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Acute Myeloid LeukemiaPhase 1NCT04167917

NTX-301 in MDS/AML

A Phase 1 study testing NTX-301 in people with Acute Myeloid Leukemia and Myelodysplastic Syndromes, run by University of Alabama at Birmingham at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

NTX-301 is a DNMT1 inhibitor. The drug is an oral drug with preclinical data that has shown preclinical anti-leukemic efficacy. This is the first clinical trial using NTX-301 in patients with myeloid malignancies. Read more about this study

Who is it for?
Ages 18 years and older, men only
What is being tested?
NTX-301
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
22 participants
Where
2 sitesUnited States
Study dates
6 Jan 2021to 24 Apr 2025
Phase
Phase 1
Are there results?
None posted in the registry

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
  • Men only
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Able to understand and comply with the study procedures, understand the risks involved in the study, and provide written informed consent before the first study-specific procedure.
  • Men or women ≥18 years with one of the following conditions that is relapsed or refractory to at least one line of therapy:
  • Acute myeloid leukemia as long as with myeloblast percentage in the marrow is ≤ 30% or the peripheral white blood cell count is less than 20,000 cells/μL in the absence of leukoreducing therapy (e.g., hydroxyurea, leukapheresis)
  • MDS classified as intermediate, high, or very high risk by International Prognostic Scoring System-Revised [IPSS-R] criteria
  • CMML classified as intermediate-2 or high risk per CMML-specific prognostic scoring system (CPSS) or clinical/molecular CPSS (CPSS-mol) criteria
  • ECOG performance status of 0, 1, or 2
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  • Adequate organ function at screening defined as follows [reasonably minor changes pre-first dose are acceptable if deemed so by the investigator]:
  • Hepatic:
  • Total bilirubin ≤2 × upper limit of normal (ULN); isolated bilirubin > 2 is acceptable if participant has a diagnosis of Gilbert's syndrome
  • Aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) ≤3 × ULN.
  • Renal:
  • estimated glomerular filtration rate (by CKD-EPI method) ≥ 40 mL/min/1.73 m2 c) Cardiac:
  • Left ventricular ejection fraction greater than 45% or the institutional lower limit of normal by either ECHO or MUGA at entry.
  • Patients must have recovered to grade 1 or less from prior toxicity or adverse events (exception of myelosuppression - neutropenia, anemia, thrombocytopenia - and alopecia). Note: Participants with treatment-related toxicities that are unlikely to resolve per the investigator may be enrolled on a case-by-case basis after discussion with the medical monitor
  • Patients must have completed any chemotherapy, radiation therapy, or biologic therapy specific to their myeloid neoplasm ≥ 2 weeks or 5 half-lives (whichever is longer)
  • Able to swallow, retain, and absorb orally administered medication
  • Expected life ≥ 4 months
  • Participants with a prior history of stem cell transplant (autologous and/or allogeneic) are allowed if all of the following are met:
  • 90 days or more have elapsed from the time of transplant
  • subject has been off systemic immunosuppressive medications (including but not limited to: cyclosporine, tacrolimus, mycophenolate mofetil, or corticosteroids) for at least 30 days prior to the first dose of NTX-301. Topical steroids are permitted
  • no signs or symptoms of acute graft versus host disease, other than Grade 1 skin involvement.
  • there are no signs or symptoms of chronic graft versus host disease requiring systemic therapy
  • Women of child-bearing potential (according to recommendations of the Clinical Trial Facilitation Group [CTFG], CTFG 2014) must not be pregnant or breastfeeding and must have a negative pregnancy test at screening. A woman is considered of childbearing potential (ie, fertile) following menarche and until becoming postmenopausal, unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy. A man is considered fertile after puberty unless permanently sterile by bilateral orchidectomy.
  • Subjects and their partners with reproductive potential must agree to use 2 highly effective contraceptive measures during the study and must agree not to become pregnant or father a child for 3 months after the last dose of study treatment. Contraceptive measures that may be considered highly effective comprise combined hormonal contraception (oral, vaginal, or transdermal) or progestogen-only hormonal contraception (oral, injectable, implantable) associated with inhibition of ovulation, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, sexual abstinence, and surgically successful vasectomy. Abstinence is acceptable only if it is consistent with the preferred and usual lifestyle of the subject. Periodic abstinence (eg, calendar, ovulation, symptothermal, or post-ovulation methods) and withdrawal are not acceptable methods of birth control.

