18F-DCFPyL PET/CT in Hepatocellular Carcinoma

NCT05009979 | Terminated Phase 2 Results FDA Drug

• 2 other identifiers: 10000080000080-C
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

Background: A radiotracer (or tracer) is a radioactive substance. It is used in Positron Emission Tomography (PET) imaging to help see specific sites in the body. Researchers want to learn if a new tracer can help them better identify hepatocellular cancer (HCC) in people. Objective: To learn if a radiotracer called piflufolastat F-18 (18F-DCFPyL), can identify sites of HCC better than current standard imaging. Eligibility: Adults aged 18 years and older who may have HCC based on previous standard imaging. Design: Participants will be screened with a medical history, physical exam, and blood tests. They will have a computed tomography (CT) and/or magnetic resonance imaging (MRI) scan. Participants will have a whole-body positron emission tomography (PET/CT) scan. The PET and CT scanners use x-rays to make pictures of the inside of the body. The PET uses a tracer to help make the pictures. Participants will get an intravenous (IV) injection of 18F-DCFPyL 1 hour before the scan. Within two weeks, participants will have a Fludeoxyglucose F 18 (18F-FDG) PET/CT scan. 18F-FDG is a commonly used tracer. They will get 18F-FDG via IV 1 hour before the scan. Participants will have a CT/magnetic resonance imaging (MRI) within 2 months of the first 18F-DCFPyL PET/CT. Participants will have standard treatment for their cancer. During treatment, they will have a tumor biopsy. If the biopsy shows they do not have HCC, they will be removed from the study. For participants who have HCC and their cancer was identified in the 18F-DCFPyL PET/CT, they will have a second 18F-DCFPyL PET/CT and 18F-FDG PET/CT. Participants will have follow-up visits every 3 months for 2 years. Then they will have yearly visits for 3 years.

Conditions

Hepatocellular Carcinoma

Keywords

Imaging; Liver Cancer; Radiotracer; PSMA

Outcome Measures

Primary Outcomes (2)

• Positive Predictive Value (PPV) Defined as the Proportion of Histopathology Positive Lesions as Measured by the Maximum Standardized Uptake (SUVmax) Value

  Positive predictive value is defined as the proportion of histopathologically positive lesions. Positron emission tomography (PET) positivity was measured by the maximum standardized uptake (SUVmax) value. The 95% confidence intervals of the positive predictive value of piflufolastat F-18 (18F-DCFPyL) positron emission tomography scan and computed tomography (PET/CT) will be reported in which the confidence limits are the 2.5th and 97.5th percentile of the 2000 bootstrap samples obtained by random sample without replacement at the participant level to account for inter-lesion correlation. For lesions within the liver, a focal abnormal area of increased 18F-DCFPyL activity higher than the surrounding liver uptake (SUVmax more than x 1.2 times than the normal liver-SUV mean) will be considered positive. For lesions outside the liver, a positive lesion is defined as focal abnormal uptake higher than the blood pool or surrounding normal organ or soft tissue background.

  Baseline, post ablation, and disease progression, an average of 3.87 months

• Point Estimates of the Positive Predictive Value of Piflufolastat F-18 (18F-DCFPyL)

  Positive predictive value of the DCFPyL PET imaging agent is defined as the proportion of radiologically positive lesions (true positives) that were PET positive, over the radiologically (CT/MRI) positive lesions that were PET positive (true positives) added to the radiologically negative lesions that were PET positive (false positives). Point estimates and 95% confidence intervals of the positive predictive values of piflufolastat F-18 (18F-DCFPyL) positron emission tomography scan and computed tomography (PET/CT) will be reported in which the confidence limits are the 2.5th and 97.5th percentile of the 2000 bootstrap samples obtained by random sample without replacement at the participant level to account for inter-lesion correlation.

  Baseline, post ablation, disease progression, an average of 3.87 months

Secondary Outcomes (4)

• Lesion Level Sensitivity

  Baseline, post ablation, and disease progression, an average of 3.87 months

• Lesion Level Specificity

  Baseline, post ablation, and disease progression, an average of 3.87 months

• Lesion Level Positive Predictive Value

  Baseline, post ablation, and disease progression, an average of 3.87 months

• Change in Piflufolastat F-18 (18F-DCFPyL) Positron Emission Tomography and Computed Tomography (PET/CT) Maximum Standardized Uptake Value (SUVmax) Between Pre- and Post-treatment

  Pre- and post-treatment hepatocellular carcinoma (HCC) 18F-DCFPyL PET scans, an average of 3.2 months.

