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Polycythemia VeraNCT06506084

Observational Study on the Use of Ropeginterferon Alfa-2b in Polycythemia Vera (ROPEG-PV)

An observational study of Polycythemia Vera, run by FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS at 37 sites in 2 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

Polycythaemia vera (PV) is associated with a reduced quality of life, a high rate of vascular events, and an intrinsic risk of disease evolution. The results of several randomised trials for the treatment with new cytoreductive agents are now available, among which a new ropegylated formulation of interferon alfa-2b… Read more about this study

Who is it for?
Ages 18 to 100 years
Conditions the study is for
Polycythemia Vera
What is being tested?
Ropeginterferon alfa-2b
How many people
429 participants
Where
37 sitesin 2 countries
Study dates
8 Sep 2022to 31 Dec 2029
How long will I take part?
24 Months
Phase
Observational
Are there results?
Not yet: the study is under way

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
  • Adult, Older adult

Study population: The first time point to assess the primary outcome defined as the achievement of CHR is after 12 months of treatment with ropeginterferon alfa-2b, is reasonable to provide evidence of at least a non-inferiority in terms of CHR with this new drug. In this framework, a sample size is calculated to test 1-sample non-inferiority of a proportion. Setting the expected proportion p of CHR under ropeginterferon alfa-2b equals to the reference value (p0) obtained with ruxolitinib (i.e., p = p0 = 0.24), a sample size of 319 is needed to demonstrate that the proportion of CHR under ropeginterferon alfa-2b is not inferior to that obtained under ruxolitinib, allowing a margin of non-inferiority (delta) of 0.07 and assuming alpha and beta errors of 5% and 10%, respectively.

Who may be able to join

  • Patients diagnosed with Polycythemia Vera by WHO 2016
  • Patient aged ≥ 18 years old
  • Patients in need of cytoreductive treatments with ropeginterferon alfa-2b in first or later lines according to the reimbursability criteria defined by the Italian National Health System
  • Patients who have signed the written informed consent for study participation.

Who cannot join

  • Any contraindication for ropeginterferon alfa-2b according to the SmPC

Trial locations and contacts

37 sites in 2 countries. Contact the study team or a site near you to ask about taking part.

TIZIANO BARBUI, MD contact

+39 0352675134

tbarbui@fondazionefrom.it

FENILI contact

sperimentazioni@fondazionefrom.it

37 sites in 2 countries

Italy32 sites, 32 recruiting

  • A.S.O. SS. Antonio e Biagio e C.Arrigo di Alessandria

    Alessandria, 15121

    Monia Marchetti, monia.marchetti@ospedale.al.it

    Monia Marchetti

    Recruiting

  • Azienda Ospedaliera Universitaria Consorziale - Policlinico, U.O. Ematologia con Trapianto

    Bari

    Pietro Ventura, trials.ematba2@gmail.com

    Alessandra Ricco, MD

    Recruiting

  • SC Ematologia, ASST Papa Giovanni XXIII

    Bergamo, Lombardy, 24127

    Maria Luisa Ferrari, 035 2673681, mlferrari@fondazionefrom.it

    Federico Lussana, MD

    Recruiting

  • Policlinico S. Orsola - Malpighi, Unità di Ematologia

    Bologna

    Francesca Palandri, MD

    Recruiting

  • ASST-Spedali Civili

    Brescia, 25123

    Ilaria Accarino, ila.accarino91@gmail.com

    Rosa Daffini

    Recruiting

  • Ospedale Businco, S.C. Ematologia e CTMO

    Cagliari

    Daniela Perra, daniela.perra82@unica.it

    Olga Mulas, MD

    Recruiting

  • Azienda Ospedaliero - Universitaria "Policlinico Vittorio Emanuele" - PO Gaspare Rodolico, Dipartimento di Ematologia con Trapianto di midollo Osseo

    Catania

    Annalisa Santisi, annalisa_santisi@hotmail.it

    Arianna Sbriglione, sbriglionearianna@gmail.com

    Recruiting

  • Azienda Ospedaliera S. Croce e Carle di Cuneo- Divisione di Ematologia,

    Cuneo, 2100

    Laura Bertolotti, laura.bertolotti@libero.it

    Davide Rapezzi, Dr

    Recruiting

  • Arcispedale Sant'Anna Azienda Ospedaliero - Universitaria di Ferrara, Unità Operativa di Ematologia

