Real World Evidence of Fedratinib Effectiveness in MF

NCT05883904 | Recruiting

• 1 other identifier: MPN0123
• Study Type: observational
• Target: 93
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis

Outcome Measures

Primary Outcomes (1)

• Splenic response

  Splenic response is defined as 50% palpatory reduction.

  6 months

Interventions

Fedratinib DRUG

Fedratinib administered after AIFA reimbusement approval

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Gruppo Italiano Malattie EMatologiche dell'Adulto: lead

Study Sites (1)

Ematologia FONDAZIONE IRCCS CA' GRANDA, OSPEDALE MAGGIORE POLICLINICO

Milan, Italy

RECRUITING

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

fedratinib

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Paola Fazi

CONTACT

00390670390528; p.fazi@gimema.it

Enrico Crea

CONTACT

00390670390514; e.crea@gimema.it

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 22, 2023
• First Posted: June 1, 2023
• Study Start: January 29, 2024
• Primary Completion: January 1, 2026
• Study Completion: January 1, 2026
• Last Updated: February 8, 2024

Record last verified: 2024-02

Locations

IT (1)