Who cannot join

  • Diagnosis or presence of any of the following:
  • acute promyelocytic leukemia
  • core-binding factor AML in first relapse
  • extramedullary leukemia
  • symptomatic or untreated Central Nervous System (CNS) disease [note that lumbar puncture (LP) is not required for study enrollment unless there is clinical suspicion for CNS disease; patients with history of CNS disease are permitted to enroll if they have previously received appropriate therapy and CNS remission has been; participants on maintenance intrathecal chemotherapy may be enrolled and continue to receive therapy]
  • Patients who are receiving any other investigational agents.
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  • Pregnant women and women who are breastfeeding are excluded from this study.
  • Patients with clinically significant illnesses which would compromise participation in the study, including, but not limited to:
  • severe or uncontrolled infection
  • known HIV infection requiring protease inhibitor therapy
  • known Hepatitis B; defined as presence of hepatitis B surface antigen (HBsAg)
  • known Hepatitis C; if Hepatitis C antibody is positive, then this is defined as positive Hepatitis C RNA polymerase chain reaction (PCR) (or comparable test)
  • uncontrolled diabetes and/or hypertension, symptomatic congestive heart failure, unstable angina pectoris, myocardial infarction within the past 6 months, uncontrolled cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements in the opinion of the investigator
  • Malabsorption syndrome or other conditions that would interfere with intestinal absorption.
  • History of a second malignancy, excluding non-melanoma skin cell cancer, within the last three years. Participants with second malignancies that were indolent, in situ or definitively treated may be enrolled even if less than three years have elapsed since treatment. Consult the monitor if there are any queries.
  • History of prior solid organ transplant
  • History of prior sensitivity reaction to any cytidine derivates

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35294

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

Other trials in United States

About this study

Official title: A Phase 1 Study of NTX-301, an Oral DNMT1 Inhibitor, in Patients With MDS and AML

NTX-301 is a DNMT1 inhibitor. The drug is an oral drug with preclinical data that has shown preclinical anti-leukemic efficacy. This is the first clinical trial using NTX-301 in patients with myeloid malignancies.

Being tested
NTX-301
Medical subject headings
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NTX-301Drug

    oral hypomethylating agent

Study groups in NCT04167917
Study groupRoleWhat they receive
NTX-301ExperimentalNot described
Receives: Drug: NTX-301

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety/tolerability: Incidence of treatment related adverse events (AEs) and dose-limiting toxicities (DLTs)

    Measured: 3 years

Secondary outcomes (9)

  1. Efficacy: Clinical Benefit Rate (CBR)

    Clinical Benefit Rate (CBR), as defined as the percentage of participants achieving a complete remission (CR), complete marrow remission (mCR), partial remission (PR), stable disease (SD) lasting at least 8 weeks, or hematologic improvement (HI), per International Working Group (IWG) criteria.

    Measured: 3 years

  2. Efficacy: Overall response rate (ORR)

    Overall response rate (ORR), defined as the percentage of participants achieving a CR, mCR, or PR, per IWG criteria.

    Measured: 3 years

  3. Efficacy: Progression free survival (PFS)

    Progression free survival (PFS), defined as time from first dose to disease progression, as defined by IWG criteria, or death due to any cause, whichever occurs earlier.

    Measured: 3 years

  4. Efficacy: Overall survival (OS)

    Overall survival (OS), defined as time from first dose to death due to any cause.

    Measured: 3 years

  5. Pharmacodynamics (PD): Global methylation (assay) in blood and/or marrow leukemia samples

    Measured: 3 years

  6. Pharmacokinetics (PK): Area under the curve (AUC)

    Measured: 3 years

  7. Pharmacokinetics (PK): Maximum plasma concentration (Cmax)

    Measured: 3 years

  8. Pharmacokinetics (PK): Time to reach maximum concentration (Tmax)

    Measured: 3 years

  9. Pharmacokinetics (PK): Half life (t1/2)

    Measured: 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Nov 2019
  2. Study start6 Jan 2021
  3. Primary completion24 Apr 2025
  4. Study completion24 Apr 2025

Resources

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
Masking
None (open label)
Enrolment
22 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04167917
Sponsor's protocol ID
IRB-300004830
Other IDs
Southern Research
Status last verified
Apr 2026
First submitted
12 Nov 2019
Last updated
1 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing NTX-301 in people with Acute Myeloid Leukemia and Myelodysplastic Syndromes, run by University of Alabama at Birmingham at 2 sites 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 24 inclusion and 17 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: finished, as of the sponsor's last update on 1 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety/tolerability: Incidence of treatment related adverse events (AEs) and dose-limiting toxicities (DLTs) (time frame: 3 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04167917.

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 1 May 2026.

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