    Ferrara

    Francesca Cura, crufnc@unife.it

    Silvia Bianchin, bncslv@unife.it

    Recruiting

  • Azienda Ospedaliera Universitaria Careggi, Divisione di Ematologia

    Florence

    Chiara Paoli, chiara.paoli@unifi.it

    Alessandro Maria Vannucchi, MD

    Recruiting

  • Azienda Ospedaliera Universitaria Policlinico "G. Martino", UOC Ematologia

    Messina

    Fabio Brunetto, protocolliematopolime@gmail.com

    Alessandro Allegra, MD

    Recruiting

  • Ospedale dell'Angelo, Dipartimento di Ematologia

    Mestre

    Simona Sannicolò, simona.sannicolo@aulss3.veneto.it

    Roberta Rocconi, MD

    Recruiting

  • Divisione Ematologia ASST, Grande Ospedale Metropolitano Niguarda

    Milan, Lombardy, 20162

    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

  • ASST MONZA Ospedale San Gerardo Clinica Ematologica

    Monza, 20900

    Alessia Picozzi, Dr, alessia.picozzihsg@gmail.com

    Elena Maria Elli, Dr

    Recruiting

  • Azienda Ospedaliera Universitaria Federico II di Napoli Divisione di Ematologia e Trapianti del Midollo

    Naples, 80131

    novypugliese@yahoo.it

    Fabrizio Pane, Prof

    Recruiting

  • Azienda Ospedaliero Universitaria Maggiore della Carità di Novara SCDU Ematologia

    Novara, 28100

    Deambrogi Clara, clara.deambrogi@med.uniupo.it

    Andrea Patriarca, Dr

    Recruiting

  • Clinica Medica I Azienda Ospedaliera di Padova

    Padova, Veneto, 35128

    Elisabetta Cosi, elisabetta.cosi@aopd.veneto.it

    Irene Bertozzi, MD

    Recruiting

  • Azienda Ospedaliera Universitaria Policlinico "P. Giaccone", Divisione di Ematologia

    Palermo

    Stefania Maniscalco Galletti, mgal.ste@gmail.com

    Marco Santoro, MD

    Recruiting

  • Divisione Ematologia, Fondazione IRCCS Policlinico San Matteo

    Pavia, Lombardy, 27100

    cto-oh@smatteo.pv.it

    Elisa Rumi, MD

    Recruiting

  • Azienda Ospedaliera Universitaria Pisana

    Pisa

    sara.galimberti@unipi.it

    Sara Galimberti, MD

    Recruiting

  • Azienda Ospedaliera San Eugenio - UOC Ematologia

    Roma

    Raoul Patricelli, raoul.patricelli@gmail.com

    Elisabetta Abruzzese, MD

    Recruiting

  • Fondazione Policlinico Universitario A. Gemelli - Università Cattolica del Sacro Cuore, UCSC Ematologia

    Roma

    Denise Soldati, denise.soldati@policlinicogemelli.it

    Giulia De Sanctis, giulia.desantis@policlinicogemelli.it

    Recruiting

  • Policlinico Umberto I, Dipartimento Ematologia, Oncologia e Dermatologia

    Roma

    Antonella Mosca, a.mosca@bce.uniroma1.it

    Ricci, r.ricci@bce.uniroma1.it

    Recruiting

  • Ospedale Casa Sollievo della Sofferenza Istituto di Ricovero e Cura a Carattere Scientifico, U.O. Ematologia

    San Giovanni Rotondo

    Daniela Valente, ematologia@operapadrepio.it

    Angelo Michele Carrella, MD

    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

    Recruiting

  • Ospedale Maggiore, SC Ematologia

    Trieste

    Maria Chiara Rossetti, mariachiara.rossetti@asugi.sanita.fvg.it

    Rossella Stella, MD

    Recruiting

  • Azienda Sanitaria Universitaria Integrata, Presidio Ospedaliero "Santa Maria della Misericordia", Clinica Ematologica

