Ampligen for Pancreatic Cancer

Not currently recruiting at 2 trial locations

Overseen By Kher Lee Ng, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: AIM ImmunoTech Inc.

Must not be taking: Investigational drugs, Immune checkpoint inhibitors

Disqualifiers: HIV, Hepatitis, Recent myocardial infarction, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 182 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of Ampligen (also known as Rintatolimod) for individuals with locally advanced pancreatic adenocarcinoma, a type of pancreatic cancer that cannot be surgically removed. Participants will receive either Ampligen combined with standard cancer treatment (chemoradiation or chemotherapy) or the standard treatment alone. The researchers aim to determine if Ampligen can improve outcomes for those whose cancer hasn't progressed after first-line therapy like FOLFIRINOX. Individuals with non-metastatic pancreatic cancer who have completed at least four months of initial therapy without disease progression might be suitable candidates. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to significant advancements in pancreatic cancer care.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, it does mention that you should not have received investigational drugs within 6 weeks before starting the study medication.

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

Research has shown that rintatolimod, also known as Ampligen, is generally safe for patients with pancreatic cancer. In studies, patients did not experience serious side effects while using rintatolimod. Early results from other research also indicate its safety when combined with other treatments for pancreatic cancer. This suggests that many people can use the treatment without major issues. However, like all medicines, it may have side effects, so discussing these with the trial team is important. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about Ampligen (rintatolimod) for pancreatic cancer because it offers a novel approach compared to the usual chemotherapy and radiation treatments. Unlike standard treatments, which typically focus on killing cancer cells directly, Ampligen works by enhancing the body's immune response, potentially offering a more targeted attack on the cancer. This intravenous treatment uses rintatolimod, a unique active ingredient that may help the immune system recognize and fight cancer cells more effectively. By combining it with or without standard care, researchers hope to improve patient outcomes and offer a new line of defense against this aggressive cancer.

What evidence suggests that this trial's treatments could be effective for pancreatic cancer?

Research has shown that rintatolimod (Ampligen), which participants in this trial may receive, might help people with pancreatic cancer live longer. In studies, it has shown promise by enhancing the immune system's ability to fight cancer. Specifically, early results indicated that 64% of patients who received rintatolimod with another drug lived more than six months, which is encouraging for this aggressive cancer. Additionally, rintatolimod helps slow the growth and spread of pancreatic cancer cells. Importantly, it is generally well-tolerated, meaning it usually doesn't cause severe side effects. ^{1 2 3 6 7}

Who Is on the Research Team?

David R Strayer, MD

Principal Investigator

AIM ImmunoTech Inc.

Are You a Good Fit for This Trial?

This trial is for adults with locally advanced pancreatic adenocarcinoma who've completed at least 4 months of FOLFIRINOX treatment without disease progression. Participants must have a life expectancy of over 3 months, weigh at least 40kg, and have a Karnofsky Performance Status of 80 or higher. They should not be pregnant or breastfeeding and must agree to use contraception during the study.

Inclusion Criteria

I am mostly self-sufficient and active.

I weigh at least 40kg.

Provide signed written informed consent and willingness, ability to comply with study requirements

Exclusion Criteria

I have a history of or currently have hepatitis A, B, or C.

Pregnant or lactating women

I need fluid removed from my abdomen at least every 2 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Ampligen in combination with SOC or SOC alone until disease progression

Up to 182 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Rintatolimod

Trial Overview

The trial is testing Ampligen (Rintatolimod) to see if it's safe and effective when given after FOLFIRINOX therapy in patients with pancreatic cancer that hasn't spread beyond the local area. It compares outcomes between those receiving Ampligen and those who do not receive any additional treatment post-FOLFIRINOX.

How Is the Trial Designed?

Group I: Ampligen / rintatolimod + SOC Chemoradiation Experimental Treatment 1 Intervention

Subjects will receive rintatolimod \[intravenous (IV)\], up to 400 mg twice weekly plus SOC chemoradiation until disease progression

Group II: Ampligen / rintatolimod + SOC Experimental Treatment 1 Intervention

Subjects will receive rintatolimod \[intravenous (IV)\], up to 400 mg twice weekly plus SOC (SOC does not include chemoradiation) until disease progression

Group III: SOC Chemoradiation Alone Active Control 1 Intervention

Subjects will receive SOC chemoradiation until evidence of disease progression.

Group IV: SOC Alone Active Control 1 Intervention

Subjects will receive SOC (SOC does not include chemoradiation) until evidence of disease progression.

Who Is Running the Clinical Trial?

AIM ImmunoTech Inc.

Lead Sponsor

Trials

15

Recruited

710+

Amarex Clinical Research

Collaborator

Trials

28

Recruited

1,700+

Published Research Related to This Trial

S-1, a newer drug for treating metastatic pancreatic cancer, showed promising results with a response rate of over 35% when combined with gemcitabine, compared to a 20% response rate with S-1 alone.

A case report of a 68-year-old man indicated that after failing multiple treatments, S-1 was well-tolerated over 14 cycles, leading to a 10-month survival with a good quality of life, suggesting its potential as an effective oral treatment option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long term survival on S-1 monotherapy in a patient with recurrent stage IV pancreatic cancer. Alsamarai, S., Zergebel, C., Zhang, J., et al.[2013]

Despite advances in surgery and chemotherapy, pancreatic cancer prognosis has not improved significantly, highlighting the need for new treatment strategies.

Combination therapies, particularly those that include immunotherapy with chemotherapy and other agents, may enhance treatment effectiveness, especially for immunogenic subtypes of pancreatic cancer, and should be prioritized in early clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immunological combination treatment holds the key to improving survival in pancreatic cancer. Sodergren, MH., Mangal, N., Wasan, H., et al.[2021]

In a phase II study involving 46 patients with advanced pancreatic and biliary carcinomas, raltitrexed was generally well tolerated, with a median survival of 4.6 months and a 1-year survival rate of 21.8%.

Only a small percentage of patients experienced improvements in quality of life (7.0%) and clinical benefit response (15.6%), indicating limited efficacy in this patient population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A phase II trial of raltitrexed (Tomudex) in advanced pancreatic and biliary carcinoma. François, E., Hebbar, M., Bennouna, J., et al.[2014]

In a phase II trial involving 25 patients with pancreatic adenocarcinoma, the combination of adjuvant gemcitabine and erlotinib showed a median recurrence-free survival (RFS) of 14 months, with 1-year and 2-year RFS rates of 56% and 26%, respectively.

The treatment was generally well-tolerated, with most side effects being grade 3 events (36% of patients) and only one case of grade 4 toxicity, indicating a manageable safety profile for this regimen.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase II study of gemcitabine and erlotinib as adjuvant therapy for patients with resected pancreatic cancer. Bao, PQ., Ramanathan, RK., Krasinkas, A., et al.[2022]

Rintatolimod (Ampligen®), a TLR3 agonist, shows potential as an antiviral treatment for pancreatic cancer cells by activating the innate immune system and enhancing interferon signaling, which could help combat SARS-CoV-2 infections in cancer patients.

The treatment increases the expression of beneficial cytokines and angiogenesis-related genes without causing fibrosis, suggesting it may help prevent severe complications associated with COVID-19 in vulnerable cancer patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rintatolimod Induces Antiviral Activities in Human Pancreatic Cancer Cells: Opening for an Anti-COVID-19 Opportunity in Cancer Patients? Mustafa, DAM., Saida, L., Latifi, D., et al.[2021]

In a study involving 27 patients with locally advanced or metastatic pancreatic cancer, treatment with the TLR-3 agonist rintatolimod was well-tolerated and led to a significant increase in peripheral B cells among long-term survivors, suggesting a potential immunomodulatory effect.

