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ROP-ET: a prospective phase III trial investigating the efficacy and safety of ropeginterferon alfa-2b in essential thrombocythemia patients with limited treatment options
JJ. Kiladjian, FF. Marin, HK. Al-Ali, A. Alvarez-Larrán, E. Beggiato, M. Bieniaszewska, M. Breccia, V. Buxhofer-Ausch, O. Cerna, AM. Crisan, CD. Danaila, V. De Stefano, K. Döhner, V. Empson, J. Gora-Tybor, M. Griesshammer, S. Grosicki, P....
Jazyk angličtina Země Německo
Typ dokumentu protokol klinické studie, časopisecké články
NLK
ProQuest Central
od 1997-03-01
Medline Complete (EBSCOhost)
od 2000-01-01 do Před 1 rokem
Nursing & Allied Health Database (ProQuest)
od 1997-03-01
Health & Medicine (ProQuest)
od 1997-03-01
Springer Nature OA/Free Journals
od 1955-03-01
- MeSH
- dospělí MeSH
- esenciální trombocytemie * farmakoterapie MeSH
- interferon alfa-2 * terapeutické užití škodlivé účinky MeSH
- interferon alfa * terapeutické užití škodlivé účinky MeSH
- klinické zkoušky, fáze III jako téma MeSH
- lidé středního věku MeSH
- lidé MeSH
- multicentrické studie jako téma MeSH
- polyethylenglykoly * terapeutické užití škodlivé účinky aplikace a dávkování MeSH
- prospektivní studie MeSH
- rekombinantní proteiny * terapeutické užití škodlivé účinky aplikace a dávkování MeSH
- senioři MeSH
- výsledek terapie MeSH
- Check Tag
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- mužské pohlaví MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- protokol klinické studie MeSH
Interferon-based therapies, such as ropeginterferon alfa-2b have emerged as promising disease-modifying agents for myeloproliferative neoplasms (MPNs), including essential thrombocythemia (ET). Current ET treatments aim to normalize hematological parameters and reduce the thrombotic risk, but they do not modify the natural history of the disease and hence, have no impact on disease progression. Ropeginterferon alfa-2b (trade name BESREMi®), a novel, monopegylated interferon alfa-2b with an extended administration interval, has demonstrated a robust and sustained efficacy in polycythemia vera (PV) patients. Given the similarities in disease pathophysiology and treatment goals, ropeginterferon alfa-2b holds promise as a treatment option for ET. The ROP-ET trial is a prospective, multicenter, single-arm phase III study that includes patients with ET who are intolerant or resistant to, and/or are ineligible for current therapies, such as hydroxyurea (HU), anagrelide (ANA), busulfan (BUS) and pipobroman, leaving these patients with limited treatment options. The primary endpoint is a composite response of hematologic parameters and disease-related symptoms, according to modified European LeukemiaNet (ELN) criteria. Secondary endpoints include improvements in symptoms and quality of life, molecular response and the safety profile of ropeginterferon alfa-2b. Over a 3-year period the trial assesses longer term outcomes, particularly the effects on allele burden and clinical outcomes, such as disease-related symptoms, vascular events and disease progression. No prospective clinical trial data exist for ropeginterferon alfa-2b in the planned ET study population and this study will provide new findings that may contribute to advancing the treatment landscape for ET patients with limited alternatives. TRIAL REGISTRATION: EU Clinical Trials Register; EudraCT, 2023-505160-12-00; Registered on October 30, 2023.
Centre d'Investigations Cliniques AP HP Hôpital Saint Louis Paris 75010 France
Centre Léon Bérard Lyon France
CIC 1427 Inserm Université Paris Cité 75010 Paris France
Clinic of Internal Hematology University Hospital Kralovske Vinohrady Prague Czech Republic
Department of Clinical Hematology Regional Institute of Oncology Iasi Romania
Department of Hematology Careggi University Hospital Florence Italy
Department of Hematology Hospital Clínic of Barcelona Barcelona Spain
Department of Hematology Jagiellonian University Hospital Kraków Poland
Department of Hematology Medical University of Lodz Lodz Poland
Department of Hematology Policlinica de Diagnostic Rapid Brasov Brasov Romania
Department of Hematooncology Copernicus Memorial Hospital Lodz Poland
Department of Internal Medicine 3 University Hospital Ulm Ulm Germany
Department of Internal Medicine 5 Medical University Innsbruck Innsbruck Austria
Department of Internal Medicine Clinical Divison of Hematology Medical University Graz Graz Austria
Department of Translational and Precision Medicine Sapienza University of Rome Rome Italy
Fondazione Policlinico Gemelli IRCCS Section of Hematology Catholic University Rome Italy
Fundeni Clinical Institute Center for Hematology and Bone Marrow Transplantation București Romania
Hospital Universitario Ramón y Cajal Madrid Madrid Spain
Laboratory of Hematology Bordeaux University Hospital Bordeaux France
Medical Clinic 3 Hematology and Internistic Oncology University Hospital Mannheim Mannheim Germany
Medical Faculty Johannes Kepler University Linz Linz Austria
Medical University of Gdańsk Gdańsk Poland
Medical University of Silesia Katowice Poland
Universidad de Alcalá Madrid Spain
University Bordeaux INSERM BMC U1034 F 33600 Pessac France
University General Hospital Attikon Athens Greece
University Hospital Halle Krukenberg Cancer Center Halle Halle Germany
Citace poskytuje Crossref.org
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