A Phase 2a, Single-dose, Open-label Study to Evaluate Diagnostic Performance and Safety of Pegsitacianine, an Intraoperative Fluorescence Imaging Agent for the Detection of Cancer, in Patients With Unknown Primary Head and Neck Cancer (ILLUMINATE STUDY)

NCT05576974 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: STU-2022-04601R01CA266146-01
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 1

Brief Summary

This is a non-randomized, open-label, single-center, safety and imaging feasibility study of Pegsitacianine, an intraoperative fluorescence imaging agent.

Conditions

Head and Neck Cancer; Unknown Primary Cancer; Head and Neck Squamous Cell Carcinoma

Outcome Measures

Primary Outcomes (2)

• Diagnostics performance and safety of 1 mg/kg Pegsitacianine in patients with HNSCC

  Pegsitacianine fluorescence from in vivo and excised tissues will be imaged using intraoperative and post-operative NIR cameras and will be correlated with the histopathological confirmation of tumor and normal tissues to determine Pegsitacianine's diagnostic performance. Patient safety will be assessed for 10 days (±48 hours) post dose. All patients will be monitored for vital signs and physical examination pretreatment and at various time points up to Day 6 (±24 hours). All patients will be monitored for comprehensive metabolic panel (CMP) and complete blood count (CBC) with differentials pretreatment and at various time points up to Day 10 (±48 hours). All patients will be monitored for TEAEs, and concomitant medication use from the start of dosing up to Day 10 (±48 hours). TEAEs will be followed closely during the study to identify any potential DLTs

  5 years

• Diagnostics performance and safety of 1 mg/kg Pegsitacianine in patients with UPC

  Pegsitacianine fluorescence from in vivo and excised tissues will be imaged using intraoperative and post-operative NIR cameras and will be correlated with the histopathological confirmation of tumor and normal tissues to determine Pegsitacianine's diagnostic performance. Patient safety will be assessed for 10 days (±48 hours). All patients will be monitored for vital signs and physical examination pretreatment and at various time points up to Day 6 (±48 hours). All patients will be monitored for comprehensive metabolic panel (CMP) and complete blood count (CBC) with differentials pretreatment and at various time points up to Day 10 (±48 hours). All patients will be monitored for TEAEs, and concomitant medication use from the start of dosing up to Day 10 (±48 hours). TEAEs will be followed closely during the study to identify any potential DLTs

  5 years

Secondary Outcomes (1)

• Pegsitacianine fluorescence imaging in HNSCC, UPC primary cancers that are HPV positive and HPV negative

  5 years

Study Arms (2)

Part 1 - Cohort A

ACTIVE COMPARATOR

In Part 1 the single dose of 1 mg/kg of pegsitacianine given 6-300 hours prior to surgery will be used to image primary tumors in patients with HNSCC to evaluated to verify the diagnostic performance of pegsitacianine fluorescence imaging for detecting primary tumors and metastatic lymph nodes.

Drug: Pegsitacianine

Part 2 - Cohort B

ACTIVE COMPARATOR

In Part 2, pegsitacianine the single dose of 1 mg/kg of given 6-300 hours prior to surgery will be used to in patients with unknown primary cancer of the head and neck. These patients typically undergo exam under anesthesia with a laryngoscopy as well as panendoscopy for identifying the source of the metastatic cancer found in the cervical nodes. The diagnostic performance of pegsitacianine fluorescence imaging for detecting primary tumors and metastatic lymph nodes in these patients will be evaluated. All the available data to date will be used to decide the additional tumor type(s), number of patients per tumor type, and number of Group(s) to be enrolled.

Drug: Pegsitacianine

Interventions

Pegsitacianine DRUG

Infusion of the Pegsitacianine-Intraoperative fluorescence imaging

Part 1 - Cohort A; Part 2 - Cohort B

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adults ≥18 years of age
• Biopsy-confirmed diagnosis, for primary or recurrent disease (or high clinical suspicion in the opinion of the Investigator)
• Part 1: Stage 1 to 4 HNSCC
• Part 2: UPC squamous cell carcinoma of the head and neck with metastatic disease to at least a single cervical node, AND no biopsy proven evidence of the primary cancer's location.
• Acceptable hematologic status (as standard surgery protocol requires, as determined by the Investigator), kidney function and liver function. Elevations of creatinine, alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase, or total bilirubin \>1.5× the upper limit of normal \[ULN\] must be determined to be not clinically significant by the Investigator and approved by the Medical Monitor.
• Documented negative serum pregnancy test for women of childbearing potential (i.e., premenopausal women with intact reproductive organs and women \<2 years after menopause)
• Male patients and female patients of child-bearing potential (i.e., premenopausal women with intact reproductive organs and women \<2 years after menopause) must agree to and comply with using medically acceptable contraception including surgical sterilization (e.g., hysterectomy, bilateral oophorectomy, bilateral tubal ligation), intrauterine device, oral contraceptive, contraceptive patch, long acting injectable contraceptive, partner's vasectomy, double-barrier method (condom or diaphragm plus spermicide or condom plus diaphragm), or abstinence during the trial and for 6 months thereafter
• Agree to abstain from alcohol consumption from 72 hours before Pegsitacianine administration through completion of Study Day 10 (±48 hours) visit in Part 1 and Part 2.
• Adequate potential for follow up

You may not qualify if:

• Tumors at sites of which the surgeon would assess that in vivo intraoperative imaging would not be feasible.
• Life expectancy \<12 weeks
• Karnofsky Performance Status \<70%
• Hepatic impairment (Child-Pugh score \>5) or significant liver disease including active hepatitis or cirrhosis
• Lab values or any sign, symptom, or medical condition that in the opinion of the PI would prevent surgical resection
• Medical or psychiatric conditions that compromise the patient's ability to give informed consent.
• Pregnant or lactating women
• Receiving or planned to receive, during the duration of the study, concomitant medication with a high chance of hepatotoxicity, as judged by the PI based on standard protocols within the study center
• Alcohol consumption within 72 hours before Pegsitacianine administration
• Received an investigational agent within the shorter of 5 half-lives or 30 days before Pegsitacianine dosing
• Inability to adhere to the schedule of assessments or any circumstance that would interfere with the validity of assessments performed in the study
• The PI considers that the patient should not participate in the study

Sponsors & Collaborators

• University of Texas Southwestern Medical Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

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Related Links

• Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 5.0. National Cancer Institute 27NOV2017
• Declaration of Helsinki
• ICH E6 (R2). Integrated addendum to ICH E6(R1): Guideline for good clinical practice. 09NOV2016
• FDA Bioanalytical Method Validation Guidance for Industry, May 2018
• EAFUS - Everything Added to Food in the United States. PEG: ASP, Doc#1285, CAS# 25322-68-3. Polymethacrylate: ASP, Doc# 1406, CAS# 54193-36-1

MeSH Terms

Conditions

Head and Neck Neoplasms; Neoplasms, Unknown Primary; Squamous Cell Carcinoma of Head and Neck

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Neoplasm Metastasis; Neoplastic Processes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Carcinoma, Squamous Cell; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type

Central Study Contacts

Baran Sumer, MD

CONTACT

2146482904; Baran.Sumer@UTSouthwestern.edu

Sindhu Voorugonda, MBBS

CONTACT

2146488096; Sindhunikhila.voorugonda@utsouthwestern.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Masking Details: Open Label
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor and Chief, Division of Head and Neck Oncology

Model Details: In Part 1 the single dose of 1 mg/kg of pegsitacianine given 6-300 hours prior to surgery will be used to image primary tumors in patients with HNSCC to evaluated to verify the diagnostic performance of pegsitacianine fluorescence imaging for detecting primary tumors and metastatic lymph nodes. In Part 2, pegsitacianine the single dose of 1 mg/kg of given 6-300 hours prior to surgery will be used to in patients with unknown primary cancer of the head and neck. These patients typically undergo exam under anesthesia with a laryngoscopy as well as panendoscopy for identifying the source of the metastatic cancer found in the cervical nodes. Subjects for Part 2 may begin dosing at any time during Part 1 thus the two parts can run concurrently.

Study Record Dates

• First Submitted: October 5, 2022
• First Posted: October 13, 2022
• Study Start: April 17, 2024
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: April 14, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)