Fedratinib combined with ropeginterferon alfa-2b in patients with myelofibrosis (FEDORA): study protocol for a multicentre, open-label, Bayesian phase II trial

Study Overview: FEDORA Trial

Background

Myelofibrosis (MF) is a serious blood disease with a median survival of only 6.5 years. The common gene mutation in these patients is JAK2V617F, which causes continuous activation of certain proteins. Fedratinib is a JAK2 inhibitor that is taken orally and has shown promise in treating MF. However, combining it with other treatments may be necessary for better results. Ropeginterferon alfa-2b is a newer type of interferon that is well-tolerated and effective in MF, but we need to understand how it works with fedratinib. The FEDORA trial aims to evaluate the safety and effectiveness of this combination in patients needing treatment.

Methods

FEDORA is a phase II trial involving multiple centers, focusing on the combination of fedratinib and ropeginterferon alfa-2b. We plan to enroll 30 patients aged 18 and older with specific types of MF. The main goal is to assess how well patients tolerate the treatment. If patients experience severe side effects in the first four months, the treatment may be considered intolerable. We will adjust doses for each patient to find the best combination for them.

Discussion

This trial uses a Bayesian design to gather evidence on the safety and effectiveness of the treatment combination. The results will help determine if a larger phase III trial is needed.

Trial Registration

EudraCT number: 2021-004056-42. ISRCTN: 88,102,629.

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