Pafolacianine Imaging for Gastrointestinal Cancer

Overseen By Chao Family Comprehensive Cancer Center University of California, Irvine

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: University of California, Irvine

Disqualifiers: Inoperable cancer, Anaphylactic history, Pregnancy, others

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

Compensation: Varies

Number of Visits: 1

Duration: 1 day

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how well the drug pafolacianine helps doctors identify cancerous areas during gastrointestinal cancer surgery. The researchers aim to improve cancer removal by clearly identifying cancerous tissues. Participants will receive a single dose of the drug before surgery to determine if it effectively highlights cancer spots. The trial seeks individuals diagnosed with gastrointestinal cancer who are scheduled for surgical removal. As an Early Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants a chance to contribute to groundbreaking advancements in cancer detection.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that pafolacianine is likely to be safe for humans?

Research has shown that pafolacianine, also known as CYTALUX™, has been tested in people before. In other studies, patients received the same dose of pafolacianine as in this trial. The safety profile, summarizing side effects, was similar for patients with ovarian cancer and those with other types of cancer. Common side effects reported include nausea, vomiting, and stomach pain. Some people also experienced skin redness, high blood pressure, and chest discomfort.

No data exists on the drug's effects on pregnant women or unborn babies. However, the FDA has approved the medicine for imaging certain cancers, indicating it has been found safe for those specific uses. Although this study is in the early stages, available information suggests it is generally well-tolerated. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for gastrointestinal cancer, which often involve chemotherapy and surgery, pafolacianine (CYTALUX) offers a novel approach by enhancing surgical imaging. This treatment uses a fluorescent dye that targets cancer cells, making them glow under special lighting during surgery. This targeted imaging can help surgeons see and remove cancerous tissues more effectively, potentially leading to better outcomes. Researchers are excited because this could mean more precise surgeries and a reduction in the likelihood of cancer recurrence.

What evidence suggests that pafolacianine is effective for identifying cancerous lesions in gastrointestinal cancer?

Research has shown that pafolacianine, administered to participants in this trial, helps identify cancerous areas by making cancer cells light up during surgery. Previous studies demonstrated its success in detecting hidden cancer in the peritoneum, the lining inside the abdomen. Pafolacianine is approved for use in certain situations and shows promise in enhancing surgical precision by highlighting cancerous tissues. This aids surgeons in removing more cancer cells, potentially improving outcomes for patients with gastrointestinal cancers. ^{6 7 8 9 10}

Who Is on the Research Team?

Maheswari Senthil, MD

Principal Investigator

Chao Family Comprehensive Cancer Center

Are You a Good Fit for This Trial?

This trial is for individuals with gastrointestinal cancers and peritoneal carcinomatosis. Participants should be candidates for cytoreductive surgery, which aims to remove visible tumors from the abdomen.

Inclusion Criteria

I am a woman and have had negative pregnancy tests before receiving study treatment or surgery.

Ability to understand the requirements of the study, provide written informed consent for participation in the study and authorization of use and disclosure of protected health information, and agree to abide by study restrictions

My cancer originates in the gastrointestinal tract and I am scheduled for surgery to remove it.

Exclusion Criteria

Clinically significant abnormalities on ECG

My liver tests are more than three times the normal limit.

Known sensitivity to fluorescent light

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous infusion of pafolacianine (CYTALUX) prior to intraoperative imaging during cytoreductive surgery

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of treatment-emergent adverse events

30 days

Long-term Follow-up

Participants are monitored for primary outcome measures such as sensitivity, specificity, and accuracy over a longer period

1 year

What Are the Treatments Tested in This Trial?

Interventions

• Pafolacianine

Trial Overview

The study tests CYTALUX (pafolacianine) used during surgery to see if it can better highlight cancerous tissues using a special camera that detects its fluorescent glow under near-infrared light.

How Is the Trial Designed?

Group I: Pafolacianine (CYTALUX) Experimental Treatment 1 Intervention

All subjects will receive one single intravenous infusion of 0.025 mg/kg of pafolacianine (CYTALUX) injection prior to anticipated intraoperative imaging planned for cytoreductive surgery.

Who Is Running the Clinical Trial?

University of California, Irvine

Lead Sponsor

Trials

580

Recruited

4,943,000+

On Target Laboratories

Collaborator

Trials

2

Recruited

30+

Published Research Related to This Trial

In a study of 15 patients with advanced gastric cancer, MR imaging using tap water as an oral contrast agent was found to be as effective as gadopentetate dimeglumine (Gd-DTPA) for evaluating the cancer, suggesting that Gd-DTPA may not be necessary.

