NCT05009979

18F-DCFPyL PET/CT in Hepatocellular Carcinoma

Terminated Phase 2 Results posted Last updated 28 August 2025

What this trial tests

Phase 2 trial testing F18-FDG in Hepatocellular Carcinoma in 8 participants. Terminated before completion.

Timeline

18 January 2023

Primary endpoint
14 November 2024

20 November 2024

Quick facts

Lead sponsor	National Cancer Institute (NCI)
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	diagnostic
Enrollment	8
Start date	18 January 2023
Primary completion	14 November 2024
Estimated completion	20 November 2024
Sites	1 location across United States

Drugs / interventions tested

• F18-FDG — full drug profile →
• MRI — full drug profile →
• CT
• F18-DCFPyL — full drug profile →
• Tumor biopsy

Conditions studied

• Hepatocellular Carcinoma — all drugs for Hepatocellular Carcinoma →

Sponsor

National Cancer Institute (NCI)

Who can join

18 and older, any sex, with Hepatocellular Carcinoma. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Positive Predictive Value (PPV) Defined as the Proportion of Histopathology Positive Lesions as Measured by the Maximum Standardized Uptake (SUVmax) Value Primary · Baseline, post ablation, and disease progression, an average of 3.87 months

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 at baseline

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	0.875	0.64 – 1.10

Point Estimates of the Positive Predictive Value of Piflufolastat F-18 (18F-DCFPyL) Primary · Baseline, post ablation, disease progression, an average of 3.87 months

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 percen

Baseline

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	0.64	0.39 – 0.89

Lesion Level Sensitivity Secondary · Baseline, post ablation, and disease progression, an average of 3.87 months

Lesion level sensitivity is defined as true positive (TP)/\[TP+ false negative (FN\], being TP = true positive lesions (i.e., positron emission tomography (PET) positive lesions that are histologically positive) and FN = false negative lesions (i.e., PET negative lesions that are histologically positive) . The lesion level sensitivity of piflufolastat F-18 (18F-DCFPyL) positron emission tomography scan and a computed tomography (PET/CT) and CT/magnetic resonance imaging (MRI) will be calculated and compared. The confidence interval for each estimate will be obtained from the bootstrap samples

Sensitivity with CT/MRI at baseline

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	1.0	1.0 – 1.0

Sensitivity with 18F-DCFPyL PET/CT at baseline

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	0.87	0.70 – 1.04

Lesion Level Specificity Secondary · Baseline, post ablation, and disease progression, an average of 3.87 months

Lesion level specificity is defined as true negative (TN)/\[TN+ false positive (FP\], being TN = true negative (i.e., positron emission tomography (PET) negative lesions that are histopathologically negative, and FP = false positive (i.e., PET positive lesions that are histopathologically negative). The lesion level specificity of piflufolastat F-18 (18F-DCFPyL) positron emission tomography scan and a computed tomography (PET/CT) and CT/magnetic resonance imaging (MRI) will be calculated and compared. The confidence interval for each estimate will be obtained from the bootstrap samples and the

Specificity with CT/MRI at baseline

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	NA	NA – NA

Specificity with 18F-DCFPyL PET/CT at baseline

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	NA	NA – NA

Lesion Level Positive Predictive Value Secondary · Baseline, post ablation, and disease progression, an average of 3.87 months

Lesion level positive predictive value is defined as true positive (TP/\[TP+ false positive (FP\], being TP = true positive (i.e., positron emission tomography (PET) positive lesions that are histopathologically positive) and FP = false positive (i.e., PET positive lesions that are histopathologically negative) . The lesion level positive predictive value of piflufolastat F-18 (18F-DCFPyL) positron emission tomography scan and a computed tomography (PET/CT) and CT/magnetic resonance imaging (MRI) will be calculated and compared. The confidence interval for each estimate will be obtained from t

Positive Predictive Value with CT/MRI at baseline

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	0.57	0.31 – 0.83

Positive Predictive Value with 18F-DCFPyL PET/CT at baseline

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	0.50	0.23 – 0.76

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

Change in 18F-DCFPyL PET/CT maximum standardized uptake value (SUVmax) between pre- and post-treatment tumor or tumor bed will be compared by paired Wilcoxon test for participants who undergo local treatment for hepatocellular carcinoma. Positive uptake is defined as a focal abnormal area of increased 18F-DCFPyL activity higher than the surrounding liver uptake standard update value (SUV)max more than x 1.2 times than the normal liver-SUV mean). Negative uptake is defined as tumor uptake less than adjacent background soft tissue, or less than blood pool for lymph nodes.

Tumor pre-treatment

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	10.72	1.88 – 12.60

Tumor post-treatment

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	4.61	2.34 – 6.87

Tumor bed pre-treatment

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	10.72	1.88 – 12.60

Tumor bed post-treatment

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	4.61	2.34 – 6.87

Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0) Secondary · Document adverse events from the first study intervention through 3 days after the agent was last administered, an average of 3.87 months

Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent

Group	Value	95% CI
Arm 1, Cohort 1, Baseline and Post-treatment Imaging With Piflufolastat F-18 (18F-DCFPyL)	7

Adverse events — posted to ClinicalTrials.gov

Time frame: All-Cause Mortality was monitored/assessed an average of 3.87 months. Adverse Events were monitored/assessed from the first study intervention through 3 days after the agent was last administered, an average of 3.87 months.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

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

Serious: 1/8 (13%)

Deaths: 0/8

Serious adverse events (1 terms)

Reaction	System	Arm 1, Cohort 1, Baseline …
Ascites	Gastrointestinal disorders	—

Other adverse events (17 terms — click to expand)

Reaction	System	Arm 1, Cohort 1, Baseline …
White blood cell decreased	Investigations	—
Alkaline phosphatase increased	Investigations	—
Anemia	Blood and lymphatic system disorders	—
Aspartate aminotransferase increased	Investigations	—
Lymphocyte count decreased	Investigations	—
Neutrophil count decreased	Investigations	—
Platelet count decreased	Investigations	—
Abdominal pain	Gastrointestinal disorders	—
Fatigue	General disorders	—
Hypoalbuminemia	Metabolism and nutrition disorders	—
Alanine aminotransferase increased	Investigations	—
Anorexia	Metabolism and nutrition disorders	—
Bruising	Injury, poisoning and procedural complications	—
Cough	Respiratory, thoracic and mediastinal disorders	—
Hypokalemia	Metabolism and nutrition disorders	—
Hyponatremia	Metabolism and nutrition disorders	—
Portal vein thrombosis	Hepatobiliary disorders	—

Most-reported serious reactions: Ascites.

Data from ClinicalTrials.gov NCT05009979 adverse events section.

Sponsor's own description

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.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. A Primer on Radiopharmaceutical Therapy.
   Salerno KE, Roy S, Ribaudo C, Fisher T, et al · · 2023 · cited 25× · PMID 35970373 · DOI 10.1016/j.ijrobp.2022.08.010
2. Functional Imaging of Liver Cancer (FLIC): Study protocol of a phase 2 trial of 18F-DCFPyL PET/CT imaging for patients with hepatocellular carcinoma.
   Mena E, Shih J, Chung JY, Jones J, et al · · 2022 · cited 6× · PMID 36367894 · DOI 10.1371/journal.pone.0277407