Reparixin for Myelofibrosis
This study is testing a drug called reparixin for people with myelofibrosis (a bone marrow disorder where scar tissue replaces normal bone marrow, affecting blood cell production). This includes primary myelofibrosis (PMF), or myelofibrosis that developed after essential thrombocythemia (ET-MF) or polycythemia vera (PV-MF). Reparixin works by blocking a specific protein (kinase inhibitor). You might be able to join if you are 18 or older, have a confirmed diagnosis of these types of myelofibrosis, and have intermediate-2 or high-risk disease. The study will look at how well reparixin works to improve your condition after 6 cycles (24 weeks) of treatment. The current status of this study is unclear.
- Study design
- This is an open-label, Phase 2 study, meaning both you and your doctors will know you are receiving reparixin. It plans to enroll 26 participants.
- What's involved
- Eligible patients will take oral reparixin three times daily for 6 cycles (24 weeks). After this, if your condition is stable, you may continue taking reparixin once daily.
- Compensation
- Not stated in the trial record.
- Follow-up
- The main goal for measuring how well the treatment works is at Cycle 6 (24 weeks). Patients may continue treatment after this if their disease is stable.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT05835466
Reparixin in Patients With Myelofibrosis Myeloproliferative Neoplasms Research Consortium (MPN-RC 120)
Recruiting
PHASE2Ages 18+InterventionalTreatmentIcahn School of Medicine at Mount SinaiInvestigator-initiated
~10 participants
Updated 2026-05-06 on ClinicalTrials.gov
What's tested:reparixin
At a glance
Recruiting sites
9 of 9 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Efficacy of reparixin treatment per IWG/ELN criteria
Measured over Cycle 6 (each cycle is 4 weeks) Response Assessment
Conditions
Where it's being run
9 sites across 5 statesNew York4
Ohio2
Florida1
Georgia1
North Carolina1
Study leadership
- Marina Kremyanskaya, PhD, MD · STUDY_CHAIR · Icahn School of Medicine at Mount Sinai
- Aaron Gerds, MD, MS · STUDY_CHAIR · Cleveland Clinic Taussig Cancer Institute
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Be ≥ 18 years of age at time of signing the informed consent form (ICF)
Willing to voluntarily sign the ICF
Have a pathologically confirmed diagnosis of PMF, post-ET-MF, or post-PV-MF as per the World Health Organization (WHO) diagnostic criteria with intermediate-2 or higher risk disease by DIPSS
Have an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
Willing to undergo a bone marrow biopsy at screening
Be refractory/resistant to or intolerant of/inappropriate for JAKi therapy as defined by at least one of the following:
Treatment for ≥ 3 months with inadequate efficacy as demonstrated by persistent palpable splenomegaly ≥ 5cm or symptoms related to splenomegaly,
Treatment for ≥ 28 days complicated by either:
Development of a red blood cell transfusion requirement (at least 2 units/month for 2 months)
CTCAE grade ≥ 3 AEs of thrombocytopenia, anemia, hematoma, or hemorrhage while being treated with a JAKi
Development of non-hematological toxicity that makes patient intolerant of JAKi therapy
In the Investigator's judgment, are not candidates for available approved JAKi
Recovery to ≤ Grade 1 or baseline of any toxicities due to prior systemic treatments, excluding alopecia
At least two weeks must have elapsed between the last dose of any MF-directed drug treatments or other investigational therapies and start of reparixin
Have adequate organ function as demonstrated by the following:
ALT (SGPT) and/or AST (SGOT) ≤ 3x upper limit of normal (ULN), or ≤ 4 x ULN (if upon judgment of the treating physician, it is believed to be due to MF-related EMH);
Direct bilirubin ≤ 1.5 x ULN; or ≤ 2x ULN (if upon judgment of the treating physician, it is believed to be due to MF-related EMH or documented Gilbert's syndrome);
Creatinine clearance ≥ 40 mL/min;
Platelet count ≥ 25 x 109/L;
Bone marrow and peripheral blood blast count \< 10%;
ANC ≥ 1000 mm3.
Life expectancy of at least six months
Women of childbearing potential (WCBP) and men must agree to use adequate contraception prior to study entry, for the duration of study participation, and for 120 days following completion of therapy. WCBP must also have a negative serum pregnancy test at screening and Cycle 1 Day 1. Should a woman become pregnant or suspect she is pregnant while participating, she should inform her treating physician immediately. (Section 5.9.2)
Ability to adhere to the study visit schedule and all protocol requirements
Exclusion
History of stroke, unstable angina, myocardial infarction, or ventricular arrhythmia requiring medication or mechanical control within the last 6 months
Other invasive malignancies within the last 3 years, except non-melanoma skin cancer and localized cured prostate and cervical cancer
Moderate or severe cardiovascular disease meeting one or both of the below criteria:
Presence of cardiac disease, including a myocardial infarction within 6 months prior to study entry, unstable angina pectoris, New York Heart Association Class III/IV congestive heart failure, or uncontrolled hypertension
Documented major electrocardiogram (ECG) abnormalities (not responding to medical treatments)
Presence of active serious infection
Any serious, unstable medical or psychiatric condition that would prevent (as judged by the Investigator) the participant from signing the ICF or any condition, including the presence of laboratory abnormalities, which places the participant at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
Participants who have undergone a hematopoietic cell transplant (HCT) within 100 days of the first dose of study therapy, participants on immunosuppressive therapy post-HCT at screening, use of calcineurin inhibitors within 4 weeks prior to first dose of study therapy, or participants with clinically significant graft-versus-host disease (GVHD)
Known history of human immunodeficiency virus (HIV), or known active hepatitis A, B, or C infection
Impairment of gastrointestinal (GI) function or GI disease that could significantly alter the absorption of reparixin, including any unresolved nausea, vomiting, or diarrhea \> CTCAE grade 1
Is or has an immediate family member (e.g., spouse, parent/legal guardian, sibling, or child) who is investigational site or sponsor staff directly involved with this trial, unless prospective institutional review board (IRB) approval (by chair or designee) is given allowing exception to this criterion for a specific participant
Organ transplant recipients other than bone marrow transplant
Women who are pregnant or lactating
History of splenectomy
Known hypersensitivity to sulfonamides
Known hypersensitivity to non-steroidal anti-inflammatory drugs (NSAID), including ibuprofen
What this trial measures
- Efficacy of reparixin treatment per IWG/ELN criteriaCycle 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 Molecular remission