NCT07006025

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

Phase 1 Study of Oral Tetrahydrouridine-Decitabine to Treat Relapsed or Refractory Myelodysplastic Syndromes

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Treebough Therapies · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Detailed 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.

Conditions

• MDS

Interventions

Type	Name	Description
DRUG	Decitabine	Oral tetrahydrouridine and oral decitabine capsule

Timeline

• Start date: 2025-06-03
• Primary completion: 2026-12-30
• Completion: 2027-03-30
• First posted: 2025-06-05
• Last updated: 2026-01-15

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study