NCT05835466

Phase II Study of Reparixin in Patients With Myelofibrosis Myeloproliferative Neoplasms Research Consortium [MPN-RC 120]

Recruiting now Phase 2 Last updated 5 May 2026

What this trial tests

Phase 2 trial testing reparixin in Myelofibrosis (PMF) in 10 participants. Currently enrolling.

Timeline

24 July 2023

Primary endpoint
31 December 2027

1 December 2028

Quick facts

Lead sponsor	Icahn School of Medicine at Mount Sinai
Phase	Phase 2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	10
Start date	24 July 2023
Primary completion	31 December 2027
Estimated completion	1 December 2028
Sites	9 locations across United States

Drugs / interventions tested

• reparixin — full drug profile →

Conditions studied

• Myelofibrosis (PMF) — all drugs for Myelofibrosis (PMF) →
• Post Essential Thrombocythemia Myelofibrosis (ET-MF) — all drugs for Post Essential Thrombocythemia Myelofibrosis (ET-MF) →
• Post Polycythemia Vera Related Myelofibrosis (PV-MF) — all drugs for Post Polycythemia Vera Related Myelofibrosis (PV-MF) →

Sponsor

Icahn School of Medicine at Mount Sinai

Who can join

18 and older, any sex, with Myelofibrosis (PMF) or Post Essential Thrombocythemia Myelofibrosis (ET-MF). Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Efficacy of reparixin treatment per IWG/ELN criteria
  Time frame: Cycle 6 (each cycle is 4 weeks) Response Assessment
  To estimate the efficacy of reparixin treatment in DIPSS intermediate-2 or high-risk subjects with PMF, post PV-MF, or post ET-MF as assessed by IWG/ELN criteria. The IWG/ELN criteria: CR (complete remission), PR (partial remission), Clinical improvement, Anemia response, Spleen response, Symptoms response, PD (progressive disease), SD (stable disease), Relapse, Cytogenetic remission, and Molecula

Sponsor's own description

This is an open label, phase II study to assess the efficacy, safety, and tolerability of Reparixin in patients with DIPSS intermediate-2, or high-risk primary myelofibrosis (PMF), post essential thrombocythemia/polycythemia vera related MF (Post ET/PV MF) after prior treatment, and those who are ineligible or refuse treatment, with a Janus kinase inhibitor (JAKi). 26 patients will be enrolled. Eligible patients will receive oral reparixin three times daily on a 4-week cycle for a core study period of 6 cycles (24 weeks). After cycle 6, patients may continue receiving reparixin once daily on a 4-week cycle if at least stable disease (SD) is met by IWG-MRT criteria until loss of response, disease progression, unacceptable toxicity, patient/physician withdrawal, or termination of study by sponsor.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. Novel approaches in myelofibrosis.
   Koschmieder S. · · 2024 · cited 7× · PMID 39670187 · DOI 10.1002/hem3.70056
2. The Variation in the Traits Ameliorated by Inhibitors of JAK1/2, TGF-β, P-Selectin, and CXCR1/CXCR2 in the <i>Gata1</i><sup>low</sup> Model Suggests That Myelofibrosis Should Be Treated by These Drugs in Combination.
   Gobbo F, Martelli F, Di Virgilio A, Demaria E, et al · · 2024 · cited 3× · PMID 39062946 · DOI 10.3390/ijms25147703
3. Neutrophils as critical orchestrators of chronic inflammation.
   Torfs K, Vermeersch G, Gouwy M, Devos T, et al · · 2026 · cited 2× · PMID 41530536 · DOI 10.1038/s41423-025-01380-w
4. Chronic Inflammation in Primary Myelofibrosis: In-Depth Insights Into Pathogenesis and Promising Anti-Inflammatory Therapeutic Strategies.
   Chen M, Zheng C, Zhang Y, He J, et al · · 2025 · PMID 41268532 · DOI 10.1155/mi/9967975