Ignite Creation Date:
2024-10-26 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 3:35 PM
Study NCT ID:
NCT06506084
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-07-11
Brief Title:
Observational Study on the Use of Ropeginterferon Alfa-2b in Polycythemia Vera ROPEG-PV
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Observational Study on the Use of Ropeginterferon Alfa-2b in Polycythemia Vera ROPEG-PV
Status:
RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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
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 ie patients intolerant to HU women of childbearing age who plan pregnancy and patients with history of skin cancer
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 ie patients intolerant to HU women of childbearing age who plan pregnancy and patients with history of skin cancer
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None