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

Last updated: January 31, 2025
Sponsor: FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS
Overall Status: Active - Recruiting

Phase

N/A

Condition

Red Blood Cell Disorders

Leukemia (Pediatric)

Bone Marrow Disorder

Treatment

Ropeginterferon alfa-2b

Clinical Study ID

NCT06506084
ROPEG-PV
  • Ages 18-100
  • All Genders

Study 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 (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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patients diagnosed with Polycythemia Vera by WHO 2016

  • Patient aged ≥ 18 years old

  • Patients in need of cytoreductive treatments with ropeginterferon alfa-2b in firstor later lines according to the reimbursability criteria defined by the ItalianNational Health System

  • Patients who have signed the written informed consent for study participation.

Exclusion

Exclusion Criteria:

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

Study Design

Total Participants: 319
Treatment Group(s): 1
Primary Treatment: Ropeginterferon alfa-2b
Phase:
Study Start date:
September 08, 2022
Estimated Completion Date:
December 31, 2026

Study 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).

Connect with a study center

  • UOC Ematologia, ASST Papa Giovanni XXIII

    Bergamo, Lombardia 24127
    Italy

    Active - Recruiting

  • Divisione Ematologia ASST, Grande Ospedale Metropolitano Niguarda

    Milano, Lombardia 20162
    Italy

    Active - Recruiting

  • Divisione Ematologia, Fondazione IRCCS Policlinico San Matteo

    Pavia, Lombardia 27100
    Italy

    Active - Recruiting

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

    Varese, Lombardia 21100
    Italy

    Active - Recruiting

  • Clinica Medica I Azienda Ospedaliera di Padova

    Padova, Veneto 35128
    Italy

    Active - Recruiting

  • Divisione Ematologia, Ospedale Borgo Roma

    Verona, Veneto 37134
    Italy

    Active - Recruiting

  • Divisione Ematologia, Ospedale San Bortolo

    Vicenza, Veneto
    Italy

    Active - Recruiting

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

    Alessandria, 15121
    Italy

    Active - Recruiting

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

    Bari,
    Italy

    Active - Recruiting

  • Policlinico S. Orsola - Malpighi, Unità di Ematologia

    Bologna,
    Italy

    Active - Recruiting

  • ASST-Spedali Civili

    Brescia, 25123
    Italy

    Active - Recruiting

  • Ospedale Businco, S.C. Ematologia e CTMO

    Cagliari,
    Italy

    Active - Recruiting

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

    Catania,
    Italy

    Active - Recruiting

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

    Cuneo, 2100
    Italy

    Active - Recruiting

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

    Ferrara,
    Italy

    Active - Recruiting

  • Azienda Ospedaliera Universitaria Careggi, Divisione di Ematologia

    Firenze,
    Italy

    Active - Recruiting

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

    Messina,
    Italy

    Active - Recruiting

  • Ospedale dell'Angelo, Dipartimento di Ematologia

    Mestre,
    Italy

    Active - Recruiting

  • Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Ematologia

    Milano,
    Italy

    Active - Recruiting

  • Ospedale San Raffaele, Unità Operativa di Ematologia e Trapianto Midollo Osseo

    Milano,
    Italy

    Active - Recruiting

  • ASST MONZA Ospedale San Gerardo Clinica Ematologica

    Monza, 20900
    Italy

    Active - Recruiting

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

    Napoli, 80131
    Italy

    Active - Recruiting

  • Azienda Ospedaliero Universitaria Maggiore della Carità di Novara SCDU Ematologia

    Novara, 28100
    Italy

    Active - Recruiting

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

    Palermo,
    Italy

    Active - Recruiting

  • Azienda Ospedaliera Universitaria Pisana

    Pisa,
    Italy

    Active - Recruiting

  • Azienda Ospedaliera San Eugenio - UOC Ematologia

    Roma,
    Italy

    Active - Recruiting

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

    Roma,
    Italy

    Active - Recruiting

  • Policlinico Umberto I, Dipartimento Ematologia, Oncologia e Dermatologia

    Roma,
    Italy

    Active - Recruiting

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

    San Giovanni Rotondo,
    Italy

    Active - Recruiting

  • A.O.U. Città della Salute e della Scienza di Torino - Ospedale Molinette- S.C. Ematologia U

    Torino, 10126
    Italy

    Active - Recruiting

  • Ospedale Maggiore, SC Ematologia

    Trieste,
    Italy

    Active - Recruiting

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

    Udine,
    Italy

    Active - Recruiting

  • University Medical Center, Department of Hematology and Transplantation

    Gdańsk,
    Poland

    Site Not Available

  • Pratia Onkologia, Department of Hematology and Cancer Prevention

    Katowice,
    Poland

    Site Not Available

  • Jagiellonian University Hospital, Department of Haematology

    Krakow,
    Poland

    Site Not Available

  • Copernicus Hospital

    Lodz,
    Poland

    Site Not Available

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

    Wrocław,
    Poland

    Site Not Available

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