Fedratinib in Myelodysplastic /Myeloproliferative Neoplasms (MDS/MPNs) and Chronic Neutrophilic Leukemia (CNL)

NCT05177211 | Active Not Recruiting Phase 2 Results DMC FDA Drug

• 1 other identifier: MCC-20963
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 3

Brief 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).

Conditions

Myeloproliferative Neoplasm; Chronic Neutrophilic Leukemia; MDS

Keywords

MPNs; CNL

Outcome Measures

Primary Outcomes (1)

• 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.

  Up to 24 weeks

Secondary Outcomes (4)

• Proportion of Patients Achieving Spleen Response at 12 Weeks

  at 12 weeks

• Proportion of Patients Achieving Spleen Response at 24 Weeks

  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

  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

  at 24 weeks

Other Outcomes (2)

• Patient's Global Impression of Change (PGIC) at Week 12

  at week 12

• Patient's Global Impression of Change (PGIC) at Week 24

  at week 24

Study Arms (1)

Treatment with Fedratinib

EXPERIMENTAL

Participants will taken Fedratinib by mouth once a day every day of each 28 day cycle.

Drug: Fedratinib Pill

Interventions

Fedratinib Pill DRUG

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.

Also known as: Inrebic

Treatment with Fedratinib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient must understand and voluntarily sign an ICF prior to any study-related assessments/ procedures being conducted
• 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)
• 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

You may not qualify if:

• Any of the laboratory abnormalities as listed in the protocol.
• Patient is pregnant or lactating female
• 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
• 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).
• a. 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.

Sponsors & Collaborators

• Bristol-Myers Squibb: collaborator
• H. Lee Moffitt Cancer Center and Research Institute: lead

Study Sites (3)

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

University of Michigan Rogel Cancer Center

Ann Arbor, Michigan, 48109, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Related Links

• Moffitt Cancer Center Clinical Trials website

MeSH Terms

Conditions

Myeloproliferative Disorders; Leukemia, Neutrophilic, Chronic

Interventions

fedratinib

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Andrew T. Kuykendall, MD
• Organization: Moffitt Cancer Center

Andrew.Kuykendall@moffitt.org

813-745-4639

Study Officials

• Andrew Kuykendall, MD

  Moffitt Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2021
• First Posted: January 4, 2022
• Study Start: March 1, 2022
• Primary Completion: April 10, 2025
• Study Completion: April 1, 2026
• Last Updated: April 17, 2026
• Results First Posted: April 17, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)