Pafolacianine for Identifying Lesions in Childhood Cancer

Overseen By Gina Lewis

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Mayo Clinic

Must not be taking: Folic acid

Disqualifiers: Renal failure, Liver failure, Pregnancy, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: 1

Duration: Day 0 to 1

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests how well a new imaging agent, pafolacianine, identifies cancerous lesions in children with solid tumors during surgery. Pafolacianine illuminates tumor cells with a special camera, aiding doctors in seeing and removing cancerous areas. The trial seeks children and teens diagnosed with or suspected of having solid tumors in areas like the neck, chest, or abdomen who plan to undergo surgery to remove the tumor. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial requires participants to stop taking folic acid, folate supplements, and multi-vitamins containing folate 48 hours before receiving the study drug. Other medications are not specifically mentioned, so it's best to discuss with the trial team.

What prior data suggests that this imaging technique is safe for children and adolescents?

Research has shown that pafolacianine, a special dye used in medical imaging, is generally safe for patients. It was first approved for ovarian cancer after studies found it helped doctors see cancerous areas more clearly. In these studies, the safety of pafolacianine was similar for patients with ovarian cancer and those with other types of tumors, suggesting it is likely safe for children and teenagers with solid tumors. While any medication can have side effects, the safety demonstrated in these studies provides some reassurance about its use in current trials. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment?

Unlike the standard treatments for childhood cancer, which often rely on surgery, chemotherapy, or radiation, pafolacianine offers a new approach by enhancing the ability to identify cancerous lesions during surgery. This treatment is unique because it uses a fluorescence-guided technique where pafolacianine is administered intravenously and highlights cancerous tissues under near-infrared light, making them easier for surgeons to spot and remove. Researchers are excited about this method because it has the potential to improve surgical accuracy, reduce the likelihood of leaving behind cancerous tissue, and ultimately improve patient outcomes.

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

Research has shown that pafolacianine helps identify cancerous areas by targeting folate receptors, which are often more numerous on cancer cells. Studies in adults have demonstrated that using pafolacianine during surgery can help surgeons see tumors more clearly, potentially improving the removal of cancerous tissue. In this trial, participants will receive pafolacianine and undergo near-infrared (NIR) fluorescent imaging during standard care surgery. Early findings suggest that this imaging agent, when used with NIR technology, makes cancer cells light up, making them easier to spot during operations. The goal of using pafolacianine in children is to achieve similar results, improving the accuracy of surgeries for solid tumors. This approach could lead to better outcomes by ensuring more complete removal of tumors. ^{1 4 6 7 8}

Who Is on the Research Team?

Stephanie F. Polites, MD, MPH

Principal Investigator

Mayo Clinic

Are You a Good Fit for This Trial?

This trial is for children and adolescents with primary or metastatic solid tumors. Participants must have a type of cancer that could potentially overexpress folate receptors, making them suitable for the imaging agent being tested.

Inclusion Criteria

Willingness of research participant or legal guardian/representative to give written informed consent

I am between 6 months and 17 years old.

I am suspected to have or diagnosed with a solid tumor and surgery is planned.

Exclusion Criteria

My kidneys are not working properly.

I am being evaluated for liver failure or am on the liver transplant list.

I cannot or will not use birth control.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive pafolacianine IV and undergo NIR fluorescent imaging during standard of care surgery

Day 0 to 1

1 visit (in-person)

Follow-up

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

Up to 30 days after surgery

What Are the Treatments Tested in This Trial?

Interventions

• Pafolacianine

Trial Overview The study tests pafolacianine, an imaging agent used during surgery to light up cancer cells in kids. It's paired with near-infrared (NIR) imaging to help surgeons see and remove tumors more effectively than with standard methods.

How Is the Trial Designed?

Group I: Diagnostic (pafolacianine + NIR) Experimental Treatment 4 Interventions

Patients receive pafolacianine IV over 60 to 90 minutes and undergo NIR fluorescent imaging during standard of care (SOC) surgery on study. Patients also undergo collection of tissue during SOC surgery on study.

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

Published Research Related to This Trial

The Patient-derived xenograft (PDX) for Childhood Cancer Therapeutics (PCAT) portal provides a comprehensive resource for studying pediatric cancer, featuring 324 PDX models along with clinical and genomic data to aid in the development of new treatments.

PCAT includes curated preclinical testing results for 68 models and 79 therapeutic agents, allowing researchers to identify effective therapies and compare patient tumors with PDX models, enhancing the understanding of pediatric cancer biology.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

PCAT: an integrated portal for genomic and preclinical testing data of pediatric cancer patient-derived xenograft models. Yang, J., Li, Q., Noureen, N., et al.[2021]

Cure rates for children with cancer have plateaued around 70%, highlighting the need for new drug approaches, especially for hard-to-treat cancers like stage IV neuroblastoma and relapsed leukemia.

Novel agents, including cell cycle inhibitors like clofarabine and nelarabine, as well as targeted therapies such as tyrosine kinase inhibitors and monoclonal antibodies, show promise in improving treatment outcomes for high-risk pediatric cancers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Educational paper. The development of new therapies for pediatric oncology. Horton, TM., Berg, SL.[2021]

Citations

1. cancer.gov cancer.gov/clinicaltrials/NCI-2025-02149

An Imaging Agent (Pafolacianine) for Identifying Lesions in ... This phase III trial studies how well pafolacianine works for identifying cancerous lesions in children and adolescent patients with primary solid tumors or ...

2. withpower.com withpower.com/trial/phase-3-childhood-malignant-solid-neoplasm-3-2025-49099

Pafolacianine for Identifying Lesions in Childhood Cancer This phase III trial studies how well pafolacianine works for identifying cancerous lesions in children and adolescent patients with primary solid tumors or ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06235125?term=PAFOLACIANINE&viewType=Table&rank=1

Study Details | NCT06235125 | Safety and Feasibility of ... Findings from the ELUCIDATE trial in adults demonstrated that intraoperative molecular imaging with CYTALUX (pafolacianine) improves surgical outcomes by ...

4. oncotarget.com oncotarget.com/article/28772/text/

Widespread folate receptor expression in pediatric and ... Advances in fluorescence-guided surgery have shown promise for enhancing tumor identification and negative-margin excision but have been limited ...

5. mayo.edu mayo.edu/research/clinical-trials/cls-20581064

Pafolacianine for Localization of Pediatric Extracranial ... Phase 4 trials look at long-term safety and effectiveness ... identifying lesions in children and adolescent patients with primary or metastatic ...

6. cancer.gov cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2025-02149

An Imaging Agent (Pafolacianine) for Identifying Lesions in ... This phase III trial studies how well pafolacianine works for identifying cancerous lesions in children and adolescent patients with primary solid tumors or ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12536893/

Widespread folate receptor expression in pediatric and ... Pafolacianine was initially approved for use in ovarian cancer in 2021 after a large phase III clinical trial demonstrated a more complete ...

8. rxlist.com rxlist.com/cytalux-drug.htm

Cytalux (Pafolacianine Injection): Side Effects, Uses, ... 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 ...