TTX-MC138 for Advanced Cancer

Not currently recruiting at 3 trial locations

Overseen By Susan Duggan

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: TransCode Therapeutics

Must not be taking: Traditional medicines

Disqualifiers: CNS metastases, Cardiovascular disease, Autoimmune, others

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

Compensation: Varies

Number of Visits: 1

Duration: 28-day cycles

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called TTX-MC138, a nanoparticle-conjugated antisense oligonucleotide, to evaluate its effectiveness for people with advanced solid tumors, where the cancer has spread or cannot be surgically removed. Researchers are testing different doses to determine the safest and most effective amount. Suitable participants have previously tried other treatments without success and have a type of cancer that lacks standard treatment options. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new therapy.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, you cannot take traditional or herbal medicines for tumors that can't be stopped during the study.

Is there any evidence suggesting that TTX-MC138 is likely to be safe for humans?

Research has shown that TTX-MC138 was generally safe in earlier studies. The main goal was to ensure the treatment's safety, and researchers achieved this goal. Patients used TTX-MC138 for 2 to 12 months, with some still continuing. This indicates that any side effects were manageable and not severe enough to warrant early discontinuation. Although specific side effects aren't listed, the continued treatment by some patients suggests it is reasonably safe. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for advanced cancer focus on killing cancer cells directly, often affecting healthy cells too. TTX-MC138 is unique because it targets microRNA, molecules that help regulate gene expression, which can stop cancer from spreading without harming healthy cells. Researchers are excited because this approach could offer a more precise and potentially less harmful way to control cancer growth. Additionally, TTX-MC138 is being tested in multiple dose levels to find the most effective and safest option, a flexibility that's crucial in personalized cancer treatment.

What evidence suggests that TTX-MC138 might be an effective treatment for advanced cancer?

Research has shown that TTX-MC138 targets a molecule called miR-10b, which is linked to cancer growth. This molecule often appears in higher amounts in various spreading cancers. By blocking miR-10b, TTX-MC138 may help prevent cancer cells from spreading. In early studies, patients safely received TTX-MC138, with some continuing treatment for several months. These findings suggest that TTX-MC138 could potentially treat advanced solid tumors. Participants in this trial will receive different dose levels of TTX-MC138 to evaluate its safety and effectiveness. ^{1 2 4 5 6}

Are You a Good Fit for This Trial?

Adults over 18 with advanced solid tumors that have relapsed or are not responding to standard treatments can join this trial. They must have a life expectancy of at least 3 months, acceptable liver and blood function, and an ECOG performance status of 0-2, indicating they are still able to carry out daily activities.

Inclusion Criteria

I can take care of myself but might not be able to do heavy physical work.

My blood counts and organ functions are within the required ranges.

Measurable or evaluable disease per RECIST version 1.1

Exclusion Criteria

History of hypersensitivity to study drug components or similar drugs

I am on medication for an autoimmune disease.

I have not had major surgery within the last 28 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 4 weeks

Treatment

Participants receive TTX-MC138 in 28-day cycles with dosing on Day 1

28-day cycles

1 visit per cycle (in-person)

Survival Follow-up

Participants are monitored every 3 months for survival after treatment

every 3 months

What Are the Treatments Tested in This Trial?

Interventions

• TTX-MC138

Trial Overview The study is testing TTX-MC138's safety and effectiveness in different doses for those with advanced solid tumors. It's an early-stage trial (Phase 1/2) where everyone gets the drug; there's no comparison group.

How Is the Trial Designed?

Group I: Dose level 3 Experimental Treatment 1 Intervention

3.2 mg/kg of TTX-MC138

Group II: Dose level 2 Experimental Treatment 1 Intervention

0.8 mg/kg of TTX-MC138

Group III: Dose level 1 Experimental Treatment 1 Intervention

0.4 mg/kg of TTX-MC138

Who Is Running the Clinical Trial?

TransCode Therapeutics

Lead Sponsor

Trials

2

Recruited

20+

Published Research Related to This Trial

A novel miRNA-targeted nanodrug effectively inhibits the pro-metastatic microRNA-10b, preventing and arresting lymph node metastasis in invasive breast cancer cells, specifically MDA-MB-231.

In mouse models, this nanodrug accumulates in both primary tumors and lymph nodes, successfully preventing metastasis when administered early and halting existing lymph node metastases without affecting the growth of the primary tumor.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Context-dependent differences in miR-10b breast oncogenesis can be targeted for the prevention and arrest of lymph node metastasis. Yigit, MV., Ghosh, SK., Kumar, M., et al.[2021]

The study introduces a novel nanoparticle platform that effectively delivers dual siRNAs targeting telomerase and telomeres, showing promise for treating paclitaxel-resistant non-small-cell lung cancer (A549/PTX) by down-regulating key genes involved in tumor growth.

In vitro and in vivo experiments demonstrated that this platform enhances siRNA stability, promotes tumor cell internalization, and significantly inhibits tumor growth and neovascularization, suggesting a new strategy for overcoming drug resistance in cancer therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A tumor-activatable peptide supramolecular nanoplatform for the delivery of dual-gene targeted siRNAs for drug-resistant cancer treatment. Wu, Y., Zhong, D., Li, Y., et al.[2021]

The study developed folate-targeted hybrid lipo-polymeric nanoplexes that effectively co-deliver the anticancer drug DTX and the microRNA miR-34a, achieving a high drug entrapment efficiency of 94.8% and a sustained release profile over 48 hours.

In vivo tests showed that these nanoplexes significantly improved drug circulation and reduced toxicity, with no adverse effects observed at a dose of 10 mg/kg, while also enhancing cytotoxicity and apoptosis in breast cancer cells compared to free treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Folate targeted hybrid lipo-polymeric nanoplexes containing docetaxel and miRNA-34a for breast cancer treatment. Sharma, S., Pukale, S., Sahel, DK., et al.[2022]

A cationic lipid-based delivery system for miR-133b significantly improved the expression of this tumor suppressor in lung cancer cells, achieving a 2.3-fold increase compared to traditional delivery methods.

In vivo studies showed that this delivery system effectively targeted lung tissue, with a 30% accumulation of the treatment, leading to a 52-fold increase in mature miR-133b expression, suggesting its potential as a therapeutic approach for lung cancer.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

MicroRNA delivery by cationic lipoplexes for lung cancer therapy. Wu, Y., Crawford, M., Yu, B., et al.[2021]

The study developed a novel nanoparticle system (LbL miR708/PTX-MTNst) that effectively co-delivers the chemotherapy drug paclitaxel and microRNA 708 to suppress c-FLIP, a protein that promotes cancer cell survival and chemotherapy resistance, showing superior cytotoxicity in colorectal cancer cell lines compared to traditional treatments.

In animal models, this nanoparticle system led to significant tumor regression and increased apoptosis in colorectal carcinoma, demonstrating both efficacy in targeting tumors and safety through biocompatibility, suggesting its potential for clinical application.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Polyamino Acid Layer-by-Layer (LbL) Constructed Silica-Supported Mesoporous Titania Nanocarriers for Stimuli-Responsive Delivery of microRNA 708 and Paclitaxel for Combined Chemotherapy. Gupta, B., Ruttala, HB., Poudel, BK., et al.[2019]

Antisense oligonucleotides targeting specific point mutations in the Ha-ras oncogene can effectively inhibit tumor growth, demonstrating their potential as a targeted cancer therapy.

By using polyalkylcyanoacrylate nanoparticles to deliver these oligonucleotides, researchers achieved a 100-fold increase in efficacy, allowing for lower concentrations to effectively suppress cell proliferation and tumor growth in mice.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Antisense oligonucleotides adsorbed to polyalkylcyanoacrylate nanoparticles specifically inhibit mutated Ha-ras-mediated cell proliferation and tumorigenicity in nude mice. Schwab, G., Chavany, C., Duroux, I., et al.[2019]

The study developed nanoparticles (PAM-Ap/pMiR-34a NPs) that effectively deliver MicroRNA-34a, a tumor suppressor, specifically targeting non-small cell lung cancer (NSCLC) cells, enhancing cellular uptake and gene transfection efficiency.

These targeted nanoparticles significantly inhibited lung cancer cell growth, migration, and invasion, while also inducing apoptosis, suggesting a promising new therapeutic strategy for treating NSCLC.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Aptamer-Dendrimer Bioconjugates for Targeted Delivery of miR-34a Expressing Plasmid and Antitumor Effects in Non-Small Cell Lung Cancer Cells. Wang, H., Zhao, X., Guo, C., et al.[2022]

In a murine model of metastatic breast cancer, combining a miR-10b-inhibitory nanodrug with low-dose anthracycline led to complete and lasting regressions of metastatic disease, highlighting a potential new treatment strategy.

The study found that miR-10b is overexpressed in highly metastatic cells, suggesting it could serve as a specific therapeutic target for treating metastasis, as the nanodrug demonstrated strong anti-proliferative and pro-apoptotic effects on these cells.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Combining miR-10b-Targeted Nanotherapy with Low-Dose Doxorubicin Elicits Durable Regressions of Metastatic Breast Cancer. Yoo, B., Kavishwar, A., Ross, A., et al.[2023]

Citations

1. ir.transcodetherapeutics.com ir.transcodetherapeutics.com/2025-10-14-TransCode-Therapeutics-presents-preliminary-data-from-its-completed-Phase-1a-study-with-TTX-MC138-in-metastatic-disease-at-ESMO

News Releases (NASDAQ: RNAZ) announces completion of Phase 1a clinical trial with TTX-MC138, an investigational inhibitor of microRNA-10b, or miR-10b. By ...

2. go.drugbank.com go.drugbank.com/drugs/DB18628

TTX-MC138: Uses, Interactions, Mechanism of Action TTX-MC138 is an antisense oligonucleotide targeting miR-10b. It is under investigation for the treatment of various metastatic cancers.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06260774

Study of TTX-MC138 in Subjects With Advanced Solid ... The study will consist of 2 phases, dose escalation and dose expansion, with 3 study periods: Screening (up to 28 days), Treatment (28-day treatment cycles, ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12010629/

MicroRNA in cancer therapy: breakthroughs and ... This review examines the dual role of miRNAs in solid and hematological malignancies, starting from their dysregulation in various cancer types.

5. prnewswire.com prnewswire.com/news-releases/transcode-therapeutics-presents-preliminary-data-from-its-completed-phase-1a-study-with-ttx-mc138-in-metastatic-disease-at-esmo-302582916.html

TransCode Therapeutics presents preliminary data from its ... Safety primary endpoint achieved Median treatment duration of 4 months with a range of 2 to 12 months and three patients remain on trial ...

6. ir.transcodetherapeutics.com ir.transcodetherapeutics.com/2024-09-27-TransCode-Therapeutics-Announces-First-Patients-Treated-in-Phase-1-Clinical-Trial-with-First-in-Class-Lead-Therapeutic-Candidate

News Releases TTX-MC138 is an antisense oligonucleotide conjugated to TransCode's proprietary TTX delivery system designed to inhibit microRNA-10b, a known driver of ...