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Myelofibrosis (PMF)Phase 2NCT05835466

Reparixin in Patients With Myelofibrosis Myeloproliferative Neoplasms Research Consortium (MPN-RC 120)

A Phase 2 study testing reparixin in people with Myelofibrosis (PMF) and Post Essential Thrombocythemia Myelofibrosis (ET-MF), run by Icahn School of Medicine at Mount Sinai at 9 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

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… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
reparixin
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
9 sitesUnited States
Study dates
24 Jul 2023to 1 Dec 2028
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
  • o A bone marrow biopsy obtained within 90 days of screening without intervening treatments and approved by the study chair may suffice.
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  • 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
  • o Participants may continue hydroxyurea until the day prior to C1D1 if needed for disease control
  • 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)
  • o Men must agree to use a condom and not father a child or donate sperm for the duration of the study and for 120 days after the last dose of study therapy
  • Ability to adhere to the study visit schedule and all protocol requirements

Who cannot join

  • 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
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  • 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)
  • o Note: The use of topical steroids or < 10mg oral prednisone for ongoing skin GVHD is permitted
  • 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
  • o Hypersensitivity to sulphanilamide antibiotics alone (e.g. sulfamethoxazole) does not qualify for exclusion
  • Known hypersensitivity to non-steroidal anti-inflammatory drugs (NSAID), including ibuprofen

Trial locations and contacts

9 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Gillian Sanchez contact

(917) 581-1774

gillian.sanchez@mssm.edu

Shakira Forde contact

212-824-8334

shakira.forde@mssm.edu

9 sites in 1 country

United States9 sites, 9 recruiting

  • Emory University

    Atlanta, Georgia, 30322

    Anthony Hunter, MD

    Recruiting

  • Roswell Park Cancer Institute

    Buffalo, New York, 14263

    Eunice Wang, MD

    Recruiting

  • The Cleveland Clinic Foundation

    Cleveland, Ohio, 44195

    Aaron Gerds, MD

    Recruiting

  • The Ohio State University

    Columbus, Ohio, 43210

    Shivani Handa, MD

    Recruiting

  • Ruttenberg Treatment Center

    New York, New York, 10029

    Marina Kremyanskaya

    Recruiting

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

    Brian Chernak, MD

    Recruiting

  • NewYork-Presbyterian/Weill Cornell Medical Center

    New York, New York, 10065

    Ghaith Abu-Zeinah, MD

    Recruiting

  • Moffitt Cancer Center

    Tampa, Florida, 33612

    Andrew Kuykendall, MD

    Recruiting

  • Wake Forest Baptist Health Comprehensive Cancer Center

    Winston-Salem, North Carolina, 27157

    Rupali Bhave, MD

    Recruiting

Other trials in United States

About this study

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

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.

Being tested
reparixin
Medical subject headings
Primary Myelofibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • reparixinDrug

    reparixin at 1200mg TID three times per day.

Study groups in NCT05835466
Study groupRoleWhat they receive
ReparixinExperimentalEligible 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.
Receives: Drug: reparixin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Efficacy of reparixin treatment per IWG/ELN criteria

    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

    Measured: Cycle 6 (each cycle is 4 weeks) Response Assessment

Secondary outcomes (7)

  1. Response Assessment of IWG/ELN

    Response by IWG/ELN criteria at the end of Cycle 6. 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

    Measured: end of Cycle 6 (each cycle is 4 weeks)

  2. Response Assessment of IWG/ELN

    Response by IWG/ELN criteria at the end of Cycle 12. 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

    Measured: end of Cycle 12 (each cycle is 4 weeks)

  3. Bone marrow fibrosis grade

    Bone marrow fibrosis grade at the end of Cycle 6. Bone marrow fibrosis (MF) is graded as MF-0 to MF-3, with higher number indicating more disease.

    Measured: end of Cycle 6 (each cycle is 4 weeks)

  4. Bone marrow fibrosis grade

    Bone marrow fibrosis grade at the end of Cycle 12. Bone marrow fibrosis (MF) is graded as MF-0 to MF-3, with higher number indicating more disease.

    Measured: end of Cycle 12 (each cycle is 4 weeks)

  5. Number of Adverse Events

    To assess the safety of reparixin as measured by the adverse event profile of CTCAE v5.0.

    Measured: End of study (24 weeks) plus 3 months

  6. Change in Spleen Volume

    Change in spleen volume by imaging after cycle 6 as compared to baseline spleen volume.

    Measured: Baseline and cycle 6 (each cycle is 4 weeks)

  7. Change in Spleen Volume

    Change in spleen volume by imaging after cycle 12 as compared to baseline spleen volume.

    Measured: Baseline and cycle 12 (each cycle is 4 weeks)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Apr 2023
  2. Study start24 Jul 2023
  3. Primary completion31 Dec 2027 (estimated)
  4. Study completion1 Dec 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05835466
Sponsor's protocol ID
STUDY-22-01764
Status last verified
May 2026
First submitted
29 Mar 2023
Last updated
6 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing reparixin in people with Myelofibrosis (PMF) and Post Essential Thrombocythemia Myelofibrosis (ET-MF), run by Icahn School of Medicine at Mount Sinai at 9 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 27 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 6 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Gillian Sanchez ((917) 581-1774, gillian.sanchez@mssm.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Efficacy of reparixin treatment per IWG/ELN criteria (time frame: Cycle 6 (each cycle is 4 weeks) Response Assessment). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05835466.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 May 2026.

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