The goal of this clinical trial is to learn about the safety and effectiveness of the combination drug Tetrahydrouridine (THU) and decitabine (DEC) to treat patients with relapsed or refractory myelodysplastic syndrome. The main questions it aims to answer are: * Does the combination drug exhibit hematological and nonhematological toxicity? * Does the combination drug improve health status and reduce the number of days of hospitalization? Participants will: * Take tetrahydrouridine and decitabine once a week for 24 weeks * Visit the clinic once every 4 weeks for checkups and tests * Keep a diary of their symptoms
This is a single-arm, open-label Phase 1 study of oral THU/decitabine to treat relapsed or refractory MDS. Patients will be treated for 24 weeks in the absence of clear evidence of progressive disease. The primary endpoint is safety. The secondary endpoints will include assessment of response rates by International Working Group (IWG) criteria, quality of life (QoL), and number of days of hospitalization. DNA-methyltransferase 1 (DNMT1) protein levels will be measured in bone marrow and peripheral blood white cells.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Oral tetrahydrouridine and oral decitabine capsule
Montefiore Einstein Medical Center
The Bronx, New York, United States
RECRUITINGWhite blood cell measurement
Measure neutrophils count
Time frame: every 4 weeks
Platelet count
Measure platelets in the blood
Time frame: every 4 week
Response for high-risk MDS
Measure using International Working Group (IWG) 2023 response criteria
Time frame: every 4 weeks
Hospitalization
Record the number of days of hospitalization
Time frame: every 4 weeks
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