Trial Outcomes & Findings for Proof of Concept Study Concerning Efficacy of P03277 MR Imaging in HCC Diagnosis (NCT NCT02973516)

NCT ID: NCT02973516

Last Updated: 2021-10-26

Results Overview

Sensitivity and specificity of diagnosis obtained by gadopiclenol imaging versus standard of reference

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 41 participants
• Primary outcome timeframe: One day procedure
• Results posted on: 2021-10-26

Participant Flow

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),

Participant milestones

Measure	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.	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.
Overall Study STARTED	30	10
Overall Study COMPLETED	30	10
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Proof of Concept Study Concerning Efficacy of P03277 MR Imaging in HCC Diagnosis

Baseline characteristics by cohort

Measure	Cohort 1 n=29 Participants 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.	Cohort 2 n=10 Participants 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.	Total n=39 Participants Total of all reporting groups
Age, Continuous	61.1 years STANDARD_DEVIATION 6.5 • n=5 Participants	60.7 years STANDARD_DEVIATION 15.4 • n=7 Participants	61 years STANDARD_DEVIATION 9.3 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	2 Participants n=7 Participants	8 Participants n=5 Participants
Sex: Female, Male Male	23 Participants n=5 Participants	8 Participants n=7 Participants	31 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) White	24 Participants n=5 Participants	8 Participants n=7 Participants	32 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Body Mass Index	27.07 kg/m^2 STANDARD_DEVIATION 6.08 • n=5 Participants	27.98 kg/m^2 STANDARD_DEVIATION 5.16 • n=7 Participants	27.30 kg/m^2 STANDARD_DEVIATION 5.80 • n=5 Participants

PRIMARY outcome

Timeframe: 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.

Sensitivity and specificity of diagnosis obtained by gadopiclenol imaging versus standard of reference

Outcome measures

Measure	Cohort 1 n=29 Participants 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.	Cohort 2 n=10 Participants 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.
Diagnostic Value Evaluation for HCC Sensitivity	62 percent	80 percent
Diagnostic Value Evaluation for HCC Specificity	86 percent	100 percent
Diagnostic Value Evaluation for HCC Accuracy	74 percent	92 percent
Diagnostic Value Evaluation for HCC Positive predictive value	81 percent	100 percent
Diagnostic Value Evaluation for HCC Negative predictive value	70 percent	89 percent

Adverse Events

Cohort 1

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Cohort 2

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Cohort 1 n=30 participants at risk 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.	Cohort 2 n=10 participants at risk 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.
Gastrointestinal disorders gastric perforation	0.00% 0/30 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.	10.0% 1/10 • Number of events 1 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.
Injury, poisoning and procedural complications procedural complication	0.00% 0/30 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.	10.0% 1/10 • Number of events 1 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.

Other adverse events

Measure	Cohort 1 n=30 participants at risk 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.	Cohort 2 n=10 participants at risk 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.
General disorders Feeling hot	6.7% 2/30 • Number of events 2 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.	0.00% 0/10 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.
General disorders Injection site coldness	3.3% 1/30 • Number of events 1 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.	0.00% 0/10 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.
General disorders Injection site haematoma	3.3% 1/30 • Number of events 1 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.	0.00% 0/10 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.
General disorders Injection site pain	3.3% 1/30 • Number of events 1 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.	0.00% 0/10 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.
Investigations Blood pressure increased	3.3% 1/30 • Number of events 1 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.	0.00% 0/10 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.
Musculoskeletal and connective tissue disorders Muscle tightness	3.3% 1/30 • Number of events 1 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.	0.00% 0/10 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.
Nervous system disorders Headache	3.3% 1/30 • Number of events 1 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.	0.00% 0/10 • Adverse events were recorded from informed consent signature up to 13 weeks after gadopiclenol administration Post-injection adverse events are reported below.

Additional Information

Jing Hao, MD, Head of Global Medical Affairs and Clinical Development

Guerbet

Phone: +33 (0) 1 45 91 50 00

Email: jing.hao@guerbet.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place