MyelofibrosisERNEST-3NCT06533813
Clinical Epidemiology in Contemporary Patients With Myelofibrosis.
An observational study of Myelofibrosis, run by FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS at 27 sites in 6 countries.
Trial summary
Multicenter retrospective and prospective European observational study. At each site, all consecutive patients with a 2016- or 2022 World Health Organization (WHO) confirmed diagnosis of myelofibrosis (MF) established from 01/01/2018 to 31/12/2027 will be enrolled into the study. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Myelofibrosis
- How many people
- 617 participants
- Where
- 27 sitesin 6 countries
- Study dates
- 14 Oct 2024to 31 Dec 2028
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with a 2016- or 2022-WHO confirmed diagnosis of MF established from 01/01/2018 to 31/12/2027 will be enrolled into the study. Patients with primary and secondary to ET and PV myelofibrosis, annotated for genetic and histological features, in relation to the presence of baseline (i.e., at MF diagnosis) or treatment-related cytopenias (i.e., reduced count of blood cells manifesting as anemia, thrombocytopenia and/or neutropenia).
Who may be able to join
- Diagnosis of primary myelofibrosis (PMF) or secondary (i.e., post-ET/PV MF) myelofibrosis according to 2016- or 2022-WHO criteria ascertained between 01/01/2018 and 31/12/2027
- Age ≥ 18 years
- Signed informed consent where applicable, in line with current European General Data Protection Regulation (GDPR) directives
Who cannot join
- Diagnosis of early/prefibrotic primary myelofibrosis
- Concurrent participation to interventional clinical trials in MF
Trial locations and contacts
27 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
FENILI contact
27 sites in 6 countries
Italy18 sites, 18 recruiting
- Recruiting
A.S.O. SS. Antonio e Biagio e C.Arrigo, SC Ematologia
Alessandria, 15121
Monia Marchetti, monia.marchetti@ospedale.al.it
Monia Marchetti
- Recruiting
ASST Papa Giovanni XXIII, SC Ematologia
Bergamo
Maria Luisa Ferrari, mlferrari@fondazionefrom.it
Federico Lussana, MD
- Recruiting
Policlinico S. Orsola - Malpighi, Unità di Ematologia
Bologna
Valerio Michelucci, valeria.michelucci@studio.unibo.it
Francesca Palandri, MD
- Recruiting
Azienda Ospedaliero - Universitaria "Policlinico Vittorio Emanuele" - PO Gaspare Rodolico, Dipartimento di ematologia con Trapianto di midollo Osseo
Catania
Giuseppe Palumbo, MD
- Recruiting
AOU Careggi di Firenze, Divisione di Ematologia
Florence
Chiara Paoli, chiara.paoli@unifi.it
Alessandro M Vannucchi, MD
- Recruiting
ASST Grande Ospedale Metropolitano Niguarda, Divisione di Ematologia
Milan
Mattia La Monica, mattia.lamonica@ospedaleniguarda.it
Marianna Caramella, MD
- Recruiting
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Ematologia
Milan
Silvia Artuso, silvia.artuso@policlinico.mi.it
Alessandra Iurlo, MD
- Recruiting
Ospedale San Raffaele, Unità Operativa di Ematologia e Trapianto Midollo Osseo
Milan
Antonella Danese, danese.antonella@hsr.it
Daniele Sannipoli, MD
- Recruiting
Fondazione IRCCS San Gerardo dei Tintori
Monza
Alessia Picozzi, alessia.picozzihsg@gmail.com
Elena Elli, MD
- Recruiting
Azienda Ospedaliera Universitaria Federico II, Divisione di Ematologia
Naples
giadamc@gmail.com
novypugliese@yahoo.it
- Recruiting
Azienda Ospedaliero Universitaria Maggiore della Carità, SCDU Ematologia
Novara
Clara Deambrogi, clara.deambrogi@med.uniupo.it
Andrea Patriarca, MD
- Recruiting
Fondazione IRCCS Policlinico San Matteo, Divisione di Ematologia
Pavia
cto-oh@smatteo.pv.it
ELisa Rumi, MD
- Recruiting
Fondazione Policlinico Universitario A. Gemelli - Università Cattolica del Sacro Cuore, UCSC Ematologia
