Chronic Liver Disease, HepatoCellular Carcinoma
Conditions
Brief summary
This study is an exploratory study aiming to collect data about diagnosis efficacy (sensitivity and specificity) of P03277 triphasic liver imaging for HCC in subjects with suspected small nodules and chronic liver disease. 30 subjects will be included, having HCC confirmed or not by previous enhanced CT and/or MRI and before any biopsy for histology analysis. The standard of reference for diagnosis will be given by the site according to their standard of care and adapted from EASL/EORTC diagnostic criteria (considering previous contrast enhanced imaging (CT and/or MRI) and/or biopsy specimen analysis given on-site and/or the more recent AFP results available). 10 additional subjects will be included to explore the diagnostic efficacy for HCC at half dose of P03277.
Interventions
DRUGP03277 at 0.1 mmol/kg
P03277 will be administered at 0.1 mmol/kg BW (corresponding to 0.2 mL/kg BW) by intravenous (IV) bolus injection at 2 mL/s rate without dilution, followed by a 0.9% saline flush.
DRUGP03277 at 0.05 mmol/kg
P03277 will be administered at 0.05 mmol/kg BW (corresponding to 0.1 mL/kg BW) by intravenous (IV) bolus injection at 2 mL/s rate without dilution, followed by a 0.9% saline flush.
Sponsors
Guerbet
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
DIAGNOSTIC
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subject presenting with liver cirrhosis or chronic liver disease as shown by previous liver biopsy or by combination of clinical, endoscopic, biological, ultrasound parameters, and elastography * Subject presenting with 1 to a maximum of 3 untreated hepatic nodule(s) of less than or equal to 3 cm (long axis) previously identified and/or characterized through enhanced CT and/or MRI within a maximum of 21 days before P03277 imaging, confirmed for HCC or not
Exclusion criteria
* Subject presenting with hepatic nodule more than 3 cm in addition to nodule(s) less than or equal to 3 cm * Subject already treated for HCC by surgery or thermal ablation for which the resection or coagulation area is less than 2 cm from the new nodule(s) * Subject previously treated by transarterial chemoembolization * Subject with moderate or severe renal impairment (eGFR \< 60 mL/min/1.73 m2) based on measurement done or available within the 7 days before P03277 administration and assessed after any iodinated contrast agent administration, or with dialysis end stage renal disease (ESRD)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Diagnostic Value Evaluation for HCC | One day procedure | Sensitivity and specificity of diagnosis obtained by gadopiclenol imaging versus standard of reference |
Countries
France
Participant flow
Pre-assignment details
Among the 41 screened patients, one failed to meet randomization criteria and was screen failure. Therefore, 40 patients were included: 30 in Cohort 1 (0.1 mmol/kg) and 10 in Cohort 2 (0.05 mmol/kg),
Participants by arm
| Arm | Count |
|---|---|
| Cohort 1 Gadopiclenol administered at 0.1 mmol/kg BW (corresponding to 0.2 mL/kg BW) by intravenous (IV) bolus injection at 2 mL/s rate without dilution, followed by a 0.9% saline flush. | 29 |
| Cohort 2 Gadopiclenol administered at 0.05 mmol/kg BW (corresponding to 0.1 mL/kg BW) by intravenous (IV) bolus injection at 2 mL/s rate without dilution, followed by a 0.9% saline flush. | 10 |
| Total | 39 |
Baseline characteristics
| Characteristic | Cohort 1 | Cohort 2 | Total |
|---|---|---|---|
| Age, Continuous | 61.1 years STANDARD_DEVIATION 6.5 | 60.7 years STANDARD_DEVIATION 15.4 | 61 years STANDARD_DEVIATION 9.3 |
| Body Mass Index | 27.07 kg/m^2 STANDARD_DEVIATION 6.08 | 27.98 kg/m^2 STANDARD_DEVIATION 5.16 | 27.30 kg/m^2 STANDARD_DEVIATION 5.8 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants | 0 Participants | 2 Participants |
| Race (NIH/OMB) Black or African American | 3 Participants | 2 Participants | 5 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 24 Participants | 8 Participants | 32 Participants |
| Sex: Female, Male Female | 6 Participants | 2 Participants | 8 Participants |
| Sex: Female, Male Male | 23 Participants | 8 Participants | 31 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 30 | 0 / 10 |
| other Total, other adverse events | 8 / 30 | 0 / 10 |
| serious Total, serious adverse events | 0 / 30 | 1 / 10 |
Outcome results
Primary
Diagnostic Value Evaluation for HCC
Sensitivity and specificity of diagnosis obtained by gadopiclenol imaging versus standard of reference
Time frame: One day procedure
Population: Analysis performed on the Full Analysis Set Nodules (FASN). A total of 56 suspected nodules were characterized: 43 from cohort 1 and 13 from cohort 2.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Cohort 1 | Diagnostic Value Evaluation for HCC | Specificity | 86 percent |
| Cohort 1 | Diagnostic Value Evaluation for HCC | Positive predictive value | 81 percent |
| Cohort 1 | Diagnostic Value Evaluation for HCC | Accuracy | 74 percent |
| Cohort 1 | Diagnostic Value Evaluation for HCC | Negative predictive value | 70 percent |
| Cohort 1 | Diagnostic Value Evaluation for HCC | Sensitivity | 62 percent |
| Cohort 2 | Diagnostic Value Evaluation for HCC | Negative predictive value | 89 percent |
| Cohort 2 | Diagnostic Value Evaluation for HCC | Sensitivity | 80 percent |
| Cohort 2 | Diagnostic Value Evaluation for HCC | Specificity | 100 percent |
| Cohort 2 | Diagnostic Value Evaluation for HCC | Accuracy | 92 percent |
| Cohort 2 | Diagnostic Value Evaluation for HCC | Positive predictive value | 100 percent |