Observational Study on the Use of Ropeginterferon Alfa-2b in Polycythemia Vera (ROPEG-PV)
1 other identifier
observational
429
2 countries
37
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Sep 2022
Longer than P75 for all trials
37 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 8, 2022
CompletedFirst Submitted
Initial submission to the registry
July 11, 2024
CompletedFirst Posted
Study publicly available on registry
July 17, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 30, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 31, 2029
July 9, 2026
July 1, 2026
4.1 years
July 11, 2024
July 8, 2026
Conditions
Outcome Measures
Primary Outcomes (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
At baseline and during the follow up at 6/12/18/24 months per patient.
Secondary Outcomes (7)
Screening failure
At baseline and during the follow up at 6/12/18/24 months per patient.
Complete hematological (CHR) and clinical remission (CR) after 1 and 2 years of treatment stratified by different eligibility categories for treatment indication.
At baseline and during the follow up at 6/12/18/24 months per patient.
Dose-response effect on CHR and CR
At baseline and during the follow up at 6/12/18/24 months per patient.
Frequency of phlebotomies
At baseline and during the follow up at 6/12/18/24 months per patient.
Change in spleen size
At baseline and during the follow up at 6/12/18/24 months per patient.
- +2 more secondary outcomes
Interventions
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.
Eligibility Criteria
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.
You may qualify if:
- 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.
You may not qualify if:
- Any contraindication for ropeginterferon alfa-2b according to the SmPC
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (37)
SC Ematologia, ASST Papa Giovanni XXIII
Bergamo, Lombardy, 24127, Italy
Divisione Ematologia ASST, Grande Ospedale Metropolitano Niguarda
Milan, Lombardy, 20162, Italy
Divisione Ematologia, Fondazione IRCCS Policlinico San Matteo
Pavia, Lombardy, 27100, Italy
U.O. Ematologia, Ospedale di Circolo e Fondazione Macchi Varese
Varese, Lombardy, 21100, Italy
Clinica Medica I Azienda Ospedaliera di Padova
Padova, Veneto, 35128, Italy
Divisione Ematologia, Ospedale Borgo Roma
Verona, Veneto, 37134, Italy
Divisione Ematologia, Ospedale San Bortolo
Vicenza, Veneto, Italy
A.S.O. SS. Antonio e Biagio e C.Arrigo di Alessandria
Alessandria, 15121, Italy
Azienda Ospedaliera Universitaria Consorziale - Policlinico, U.O. Ematologia con Trapianto
Bari, Italy
Policlinico S. Orsola - Malpighi, Unità di Ematologia
Bologna, Italy
ASST-Spedali Civili
Brescia, 25123, Italy
Ospedale Businco, S.C. Ematologia e CTMO
Cagliari, Italy
Azienda Ospedaliero - Universitaria "Policlinico Vittorio Emanuele" - PO Gaspare Rodolico, Dipartimento di Ematologia con Trapianto di midollo Osseo
Catania, Italy
Azienda Ospedaliera S. Croce e Carle di Cuneo- Divisione di Ematologia,
Cuneo, 2100, Italy
Arcispedale Sant'Anna Azienda Ospedaliero - Universitaria di Ferrara, Unità Operativa di Ematologia
Ferrara, Italy
Azienda Ospedaliera Universitaria Careggi, Divisione di Ematologia
Florence, Italy
Azienda Ospedaliera Universitaria Policlinico "G. Martino", UOC Ematologia
Messina, Italy
Ospedale dell'Angelo, Dipartimento di Ematologia
Mestre, Italy
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Ematologia
Milan, Italy
Ospedale San Raffaele, Unità Operativa di Ematologia e Trapianto Midollo Osseo
Milan, Italy
ASST MONZA Ospedale San Gerardo Clinica Ematologica
Monza, 20900, Italy
Azienda Ospedaliera Universitaria Federico II di Napoli Divisione di Ematologia e Trapianti del Midollo
Naples, 80131, Italy
Azienda Ospedaliero Universitaria Maggiore della Carità di Novara SCDU Ematologia
Novara, 28100, Italy
Azienda Ospedaliera Universitaria Policlinico "P. Giaccone", Divisione di Ematologia
Palermo, Italy
Azienda Ospedaliera Universitaria Pisana
Pisa, Italy
Azienda Ospedaliera San Eugenio - UOC Ematologia
Roma, Italy
Fondazione Policlinico Universitario A. Gemelli - Università Cattolica del Sacro Cuore, UCSC Ematologia
Roma, Italy
Policlinico Umberto I, Dipartimento Ematologia, Oncologia e Dermatologia
Roma, Italy
Ospedale Casa Sollievo della Sofferenza Istituto di Ricovero e Cura a Carattere Scientifico, U.O. Ematologia
San Giovanni Rotondo, Italy
A.O.U. Città della Salute e della Scienza di Torino - Ospedale Molinette- S.C. Ematologia U
Torino, 10126, Italy
Ospedale Maggiore, SC Ematologia
Trieste, Italy
Azienda Sanitaria Universitaria Integrata, Presidio Ospedaliero "Santa Maria della Misericordia", Clinica Ematologica
Udine, Italy
University Medical Center, Department of Hematology and Transplantation
Gdansk, Poland
Pratia Onkologia, Department of Hematology and Cancer Prevention
Katowice, Poland
Jagiellonian University Hospital, Department of Haematology
Krakow, Poland
Copernicus Hospital
Lodz, Poland
Medical University, Clinical Department of Haematology, Blood Neoplasms and Bone Marrow Transplantation
Wroclaw, Poland
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
TIZIANO BARBUI, MD
FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- PROSPECTIVE
- Target Duration
- 24 Months
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 11, 2024
First Posted
July 17, 2024
Study Start
September 8, 2022
Primary Completion (Estimated)
September 30, 2026
Study Completion (Estimated)
December 31, 2029
Last Updated
July 9, 2026
Record last verified: 2026-07