Pegcetacoplan + Chemotherapy for Pancreatic Cancer

Overseen By Sarah Chatley, BS

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Roswell Park Cancer Institute

Disqualifiers: Previous chemotherapy, Brain metastases, Uncontrolled illness, others

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

Compensation: Varies

Number of Visits: 9

Duration: 16 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment combination for individuals with pancreatic cancer that has spread. It combines pegcetacoplan, which boosts the immune system's ability to fight cancer, with a chemotherapy mix called mFOLFIRINOX. The trial aims to determine if this combination can safely and effectively treat pancreatic cancer and prevent dangerous blood clots. Individuals with confirmed metastatic pancreatic cancer, who have not received prior chemotherapy for it and can manage self-injections, might be suitable for this study. 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 treatment.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss your specific medications with the trial team to get a clear answer.

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

Research has shown that pegcetacoplan has been well-tolerated in various conditions. In one study with adults, no new safety issues emerged over three years. Serious side effects occurred in about 55% of patients, but only 4.5% were linked to pegcetacoplan itself.

When combined with mFOLFIRINOX for treating pancreatic cancer, this treatment aims to enhance the immune system's ability to fight cancer and reduce the risk of blood clots, which are common in pancreatic cancer patients. While specific safety data for this combination in pancreatic cancer is not detailed, the previous safety record of pegcetacoplan offers some reassurance. As this trial is in its early stages, monitoring for any side effects is crucial. Researchers will closely observe participants to ensure the treatment's safety. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard chemotherapy options for pancreatic cancer, such as gemcitabine and nab-paclitaxel, pegcetacoplan offers a unique approach by targeting the complement system, which is part of the immune system. This treatment is combined with mFOLFIRINOX, a powerful chemotherapy regimen, to potentially enhance its effectiveness. Researchers are excited about pegcetacoplan because its mechanism could improve the immune response against cancer cells, offering hope for better outcomes in a notoriously difficult-to-treat cancer.

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

In this trial, participants will receive a combination treatment of pegcetacoplan with mFOLFIRINOX chemotherapy. Research has shown that pegcetacoplan targets a part of the immune system that might help stop tumor growth. Studies have found that this immune system component can cause issues in pancreatic cancer. Pegcetacoplan has shown promise in other conditions by improving blood health, suggesting it might help prevent blood clots—a major risk in pancreatic cancer. Although data on pegcetacoplan specifically for pancreatic cancer is limited, its mechanism offers hope for effectiveness. ^{1 6 7 8 9}

Who Is on the Research Team?

Christos Fountzilas

Principal Investigator

Roswell Park Cancer Institute

Are You a Good Fit for This Trial?

This trial is for patients with metastatic pancreatic ductal adenocarcinoma (PDAC). Participants should have PDAC that has spread from its original site. Specific eligibility criteria are not provided, but typically include factors like age, health status, and previous treatments.

Inclusion Criteria

Absolute neutrophil count ≥ 1,500/uL

Platelets ≥ 100,000/uL

Total bilirubin ≤ 1.5 x institutional upper limit of normal (ULN)

Exclusion Criteria

Unwilling or unable to follow protocol requirements

Any condition which in the investigator's opinion deems the participant an unsuitable candidate to receive study drug

Pregnant or nursing female participants

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive pegcetacoplan in combination with mFOLFIRINOX. Pegcetacoplan is administered intravenously on day 1 of cycle 1 and subcutaneously thrice weekly. mFOLFIRINOX is administered intravenously on day 1 of each cycle. Cycles repeat every 14 days for up to 8 cycles.

16 weeks

8 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment completion. Follow-up occurs at 30 days post-treatment and then every 3 months for up to 3 years.

3 years

1 visit (in-person) at 30 days, then every 3 months

What Are the Treatments Tested in This Trial?