FLASH imaging provided slightly better image quality and diagnostic accuracy for T-staging compared to other MR techniques, while HASTE and true FISP imaging showed superior contrast-to-noise ratios, indicating that a combination of these methods can enhance evaluation of advanced gastric cancer.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

MR imaging of advanced gastric cancer: comparison of various MR pulse sequences using water and gadopentetate dimeglumine as oral contrast agents. Kim, AY., Han, JK., Kim, TK., et al.[2019]

The newly developed dipeptides [18F]Gly-4F-Gln and [18F]Ala-4F-Gln rapidly convert to the active imaging agent (2S,4R)-4-[18F]fluoroglutamine in the bloodstream, indicating their potential as effective prodrugs for tumor imaging.

Both dipeptides showed rapid localization in tumor tissues during PET imaging in rat models, with [18F]Ala-4F-Gln being particularly promising for imaging glutaminolysis in tumors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Alanine and glycine conjugates of (2S,4R)-4-[18F]fluoroglutamine for tumor imaging. Zha, Z., Ploessl, K., Lieberman, BP., et al.[2019]

Fluorine 18-fluorodeoxyglucose PET (FDG-PET) is a valuable noninvasive imaging technique that enhances cancer diagnosis and treatment by detecting increased glucose metabolism in tumors, aiding in staging and assessing treatment response.

Emerging metabolic PET imaging agents, such as amino acid analogs and choline, offer greater specificity for tumor cells compared to FDG, making them useful for identifying cancers that may not show high FDG uptake, thus improving diagnostic accuracy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Metabolic positron emission tomography imaging in cancer detection and therapy response. Zhu, A., Lee, D., Shim, H.[2021]

FDG-PET scanning is a highly effective diagnostic tool for gastrointestinal cancers, particularly in staging oesophageal carcinoma and detecting recurrence in colorectal carcinoma, with sensitivity rates reaching up to 100%.

The technique is also valuable for differentiating between benign and malignant pancreatic lesions, showing high sensitivity and specificity, making it a crucial part of cancer diagnosis and treatment monitoring.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

FDG-PET scanning in the diagnosis of gastrointestinal cancers. van Kouwen, MC., Oyen, WJ., Nagengast, FM., et al.[2019]

In a Phase 3 trial involving 112 lung cancer patients, the use of pafolacianine for intraoperative molecular imaging significantly improved surgical outcomes by identifying hidden tumors and narrow margins, with 53% of participants experiencing clinically significant events compared to a 10% threshold.

No serious adverse events were reported from pafolacianine, indicating its safety, while it successfully located primary nodules that were undetectable by conventional methods in 19% of cases and revealed additional malignant lesions in 8% of subjects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pafolacianine for intraoperative molecular imaging of cancer in the lung: The ELUCIDATE trial. Sarkaria, IS., Martin, LW., Rice, DC., et al.[2023]

FAPI PET imaging outperformed traditional FDG PET/CT/MRI in detecting tumors and metastases in patients with pancreatic, gastric, and cholangiocarcinoma, based on a systematic review of 10 studies.

FAPI PET provided higher maximum standardized uptake values (SUVmax) and tumor-to-background ratios (TBRmax), indicating its potential for more accurate cancer staging and diagnosis, which supports the need for further clinical studies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

FAPI PET versus FDG PET, CT or MRI for Staging Pancreatic-, Gastric- and Cholangiocarcinoma: Systematic Review and Head-to-Head Comparisons of Diagnostic Performances. Veldhuijzen van Zanten, SEM., Pieterman, KJ., Wijnhoven, BPL., et al.[2022]

In a study of 32 patients with digestive system tumors, 68 Ga-FAPI-04 PET/CT imaging resulted in staging changes for nearly 47% of patients, indicating its potential to provide more accurate tumor assessments compared to standard imaging methods.

The use of FAPI-directed imaging led to therapeutic management changes in 25% of cases, particularly benefiting patients with hepatocellular carcinoma (HCC) and pancreatic duct adenocarcinoma (PDAC), suggesting it could enhance treatment strategies for these cancers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Impact of 68 Ga-FAPI-04 PET/CT on Staging and Therapeutic Management in Patients With Digestive System Tumors. Kosmala, A., Serfling, SE., Schlötelburg, W., et al.[2023]

In a phase III study involving 150 patients with ovarian cancer, pafolacianine successfully identified additional cancerous lesions in 33% of cases that were not detected by traditional methods, demonstrating its efficacy as an adjunctive tool during surgery.