Roma
giulia.desantis@policlinicogemelli.it
denise.soldati@policlinicogemelli.it
- Recruiting
Policlinico Umberto I, Dipartimento Ematologia, Oncologia e Dermatologia
Roma
Antonella Mosca, a.mosca@bce.uniroma1.it
Massimo Breccia, MD
- Not yet recruiting
A.O.U. Città della Salute e della Scienza di Torino - Ospedale Molinette- S.C. Ematologia U
Torino, 10126
Francesca Pirillo, francescapirillo.ospedale@gmail.com
Giulia Benevolo, Dr
- Not yet recruiting
Ospedale Cardinale G. Panico
Tricase
Anna Mele, MD
- Recruiting
Azienda Ospedaliera Universitaria Integrata - Ospedale Borgo Roma, Unità di Ematologia
Verona
massimiliano.bonifacio@univr.it
Massimiliano Bonifacio, MD
- Recruiting
Azienda ULSS 8 Berica - Ospedale San Bortolo, Divisione di Ematologia
Vicenza
studiclinici.ematologia@aulss8.veneto.it
Giuseppe Carli, MD
Germany3 sites, 3 recruiting
- Not yet recruiting
University Hospital Halle Department of Hematology/Oncology
Halle
haifa.al-ali@uk-halle.de
Haifa Kathrin Al-Ali, MD
- Recruiting
University Medicine Hannover
Hanover
florian.heidel@uni-greifswald.de
Florian Heidel, MD
- Recruiting
University Clinic for Hematology, Oncology, Hemostaseology and Palliative Care Johannes Wesling Medical Center Minden UKRUB, University of Bochum
Minden
martin.griesshammer@muehlenkreiskliniken.de
Martin Griesshammer, MD
Poland2 sites, 2 recruiting
- Recruiting
Department of Hematology, University Hospital, Jagiellonian University Medical College
Krakow
weronika.lebowa@doctoral.uj.edu.pl
Weronika Lebowa, MD
- Recruiting
Department and Clinic of Haematology, Blood Neoplasms, and Bone Marrow Transplantation, Wroclaw Medical University
Wroclaw
marta.sobas@gmail.com
Marta Sobas, MD
Spain2 sites, 2 recruiting
- Recruiting
Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS) - Hospital Clinic
Barcelona
AALVAR@clinic.cat
Alberto Alvarez-Larran, MD
- Recruiting
Hematology and Hemotherapy Department, 12 de Octubre University Hospital
Madrid
gonzalo.carreno.gomeztarragona@gmail.com
Gonzalo Carreno Gomez-Tarragona, MD
Armenia1 site, 1 recruiting
- Recruiting
Hematology Center after Prof.R.H. Yeolyan
Yerevan
badikyanmaria@gmail.com
Maria Badikyan, MD
Romania1 site, 1 recruiting
- Recruiting
Department of Hematology and Bone Marrow Transplantation, Fundeni Clinical Institute
Bucharest
daniel_coriu@yahoo.com
Daniel Coriu
About this study
Official title: Clinical Epidemiology in Contemporary Patients With Myelofibrosis (ERNEST-3): A European LeukemiaNet (ELN) Observational Study
Multicenter retrospective and prospective European observational study. At each site, all consecutive patients with a 2016- or 2022 World Health Organization (WHO) confirmed diagnosis of myelofibrosis (MF) established from 01/01/2018 to 31/12/2027 will be enrolled into the study. Yearly follow-up updates will be scheduled until the end of data collection on 31/12/2028 or until the last available patient visit, whichever comes first. At least 1 year of follow-up will be ensured from the last patient enrolled.
Read the detailed description
Myeloproliferative neoplasms (MPNs) are rare bone marrow disorders characterized by clonal proliferation of hematopoietic cell lineages, and include polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF). MF has worse prognosis, with main causes of death including acute leukemia transformation, comorbid conditions, and consequences of cytopenia. MF is characterized by progressive anemia, bone marrow fibrosis, and extramedullary hematopoiesis with splenomegaly. Moreover, the disease is associated with a heavy symptom burden including night sweats, fever, bone pain, and weight loss and worsening the quality of life.
