Single Dose Investigator Initiated Pilot Study to Investigate CYTALUX (Pafolacianine) for Intraoperative Detection of Malignant Tissue in Subjects Undergoing Surgical Resection for Cancer.

NCT07039526 | Recruiting Phase 2 FDA Drug

• 1 other identifier: STU20250049
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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 camera system in adults with a suspected primary diagnosis, or a high clinical suspicion of gastrointestinal, fore gut, pancreatic, hepatobiliary, esophageal malignancies and gyn malignancies planned for HIPEC/debulking, warranting surgery. The main question it aims to answer is: • Can CYTALUX™ (pafolacianine) injection used with near-infrared (NIR) fluorescent imaging improve the detection of malignant tissue in subjects undergoing surgical resection for cancer? Participants taking part in this study will receive the study medication. Then, during the surgery the investigator team will turn on the camera to evaluate if the tumor is visible with the help of the study medication. Taking part in this study will last about 2 months.

Conditions

Esophageal Cancer; Gastrointestinal Cancers; Gynecologic Cancers

Keywords

Pafolacianine; CYTALUX; Folate receptor-alpha; Near-infrared fluorescence imaging; Intraoperative imaging; Fluorescence-guided surgery; esophageal cancer; gastrointestinal cancer

Outcome Measures

Primary Outcomes (1)

• Proportion of subjects with one or more NIR fluorescence positive lesion not detected under normal light and/or palpation

  Proportion of subjects who have one or more NIR fluorescence positive lesion not detected under normal light and/or palpation will be measured in this outcome.

  Day 1 intra-operatively post intervention

Secondary Outcomes (1)

• Number of lesions that are simultaneously fluorescence positive and cancer positive

  Day 1 intra-operatively post intervention

Study Arms (1)

CYTALUX™ (pafolacianine) injection group

EXPERIMENTAL

Single arm study, every patient receives the study intervention.

Drug: CYTALUX™ (pafolacianine) injection preoperatively

Interventions

CYTALUX™ (pafolacianine) injection preoperatively DRUG

CYTALUX™ (pafolacianine) injection: folate analog ligand conjugated with an indole cyanine green-like dye as a solution in vials containing 1.6 mL at 2 mg/mL. Each subject will be administered a single intravenous dose of not less than 1 hours before and not more than 24 hours before initiation of intraoperative NIR fluorescent imaging.

CYTALUX™ (pafolacianine) injection group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and Female patients 18 years of age and older
• Have a primary diagnosis, or a high clinical suspicion, of gastrointestinal, fore gut, pancreatic, hepatobiliary, esophageal malignancies and gyn malignancies planned for HIPEC/debulking, warranting surgery.
• Are scheduled to undergo surgical intervention for tumor resection or diagnostic laparoscopy for assessment of disease burden.
• Willingness to stop the use of folate, folic acid, or folate-containing supplements within 48 hours before administration of Cytalux
• Willingness of research participant or legal guardian/representative to give written informed consent.

You may not qualify if:

• Any medical condition that in the opinion of the investigators could potentially jeopardize the safety of the subject
• History of anaphylactic reactions to products containing indocyanine green for near infrared imaging. Subjects 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

Sponsors & Collaborators

• John Waters: lead
• On Target Laboratories, LLC: collaborator

Study Sites (1)

Clemments University Hospital

Dallas, Texas, 75235, United States

RECRUITING

Related Links

• OTL38 for Intra-operative Imaging of Folate Receptor Positive Ovarian Cancer

MeSH Terms

Conditions

Esophageal Neoplasms; Gastrointestinal Neoplasms

Interventions

Pafolacianine

Condition Hierarchy (Ancestors)

Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Head and Neck Neoplasms; Digestive System Diseases; Esophageal Diseases; Gastrointestinal Diseases

Study Officials

• John Waters, M.D.

  University of Texas Southwestern Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

John Waters, M.D.

CONTACT

214-645-7700; john.waters@utsouthwestern.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: MD, Assistant Professor, Cardiothoracic Surgery

Study Record Dates

• First Submitted: June 17, 2025
• First Posted: June 26, 2025
• Study Start: August 19, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: September 24, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)