to moderate congenital hemophilia A; Regular prophylaxis with licensed FVIII products (SHL-FVIII, EHL-FVIII or HS-FVIII) at its recommended regimen during the last 6 months; Synovitis of elbow, knee, and/or ankle joint (score ≥ 1 for synovial hypertrophy in HEAD-US) confirmed by Blinded ultrasound examination (BLUE) at the time of screening.
Exclusion: Acute hemarthrosis at time of screening (clinical or ultrasound detected) or within the past 4 weeks before screening (clinical); Use of anticoagulant or antiplatelet drugs at the time of screening; Participation in another clinical interventional trial in the 3 months before screening; Current factor VIII inhibitor ≥0.6 BU/mL; Current immune tolerance therapy; Radiosynovectomy (RSO) or orthopedic surgery during the past 3 months or planned within the next 12 months; Known bleeding disorder other than hemophilia A; Hypersensitivity to the active substance or to any of the excipients of the IMP; History of thrombosis, myocardial infarction, other clinically relevant vascular diseases, or atrial fibrillation, or combination of risk factors that would significantly increase the cardiovascular risk during ERT or standard of care (SOC); Life expectancy <12 months at the time of screening; Patients of Asian ethnicity; Close affiliation with the investigator (e.g. a close relative) or persons working at the study site, the sponsor or involved CRO; Criteria which in the opinion of the investigator preclude participation for scientific reasons, for reasons of compliance, or for reasons of the patient’s safety; Patient is in custody by virtue of an order issued either by the judicial or the administrative authorities; Planned intensification of FVIII prophylaxis above the allowed doses specified in the protocol (Table 1, section 5.1).