Phase 1 Study of Oral Tetrahydrouridine-Decitabine to Treat Relapsed or Refractory Myelodysplastic Syndromes
Treebough Therapies
Summary
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
Description
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.
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Patients must have a diagnosis of MDS that has received one or more prior standard therapies and is relapsed or refractory * Patients must be 18 years of age or older * Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 3 * Patients must have adequate end-organ function * Patient's body weight must be ≥ 41 kg * Subjects must be able to understand and willing to sign a written informed consent document and complete study-related procedures. Exclusion Criteria: * Diagnosis of acute promyelocytic leukemia (APL) * Prior treatment with ≥…
Interventions
- DrugDecitabine
Oral tetrahydrouridine and oral decitabine capsule
Location
- Montefiore Einstein Medical CenterThe Bronx, New York