MTI-301 for Cancer

Overseen By Clinical Trials Referral Office

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Mayo Clinic

Must not be taking: QT prolonging, CYP inhibitors

Disqualifiers: Pregnancy, HIV, Hepatitis, Cardiac, others

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

Compensation: Varies

Number of Visits: 3

Duration: 24 weeks

42 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and optimal dose of a new drug, MTI-301, for individuals with solid cancers that have spread or cannot be surgically removed and have not responded to other treatments. MTI-301 blocks an enzyme that aids tumor growth and spread. The trial seeks participants with solid tumors unresponsive to standard treatments. Those with a solid tumor that has spread or cannot be removed and has not improved with other treatments may be suitable for this trial. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications that are known to interact with major enzymes or transporters at least 14 days before joining. If you're on these medications, you may need to stop or switch them, but the protocol doesn't specify all medications, so check with the trial team.

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

Research has shown that MTI-301, a new drug, is being tested for safety in patients with certain types of cancer. This drug targets an enzyme that aids tumor growth and spread, so blocking it might slow cancer progression.

As MTI-301 is in the early testing phase, limited information exists on its tolerability. However, early trials aim to determine the safest dose and observe the body's response to the treatment. Researchers closely monitor any side effects and assess how the drug is processed in the body.

For those considering joining a trial, it's important to understand that these studies are crucial for determining the safety and effectiveness of a new treatment. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard cancer treatments that often rely on chemotherapy, radiation, or immunotherapy, MTI-301 is unique because it is administered orally and may offer a more patient-friendly option. Researchers are excited about MTI-301 because it uses a novel active ingredient that targets cancer cells in a new way, potentially leading to fewer side effects and better outcomes. This approach could provide a breakthrough for patients who do not respond well to existing therapies.

What evidence suggests that MTI-301 might be an effective treatment for cancer?

Research has shown that MTI-301, the treatment under study in this trial, targets a protein called SCD1, which is linked to the growth and spread of some cancers. By blocking SCD1, MTI-301 might slow tumor growth and help prevent cancer from spreading. Early results suggest it could also enhance tumor response to other treatments, such as immune therapies, which assist the body in fighting cancer. Although more research is needed to confirm its effectiveness, these early signs are promising for patients with difficult-to-treat cancers that have spread or cannot be removed by surgery. ^{1 2 4 6 7}

Who Is on the Research Team?

Winston Tan, MD

Principal Investigator

Mayo Clinic

Are You a Good Fit for This Trial?

This trial is for patients with solid tumors that have spread or can't be surgically removed and haven't improved after treatment. Participants must have measurable disease, acceptable organ function, and no other options of curative therapy.

Inclusion Criteria

Measurable disease as defined by Response Evaluation Criteria in Solid Tumors (RECIST) criteria or evaluable disease

Absolute neutrophil count (ANC) ≥ 1500/mm^3 (obtained ≤ 28 days prior to registration)

Platelet count ≥ 100,000/mm^3 (obtained ≤ 28 days prior to registration)

Exclusion Criteria

Persons who are of childbearing potential who are unwilling to employ adequate contraception

Corrected QT (QTc) prolongation based on QTc interval prior to registration of ≥ 470 ms using the Fridericia's formula (QTcF)

Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive MTI-301 orally once daily on days 1-28 of each cycle, with cycles repeating every 28 days for up to 6 cycles

24 weeks

Regular imaging and blood sample collection throughout the study

Follow-up

Participants are monitored for safety and effectiveness after treatment completion

1 year

Follow-up every 3 months

What Are the Treatments Tested in This Trial?

Interventions

• MTI-301

Trial Overview The trial tests the safety and optimal dose of MTI-301, a drug targeting SCD1—an enzyme aiding tumor growth—in patients with advanced or stubborn solid cancers. It includes scans like CT, MRI, PET, biospecimen collection, and questionnaires.

How Is the Trial Designed?

