Leflunomide in Combination With Decitabine for Treatment of Relapsed or Refractory Myelodysplastic Syndromes

Launched by WEST VIRGINIA UNIVERSITY · Apr 3, 2025

Keywords

ClinConnect Summary

This clinical trial is investigating a new treatment approach for patients with relapsed or refractory myelodysplastic syndromes (R/R MDS), a type of blood disorder. The study is testing the safety and effectiveness of combining two medications: leflunomide, which is taken as a pill, and decitabine, which is given through an intravenous (IV) infusion. The goal is to find out the best dosages of these medications that patients can tolerate. Participants will receive decitabine daily for five days every four weeks, along with leflunomide taken daily for about two to three weeks during a 28-day cycle.

To join the trial, patients must have a confirmed diagnosis of MDS and meet certain health criteria, such as having specific blood counts. They should have previously tried at least one other treatment for their condition. Participants can expect regular check-ups during the study to monitor their health and response to the treatment. The trial is not currently recruiting, but it aims to help improve treatment options for people with this challenging condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Patient has pathologically confirmed diagnosis of MDS
• * Patient has currently measurable disease meeting the following criteria:
• • Bone marrow biopsy with more than 5% blasts, AND
• • Absolute neutrophil count (ANC) less than 1,000/mcL, and/or platelet count less than 100,000/mcL and/or hemoglobin levels less than 10g/dL
• • Patient has received one prior treatment with a DNA methyltransferase inhibitor (DNMTi), also commonly called hypomethylating agent (HMA). Patients whose MDS has IDH1/IDH2 mutations should have received at least one available IDH1/IDH2 inhibitor
• • Patient has an Eastern Cooperative Oncology Group (ECOG) performance status less than or equal to 2
• * Patient has the following required baseline laboratory data (eligibility can be based on local lab results):
• • Total serum bilirubin level less than or equal to 2 times ULN
• • Estimated glomerular filtration rate (eGFR) greater than or equal to 45 mL/min/1.73 m2
• • Patients who have undergone alloHSCT are eligible if they are more than 28 days post stem cell infusion, have no evidence of GVHD \> Grade 1, and are more than a week off all immunosuppressive therapy
• • If a female of childbearing potential, the patient has a negative serum or urine pregnancy test result within 7 days prior to the first dose of treatment. Women of non-childbearing potential are those who are postmenopausal greater than one year or who have had a bilateral tubal ligation or hysterectomy
• • If female of childbearing potential or a male patient, patient agrees to use an effective contraceptive method from the time of informed consent, during the course of the study, and for 3 months following the last dose of treatment
• • Patient understands and voluntarily signs the written informed consent prior to any study-specific procedures. A copy of the signed informed consent form will be retained by the treating institution
• Exclusion Criteria:
• • Patients receiving any other investigational agents, or concurrent chemotherapy or immunotherapy
• • Patients with progression to acute myeloid leukemia
• • Patients with other malignancies requiring systemic chemotherapy, immunotherapy or targeted therapy in the last four weeks
• • Patients with uncontrolled bacterial, viral or fungal infections (undergoing appropriate treatment and with progression of clinical symptoms)
• • Patients with active or latent tuberculosis
• • Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that per Principal Investigator's judgment would limit compliance with study requirements
• • Females who are pregnant or breast feeding
• • Any other clinical conditions that in the opinion of the investigator would make the subject unsuitable for the study