Clinical trial NCT06915727: An Imaging Agent (Pafolacianine) for Identifying Lesions in Pediatric Patients With Primary or Metastatic Solid Tumors
Official title: Pafolacianine for Localization of Pediatric Extracranial Solid Tumors
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Childhood Malignant Solid Neoplasm
Childhood Malignant Thoracic Neoplasm
Malignant Abdominal Neoplasm
Malignant Neck Neoplasm
Malignant Pelvic Neoplasm
Metastatic Childhood Malignant Solid Neoplasm
Lead sponsor: Mayo Clinic
Sponsor class: OTHER
First posted: 2025-04-08
Enrollment: 20
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

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.


Eligibility criteria: 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

Primary outcomes:
measure: Accuracy of Cytalux™ (pafolacianine) injection used with NIR fluorescent imaging for detecting lesions
timeFrame: Up to 30 days after surgery
Secondary outcomes:
measure: Time between Cytalux™ administration and NIR imaging detection intraoperatively
timeFrame: Day 0 to 1
measure: Reasons for not excising NIR avid lesions
timeFrame: Day 0 to 1
measure: Incidence of Adverse Events surgery
timeFrame: From study enrollment up to 30 days after surgery
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic in Rochester
state: Minnesota
Study officials:
affiliation: Mayo Clinic
name: Stephanie F. Polites, MD, MPH
role: PRINCIPAL_INVESTIGATOR
References: