An observational study in Polycythemia Vera, sponsored by FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS. Recruiting at 37 sites in 2 countries. Open to participants aged 18 Years to 100 Years. Per ClinicalTrials.gov, last updated 2026-07-09.
Sponsored by FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS · Observational
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.
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).
278 studies on the registry are indexed under Polycythemia Vera; 53 are open to participants now.
This study's planned enrollment of 429 is above the median of 240 across 44 observational studies indexed under Polycythemia Vera.
Browse Polycythemia Vera studies →FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS is the lead sponsor of 20 studies on the registry; 7 are open to participants now.
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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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Also known as: Besremi
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
Time frame: At baseline and during the follow up at 6/12/18/24 months per patient.
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.
Time frame: 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.
Secondary Outcomes for efficacy
Time frame: At baseline and during the follow up at 6/12/18/24 months per patient.
Dose-response effect on CHR and CR
Secondary Outcomes for efficacy
Time frame: At baseline and during the follow up at 6/12/18/24 months per patient.
Frequency of phlebotomies
Secondary Outcomes for efficacy
Time frame: At baseline and during the follow up at 6/12/18/24 months per patient.
Change in spleen size
Change in spleen size (from baseline) evaluated by palpation.
Time frame: At baseline and during the follow up at 6/12/18/24 months per patient.
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
Time frame: At baseline and during the follow up at 6/12/18/24 months per patient.
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
Time frame: At baseline and during the follow up at 6/12/18/24 months per patient.
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FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS