An Imaging Agent (Pafolacianine) for Identifying Lesions in Pediatric Patients With Primary or Metastatic Solid Tumors

NCT06915727 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: MC25070124-012249
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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 Thoracic Neoplasm; Malignant Abdominal Neoplasm; Malignant Neck Neoplasm; Malignant Pelvic Neoplasm; Metastatic Childhood Malignant Solid Neoplasm; Childhood Malignant Solid Neoplasm

Outcome Measures

Primary Outcomes (1)

• Accuracy of Cytalux™ (pafolacianine) injection used with NIR fluorescent imaging for detecting lesions

  Each excised specimen will be analyzed for histologic findings to determine accuracy of Cytalux™ (pafolacianine) injection used with near infrared (NIR) fluorescent imaging for detecting lesions. Accuracy will be assessed using sensitivity and specificity.

  Up to 30 days after surgery

Secondary Outcomes (3)

• Time between Cytalux™ administration and NIR imaging detection intraoperatively

  Day 0 to 1

• Reasons for not excising NIR avid lesions

  Day 0 to 1

• Incidence of Adverse Events surgery

  From study enrollment up to 30 days after surgery

Study Arms (1)

Diagnostic (pafolacianine + NIR)

EXPERIMENTAL

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.

Procedure: Biospecimen Collection; Procedure: Near Infrared Imaging; Drug: Pafolacianine Sodium; Procedure: Surgical Procedure

Interventions

Near Infrared Imaging PROCEDURE

Undergo NIR fluorescent imaging

Also known as: Near-Infrared Imaging, NIR Imaging

Diagnostic (pafolacianine + NIR)

Pafolacianine Sodium DRUG

Given IV

Also known as: Cytalux

Diagnostic (pafolacianine + NIR)

Surgical Procedure PROCEDURE

Undergo SOC surgery

Also known as: Operation, Surgery, Surgery Type, Surgery, Not Otherwise Specified (NOS), Surgical, Surgical Intervention, Surgical Interventions, Surgical Procedures, Type of Surgery, Surgery, NOS

Diagnostic (pafolacianine + NIR)

Biospecimen Collection PROCEDURE

Undergo tissue sample collection

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection

Diagnostic (pafolacianine + NIR)

Eligibility Criteria

• Age: 6 Months - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

RECRUITING

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Head and Neck Neoplasms; Pelvic Neoplasms

Interventions

Specimen Handling; Pafolacianine; Surgical Procedures, Operative

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques

Study Officials

• Stephanie F. Polites, MD, MPH

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Clinical Trials Referral Office

CONTACT

855-776-0015; mayocliniccancerstudies@mayo.edu

Gina Lewis

CONTACT

507-284-0850; DOSCRO2@mayo.edu

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 28, 2025
• First Posted: April 8, 2025
• Study Start: April 15, 2025
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): May 1, 2027
• Last Updated: April 1, 2026

Record last verified: 2026-03

Locations

US (1)