AMXT 1501 + DFMO for Breast Cancer and Melanoma

Recruiting at 4 trial locations

Overseen By Mark Burns, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Aminex Therapeutics, Inc.

Must be taking: LHRH agonist

Must not be taking: Biotin, Adriamycin

Disqualifiers: CNS pathology, Cardiovascular disease, Autoimmune disease, others

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

Compensation: Varies

Number of Visits: 1

92 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug combination, AMXT 1501 Dicaprate and DFMO, to determine their safety and effectiveness in treating advanced breast cancer and melanoma, both challenging to treat with current options. For breast cancer, the trial seeks patients with advanced cancer that continues to grow despite previous treatments. In the melanoma group, it targets those whose cancer cannot be surgically removed or has spread, especially if other treatments have failed. Participants will receive these drugs alongside standard therapies to assess whether the combination improves outcomes. As a Phase 1 and Phase 2 trial, this research aims to understand how the treatment works in people and measure its effectiveness in an initial, smaller group, offering participants a chance to be among the first to potentially benefit from 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, certain treatments like radiation, chemotherapy, or immunotherapy must be stopped a few weeks before starting the trial. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that the combination of AMXT 1501 and DFMO appears safe. In earlier studies, patients generally tolerated these drugs well. For those with breast cancer, significant tumor control occurred with only minor side effects after a week of treatment.

DFMO, in particular, is known for its low toxicity. Most patients managed it well, with very few needing to adjust doses or stop treatment due to side effects. However, long-term use of DFMO has been linked to ear-related issues, known as ototoxicity.

While AMXT 1501 and DFMO are still being tested for safety and effectiveness in this study, they have been used safely in other conditions. The FDA has granted them special status for treating neuroblastoma, a type of childhood cancer, which supports their potential safety.

Overall, while the treatment is still under study, early results suggest it is well-tolerated, making it a potentially safe option for those considering joining the trial. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about AMXT 1501 combined with DFMO for breast cancer and melanoma because they offer a fresh approach. Unlike traditional treatments for breast cancer, which often focus on hormone or HER2 receptor targeting, AMXT 1501 and DFMO work together to inhibit polyamine synthesis, a process crucial for cancer cell growth. This combination is unique because it targets cancer metabolism, potentially offering an effective treatment even for cancers that are resistant to current therapies. Meanwhile, for melanoma, this combo is paired with pembrolizumab, a well-known immunotherapy, which could enhance the immune system's ability to attack cancer cells more effectively. This dual approach may provide new hope for patients with limited options.

What evidence suggests that this trial's treatments could be effective for breast cancer and melanoma?

Studies have shown that combining AMXT 1501 and DFMO can significantly slow tumor growth. In this trial, participants with breast cancer (ER+ HER2-) will receive AMXT 1501 and DFMO along with Fulvestrant and Capivasertib. Early results suggest these drugs can nearly halt tumor growth in some patients after just one week. Participants with melanoma will receive AMXT 1501 and DFMO along with Pembrolizumab. Animal research indicates this treatment can effectively delay tumor growth. DFMO, a drug in this combination, has been found to extend patient survival when used after other treatments. The FDA has recognized the potential of these drugs by granting them special designations for use in certain cancers, highlighting their promise. ^{1 2 3 4 6}

Who Is on the Research Team?

Deyaa Adib, MD

Principal Investigator

Aminex Therapeutics, Inc.

Are You a Good Fit for This Trial?

This trial is for adults with advanced solid tumors: specifically, ER+/HER2- breast cancer or unresectable/metastatic cutaneous melanoma. Participants must be suitable for standard treatments like fulvestrant, capivasertib (breast cancer), or pembrolizumab (melanoma).

Inclusion Criteria

Life expectancy of at least 12 weeks

Understand and sign the informed consent form (ICF) and be willing to comply with all study procedures before any study specific procedures are conducted

Has evaluable or measurable disease by tumor Response Evaluable Criteria in Solid Tumors version 1.1 (RECIST 1.1) at the time of enrollment

Exclusion Criteria

History or presence of ECG abnormalities

Unwillingness or inability to comply with required procedures in the protocol

Serious nonmalignant disease that could compromise protocol objectives

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Phase 1b (Safety Run-In)

Evaluate safety, tolerability, dose-limiting toxicities, and identify the recommended Phase 2 dose using a 3+3 dose-escalation design

Approximately 8-12 weeks

Multiple visits (in-person)

Phase 2 (Dose Expansion)

Evaluate the efficacy of the combination regimens using objective response rate, duration of response, and other clinical measures

24 months

Regular visits (in-person and virtual)

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Periodic visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• AMXT 1501 Dicaprate
• DFMO

Trial Overview

The study tests AMXT 1501 and DFMO combined with standard therapies in two groups: one with breast cancer patients receiving fulvestrant and capivasertib, another with melanoma patients getting pembrolizumab. It aims to determine the safest dose and assess effectiveness.

How Is the Trial Designed?

Group I: Arm 2: Melanoma Experimental Treatment 3 Interventions

Arm 2: Melanoma Cohort (Cohort 2: Melanoma) AMXT 1501 oral DFMO oral Pembrolizumab IV

Group II: Arm 1: Breast Cancer (ER+ HER2-) Experimental Treatment 4 Interventions

Arm 1: Breast Cancer Cohort (Cohort 1: ER+ / HER2-) AMXT 1501 oral DFMO oral Fulvestrant IM Capivasertib PO

Who Is Running the Clinical Trial?

Aminex Therapeutics, Inc.

Lead Sponsor

Trials

4

Recruited

320+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07287917?cond=cancer

Study of AMXT 1501 and DFMO in Combination With ...

Preclinical and early clinical studies suggest that dual polyamine blockade may reduce tumor growth and reverse tumor-induced immunosuppression.

2.

esmoopen.com

esmoopen.com/article/S2059-7029(25)01445-0/fulltext

Phase I dose-escalation trial of AMXT 1501 dicaprate plus ...

Importantly, the breast cancer patient with a PR showed near-complete inhibition when measured after day 7 of AMXT 1501 plus DFMO treatment.

3.

sciencedirect.com

sciencedirect.com/science/article/pii/S2059702925014450

Phase I dose-escalation trial of AMXT 1501 dicaprate plus ...

Importantly, the breast cancer patient with a PR showed near-complete inhibition when measured after day 7 of AMXT 1501 plus DFMO treatment. These data support ...

4.

targetedonc.com

targetedonc.com/view/amxt-1501-and-dfmo-combination-achieve-fda-orphan-drug-designation

AMXT 1501 and DFMO Combination Achieve FDA Orphan ...

FDA designates AMXT 1501 and DFMO as an orphan drug for neuroblastoma, aiming to enhance treatment outcomes for pediatric patients.

5.

onclive.com

onclive.com/view/amxt-1501-plus-difluoromethylornithine-nets-fda-orphan-drug-designation-in-neuroblastoma

AMXT 1501 Plus Difluoromethylornithine Nets FDA ...

AMXT 1501 is designed to block the uptake of polyamines, which are key for tumor growth and survival. When combined with difluoromethylornithine ...

6.

trial.medpath.com

trial.medpath.com/clinical-trial/d1e2b663024b119e/nct07287917-amxt-1501-dfmo-advanced-solid-tumors

Study of AMXT 1501 and DFMO in Combination ... - MedPath

This study will evaluate the safety, tolerability, and preliminary effectiveness of AMXT 1501 and DFMO when combined with standard ...