NTX-301 for Leukemia

Not currently recruiting at 1 trial location

Overseen By Pankit Vachhani

Age: 18+

Sex: Male

Trial Phase: Phase 1

Sponsor: University of Alabama at Birmingham

Must not be taking: Protease inhibitors

Disqualifiers: Acute promyelocytic leukemia, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 3 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral drug, NTX-301, to determine its effectiveness in treating certain blood cancers known as myeloid malignancies, including specific forms of leukemia. NTX-301 blocks an enzyme that may aid cancer growth, and this trial marks its first use in patients. Patients who have tried other treatments without success, especially those with frequent relapses, might be suitable candidates. Participants should be able to take oral medication and should not have severe organ issues or other significant health problems. As a Phase 1 trial, this research aims to understand how NTX-301 works in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I have to stop taking my current medications for the NTX-301 trial?

The trial requires that participants stop taking systemic immunosuppressive medications at least 30 days before starting NTX-301. Additionally, any chemotherapy, radiation, or biologic therapy specific to the myeloid neoplasm must be completed at least 2 weeks or 5 half-lives before starting the trial. Other medications are not specifically mentioned, so it's best to discuss with the trial team.

Is there any evidence suggesting that NTX-301 is likely to be safe for humans?

Research shows that NTX-301 is undergoing its first human tests to assess safety. This drug has shown promise in lab studies by targeting a protein that aids cancer cell survival. However, as this is an early human trial, safety data from patients is not yet available.

In lab studies, the drug effectively targeted leukemia cells, outperforming some other treatments by reducing cancer cell survival. But since NTX-301 has not been administered to humans before, information about its effects and potential side effects remains limited.

This is a Phase 1 trial, focusing on determining the drug's safety for humans. Participants in this early trial will provide crucial information about its safety and tolerability. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

NTX-301 is unique because it offers a new mechanism of action for treating leukemia. Unlike standard treatments that often involve chemotherapy targeting rapidly dividing cells, NTX-301 is designed to specifically target cancer cells at a molecular level, potentially reducing harm to healthy cells. This precision approach could lead to fewer side effects and a better quality of life for patients. Researchers are excited about NTX-301 because it has the potential to be more effective and safer than current options, providing a much-needed alternative for leukemia patients.

What evidence suggests that NTX-301 might be an effective treatment for leukemia?

Research has shown that NTX-301, the investigational treatment in this trial, has potential for treating leukemia. It blocks DNMT1, a protein involved in DNA changes that can affect gene function. In lab tests, NTX-301 significantly lowered DNMT1 levels and reduced the survival and growth of various leukemia cells. This effect surpassed that of 5-azacytidine, a common treatment. NTX-301 also encouraged cancer cells to behave more like normal blood cells. Although these findings come from early research, they provide a strong rationale for studying NTX-301 in clinical trials. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

Adults (≥18 years) with certain types of blood cancers that have not responded to previous treatments can join this trial. They must be able to take oral medication, have a life expectancy of at least 4 months, and their major organs need to function well. Women who could get pregnant and men must use effective birth control. People with acute promyelocytic leukemia, severe infections, malabsorption issues, recent other cancers or organ transplants cannot participate.

Inclusion Criteria

Aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) ≤3 × ULN.

I haven't taken any immune-weakening medications for at least 30 days.

I can take pills by mouth and my body can absorb them.

Exclusion Criteria

Patients with clinically significant illnesses which would compromise participation in the study, including, but not limited to:

My AML with core-binding factor is in its first relapse.

I have been diagnosed with acute promyelocytic leukemia.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive NTX-301, an oral DNMT1 inhibitor, for the treatment of MDS and AML

3 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 years

What Are the Treatments Tested in This Trial?

Interventions

• NTX-301

Trial Overview The trial is testing NTX-301, an oral DNMT1 inhibitor designed to treat myeloid malignancies like Myelomonocytic Leukemia and Acute Myeloid Leukemia. This is the first time it's being used in humans after showing promise in preclinical studies.

How Is the Trial Designed?

Group I: NTX-301 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

University of Alabama at Birmingham

Lead Sponsor

Trials

1,677

Recruited

2,458,000+

Citations

1. nature.com nature.com/articles/s41408-022-00664-y

The preclinical efficacy of the novel hypomethylating agent ... Consistent with its observed efficacy, NTX-301 promoted greater transcriptional reprogramming toward a normal myeloid-like signature [9] than ...

2. ashpublications.org ashpublications.org/blood/article/142/Supplement%201/4158/504611/Novel-Hypomethylating-Agent-Ntx-301-Exhibits-Anti

Novel Hypomethylating Agent Ntx-301 Exhibits Anti-Leukemia ... NTX-301 greatly reduced the levels of DNMT1 and decreased viability in various AML cells, markedly more effective than 5-azacytide (5-Aza).

3. clinicaltrials.gov clinicaltrials.gov/study/NCT04167917

Study Details | NCT04167917 | NTX-301 in MDS/AML 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 ...

4. ascopubs.org ascopubs.org/doi/10.1200/JCO.2022.40.16_suppl.TPS7077

A phase 1 study of NTX-301, an oral DNMT1 inhibitor, in ... Preclinical data suggest a correlation between NTX-301 activity in leukemia xenograft models and decreased levels of DNMT1. NTX-301 offers ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10046470/

Integrative Analyses Reveal the Anticancer Mechanisms and ... In a recent companion paper, we clearly demonstrated that NTX-301 exhibited better therapeutic potential than conventional HMAs in preclinical ...