OT-101 + mFOLFIRINOX for Pancreatic Cancer

(STOP-PC Trial)

Recruiting at 1 trial location

Overseen By Cynthia Lee, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Oncotelic Inc.

Must not be taking: Anticoagulants, NSAIDs, Corticosteroids, others

Disqualifiers: Other cancers, Infections, Cardiac events, others

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 2.5 years

455 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, including investigational agents, anti-coagulants (except heparin for catheters), non-steroidal anti-inflammatory drugs, and specific drugs that affect platelet function. If you are on these medications, you may need to stop them to participate in the study.

What data supports the effectiveness of the drug OT-101 + mFOLFIRINOX for pancreatic cancer?

The modified FOLFIRINOX (mFOLFIRINOX) regimen, which is part of the treatment, has shown improved survival rates in patients with advanced pancreatic cancer compared to other treatments, although it can have significant side effects. Studies have reported response rates exceeding 30% in patients with advanced nonmetastatic pancreatic cancer, suggesting its potential effectiveness. ^{1 2 3 4 5}

Is the combination of OT-101 and mFOLFIRINOX safe for humans?

Modified FOLFIRINOX (mFOLFIRINOX) has been studied for pancreatic cancer and is generally considered safe, though it can have significant side effects. These side effects include issues like nausea, fatigue, and low blood cell counts, which are common with chemotherapy treatments. There is no specific safety data available for OT-101 in combination with mFOLFIRINOX. ^{1 4 5 6 7}

How is the drug mFOLFIRINOX different from other treatments for pancreatic cancer?

mFOLFIRINOX is a modified version of the FOLFIRINOX chemotherapy regimen, which includes a reduced dosage to lessen side effects while maintaining effectiveness. It combines four drugs—fluorouracil, leucovorin, irinotecan, and oxaliplatin—and is used as a first-line treatment for advanced pancreatic cancer, offering improved survival rates compared to some other treatments. ^{1 2 3 5 8}

What is the purpose of this trial?

The goal of this clinical study is to compare the efficacy and safety of OT-101 in combination with mFOLFIRINOX (folinic acid, 5-FU, irinotecan, oxaliplatin) to mFOLFIRINOX alone in patients with advanced and unresectable or metastatic pancreatic cancer.

Eligibility Criteria

This trial is for adults with advanced, inoperable or metastatic pancreatic cancer. Participants must have measurable disease, not be pregnant or breastfeeding, and agree to use contraception. They should have completed prior treatments with recovery from most side effects and have good performance status indicating they can carry out daily activities.

Inclusion Criteria

Provide signed written informed consent

Measurable disease per RECIST v.1.1

I will use effective birth control for 6 months after my last oxaliplatin dose.

Exclusion Criteria

Any serious medical condition, laboratory abnormality, psychiatric illness, or comorbidity that, in the judgment of the Investigator, would make the patient inappropriate for the study

Patients with abnormal electrocardiogram (ECG) at baseline (QT or QTc interval >470 ms)

I am currently taking prescribed medications.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive OT-101 in combination with mFOLFIRINOX or mFOLFIRINOX alone in 14-day cycles

2.5 years

Bi-weekly visits for treatment administration

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 year

Treatment Details

Interventions

• mFOLFIRINOX
• OT-101

Trial Overview The study tests OT-101 combined with mFOLFIRINOX (a mix of folinic acid, 5-FU, irinotecan, oxaliplatin) against mFOLFIRINOX alone in treating pancreatic cancer. The aim is to see which treatment is more effective and safe.

Participant Groups

Group I: OT-101 + mFOLFIRINOX Active Control 2 Interventions

OT-101 IV dosed on Days 4-7 plus mFOLFIRINOX (dl-LV 400 mg/m2, irinotecan 180 mg/m2 and oxaliplatin 85 mg/m2 followed by a 46-hour infusion of 5-FU 2400 mg/m2) initiated on Day 1 of a 14-day cycle

Group II: mFOLFIRINOX Only Placebo Group 1 Intervention

mFOLFIRINOX (dl-LV 400 mg/m2, irinotecan 180 mg/m2 and oxaliplatin 85 mg/m2 followed by a 46-hour infusion of 5-FU 2400 mg/m2) initiated on Day 1 of a 14-day cycle

mFOLFIRINOX is already approved in European Union, United States, Canada for the following indications:

Approved in European Union as mFOLFIRINOX for:

• Pancreatic ductal adenocarcinoma (PDAC)

Approved in United States as mFOLFIRINOX for:

• Advanced pancreatic cancer

Approved in Canada as mFOLFIRINOX for:

• Resectable pancreatic ductal adenocarcinoma

Who Is Running the Clinical Trial?

Oncotelic Inc.

Lead Sponsor

Trials

3

Recruited

540+

Findings from Research

Modified-dose FOLFIRINOX (mFOLFIRINOX) demonstrated comparable efficacy to standard-dose FOLFIRINOX (sFOLFIRINOX) in treating pancreatic cancer, with similar objective response rates and overall survival outcomes among 130 patients studied.

mFOLFIRINOX was associated with significantly lower rates of severe adverse events, such as neutropenia, anorexia, and diarrhea, suggesting it is a safer option for patients who may be concerned about toxicity while maintaining effective treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparison of efficacy and safety between standard-dose and modified-dose FOLFIRINOX as a first-line treatment of pancreatic cancer. Kang, H., Jo, JH., Lee, HS., et al.[2022]

In a study of 198 patients with advanced pancreatic cancer, the SOXIRI chemotherapy regimen showed similar overall survival (12.1 months) and progression-free survival (6.5 months) compared to the standard mFOLFIRINOX regimen (11.2 months and 6.8 months, respectively), indicating comparable efficacy.

