Fedratinib for Leukemia

Recruiting at 2 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: H. Lee Moffitt Cancer Center and Research Institute

Must not be taking: Chemotherapy, Immunomodulatory, Ruxolitinib, others

Disqualifiers: Pregnancy, Encephalopathy, Liver disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

25 Participants Needed

Trial Summary

What is the purpose of this trial?

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).

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, such as chemotherapy, immunomodulatory drugs, and some other specific treatments. However, you can continue taking hydroxyurea if it's being used to control leukocytosis and has been stable for more than 14 days before starting the study drug.

What data supports the effectiveness of the drug Fedratinib for treating leukemia?

Fedratinib is a drug that has shown effectiveness in treating myelofibrosis, a type of blood cancer, by reducing spleen size and symptoms in patients who did not respond well to another treatment called ruxolitinib. While this data is specific to myelofibrosis, it suggests that Fedratinib can be effective in managing blood-related conditions. ^{1 2 3 4 5}

Is fedratinib safe for humans?

Fedratinib has been studied for safety in both healthy volunteers and patients with myelofibrosis, showing common mild side effects like stomach issues. However, more serious side effects like anemia (low red blood cell count) and thrombocytopenia (low platelet count) have been observed in some patients. ^{1 2 3 4 6}

How is the drug Fedratinib unique in treating leukemia?

Fedratinib is unique because it is an oral medication that selectively inhibits JAK2, a protein involved in blood cell production, and is primarily used for myelofibrosis, a related blood disorder. This makes it different from other treatments that may not target JAK2 specifically or are not taken orally. ^{1 2 3 4 7}

Research Team

Andrew Kuykendall, MD

Principal Investigator

Moffitt Cancer Center

Eligibility Criteria

Adults with certain blood disorders like MDS/MPNs or CNL, who can swallow pills and have not been treated with fedratinib before. They must understand the study and agree to use effective contraception. Excluded are those with thiamine deficiency, serious infections, on specific drugs that affect fedratinib, pregnant women, or those with other major health issues.

Inclusion Criteria

I am 18 years old or older.

Patient must be able to adhere to the study visit schedule and other protocol requirements

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

Exclusion Criteria

I had major surgery or radiation therapy within the last four weeks.

I agree to use effective contraception while on the study treatment.

Patient with known human immunodeficiency virus (HIV), active infectious Hepatitis B (Hep B), and/or active Hepatitis C (Hep C)

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Fedratinib by mouth once a day every day of each 28-day cycle

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Fedratinib

Trial Overview The trial is testing the effectiveness of a pill called fedratinib for treating blood disorders such as MDS/MPNs and CNL. It aims to see how safe it is for patients and if there are any side effects when taking this medication.

Participant Groups

Group I: Treatment with Fedratinib Experimental Treatment 1 Intervention

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

Fedratinib is already approved in United States, European Union for the following indications:

Approved in United States as Inrebic for:

• Myelofibrosis

Approved in European Union as Inrebic for:

• Myelofibrosis

Who Is Running the Clinical Trial?

H. Lee Moffitt Cancer Center and Research Institute

Lead Sponsor

Trials

576

Recruited

145,000+

Bristol-Myers Squibb

Industry Sponsor

Trials

2,731

Recruited

4,127,000+

Headquarters

New York City, USA

Known For

Oncology & Cardiovascular

Top Products

Eliquis, Opdivo, Revlimid, Orencia

Christopher Boerner

Bristol-Myers Squibb

Chief Executive Officer since 2023

PhD in Business Administration from the Haas School of Business, University of California, Berkeley; BA in Economics and History from Washington University in St. Louis

Deepak L. Bhatt

Bristol-Myers Squibb

Chief Medical Officer since 2024

MD from Yale University; MSc in Clinical Epidemiology from the University of Pennsylvania

Findings from Research

In the phase II JAKARTA2 study involving 97 patients with intermediate- or high-risk myelofibrosis who were resistant or intolerant to ruxolitinib, fedratinib demonstrated a spleen volume response rate of 31%, indicating its efficacy in reducing spleen size.

Despite the positive outcomes, the treatment was associated with significant side effects, including grade 3-4 anemia in 38% of patients and thrombocytopenia in 22%, highlighting the need for careful monitoring during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fedratinib in patients with myelofibrosis previously treated with ruxolitinib: An updated analysis of the JAKARTA2 study using stringent criteria for ruxolitinib failure. Harrison, CN., Schaap, N., Vannucchi, AM., et al.[2022]

References

1. England pubmed.ncbi.nlm.nih.gov

Current and future role of fedratinib in the treatment of myelofibrosis. [2022]

2. New Zealand pubmed.ncbi.nlm.nih.gov

Fedratinib: First Approval. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Fedratinib in patients with myelofibrosis previously treated with ruxolitinib: An updated analysis of the JAKARTA2 study using stringent criteria for ruxolitinib failure. [2022]

4. Germany pubmed.ncbi.nlm.nih.gov

Pharmacokinetics and tolerability of fedratinib, an oral, selective Janus kinase 2 inhibitor, in subjects with renal or hepatic impairment. [2022]

5. Germany pubmed.ncbi.nlm.nih.gov

Effect of fluconazole on the pharmacokinetics of a single dose of fedratinib in healthy adults. [2022]

6. England pubmed.ncbi.nlm.nih.gov

A randomized, placebo-controlled study of the pharmacokinetics, pharmacodynamics, and tolerability of the oral JAK2 inhibitor fedratinib (SAR302503) in healthy volunteers. [2022]

7. Germany pubmed.ncbi.nlm.nih.gov

Population pharmacokinetics of fedratinib in patients with myelofibrosis, polycythemia vera, and essential thrombocythemia. [2022]