Myeloproliferative NeoplasmPhase 2NCT05177211
Fedratinib in Myelodysplastic /Myeloproliferative Neoplasms (MDS/MPNs) and Chronic Neutrophilic Leukemia (CNL)
A Phase 2 study testing Fedratinib Pill in people with Myeloproliferative Neoplasm and Chronic Neutrophilic Leukemia, run by H. Lee Moffitt Cancer Center and Research Institute at 3 sites in United States.
Trial summary
The purpose of the study is to evaluate the effectiveness, safety, and tolerability of a study drug called fedratinib in participants with myelodysplastic/myeloproliferative neoplasms (MDS/MPNs) and chronic neutrophilic leukemia (CNL). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myeloproliferative Neoplasm, Chronic Neutrophilic Leukemia, MDS
- What is being tested?
- Fedratinib Pill
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 3 sitesUnited States
- Study dates
- 1 Mar 2022to 9 Jan 2026
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Patient must understand and voluntarily sign an ICF prior to any study-related assessments/ procedures being conducted
- 18 years of age or older on day of signing informed consent.
- Morphologically confirmed diagnosis of one of the following in accordance with WHO (2016) diagnostic criteria:
- Atypical Chronic Myeloid Leukemia (aCML), BCR-ABL1 negative
- Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable (MDS/MPN-U)
- Myelodysplastic Syndrome/Myeloproliferative Neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T)
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- Chronic Neutrophilic Leukemia (CNL).
- Palpable splenomegaly ≥ 5 cm below left costal margin (LCM), spleen volume ≥ 450 cc, AND/OR MPN-SAF TSS > 10.
- Has an Eastern Cooperative Oncology Group (ECOG) Performance Score (PS) of 0, 1 or 2
- Able to adhere to the study visit schedule and other protocol requirements.
- Females of childbearing potential (FCBP) must have a negative serum pregnancy test at screening. A FCBP is considered when a sexually mature female: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 12 consecutive months.
- A FCBP must agree to use of two methods of highly effective contraception, be surgically sterile, or abstain from heterosexual activity for the course of the study through 30 days after the last dose of study treatment.
- Male patients must agree to use an adequate method of contraception starting with the first dose of study therapy through 90 days after the last dose of study therapy. Men must agree to not donate sperm during study therapy and for 30 days after the last dose of study therapy
Who cannot join
- Any of the laboratory abnormalities as listed in the protocol.
- Patient is pregnant or lactating female
- Patient is a woman of childbearing potential as previously defined in inclusion #7, unless using effective contraception while on study treatment
- Patient is a man who is a partner with of a woman of childbearing potential, unless they agree to use effective contraception while on study treatment as previously defined in inclusion #9.
- Patient with prior history of encephalopathy, including Wernicke's Encephalopathy (WE)
- Patient has signs or symptoms of encephalopathy, including Wernicke's Encephalopathy (e.g., severe ataxia, ocular paralysis or cerebellar signs) in which case thiamine deficiency needs to be excluded and a brain MRI might be required to exclude possible Wernicke's encephalopathy
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- Patient has thiamine deficiency if not corrected before enrollment on the study
- Patient with concomitant treatment with or use of pharmaceutical or herbal agents known to be moderate or strong inducers of CYP3A4 or dual CYP3A4 and CYP2C19 inhibitors. For a list of moderate or strong inhibitors or inducers of CYP3A4, see table 3-2 and 3-3 at https://www.fda.gov/drugs/drug-interactions-labeling/drug-development-and-drug-interactions-table-substrates-inhibitors-and-inducers.
- Patient on any chemotherapy, immunomodulatory drug therapy (e.g., lenalidomide, pomalidomide, thalidomide, interferon-alpha), ruxolitinib, anagrelide, corticosteroids >10 mg/day prednisone or equivalent. Patients may remain on hydroxyurea (e.g., hydrea) if it is being employed to control leukocytosis as long as the patient has been on a stable dose for > 14 days prior to initiation of fedratinib.
- Prior treatment with fedratinib
- Patient on treatment with myeloid growth (e.g., G-CSF) factor within 14 days prior to initiation of fedratinib
- Patient on treatment with aspirin with doses > 150 mg daily.
- Patient with diagnosis of chronic liver disease (e.g., chronic alcoholic liver disease, autoimmune hepatitis, sclerosing cholangitis, primary biliary cirrhosis).
- Patients with active (uncontrolled, metastatic) second malignancies are excluded.
- Patient with uncontrolled congestive heart failure (New York Heart Association Classification 3 or 4).
- Patient with known human immunodeficiency virus (HIV), active infectious Hepatitis B (Hep B), and/or active Hepatitis C (Hep C).