While both regimens had similar safety profiles, SOXIRI was associated with a higher incidence of anemia (41.4% vs. 24%), suggesting that while effective, it may have specific side effects that need to be monitored.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of SOXIRI versus mFOLFIRINOX in advanced pancreatic cancer. Li, X., Huang, J., Wang, F., et al.[2023]

The modified FOLFIRINOX regimen is effective and well-tolerated for patients with advanced nonmetastatic pancreatic cancer, showing a high rate of surgical resection (72% attempted, 51.1% accomplished) with manageable toxicity, primarily diarrhea in 14% of patients.

Patients who underwent surgical resection after treatment with mFOLFIRINOX experienced a median progression-free survival of 18 months, significantly longer than the 8 months for those who did not have surgery, indicating a potential survival benefit from this approach.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Neoadjuvant modified (m) FOLFIRINOX for locally advanced unresectable (LAPC) and borderline resectable (BRPC) adenocarcinoma of the pancreas. Blazer, M., Wu, C., Goldberg, RM., et al.[2022]

Modified FOLFIRINOX shows similar effectiveness in treating metastatic pancreatic adenocarcinoma (MPA) compared to standard FOLFIRINOX, with no significant differences in overall survival or progression-free survival among patients.

While modified FOLFIRINOX had a lower dose reduction rate, it was associated with a slightly higher incidence of severe toxicity, indicating that patient selection may influence treatment outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Retrospective comparison of the efficacy and the toxicity of standard and modified FOLFIRINOX regimens in patients with metastatic pancreatic adenocarcinoma. de Jesus, VHF., Camandaroba, MPG., Donadio, MDS., et al.[2023]

The modified FOLFIRINOX (mFOLFIRINOX) regimen demonstrated a high treatment efficacy in patients with locally advanced pancreatic cancer (LAPC) and metastatic pancreatic cancer (MPC), achieving a response rate of 55.2% among 29 evaluable patients.

The treatment was well-tolerated, with only 9 patients experiencing grade 3 or 4 adverse effects, and no patients discontinued treatment due to side effects, indicating a favorable safety profile for this regimen.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Modified FOLFIRINOX for advanced pancreatic cancer: a tertiary center experience from China]. Bai, X., Su, R., Ma, T., et al.[2018]

The modified FOLFIRINOX (mFOLFIRINOX) regimen showed a tolerable safety profile with lower hematological toxicity compared to the full-dose FOLFIRINOX, while still maintaining comparable efficacy in treating advanced pancreatic ductal adenocarcinoma (PDAC).

In a study of 18 patients, 80% achieved at least stable disease, with a median overall survival of 9.3 months and progression-free survival of 7.2 months, indicating that mFOLFIRINOX can be an effective treatment option for fit patients with advanced PDAC.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and Efficacy of Modified FOLFIRINOX for Advanced Pancreatic Adenocarcinoma: A UK Single-Centre Experience. Ghorani, E., Wong, HH., Hewitt, C., et al.[2023]

In a phase III trial involving 80 patients with metastatic pancreatic adenocarcinoma, modified FOLFIRINOX (mFOLFIRINOX) significantly improved overall survival compared to S-1, with median survival rates of 9.2 months versus 4.9 months, respectively.

While mFOLFIRINOX showed higher efficacy with better response and disease control rates, it also resulted in more severe adverse events, with 56% of patients experiencing grade 3-4 side effects compared to 17% in the S-1 group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Modified FOLFIRINOX versus S-1 as second-line chemotherapy in gemcitabine-failed metastatic pancreatic cancer patients: A randomised controlled trial (MPACA-3). Go, SI., Lee, SC., Bae, WK., et al.[2022]

In a study of 318 patients with metastatic pancreatic cancer, the modified FOLFIRINOX (mFFX) regimen showed similar overall survival and progression-free survival compared to the original FOLFIRINOX (oFFX) regimen, indicating that mFFX is just as effective.

mFFX was associated with significantly fewer adverse events, such as thrombopenia and liver dysfunction, making it a safer option for patients compared to oFFX.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Multicenter Retrospective Analysis of Original versus Modified FOLFIRINOX in Metastatic Pancreatic Cancer: Results of the NAPOLEON Study. Nakazawa, J., Tsuruta, N., Shimokawa, M., et al.[2023]

References

1. China pubmed.ncbi.nlm.nih.gov

Comparison of efficacy and safety between standard-dose and modified-dose FOLFIRINOX as a first-line treatment of pancreatic cancer. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Efficacy and safety of SOXIRI versus mFOLFIRINOX in advanced pancreatic cancer. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Neoadjuvant modified (m) FOLFIRINOX for locally advanced unresectable (LAPC) and borderline resectable (BRPC) adenocarcinoma of the pancreas. [2022]

4. China pubmed.ncbi.nlm.nih.gov

Retrospective comparison of the efficacy and the toxicity of standard and modified FOLFIRINOX regimens in patients with metastatic pancreatic adenocarcinoma. [2023]

5. China pubmed.ncbi.nlm.nih.gov

[Modified FOLFIRINOX for advanced pancreatic cancer: a tertiary center experience from China]. [2018]

6. Switzerland pubmed.ncbi.nlm.nih.gov

Safety and Efficacy of Modified FOLFIRINOX for Advanced Pancreatic Adenocarcinoma: A UK Single-Centre Experience. [2023]

7. England pubmed.ncbi.nlm.nih.gov

Modified FOLFIRINOX versus S-1 as second-line chemotherapy in gemcitabine-failed metastatic pancreatic cancer patients: A randomised controlled trial (MPACA-3). [2022]

8. Switzerland pubmed.ncbi.nlm.nih.gov

Multicenter Retrospective Analysis of Original versus Modified FOLFIRINOX in Metastatic Pancreatic Cancer: Results of the NAPOLEON Study. [2023]