Recruiting
Phase 3

Pafolacianine

Sponsor:

Mayo Clinic

Code:

NCT06915727

Conditions

Childhood Malignant Solid Neoplasm

Childhood Malignant Thoracic Neoplasm

Malignant Abdominal Neoplasm

Malignant Neck Neoplasm

Malignant Pelvic Neoplasm

Eligibility Criteria

Sex: All

Age: 0 - 17

Healthy Volunteers: Not accepted

Interventions

Biospecimen Collection

Near Infrared Imaging

Pafolacianine Sodium

Surgical Procedure

Study Details

Brief summary:

This phase III trial studies how well pafolacianine works for identifying cancerous lesions in children and adolescent patients with primary solid tumors or solid tumors that have spread from where they first started (primary site) to other places in the body (metastatic). Pafolacianine is a fluorescent imaging agent that targets folate receptors which are overexpressed in many cancers and is used with near infrared (NIR) imaging during surgery to identify tumor cells. NIR uses a special camera that uses wavelengths in the infrared range to visualize and locate the tumor cells that are lit up by the pafolacianine. Giving pafolacianine for NIR imaging may work better than other imaging agents in identifying cancerous lesions in pediatric patients with solid tumors.

Conditions

Childhood Malignant Solid Neoplasm

Childhood Malignant Thoracic Neoplasm

Malignant Abdominal Neoplasm

Malignant Neck Neoplasm

Malignant Pelvic Neoplasm

Study ID

NCT06915727

Start date

Apr 15, 2025

Status verified date

Mar, 2026

Completion date

May 1, 2027

Anticipated

Primary completion date

May 1, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 17

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Willingness of research participant or legal guardian/representative to give written informed consent
  • Age 6 months to 17 years
  • Have a diagnosis, or a high clinical suspicion, of a solid tumor in the neck, chest, abdomen, pelvis, or extremities with a plan for surgical resection including embryonal tumors, sarcomas, neurogenic tumors, carcinomas, mesenchymal tumors, germ cell tumors. Children may undergo surgical resection or biopsy of a solid tumor that is suspected to be malignant without a separate preoperative biopsy; therefore, the tumor type may not be definitively known preoperatively

Exclusion Criteria:

  • Previous exposure to Cytalux™ (pafolacianine) injection
  • Any medical condition that in the judgement of the investigators could potentially jeopardize the safety of the participant
  • History of anaphylactic reactions to products containing indocyanine green for near infrared imaging. Participants with a medical history of 'idiopathic anaphylaxis' will require evaluation
  • History of allergy to any of the components of Cytalux™ (pafolacianine) injection
  • Presence of any psychological, familial, sociological condition or geographical challenges potentially hampering compliance with the study protocol or follow-up schedule
  • Received an investigational agent in another investigational drug or vaccine trial within 30 days prior to the administration of the study drug. Investigational trials involving administration of drugs or vaccines which are already Food and Drug Administration (FDA)-approved, such as Children's Oncology Group cooperative trials, are okay
  • Known sensitivity to fluorescent light
  • Pregnancy
  • Unable or unwilling to discontinue folic acid, folate supplements, and multi-vitamins containing folate 48 hours prior to drug administration
  • Renal failure on dialysis or estimated glomerular filtration rate (eGFR) < 30 mL/min/1.72m\^2 as measured by the U25 eGFR equation for patients ≥1 year(s) of age. For patients < 1 year of age, creatinine > 2x the upper limit of normal will serve as an exclusion criteria. Creatinine will be measured by enzymatic assay with calibration traceable to the international standard reference materials and minimal bias compared to isotope-dilution mass spectrometry (IDMS) reference methodology.
  • Undergoing workup for liver failure or listed for liver transplant. Patients with Gilbert's syndrome will be excluded if impaired liver function defined as values > 3x the upper limit of normal (ULN) for alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP), or total bilirubin
  • Unable or unwilling to use adequate birth control/contraceptive measures or abstain from sexual activity for 30 days following the study intervention
  • Anticipated need to donate ova or sperm within 30 days following study intervention

Study Design

Enrollment

20 participants

Anticipated

Intervention Model

Single group

Primary purpose

Diagnostic

Interventions and Outcome Measures

Arms

experimental: Diagnostic (pafolacianine + NIR)

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.

Interventions

Biospecimen Collection

Undergo tissue sample collection

Near Infrared Imaging

Undergo NIR fluorescent imaging

Pafolacianine Sodium

Given IV

Surgical Procedure

Undergo SOC surgery

Primary outcome measure

  • Accuracy of Cytalux™ (pafolacianine) injection used with NIR fluorescent imaging for detecting lesions [ Time Frame: Up to 30 days after surgery ]

Central Contacts and Locations

Central contacts

Clinical Trials Referral Office

855-776-0015mayocliniccancerstudies@mayo.edu

Gina Lewis

507-284-0850DOSCRO2@mayo.edu

Locations

Mayo Clinic in Rochester

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Gina Lewis

507-284-0850DOSCRO2@mayo.edu

Clinical Trials Referral Office

855-776-0015mayocliniccancerstudies@mayo.edu

Principal Investigator:

Stephanie F. Polites, MD, MPH

More Information

Sponsor

Mayo Clinic

Last update posted

Apr 1, 2026

Last verified

Mar, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Mayo Clinic on 2026-04-01.