Interventions

• Fluorouracil
• Irinotecan
• Oxaliplatin
• Pegcetacoplan

Trial Overview The trial tests pegcetacoplan combined with mFOLFIRINOX chemotherapy (oxaliplatin, irinotecan, leucovorin, fluorouracil) in treating metastatic PDAC. It aims to see if this combination can limit tumor growth and improve immune response against cancer cells while also reducing blood clot risks.

How Is the Trial Designed?

Group I: Treatment (pegcetacoplan, mFOLFIRINOX) Experimental Treatment 9 Interventions

Patients receive pegcetacoplan IV over 30 minutes on day 1 of cycle 1 and prior to mFOLFIRINOX and self-administered SC thrice weekly (every 2 days), when due on days in the office it will be given IV over 30 minutes prior to mFOLFIRINOX. Patients receive oxaliplatin IV over 2 hours, irinotecan and leucovorin IV concurrently over 90 minutes, and fluorouracil IV over 48 hours on day 1 of each cycle. Cycles repeat every 14 days for up to 8 cycles in the absence of disease progression or unacceptable toxicity. Patients may undergo ascites, pleural fluid (if present) and blood sample collection and CT or MRI throughout the study. Additionally, patients may undergo tumor biopsy throughout the study.

Who Is Running the Clinical Trial?

Roswell Park Cancer Institute

Lead Sponsor

Trials

427

Recruited

40,500+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07214298

Study Details | NCT07214298 | Pegcetacoplan in ... This phase I/II trial tests the effect of pegcetacoplan in combination with oxaliplatin, irinotecan, leucovorin, and fluorouracil (mFOLFIRINOX) ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12165795/

Complement pathway activation mediates pancreatic cancer ... These studies demonstrate that complement activation contributes to the skeletal muscle pathology and dysfunction in PDAC.

3. onclive.com onclive.com/view/pegcetacoplan-outperforms-current-standard-of-care-in-paroxysmal-nocturnal-hemoglobinuria

Pegcetacoplan Outperforms Current Standard of Care in ... Regarding the secondary end points, transfusion avoidance was achieved in 85% of patients (n = 35) with pegcetacoplan versus 15% of patients (n ...

4. hcplive.com hcplive.com/view/gemcitabine_asco_trials

Treatment with Gemcitabine (Gemzar) for Six Months After ... Response data were reported for 23 patients. There were five partial responses (22%), four confirmed partial responses (17%), 13 patients with stable disease ( ...

5. delta.larvol.com delta.larvol.com/Products/?ProductId=c95fec1a-83ff-4dfd-af1f-b55a96d02faa

Empaveli (pegcetacoplan SC) / Apellis, SOBI Delicious. October 20, 2025. New One-Year Data Reinforcing the Robust and Sustained Efficacy of EMPAVELI (pegcetacoplan) in C3G and Primary IC-MPGN to be ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38573482/

Safety and Efficacy of Pegcetacoplan in Adult Patients with ... Conclusion: Pegcetacoplan sustained long-term improvements in hemoglobin concentrations, fatigue reduction, and transfusion burden. Long-term ...

7. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2021/215014Orig1s000IntegratedR.pdf

215014Orig1s000 - accessdata.fda.gov should include an integrated safety dataset and patient level data including data on pegcetacoplan dosing, meningococcal and pneumococcal vaccination status ...

8. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40720060/

Efficacy and Safety Maintained up to 3 Years in Adults with ... No new safety concerns were identified over 3 years. Serious adverse events were reported in 73 (55.3%) patients, deemed pegcetacoplan related in 6 (4.5%) ...

9. sfj-pharma.com sfj-pharma.com/apellis-reports-positive-top-line-results-from-phase-3-head-to-head-study-of-pegcetacoplan-apl-2-2/

Apellis Reports Positive Top-line Results from Phase 3 ... Top-line data show that pegcetacoplan met the study's primary efficacy endpoint, demonstrating superiority to eculizumab with a statistically significant ...