The use of pafolacianine was found to be safe, with no serious drug-related adverse events reported, although 30% of patients experienced mild side effects like nausea and abdominal pain.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Phase III Study of Pafolacianine Injection (OTL38) for Intraoperative Imaging of Folate Receptor-Positive Ovarian Cancer (Study 006). Tanyi, JL., Randall, LM., Chambers, SK., et al.[2023]

In a study of 33 patients with suspected recurrent pancreatic ductal adenocarcinoma (PDAC), both early (20 min) and late (60 min) 68Ga-FAPI-PET imaging provided similar detection rates for most lesions, indicating that either timepoint can be effective for imaging.

However, early imaging was more effective for detecting cholestatic lesions, suggesting that using both timepoints may enhance diagnostic accuracy and help differentiate between types of lesions in recurrent PDAC.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparison of early and late 68Ga-FAPI-46-PET in 33 patients with possible recurrence of pancreatic ductal adenocarcinomas. Hoppner, J., van Genabith, L., Hielscher, T., et al.[2023]

In a study of 38 patients with suspected lung cancer, preoperative staining for folate receptors (FRα and FRβ) was found to correlate with the effectiveness of pafolacianine fluorescence during surgery, indicating that higher FRα expression is associated with better fluorescence detection of malignant tumors.

The results showed that 95% of malignant tumors fluoresced during surgery, while none of the benign tumors did, suggesting that FRα IHC could be a useful tool for selecting patients who would benefit from pafolacianine-guided surgery, warranting further investigation in larger clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prospective validation of tumor folate receptor expression density with the association of pafolacianine fluorescence during intraoperative molecular imaging-guided lung cancer resections. Azari, F., Zhang, K., Kennedy, G., et al.[2023]

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07039526

Study Details | NCT07039526 | Single Dose Investigator ...

The goal of clinical trial is to test an FDA approved drug called Pafolacianine that attaches to cancer cells and lights up when seen through a special ...

2.

cancer.gov

cancer.gov/clinicaltrials/NCI-2024-06351

Pafolacianine Sodium for the Intraoperative Imaging of ...

This phase I trial studies whether pafolacianine sodium (cytalux) works better than the standard approach at identifying cancer lesions that should be removed ...

3.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12035792/

Real‑world application of Cytalux for targeted imaging ...

In conclusion, the use of Cytalux in real-world settings demonstrates significant efficacy in identifying occult peritoneal disease in a ...

4.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07124351

Study Details | NCT07124351 | Intraoperative Imaging of ...

This is an open-label study in adults to determine feasibility of using CYTALUX™ (pafolacianine) injection with near-infrared (NIR) fluorescent imaging for ...

5.

withpower.com

withpower.com/trial/phase-1-gastrointestinal-neoplasms-7-2025-7bae7

Pafolacianine Imaging for Gastrointestinal Cancer

This Phase 4 medical study run by Cedars-Sinai Medical Center is evaluating whether CYTALUX™ (pafolacianine) will have tolerable side effects & efficacy for ...

6.

cytalux.com

cytalux.com/important-safety-information/

CYTALUX Important Safety Information

There are no available human data to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes.

7.

cytaluxhcp.com

cytaluxhcp.com/important-safety-information/

CYTALUX Important Safety Information

Adverse reactions including nausea, vomiting, abdominal pain, flushing, hypersensitivity, elevation in blood pressure, dyspepsia, and chest discomfort were ...

8.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07124351

Study Details | NCT07124351 | Intraoperative Imaging of ...

This is an open-label study in adults to determine feasibility of using CYTALUX™ (pafolacianine) injection with near-infrared (NIR) fluorescent imaging for ...

9.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/label/2022/214907s002lbl.pdf

Cytalux - accessdata.fda.gov

Overall, the safety profile observed in patients treated with CYTALUX 0.025 mg/kg was similar between patients with ovarian cancer and patients with known or ...

10.

reference.medscape.com

reference.medscape.com/drug/cytalux-pafolacianine-4000180

Cytalux (pafolacianine) dosing, indications, interactions, ...

Medscape - Cancer imaging dosing for Cytalux (pafolacianine), frequency-based adverse effects, comprehensive interactions, contraindications, ...