Other Outcomes (1)

• Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)

  Document adverse events from the first study intervention through 3 days after the agent was last administered, an average of 3.87 months

Study Arms (1)

Arm 1, Cohort 1 Baseline and Post-treatment Imaging with Piflufolastat F-18 (18F-DCFPyL)

EXPERIMENTAL

Piflufolastat F-18 (18F-DCFPyL) positron emission tomography computed tomography (PET/CT) imaging, CT and/or magnetic resonance imaging (MRI) and standard of care local ablative treatment

Drug: F18-FDG; Device: MRI; Device: CT; Drug: F18-DCFPyL; Procedure: Tumor biopsy

Interventions

F18-FDG DRUG

Within approximately 2 weeks of each piflufolastat F-18 (18F-DCFPyL) positron emission tomography/ computed tomography (PET/CT) scan, participants will be scanned with a fludeoxyglucose F 18 (18F-FDG) PET/CT imaging at the NIH Clinical Center using standard procedures. The 18F-FDG PET/CT imaging performed will allow the localization of viable tumor sites and characterize their FDG metabolism for comparison with 18F-DCFPyL imaging. The 18F-FDG PET/CT imaging will consist of an 18F-FDG injection and PET/CT imaging performed approximately 1 hour post 18F-FDG injection. A corresponding low dose CT scan for attenuation correction and co-registration purposes will be performed prior to the PET image.

Also known as: Fludeoxyglucose F 18

Arm 1, Cohort 1 Baseline and Post-treatment Imaging with Piflufolastat F-18 (18F-DCFPyL)

MRI DEVICE

A standard of care computed tomography (CT) and/or magnetic resonance imaging (MRI) will be performed within 2 months of each piflufolastat F-18 (18F-DCFPyL) positron emission tomography (PET/CT).

Also known as: Magnetic resonance imaging

Arm 1, Cohort 1 Baseline and Post-treatment Imaging with Piflufolastat F-18 (18F-DCFPyL)

CT DEVICE

A standard of care computed tomography (CT) and/or magnetic resonance imaging (MRI) will be performed within 2 months of each piflufolastat F-18 (18F-DCFPyL) positron emission tomography (PET/CT).

Also known as: Computed tomography

Arm 1, Cohort 1 Baseline and Post-treatment Imaging with Piflufolastat F-18 (18F-DCFPyL)

F18-DCFPyL DRUG

Each subject will receive a single intravenous (IV) dose of piflufolastat F-18 (18F-DCFPyL) by bolus injection at a rate of approximately 1 ml/3-5 sec. The maximum amount of injected active drug will be less than 4.02 microgram. The target administered activity will be 9 mCi. The 18F-DCFPyL positron emission tomography and computed tomography (PET/CT) imaging will consist of the 18F-DCFPyL injection, followed by approximately 45 min dynamic CT imaging of a single bed position (including the liver lesion), and a static whole-body PET/CT imaging (top of head to mid-thighs) performed at 1 hour (+/-10 minutes) post 18F-DCFPyL injection. Only a single injection of 18F-DCFPyL is required. The initial 45 minutes dynamic regional scan will be used to determine the kinetics of 18F-DCFPyL within the tumor as compared with normal liver and other background.

Also known as: piflufolastat F-18

Arm 1, Cohort 1 Baseline and Post-treatment Imaging with Piflufolastat F-18 (18F-DCFPyL)

Tumor biopsy PROCEDURE

Principal investigator discretion pre-treatment or during surgical resection.

Arm 1, Cohort 1 Baseline and Post-treatment Imaging with Piflufolastat F-18 (18F-DCFPyL)

Eligibility Criteria

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

You may qualify if:

• High radiological suspicion of hepatocellular carcinoma (LR4 (probably hepatocellular carcinoma) or LR5 (definitely hepatocellular carcinoma) based on the most current version of Liver Imaging Reporting \& Data System (LI-RADS) with at least one measurable lesion on standard imaging modality computed tomography (CT) and/or magnetic resonance imaging (MRI).
• Eligible for local therapies (included but not limited to surgical resection, stereotactic radiation therapy, transarterial chem/radio/bland embolization, microwave ablation, radiofrequency ablation).
• Ability to take oral medication and be willing to adhere to the study intervention regimen.
• Age \>=18 years.
• Eastern Cooperative Oncology Group (ECOG) performance status \<=2.
• Known human immunodeficiency virus (HIV)-infected individuals must be on effective anti-retroviral therapy with undetectable viral load within 6 months.
• Known chronic hepatitis B virus (HBV) infected individuals, must be on suppressive therapy with undetectable viral load.
• Individuals with a history of hepatitis C virus (HCV) infection must have been treated and cured.
• The effects of piflufolastat F-18 (18F-DCFPyL) (study drug) on the developing human fetus are unknown. For this reason and because this agent as well as other therapeutic agents used in this trial are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for 2 months after each study positron emission tomography and computed tomography (PET/CT) imaging. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
• Ability of subject to understand and the willingness to sign a written informed consent document

You may not qualify if:

• History of allergic reactions attributed to compounds of similar chemical or biologic composition to 18F-DCFPyL, or other agents used in study.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
• Subjects with severe claustrophobia unresponsive to oral anxiolytics.
• Other medical conditions deemed by the principal investigator (or associates) to make the subject unsafe/ineligible for protocol procedures.
• Subjects weighing \> 350 lbs. (weight limit for scanner table), or unable to fit within the imaging gantry
• Serum creatinine \> 2 times the upper limit of normal
• Pregnant women are excluded from this study because 18F-DCFPyL is an agent with the potential for teratogenic or abortifacient effects. as well as other agents used in this trial are known to be teratogenic.

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

Related Publications (1)

• Mena E, Shih J, Chung JY, Jones J, Rabiee A, Monge C, Turkbey B, Lindenberg L, Salerno KE, Kassin M, Wood B, Hernandez J, Maass-Moreno R, Saboury B, Jakhete N, Molitoris JK, Unger KR, Choyke PL, Escorcia FE. Functional Imaging of Liver Cancer (FLIC): Study protocol of a phase 2 trial of 18F-DCFPyL PET/CT imaging for patients with hepatocellular carcinoma. PLoS One. 2022 Nov 11;17(11):e0277407. doi: 10.1371/journal.pone.0277407. eCollection 2022.

  PMID: 36367894 DERIVED

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Carcinoma, Hepatocellular; Liver Neoplasms

Interventions

Fluorodeoxyglucose F18; Magnetic Resonance Imaging; Tomography, X-Ray Computed; 2-(3-(1-carboxy-5-((6-fluoropyridine-3-carbonyl)amino)pentyl)ureido)pentanedioic acid

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Digestive System Diseases; Liver Diseases

Intervention Hierarchy (Ancestors)

Deoxyglucose; Deoxy Sugars; Carbohydrates; Tomography; Diagnostic Imaging; Diagnostic Techniques and Procedures; Diagnosis; Image Interpretation, Computer-Assisted; Radiographic Image Enhancement; Image Enhancement; Photography; Radiography; Tomography, X-Ray

Results Point of Contact

• Title: Dr. Esther Mena Gonzalez
• Organization: National Cancer Institute

esther.menagonzalez@nih.gov

240-760-6111

Study Officials

• Esther Mena Gonzalez, M.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: August 17, 2021
• First Posted: August 18, 2021
• Study Start: January 18, 2023
• Primary Completion: November 14, 2024
• Study Completion: November 20, 2024
• Last Updated: August 28, 2025
• Results First Posted: August 28, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Clinical data available during the study and indefinitely. Genomic data is available once genomic data are uploaded per protocol Genomic Data Sharing (GDS) plan for as long as database is active.
• Access Criteria: Clinical data will be made available via subscription to Biomedical Translational Research Information System (BTRIS) and with the permission of the study principal investigator (PI). Genomic data is made available via the database of Genotypes and Phenotypes (dbGaP) through requests to the data custodians.

Locations

US (1)