    Udine

    Laura Calò, laura.calo@asufc.sanita.fvg.it

    Mario Tiribelli, MD

    Recruiting

  • U.O. Ematologia, Ospedale di Circolo e Fondazione Macchi Varese

    Varese, Lombardy, 21100

    Roberta Mattarucchi, roberta.mattarucchi@asst-settelaghi.it

    Margherita Maffioli, MD

    Recruiting

  • Divisione Ematologia, Ospedale Borgo Roma

    Verona, Veneto, 37134

    massimiliano.bonifacio@univr.it

    Massimiliano Bonifacio, MD

    Recruiting

  • Divisione Ematologia, Ospedale San Bortolo

    Vicenza, Veneto

    MD, studiclinici.ematologia@aulss8.veneto.it

    Giuseppe Carli, MD

    Recruiting

Other trials in Italy

Poland5 sites, 5 recruiting

  • University Medical Center, Department of Hematology and Transplantation

    Gdansk

    Marta Sobas

    Maria Bieniaszewska, MD

    Recruiting

  • Pratia Onkologia, Department of Hematology and Cancer Prevention

    Katowice

    Marta Sobas

    Sebastian Grosicki, MD

    Recruiting

  • Jagiellonian University Hospital, Department of Haematology

    Krakow

    Marta Sobas

    Tomasz Sasha, MD

    Recruiting

  • Copernicus Hospital

    Lodz

    Marta Sobas

    Joanna Gora-Tybor, MD

    Recruiting

  • Medical University, Clinical Department of Haematology, Blood Neoplasms and Bone Marrow Transplantation

    Wroclaw

    Marta Sobas

    Marta Sobas, MD

    Recruiting

Other trials in Poland

About this study

Polycythaemia vera (PV) is associated with a reduced quality of life, a high rate of vascular events, and an intrinsic risk of disease evolution. The results of several randomised trials for the treatment with new cytoreductive agents are now available, among which a new ropegylated formulation of interferon alfa-2b (ropeginterferon alfa-2b) have been recently approved in Europe and USA [EMA (2019), FDA (2021) and AIFA (2022)]. The use of this drug in clinical practice is an opportunity for a prospective observational study in a rare disease such as PV; the aim is to evaluate its impact in the practical management of these patients.

Therefore, the main objectives of the present study are to determine:

(i) to what extent ropeginterferon alfa-2b can be prescribed and tolerated in patients with PV; (ii) the risk-benefit of ropeginterferon alfa-2b in patients with PV, followed-up in real-world clinical practice.

Read the detailed description

Classical Philadelphia-negative myeloproliferative neoplasms (Ph-neg MPNs) including polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF) are characterized by uncontrolled clonal proliferation of multipotent bone marrow progenitors, sustained by acquired mutations in JAK2, CALR and MPL genes.

Natural history of PV is marked by life threatening outcomes such as thrombosis, bleeding and clonal evolution towards myelofibrosis and acute myeloid leukemia. Treatment-relevant risk stratification is designed to estimate the likelihood of thrombotic complications, which is estimated to occur before or after diagnosis in 20-30% of patients according disease and patient-related risk factors. The cornerstone of treatment in PV includes scheduled phlebotomy, with a hematocrit (Hct) target of <45% and low-dose aspirin in all patients, regardless of risk category. There is currently broad consensus regarding the need for cytoreductive drugs in high-risk patients with PV identified by age >60 years and prior history of thrombosis.

The results of several andomized trials for the treatment of PV are now available, and, in addition to the standard drug hydroxyurea (HU), both a new ropegylated formulation of interferon alfa-2b3 and ruxolitinib4 are now available have been approved in Europe and US and European LeukemiaNet (ELN) investigators have recently provided recommendations for the use of these drugs in clinical practice in low-risk as well as high-risk patients.

After approval by EMA (2019) and FDA (2021), the drug (ROPEGINTERFERON ALFA-2B) was very recently approved and reimbursed by AIFA (2022) in some subgroups of patients with PV. The use of this drug in clinical practice is an opportunity for a prospective observational study in a rare disease such as PV; the aim is to evaluate its impact in the practical management of these patients, according to Determinazione AIFA 20 marzo 2008 about observational clinical studies, and Decreto Ministeriale 17 dicembre 2004 on non-profit studies.

