Oral tetrahydrouridine plus decitabine for relapsed or refractory MDS

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This single-arm, open-label Phase 1 trial gives oral tetrahydrouridine (THU) with decitabine once weekly for up to 24 weeks to adults with relapsed or refractory myelodysplastic syndromes. The primary goal is to define safety and capture hematologic and nonhematologic toxicities. Secondary outcomes include response rates by International Working Group criteria, quality of life measures, number of days hospitalized, and measurement of DNMT1 protein levels in bone marrow and peripheral blood. Participants keep symptom diaries and attend clinic visits approximately every four weeks for testing and follow-up.

Why it matters

Who can join: the full rules

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

Where it's running

• Montefiore Einstein Medical Center
  The Bronx, New York, United States Recruiting

Study contacts

• Principal investigator: Mendel Goldfinger, MD — Montefiore/Einstein Cancer Center
• Study coordinator: Davina Hoban
• Email: dhoban@montefiore.org
• Phone: 201-618-4725

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.