- Patients with known HIV are eligible if the following criteria are met: i. Patient has CD4+ T-cell count ≥ 350 cells/µL ii. Patient is on established anti-retroviral therapy (ART) (with medications that are not specifically excluded due to potential interactions within this study) for at least four weeks prior to study enrollment and have an HIV viral load less than 400 copies/mL prior to enrollment.
- Patients with a history of Hep C infection are eligible if: i. Patient has completed curative antiviral treatment and has hepatitis C viral load below the limit of quantification ii. Pt has Hep C antibody positive but Hep C RNA negative due to prior treatment or natural resolution.
- Patient with serious active infection requiring IV anti-microbials.
- Patient with presence of any significant gastric or other disorder that would inhibit absorption of oral medication.
- Patient is unable to swallow capsules.
- Patient with participation in any study of an investigational agent (drug, biologic, device) within 30 days prior to start of fedratinib.
- Patient has any condition including the presence of laboratory abnormalities, which places the patient at unacceptable risk if he/she were to participate in the study.
- Patient has any condition that confounds the ability to interpret data from the study.
- Any major surgery or radiation therapy within four weeks.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of Michigan Rogel Cancer Center
Ann Arbor, Michigan, 48109
Cleveland Clinic
Cleveland, Ohio, 44195
Moffitt Cancer Center
Tampa, Florida, 33612
About this study
Official title: A Phase 2 Study of Fedratinib in Myelodysplastic /Myeloproliferative Neoplasms (MDS/MPNs) and Chronic Neutrophilic Leukemia (CNL)
The purpose of the study is to evaluate the effectiveness, safety, and tolerability of a study drug called fedratinib in participants with myelodysplastic/myeloproliferative neoplasms (MDS/MPNs) and chronic neutrophilic leukemia (CNL).
- Being tested
- Fedratinib Pill
- Keywords
- MPNs, CNL
- Medical subject headings
- Myeloproliferative Disorders, Leukemia, Neutrophilic, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fedratinib PillDrug
Also called Inrebic
Participants will be given Fedratinib at a dose of 400 mg PO once daily (4-100 mg capsules). Fedratinib can be given at any time during the day, but patients are advised to take the dose at the same approximate time every day.
| Study group | Role | What they receive |
|---|---|---|
| Treatment with Fedratinib | Experimental | Participants will taken Fedratinib by mouth once a day every day of each 28 day cycle. Receives: Drug: Fedratinib Pill |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Clinical Response Rate
Response rate of fedratinib in MDS/MPN and CNL. Investigators will measure the proportion of patients achieving a clinical response from baseline to week 24 as defined by Complete Response (CR), Partial Response (PR) or Clinical Benefit (CB) by modified MDS/MPN IWG Proposed response criteria.
Measured: Up to 24 weeks
Secondary outcomes (4)
Proportion of Patients Achieving Spleen Response at 12 Weeks
Participants spleen size will be measured and compared to baseline spleen size measurement. Spleen response is defined as 50% reduction in palpable splenomegaly of a spleen that is at least 10cm at baseline, a spleen that is palpable at more than 5 cm at baseline becoming non-palpable, or, in cases where volumetric evaluation is feasible, a ≥ 35% reduction in spleen volume in patients with a baseline spleen volume > 450 cc. Spleen responses by palpation will need to correlate with a 50% reduction in longest diameter of the spleen by imaging.
Measured: at 12 weeks
Proportion of Patients Achieving Spleen Response at 24 Weeks
Participants spleen size will be measured and compared to baseline spleen size measurement. Spleen response is defined as 50% reduction in palpable splenomegaly of a spleen that is at least 10cm at baseline, a spleen that is palpable at more than 5 cm at baseline becoming non-palpable, or, in cases where volumetric evaluation is feasible, a ≥ 35% reduction in spleen volume in patients with a baseline spleen volume > 450 cc. Spleen responses by palpation will need to correlate with a 50% reduction in longest diameter of the spleen by imaging.
Measured: at 24 weeks
Proportion of Patients Who Have a 50% Reduction in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) at 12 Weeks
Reduction in MPN-SAF TSS score will be measured by comparing TSS score at day 84 assessment to TSS score on cycle 1 day 1. Only patients with baseline TSS ≥ 10 will be eligible for symptom response. Patients who do not have a TSS score performed at day 84 assessment or who have discontinued study drug prior to day 84 assessment will be considered to not have 50% reduction in TSS.
The MPN-SAF TSS is a questionnaire with nine items containing the most common symptoms reported by patients (concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss and fever), plus one item ("worst fatigue") from the "Brief Fatigue Inventory (BFI)". Each item has a score that ranges from 0 (absent/as good as it can be) to 10 (worst imaginable/as bad as it can be). A higher score therefore indicates more severe symptoms
Measured: at 12 weeks
Proportion of Patients Who Have a 50% Reduction in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) at 24 Weeks
Reduction in MPN-SAF TSS score will be measured by comparing TSS score at day 84 assessment to TSS score on cycle 1 day 1. Only patients with baseline TSS ≥ 10 will be eligible for symptom response. Patients who do not have a TSS score performed at day 84 assessment or who have discontinued study drug prior to day 84 assessment will be considered to not have 50% reduction in TSS.