On the beginning of 2013 the European Registry for Myeloproliferative Neoplasms (ERNEST) observational study was launched and approved by several IRBs of European hematological centers. The study focused on overt Primary (PMF) and Secondary myelofibrosis (SMF; i.e., post-Essential Thrombocythemia myelofibrosis (post-ET MF) and post-polycythemia (post-PV MF)) and aimed at describing the clinical epidemiology of large series of patients observed in clinical practice. This research was justified as the landscape of both pathophysiological and clinical knowledge in MPNs was rapidly evolving, prompting to revise diagnostic criteria, prognostication and therapy recommendations.
ERNEST retrospectively enrolled 1292 patients in whom the proposed prognostic models were confirmed to differentiate treatments in clinical practice, while ERNEST-2 reported results on critical events observed in 1010 of these cases during a median follow-up period of 5.4 years.4,5 The two studies closed in December 2022.
In the last decade, new diagnostic and prognostic findings have been accumulated and the availability of new approved drugs, based on results of several new clinical trials, influenced the therapy decision making in the real-world clinical practice. Therefore, the continuation of observational studies in present ERNEST-3 on large multicenter case series of patients with MF is timely and might refine the results of clinical trials.
The purpose of this study observational retrospective/prospective study is to gain information on MF associated cytopenias that represent a significant challenge in the contemporary patients with MF. Currently, there are few agents aimed at treating cytopenic MF, including immunomodulatory drugs, hypomethylating agents, and JAK inhibitors such as momelotinib and pacritinib, and development of new agents specifically tailored to this patient population remains an unmet need. Therefore, this study can provide data on these patients, focusing on clinical status, quality of life, comorbidities, and treatment results over time.
- Conditions
- Myelofibrosis
- Medical subject headings
- Primary Myelofibrosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival according to the presence of cytopenias at diagnosis
Obtained by medical health records normally filled out in clinical practice
Measured: At diagnosis during the baseline
Secondary outcomes (8)
Frequency and types of cytopenias
Obtained by medical health records normally filled out in clinical practice
Measured: At baseline; 1 year follow-up.
Treatments response and duration (according to modified IWG-MRT and ELN 2013 criteria, overall and by presence of cytopenias
Obtained by medical health records normally filled out in clinical practice
Measured: At baseline; 1 year follow-up.
Incidence of major CV events (thrombosis and bleeding), overall and by presence of cytopenias and treatments received
Obtained by medical health records normally filled out in clinical practice
Measured: At baseline; 1 year follow-up.
Incidence of disease progressions (MF accelerated phase, MF blast phase/acute myeloid leukemia (AML)), overall and by presence of cytopenias and treatments received
Obtained by medical health records normally filled out in clinical practice
Measured: At baseline; 1 year follow-up.
Incidence of secondary neoplasia, overall, by site and by presence of cytopenias and treatments received
Obtained by medical health records normally filled out in clinical practice
Measured: At baseline; 1 year follow-up.
Incidence of infections, overall and by presence of cytopenias and treatments received
Obtained by medical health records normally filled out in clinical practice
Measured: At baseline; 1 year follow-up.
Incidence of adverse events (AEs) of interest, overall, by type, severity and treatments received
Obtained by medical health records normally filled out in clinical practice
Measured: At baseline; 1 year follow-up.
Incidence of death, overall, by cause and by presence of cytopenias and treatments received
Obtained by medical health records normally filled out in clinical practice
Measured: At baseline; 1 year follow-up.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2024
- Study start14 Oct 2024
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MyelofibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Retrospective
- Enrolment
- 617 participants (estimated)
Sponsor and investigators
- Lead sponsor
- FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS
- Sponsor type
- University, hospital or other
- Collaborators
- GlaxoSmithKline
- Responsible party
- Sponsor
- Investigators
- TIZIANO BARBUI, MD, Study director, FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06533813
- Sponsor's protocol ID
- ERNEST-3
- Acronym
- ERNEST-3
- Status last verified
- Jul 2026
- First submitted
- 19 Jul 2024
- Last updated
- 9 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No