Patients receiving rintatolimod had a median progression-free survival of 13 months and a median overall survival of 19 months, both significantly better than matched controls, indicating that rintatolimod may improve survival outcomes in pancreatic cancer patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rintatolimod (Ampligen®) Enhances Numbers of Peripheral B Cells and Is Associated with Longer Survival in Patients with Locally Advanced and Metastasized Pancreatic Cancer Pre-Treated with FOLFIRINOX: A Single-Center Named Patient Program. El Haddaoui, H., Brood, R., Latifi, D., et al.[2023]

Rintatolimod (Ampligen®), a TLR-3 agonist, shows promise in boosting the immune response against pancreatic cancer, particularly in cells expressing TLR-3, as demonstrated in a study of 13 tissue samples and three human pancreatic cancer cell lines.

In the CFPAC-1 cell line, rintatolimod treatment significantly reduced cell proliferation and showed a trend towards decreased cell migration, suggesting a potential direct anti-tumor effect linked to TLR-3 signaling.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rintatolimod: a potential treatment in patients with pancreatic cancer expressing Toll-like receptor 3. Haddaoui, HE., van Eijck, CW., Doukas, M., et al.[2023]

In a phase I trial involving 50 patients with advanced pancreatic cancer, the combination of nivolumab, nab-paclitaxel, and gemcitabine showed a manageable safety profile, with 48 patients experiencing grade 3/4 treatment-emergent adverse events, but only one dose-limiting toxicity reported.

The treatment resulted in a median overall survival of 9.9 months and an overall response rate of 18%, indicating limited efficacy, which led to the conclusion that further investigation of this combination is not supported.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Open-label, Phase I Study of Nivolumab Combined with nab-Paclitaxel Plus Gemcitabine in Advanced Pancreatic Cancer. Wainberg, ZA., Hochster, HS., Kim, EJ., et al.[2022]

Citations

1.

aimimmuno.com

aimimmuno.com/aim-immunotech-reports-positive-mid-year-safety-and-efficacy-data-from-phase-2-study-evaluating-ampligen-rintatolimod-in-combination-with-astrazenecas-imfinzi-durvalumab-for-the-trea/

Mid-year report of combination therapy ...

AIM ImmunoTech Reports Positive Mid-year Safety and Efficacy Data from Phase 2 Study Evaluating Ampligen® (rintatolimod) in Combination with ...

2.

targetedonc.com

targetedonc.com/view/rintatolimod-plus-durvalumab-shows-encouraging-early-outcomes-in-metastatic-pancreatic-cancer

Rintatolimod Plus Durvalumab Shows Encouraging Early ...

Further, 64% have achieved OS greater than 6 months. This early survival data is considered better than expected in this aggressive disease ...

3.

aacrjournals.org

aacrjournals.org/clincancerres/article/30/16/3447/746780/Rintatolimod-in-Advanced-Pancreatic-Cancer

Rintatolimod in Advanced Pancreatic Cancer Enhances ...

This investigation revealed that rintatolimod reduces proliferation and migration rates in pancreatic cancer cells with high TLR-3 mRNA ...

4.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC8227153/

Rintatolimod Induces Antiviral Activities in Human ...

In addition, Rintatolimod is a well-tolerated drug that did not show severe adverse events when it was given to pancreatic cancer patients (data not shown).

5.

withpower.com

withpower.com/trial/phase-2-adenocarcinoma-8-2022-8b3ef

Ampligen for Pancreatic Cancer · Info for Participants

Research shows that Rintatolimod (Ampligen) may help improve survival in pancreatic cancer patients by boosting the immune system. In a study, patients treated ...

6.

clinicaltrialsarena.com

clinicaltrialsarena.com/news/aim-immunotech-ampligen/

AIM ImmunoTech reports Ampligen trial data for pancreatic ...

AIM ImmunoTech has announced encouraging data from the ongoing Phase II trial of its Ampligen (rintatolimod) drug plus AstraZeneca's Imfinzi ...

7.

sec.gov

sec.gov/Archives/edgar/data/946644/000149315225011441/ex99-1.htm

Press Release dated July 28, 2025

Exhibit 99.1. AIM ImmunoTech Reports Positive Mid-year Safety and Efficacy Data from Phase 2 Study Evaluating Ampligen® (rintatolimod) in ...