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

NCT07006025 | Recruiting Phase 1 FDA Drug

• 1 other identifier: 2024-00001
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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

Conditions

MDS

Outcome Measures

Primary Outcomes (2)

• White blood cell measurement

  Measure neutrophils count

  every 4 weeks

• Platelet count

  Measure platelets in the blood

  every 4 week

Secondary Outcomes (2)

• Response for high-risk MDS

  every 4 weeks

• Hospitalization

  every 4 weeks

Study Arms (1)

Active

EXPERIMENTAL

Oral capsules of THU followed 1 hour later by decitabine

Drug: Decitabine

Interventions

Decitabine DRUG

Oral tetrahydrouridine and oral decitabine capsule

Also known as: Decagen

Active

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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
• \. 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

Sponsors & Collaborators

• Treebough Therapies: lead
• EpiDestiny, Inc.: collaborator

Study Sites (1)

Montefiore Einstein Medical Center

The Bronx, New York, 10467, United States

RECRUITING

MeSH Terms

Interventions

Decitabine

Intervention Hierarchy (Ancestors)

Azacitidine; Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• Mendel Goldfinger, MD

  Montefiore/Einstein Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Davina Hoban

CONTACT

201-618-4725; dhoban@montefiore.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single arm, open label

Study Record Dates

• First Submitted: May 20, 2025
• First Posted: June 5, 2025
• Study Start: June 3, 2025
• Primary Completion (Estimated): December 30, 2026
• Study Completion (Estimated): March 30, 2027
• Last Updated: January 15, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)