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

Launched by EPIDESTINY, INC. · May 28, 2025

Keywords

ClinConnect Summary

This clinical trial is exploring a new treatment for patients with relapsed or refractory myelodysplastic syndromes (MDS), a type of blood disorder. The study will test the combination of two drugs, tetrahydrouridine (THU) and decitabine (DEC), to see how safe and effective they are. Researchers want to find out if this combination can cause side effects, help improve patients' health, and reduce the number of days they need to stay in the hospital.

To participate in this trial, patients must be at least 18 years old and have been diagnosed with MDS that hasn’t responded to previous treatments. They should also have a certain level of health that allows them to participate in the study. If eligible, participants will take the study drugs once a week for 24 weeks and will visit the clinic every four weeks for checkups and tests. Additionally, they'll keep a diary to track any symptoms they experience. Please note that the trial is not yet recruiting participants, and there are specific criteria to ensure the safety and health of those involved.

Gender

ALL

Eligibility criteria

• 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 ≥4 28-day cycles of parenteral or oral decitabine
• • No other disease-directed therapy, save for hydroxyurea, including experimental or investigational drug therapy for 14 days prior to study entry (hydroxyurea should be discontinued ≥24 hours prior to initiation of study drug)
• • Requiring concomitant treatment with drugs that are cytidine deaminase (CDA) substrates and/or inhibitors, e.g., cytarabine, 5-azacytidine, gemcitabine
• • Currently pregnant or breastfeeding. Females of childbearing potential must have a negative serum pregnancy test within 72 hours of treatment start.
• • Uncontrolled intercurrent illness that could limit life expectancy or ability to complete study correlates
• • Women of Childbearing Potential (WOCBP) who are unwilling to agree to use dual contraceptive measures (i.e., hormonal or barrier method of birth control; abstinence, condom) prior to study entry, for the duration of study participation, and until 6 months after taking the last dose of THU/decitabine
• • Sexually active male who is unwilling to use a condom when engaging in any sexual contact with a WOCBP, beginning at the screening visit and continuing until 6 months after taking the last dose of THU/decitabine
• • 9. Patients with uncontrolled active human retrovirus (HIV) infection, as this will further increase the risk for opportunistic infections. However, patients with HIV with undetectable viral load by polymerase chain reaction (PCR), without opportunistic infection, and on a stable regimen of antiretroviral therapy are eligible