Phase 1 Study of Oral Tetrahydrouridine and Decitabine for Treating Relapsed or Refractory Myelodysplastic Syndromes

What this Trial Is About

Researchers are evaluating the safety and effectiveness of a combination drug made of tetrahydrouridine THU and decitabine DEC for patients with relapsed or refractory myelodysplastic syndrome MDS. This single-arm, open-label Phase 1 study aims to find out if the treatment causes blood-related or other side effects, and whether it improves patients health and reduces hospital stays. The study also measures protein levels related to the disease and tracks quality of life. Participants will take oral capsules of THU followed one hour later by decitabine once a week for 24 weeks, provided there is no clear evidence the disease is progressing. There is no placebo group as all patients receive the treatment. The primary goal is to assess safety, while secondary goals include response rates, hospital days, and quality of life. Protein levels will be checked in bone marrow and blood cells. During the study, participants visit the clinic every 4 weeks for checkups, blood tests measuring white blood cells and platelets, and other assessments. They will keep a diary to record symptoms throughout the study. Researchers will monitor hospitalizations, disease response, and safety indicators. The study is planned to run until March 2027, with regular monitoring to understand how the treatment affects patients over time.

A Study of Oral Tetrahydrouridine-Decitabine in Relapsed or Refractory Myelodysplastic Syndromes (MDS)