Group I: Treatment (MTI-301) Experimental Treatment 6 Interventions

Patients receive MTI-301 PO QD on days 1-28 of each cycle. Cycles repeat every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo CT, PET/CT, or MRI and collection of blood samples throughout the study, and undergo tissue sample collection at baseline and at disease progression.

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Published Research Related to This Trial

In a phase I study involving 26 patients with refractory solid tumors, the combination of docetaxel and temsirolimus was limited by severe dose-limiting toxicities (DLTs), such as grade 4 neutropenia and grade 3 diarrhea, preventing the determination of a maximal tolerated dose (MTD).

The study's innovative Bayesian Optimal Interval design indicated similar DLT rates at different dose levels, but ultimately, patients could not tolerate the treatment regimen without dose reductions, suggesting that this combination should not be pursued further at the tested doses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase I Study of Docetaxel and Temsirolimus in Refractory Solid Tumors. Amin, M., Gao, F., Terrero, G., et al.[2023]

In a 24-week study involving 507 patients with active rheumatoid arthritis, tofacitinib dosages of 3 mg twice daily or higher significantly improved symptoms compared to placebo, with ACR20 response rates exceeding 50%.

Tofacitinib was generally well-tolerated, with manageable side effects such as diarrhea and upper respiratory infections, and showed sustained efficacy in improving various health measures over the study period.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A phase IIb dose-ranging study of the oral JAK inhibitor tofacitinib (CP-690,550) versus placebo in combination with background methotrexate in patients with active rheumatoid arthritis and an inadequate response to methotrexate alone. Kremer, JM., Cohen, S., Wilkinson, BE., et al.[2022]

The study found that an oral drug combining paclitaxel and HM30181A is well tolerated in patients with advanced cancers, with only one case of significant toxicity (grade 3 neutropenia) reported at a dose of 240 mg/m².

The recommended phase II dose for this combination therapy is 300 mg/m² of paclitaxel, which effectively achieves the desired plasma concentration for treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Phase I Study of Oral Paclitaxel with a Novel P-Glycoprotein Inhibitor, HM30181A, in Patients with Advanced Solid Cancer. Lee, HJ., Heo, DS., Cho, JY., et al.[2021]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06911008

NCT06911008 | MTI-301 for the Treatment of Metastatic or ... MTI-301 may disrupt the activity of SCD1, which may lead to reduced tumor growth and/or spread. PRIMARY OBJECTIVE:

2. cancer.gov cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2025-01930

MTI-301 for the Treatment of Metastatic or Unresectable ... This phase I trial tests the safety, side effects, and best dose of MTI-301 in treating patients with solid cancers that have spread from where they first ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06911008?term=AREA%5BBasicSearch%5D(PIPERIDINE)&rank=4

MTI-301 for the Treatment of Metastatic or Unresectable ... MTI-301 may disrupt the activity of SCD1, which may lead to reduced tumor growth and/or spread. PRIMARY OBJECTIVE:

4. reporter.nih.gov reporter.nih.gov/project-details/10913640

Modulation of cancer induced immune suppression via ... Therapeutically useful, MTI-301 increases the immunogenicity of poorly immunogenic tumors thereby sensitizing to immune checkpoint blockade, leading to dramatic ...

5. mayo.edu mayo.edu/research/centers-programs/cancer-research/specialized-programs-research-excellence-spore-grants/hepatobiliary/clinical-trials

Clinical trials A Phase 1 clinical trial will test the safety of a novel first-in-class SCD1 inhibitor in cancer called MTI-301 (also known as SSI-4).

6. withpower.com withpower.com/trial/phase-1-neoplasms-3-2025-efc2e

MTI-301 for Cancer · Info for Participants This phase I trial tests the safety, side effects, and best dose of MTI-301 in treating patients with solid cancers that have spread from where they first ...

7. synapse.patsnap.com synapse.patsnap.com/drug/122e8b1ed3a64489bb6e8ecf7a913483

MTI-301 - Drug Targets, Indications, Patents ... Safety, Tolerability, Pharmacokinetics, and Activity of MTI-301 in Patients With Advanced Malignancy. 100 Clinical Results associated with MTI-301. Login to ...