Real World Evidence of Fedratinib Effectiveness in MF (REALFed)

Observational Study Protocol REALFed - Real World Evidence of Fedratinib Effectiveness in Myelofibrosis

This is a multicenter prospective and retrospective observational clinical study in patients with primary or post polycythemia vera or post essential thrombocythemia myelofibrosis to test the efficacy of fedratinib in the rea world. Participants will be managed according to the clinical practice of the participating Center. All Centers will be Italian Hematology Units belonging to the GIMEMA Organization in Italy.

Study Overview

• Status: Recruiting
• Conditions: Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis
• Intervention / Treatment: Drug: Fedratinib

Detailed Description

This is a multicenter prospective and retrospective observational clinical study with the aim of assessing the efficacy of fedratinib upon AIFA approval in patients with primary myelofibrosis or post polycythemia vera or post essential thrombocythemia myelofibrosis. Patients must meet current diagnostic criteria of MF, according to the WHO (World Health Organization) classification version 5th or the ICC (International Consensus Conference) either published in 2022 or post- polycythemia vera and post-essential thrombocythemia myelofibrosis (according to the ICC classification 2022). Patients enter the study at diagnosis or already in follow-up, at any stage of disease, except if transformed to blast phase. Patients who received fedratinib after June 2022 (time of AIFA reimbursement in Italy) will be enrolled and will be included both those ruxolitinib-naïve and ruxolitinib-exposed.

• Study Type: Observational
• Enrollment (Estimated): 93

Contacts and Locations

• Study Contact: Name: Paola Fazi; Phone Number: 00390670390528; Email: p.fazi@gimema.it
• Study Contact Backup: Name: Enrico Crea; Phone Number: 00390670390514; Email: e.crea@gimema.it

Study Locations

• Italy
  • Milano, Italy
    • Recruiting
    • Ematologia FONDAZIONE IRCCS CA' GRANDA, OSPEDALE MAGGIORE POLICLINICO
    • Contact: Francesco Passamonti

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients affected by MF, according to the WHO classification version 5th or the ICC ) or post- polycythemia vera and post-essential thrombocythemia myelofibrosis according to the ICC who received fedratinib after June 2022 (time of AIFA reimbursement in Italy) will be enrolled and will be included both those ruxolitinib-naïve and ruxolitinib-exposed.

Description

Inclusion Criteria:

• Patients aged 18 years or older.
• Patients diagnosed with primary myelofibrosis [according to the WHO (World Health Organization) classification version 5th or the ICC (International Consensus Conference) either published in 2022 or post-polycythemia vera and post-essential thrombocythemia myelofibrosis (according to the ICC classification 2022)].
• Patients who met the reimbursement criteria for fedratinib, in accordance with the AIFA (Agenzia Italiana del Farmaco) after June 2022.
• Patients eligible or not for stem cell transplant (SCT) or patients already undergoing SCT.
• Patients on non-JAKi cytoreductive treatment.
• Patients with palpable splenomegaly at baseline of fedratinib treatment.
• Informed consent signed, if applicable.

Exclusion Criteria:

• Diagnosis of MPN, unclassifiable, myelodysplastic/myeloproliferative neoplasms, myelodysplastic syndromes, essential thrombocythemia, polycythemia vera.
• Blast phase of MF.
• Patients with platelets <50 x10^9/L at baseline of fedratinib treatment.
• Patients ruxolitinib-exposed for other diseases.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Splenic response	Splenic response is defined as 50% palpatory reduction.	6 months

Collaborators and Investigators

• Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

Study record dates

Study Major Dates

• Study Start (Actual): January 29, 2024
• Primary Completion (Estimated): January 1, 2026
• Study Completion (Estimated): January 1, 2026

Study Registration Dates

• First Submitted: May 22, 2023
• First Submitted That Met QC Criteria: May 22, 2023
• First Posted (Actual): June 1, 2023

Study Record Updates

• Last Update Posted (Actual): February 8, 2024
• Last Update Submitted That Met QC Criteria: February 6, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Site; Bone Marrow Diseases; Hematologic Diseases; Hemorrhagic Disorders; Myeloproliferative Disorders; Blood Coagulation Disorders; Blood Platelet Disorders; Bone Marrow Neoplasms; Hematologic Neoplasms; Primary Myelofibrosis; Thrombocytosis; Thrombocythemia, Essential; Polycythemia Vera; Polycythemia
• Other Study ID Numbers: MPN0123

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No