The MPN-SAF TSS is a questionnaire with nine items containing the most common symptoms reported by patients (concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss and fever), plus one item ("worst fatigue") from the "Brief Fatigue Inventory (BFI)". Each item has a score that ranges from 0 (absent/as good as it can be) to 10 (worst imaginable/as bad as it can be). A higher score therefore indicates more severe symptoms
Measured: at 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jan 2022
- Study start1 Mar 2022
- Primary completion10 Apr 2025
- Study completion9 Jan 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myeloproliferative NeoplasmStudies of the same condition, open and under way.
- More trials run by H. Lee Moffitt Cancer Center and Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 25 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Bristol-Myers Squibb
- Responsible party
- Sponsor
- Investigators
- Andrew Kuykendall, MD, Principal investigator, Moffitt Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05177211
- Sponsor's protocol ID
- MCC-20963
- Status last verified
- May 2026
- First submitted
- 15 Dec 2021
- Last updated
- 9 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of Fedratinib in Myelodysplastic /Myeloproliferative Neoplasms (MDS/MPNs) and Chronic Neutrophilic Leukemia (CNL)
- ClinicalTrials.gov ID
- NCT05177211
- Acronym / sponsor ID
- MCC-20963
- Keywords
- MPNs, CNL
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- Bristol-Myers Squibb
- Study start
- 1 Mar 2022 (actual)
- Primary completion
- 10 Apr 2025 (actual)
- Study completion
- 9 Jan 2026 (actual)
- First posted
- 4 Jan 2022 (actual)
- Results
- Posted 17 Apr 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to evaluate the effectiveness, safety, and tolerability of a study drug called fedratinib in participants with myelodysplastic/myeloproliferative neoplasms (MDS/MPNs) and chronic neutrophilic leukemia (CNL).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment with Fedratinib | Experimental | Drug: Fedratinib PillParticipants will taken Fedratinib by mouth once a day every day of each 28 day cycle. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Patient must understand and voluntarily sign an ICF prior to any study-related assessments/ procedures being conducted
- 18 years of age or older on day of signing informed consent.
- Morphologically confirmed diagnosis of one of the following in accordance with WHO (2016) diagnostic criteria:
- Palpable splenomegaly ≥ 5 cm below left costal margin (LCM), spleen volume ≥ 450 cc, AND/OR MPN-SAF TSS > 10.
- Has an Eastern Cooperative Oncology Group (ECOG) Performance Score (PS) of 0, 1 or 2
Key exclusion criteria (25 listed)
- Any of the laboratory abnormalities as listed in the protocol.
- Patient is pregnant or lactating female
- Patient is a woman of childbearing potential as previously defined in inclusion #7, unless using effective contraception while on study treatment
- Patient is a man who is a partner with of a woman of childbearing potential, unless they agree to use effective contraception while on study treatment as previously defined in inclusion #9.
- Patient with prior history of encephalopathy, including Wernicke's Encephalopathy (WE)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fedratinib PillAlso: InrebicParticipants will be given Fedratinib at a dose of 400 mg PO once daily (4-100 mg capsules). Fedratinib can be given at any time during the day, but patients are advised to take the dose at the same approximate time every day. | Drug | Treatment with Fedratinib |
Primary outcome
Objective Clinical Response Rate
Time frame: Up to 24 weeks
Response rate of fedratinib in MDS/MPN and CNL. Investigators will measure the proportion of patients achieving a clinical response from baseline to week 24 as defined by Complete Response (CR), Partial Response (PR) or Clinical Benefit (CB) by modified MDS/MPN IWG Proposed response criteria.
4 secondary outcomes
Proportion of Patients Achieving Spleen Response at 12 Weeks
Time frame: at 12 weeks
Proportion of Patients Achieving Spleen Response at 24 Weeks
Time frame: at 24 weeks
Proportion of Patients Who Have a 50% Reduction in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) at 12 Weeks
Time frame: at 12 weeks
Proportion of Patients Who Have a 50% Reduction in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) at 24 Weeks
Time frame: at 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrew Kuykendall, MD principal investigator
Moffitt Cancer Center
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fedratinib Pill in people with Myeloproliferative Neoplasm and Chronic Neutrophilic Leukemia, run by H. Lee Moffitt Cancer Center and Research Institute at 3 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 13 inclusion and 25 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: finished, as of the sponsor's last update on 9 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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?
Yes. Results are posted in the registry (first posted 17 Apr 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Clinical Response Rate (time frame: Up to 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Jun 2026.