Ignite Creation Date:
2024-10-26 @ 3:36 PM
Last Modification Date:
2024-10-26 @ 3:36 PM
Study NCT ID:
NCT06533813
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-07-19
Brief Title:
Clinical Epidemiology in Contemporary Patients With Myelofibrosis
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Clinical Epidemiology in Contemporary Patients With Myelofibrosis ERNEST-3 A European LeukemiaNet ELN Observational Study
Status:
NOT_YET_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:
ERNEST-3
Brief Summary:
Multicenter retrospective and prospective European observational study At each site all consecutive patients with a 2016- or 2022 World Health Organization WHO confirmed diagnosis of myelofibrosis MF established from 01012018 to 31122027 will be enrolled into the study Yearly follow-up updates will be scheduled until the end of data collection on 31122028 or until the last available patient visit whichever comes first At least 1 year of follow-up will be ensured from the last patient enrolled
Detailed Description:
Myeloproliferative neoplasms MPNs are rare bone marrow disorders characterized by clonal proliferation of hematopoietic cell lineages and include polycythemia vera PV essential thrombocythemia ET and myelofibrosis MF MF has worse prognosis with main causes of death including acute leukemia transformation comorbid conditions and consequences of cytopenia MF is characterized by progressive anemia bone marrow fibrosis and extramedullary hematopoiesis with splenomegaly Moreover the disease is associated with a heavy symptom burden including night sweats fever bone pain and weight loss and worsening the quality of life
On the beginning of 2013 the European Registry for Myeloproliferative Neoplasms ERNEST observational study was launched and approved by several IRBs of European hematological centers The study focused on overt Primary PMF and Secondary myelofibrosis SMF ie post-Essential Thrombocythemia myelofibrosis post-ET MF and post-polycythemia post-PV MF and aimed at describing the clinical epidemiology of large series of patients observed in clinical practice This research was justified as the landscape of both pathophysiological and clinical knowledge in MPNs was rapidly evolving prompting to revise diagnostic criteria prognostication and therapy recommendations
ERNEST retrospectively enrolled 1292 patients in whom the proposed prognostic models were confirmed to differentiate treatments in clinical practice while ERNEST-2 reported results on critical events observed in 1010 of these cases during a median follow-up period of 54 years45 The two studies closed in December 2022
In the last decade new diagnostic and prognostic findings have been accumulated and the availability of new approved drugs based on results of several new clinical trials influenced the therapy decision making in the real-world clinical practice Therefore the continuation of observational studies in present ERNEST-3 on large multicenter case series of patients with MF is timely and might refine the results of clinical trials
The purpose of this study observational retrospectiveprospective study is to gain information on MF associated cytopenias that represent a significant challenge in the contemporary patients with MF Currently there are few agents aimed at treating cytopenic MF including immunomodulatory drugs hypomethylating agents and JAK inhibitors such as momelotinib and pacritinib and development of new agents specifically tailored to this patient population remains an unmet need Therefore this study can provide data on these patients focusing on clinical status quality of life comorbidities and treatment results over time
On the beginning of 2013 the European Registry for Myeloproliferative Neoplasms ERNEST observational study was launched and approved by several IRBs of European hematological centers The study focused on overt Primary PMF and Secondary myelofibrosis SMF ie post-Essential Thrombocythemia myelofibrosis post-ET MF and post-polycythemia post-PV MF and aimed at describing the clinical epidemiology of large series of patients observed in clinical practice This research was justified as the landscape of both pathophysiological and clinical knowledge in MPNs was rapidly evolving prompting to revise diagnostic criteria prognostication and therapy recommendations
ERNEST retrospectively enrolled 1292 patients in whom the proposed prognostic models were confirmed to differentiate treatments in clinical practice while ERNEST-2 reported results on critical events observed in 1010 of these cases during a median follow-up period of 54 years45 The two studies closed in December 2022
In the last decade new diagnostic and prognostic findings have been accumulated and the availability of new approved drugs based on results of several new clinical trials influenced the therapy decision making in the real-world clinical practice Therefore the continuation of observational studies in present ERNEST-3 on large multicenter case series of patients with MF is timely and might refine the results of clinical trials
The purpose of this study observational retrospectiveprospective study is to gain information on MF associated cytopenias that represent a significant challenge in the contemporary patients with MF Currently there are few agents aimed at treating cytopenic MF including immunomodulatory drugs hypomethylating agents and JAK inhibitors such as momelotinib and pacritinib and development of new agents specifically tailored to this patient population remains an unmet need Therefore this study can provide data on these patients focusing on clinical status quality of life comorbidities and treatment results over time
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