MDSPhase 1NCT07006025
A Study of Oral Tetrahydrouridine-Decitabine in Relapsed or Refractory Myelodysplastic Syndromes (MDS)
A Phase 1 study testing Decitabine in people with MDS, run by Treebough Therapies at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- MDS
- What is being tested?
- Decitabine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Jun 2025to 30 Mar 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Montefiore Einstein Medical Center
The Bronx, New York, 10467
Davina Hoban, 201-618-4725, dhoban@montefiore.org
Mendel Goldfinger, MD
About this study
Official title: Phase 1 Study of Oral Tetrahydrouridine-Decitabine to Treat Relapsed or Refractory Myelodysplastic Syndromes
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
Read the 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
- Being tested
- Decitabine
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DecitabineDrug
Also called Decagen
Oral tetrahydrouridine and oral decitabine capsule
| Study group | Role | What they receive |
|---|---|---|
| Active | Experimental | Oral capsules of THU followed 1 hour later by decitabine Receives: Drug: Decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
White blood cell measurement
Measure neutrophils count
Measured: every 4 weeks
Platelet count
Measure platelets in the blood
Measured: every 4 week
Secondary outcomes (2)
Response for high-risk MDS
Measure using International Working Group (IWG) 2023 response criteria
Measured: every 4 weeks
Hospitalization
Record the number of days of hospitalization
Measured: every 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2025
- Study start3 Jun 2025
- Primary completion30 Dec 2026 (estimated)
- Study completion30 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MDSStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single arm, open label
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Treebough Therapies
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- EpiDestiny, Inc.
- Responsible party
- Sponsor
- Investigators
- Mendel Goldfinger, MD, Principal investigator, Montefiore/Einstein Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07006025
- Sponsor's protocol ID
- 2024-00001
- Status last verified
- Jan 2026
- First submitted
- 20 May 2025
- Last updated
- 15 Jan 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Study of Oral Tetrahydrouridine-Decitabine to Treat Relapsed or Refractory Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT07006025
- Acronym / sponsor ID
- 2024-00001
- Conditions
- MDS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Treebough TherapiesIndustry (pharma, biotech, devices)
- Collaborators
- EpiDestiny, Inc.
- Study start
- 3 Jun 2025 (actual)
- Primary completion
- 30 Dec 2026 (estimated)
- Study completion
- 30 Mar 2027 (estimated)
- First posted
- 5 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single arm, open label
| Arm | Type | Receives |
|---|---|---|
| Active | Experimental | Drug: DecitabineOral capsules of THU followed 1 hour later by decitabine |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DecitabineAlso: DecagenOral tetrahydrouridine and oral decitabine capsule | Drug | Active |
Primary outcome
White blood cell measurement
Time frame: every 4 weeks
Measure neutrophils count
Platelet count
Time frame: every 4 week
Measure platelets in the blood
2 secondary outcomes
Response for high-risk MDS
Time frame: every 4 weeks
Hospitalization
Time frame: every 4 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Mendel Goldfinger, MD principal investigator
Montefiore/Einstein Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Decitabine in people with MDS, run by Treebough Therapies at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Davina Hoban (201-618-4725, dhoban@montefiore.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
White blood cell measurement (time frame: every 4 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jan 2026.