It is not entirely known which is the percentage of patients who, after careful screening as required in good clinical practice, will fail the indications for concomitant clinical or laboratory abnormalities. Furthermore, the proportion of patients who discontinue the drug during follow-up for intolerance or other reasons is currently unknown and data on the benefit-risk ratio are limited.

Moreover, it should be noted that the haematological and clinical responses obtained in clinical trials not always are replicated in the studies of the real-world clinical practice. In fact, daily management of PV patients does not require the same stringent enrollment and follow-up criteria as instead are necessary in clinical trials. Our proposal may also contribute to better implement the results following the recent guidelines, particularly in some subgroups of patients in which AIFA has established the use with reimbursement by Italian National Health System (NHS) (i.e., patients intolerant to HU, women of childbearing age who plan pregnancy and patients with history of skin cancer).

Being tested
Ropeginterferon alfa-2b
Medical subject headings
Polycythemia Vera

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Ropeginterferon alfa-2bDrug

    Also called Besremi

    Data will be collected at each visit during the observational study (total duration: 24 months). In accordance with routine clinical practice in this patient population, visits are expected to take place every 6 months.

    Patients who discontinue ropeginterferon alfa-2b treatment at any time will no longer be followed up.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Complete hematological remission (CHR)* after 1 and 2 years of treatment.

    *HCT lower than 45% without phlebotomies in the past 3 months; platelet count ≤ 400x109/L, WBC count <10 x109/L

    Measured: At baseline and during the follow up at 6/12/18/24 months per patient.

Secondary outcomes (7)

  1. Screening failure

    Percentage of patients who, after careful screening as required in good clinical practice, will fail the indications for concomitant clinical or laboratory abnormalities.

    Measured: At baseline and during the follow up at 6/12/18/24 months per patient.

  2. Complete hematological (CHR) and clinical remission (CR) after 1 and 2 years of treatment stratified by different eligibility categories for treatment indication.

    Secondary Outcomes for efficacy

    Measured: At baseline and during the follow up at 6/12/18/24 months per patient.

  3. Dose-response effect on CHR and CR

    Secondary Outcomes for efficacy

    Measured: At baseline and during the follow up at 6/12/18/24 months per patient.

  4. Frequency of phlebotomies

    Secondary Outcomes for efficacy

    Measured: At baseline and during the follow up at 6/12/18/24 months per patient.

  5. Change in spleen size

    Change in spleen size (from baseline) evaluated by palpation.

    Measured: At baseline and during the follow up at 6/12/18/24 months per patient.

  6. Incidence of any of the following: o arterial and venous thrombotic events; o hemorrhagic events; o disease related symptoms; o disease evolutions into myelofibrosis and acute leukemia; o secondary malignancies.

    Secondary Outcomes for efficacy

    Measured: At baseline and during the follow up at 6/12/18/24 months per patient.

  7. Incidence, causality and intensity of any adverse event occurring during study period (as defined by MedDRA code and graded according to Common Terminology Criteria for Adverse Events (CTCAE last version)).

    Secondary Outcomes for safety

    Measured: At baseline and during the follow up at 6/12/18/24 months per patient.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Jul 2024
  2. Study start8 Sep 2022
  3. Primary completion30 Sep 2026 (estimated)
  4. Study completion31 Dec 2029 (estimated)

Resources

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 only
Time perspective
Prospective
Enrolment
429 participants (estimated)
Target duration
24 Months
IDs, dates and publications
ClinicalTrials.gov ID
NCT06506084
Sponsor's protocol ID
ROPEG-PV
Status last verified
Jul 2026
First submitted
11 Jul 2024
Last updated
9 Jul 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

An observational study of Polycythemia Vera, run by FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS at 37 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 9 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 37 sites in Italy, Poland. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact TIZIANO BARBUI, MD (+39 0352675134, tbarbui@fondazionefrom.it), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Complete hematological remission (CHR)* after 1 and 2 years of treatment. (time frame: At baseline and during the follow up at 6/12/18/24 months per patient.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06506084.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jul 2026.

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