Actively Recruiting
Phase 1 Study of Oral Tetrahydrouridine-Decitabine to Treat Relapsed or Refractory Myelodysplastic Syndromes
Led by Treebough Therapies · Updated on 2026-01-15
20
Participants Needed
1
Research Sites
12 weeks
Total Duration
On this page
Sponsors
T
Treebough Therapies
Lead Sponsor
E
EpiDestiny, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of a combination drug made of tetrahydrouridine (THU) and decitabine (DEC) for patients with relapsed or refractory myelodysplastic syndrome (MDS). This single-arm, open-label Phase 1 study aims to find out if the treatment causes blood-related or other side effects, and whether it improves patients' health and reduces hospital stays. The study also measures protein levels related to the disease and tracks quality of life. Participants will take oral capsules of THU followed one hour later by decitabine once a week for 24 weeks, provided there is no clear evidence the disease is progressing. There is no placebo group as all patients receive the treatment. The primary goal is to assess safety, while secondary goals include response rates, hospital days, and quality of life. Protein levels will be checked in bone marrow and blood cells. During the study, participants visit the clinic every 4 weeks for checkups, blood tests measuring white blood cells and platelets, and other assessments. They will keep a diary to record symptoms throughout the study. Researchers will monitor hospitalizations, disease response, and safety indicators. The study is planned to run until March 2027, with regular monitoring to understand how the treatment affects patients over time.
CONDITIONS
Brief Title
A Study of Oral Tetrahydrouridine-Decitabine in Relapsed or Refractory Myelodysplastic Syndromes (MDS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of myelodysplastic syndrome (MDS) that has relapsed or is refractory after one or more prior standard therapies
- Age 18 years or older
- Eastern Cooperative Oncology Group (ECOG) performance status of 3 or less
- Adequate end-organ function
- Body weight of at least 41 kg
- Ability to understand and willing to sign informed consent and complete study procedures
You will not qualify if you...
- Diagnosis of acute promyelocytic leukemia (APL)
- Prior treatment with 4 or more 28-day cycles of parenteral or oral decitabine
- No other disease-directed therapy, except hydroxyurea, within 14 days prior to study entry
- Use of drugs that are cytidine deaminase (CDA) substrates or inhibitors during the study
- Currently pregnant or breastfeeding
- Females of childbearing potential without a negative pregnancy test within 72 hours before treatment
- Uncontrolled illness limiting life expectancy or study completion
- Women of childbearing potential unwilling to use dual contraceptive methods during and 6 months after the study
- Sexually active males unwilling to use condoms during and 6 months after the study
- Uncontrolled active HIV infection (patients with undetectable viral load and stable therapy are eligible)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 24 weeks in the absence of progressive disease
Participants receive oral tetrahydrouridine followed by oral decitabine capsules to treat relapsed or refractory myelodysplastic syndromes.
Visits every 4 weeks during treatment
Trial Site Locations
Total: 1 location
1
Montefiore Einstein Medical Center
The Bronx, New York, United States, 10467
Actively Recruiting
Research Team
D
Davina Hoban
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here