Evaluating the effectiveness of fedratinib in myelofibrosis patients
Observational Study Protocol REALFed - Real World Evidence of Fedratinib Effectiveness in Myelofibrosis
This study is trying to see how well fedratinib works for people with myelofibrosis, including those with different types of the condition.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 93 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Gruppo Italiano Malattie EMatologiche dell'Adulto Academic / other |
| Drugs / interventions | fedratinib, ruxolitinib |
| Locations | 1 site (Milano) |
| Trial ID | NCT05883904 on ClinicalTrials.gov |
What this trial studies
This observational study aims to assess the real-world effectiveness of fedratinib in patients diagnosed with primary myelofibrosis or post-polycythemia vera and post-essential thrombocythemia myelofibrosis. It will include both prospective and retrospective data collection from multiple Italian Hematology Units belonging to the GIMEMA Organization. Participants will be managed according to the clinical practices of their respective centers, and the study will enroll patients who meet specific diagnostic criteria and reimbursement guidelines for fedratinib. The study will include patients at various stages of the disease, except those in blast phase.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 and older diagnosed with primary myelofibrosis or post-polycythemia vera or post-essential thrombocythemia myelofibrosis.
Not a fit: Patients with unclassifiable myeloproliferative neoplasms, myelodysplastic syndromes, or those in blast phase of myelofibrosis may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the effectiveness of fedratinib for treating myelofibrosis, potentially improving patient outcomes.
How similar studies have performed: Other studies have shown promising results with similar treatments in myelofibrosis, but this specific approach is being evaluated in a real-world setting, making it a novel contribution.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Milano
- Ematologia FONDAZIONE IRCCS CA' GRANDA, OSPEDALE MAGGIORE POLICLINICO — Milano, Italy (Recruiting)
Study contacts
- Study coordinator: Paola Fazi
- Email: p.fazi@gimema.it
- Phone: 00390670390528
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.