Trial Outcomes & Findings for Hyperpolarized C-13 Pyruvate as a Biomarker in Patients With Advanced Solid Tumor Malignancies (NCT NCT02913131)

NCT ID: NCT02913131

Last Updated: 2022-04-22

Results Overview

The Signal-to-noise ratio with respect to intra-tumoral hyperpolarized (HP) C-13 Lactate/Pyruvate (lac/pyr) ratio detected within target liver or other intra-abdominal metastasis in patients with advanced solid tumor malignancies enrolled in the feasibility cohort will be determined

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

23 participants

Primary outcome timeframe

1 day

Results posted on

2022-04-22

Participant Flow

Participants in the feasibility cohort (Part A) who completed the scan were permitted the option to enroll in the Biomarker cohort (Part B).

Participant milestones

Participant milestones
Measure
Part A: Feasibility Run-In
Patients with advanced solid tumor malignancies with at least one liver metastasis will be enrolled with iterative adjustment of coil design to optimize imaging parameters including spatial resolution and signal-to-noise ratio (SNR) of hyperpolarized pyruvate / lactate within the target metastatic lesion(s).
Part B: Biomarker Cohort
Patients with advanced solid tumor malignancies and the presence of at least one liver metastasis amenable to hyperpolarized C-13 pyruvate metabolic magnetic resonance (MR) imaging who are planning on being treated with agent targeting phosphatidylinositol 3-kinase (PI3K)/Mechanistic target of rapamycin (mTOR) pathway will be enrolled.
Overall Study
STARTED
18
5
Overall Study
COMPLETED
18
5
Overall Study
NOT COMPLETED
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Hyperpolarized C-13 Pyruvate as a Biomarker in Patients With Advanced Solid Tumor Malignancies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Part A: Feasibility Run-In
n=18 Participants
Patients with advanced solid tumor malignancies with at least one liver metastasis will be enrolled with iterative adjustment of coil design to optimize imaging parameters including spatial resolution and signal-to-noise ratio (SNR) of hyperpolarized pyruvate / lactate within the target metastatic lesion(s).
Part B: Biomarker Cohort
n=5 Participants
Patients with advanced solid tumor malignancies and the presence of at least one liver metastasis amenable to hyperpolarized C-13 pyruvate metabolic MR imaging who are planning on being treated with agent targeting PI3K/mTOR pathway will be enrolled.
Total
n=23 Participants
Total of all reporting groups
Age, Customized
30-39 years old
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Age, Customized
40-49 years old
2 Participants
n=5 Participants
0 Participants
n=7 Participants
2 Participants
n=5 Participants
Age, Customized
50-59 years old
5 Participants
n=5 Participants
1 Participants
n=7 Participants
6 Participants
n=5 Participants
Age, Customized
60-69 years old
4 Participants
n=5 Participants
1 Participants
n=7 Participants
5 Participants
n=5 Participants
Age, Customized
70-79 years old
6 Participants
n=5 Participants
2 Participants
n=7 Participants
8 Participants
n=5 Participants
Sex: Female, Male
Female
13 Participants
n=5 Participants
2 Participants
n=7 Participants
15 Participants
n=5 Participants
Sex: Female, Male
Male
5 Participants
n=5 Participants
3 Participants
n=7 Participants
8 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
17 Participants
n=5 Participants
5 Participants
n=7 Participants
22 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 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
4 Participants
n=5 Participants
3 Participants
n=7 Participants
7 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
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Race (NIH/OMB)
White
12 Participants
n=5 Participants
2 Participants
n=7 Participants
14 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
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Region of Enrollment
United States
18 participants
n=5 Participants
5 participants
n=7 Participants
23 participants
n=5 Participants

PRIMARY outcome

Timeframe: 1 day

Population: Data for this endpoint was not collected

The Signal-to-noise ratio with respect to intra-tumoral hyperpolarized (HP) C-13 Lactate/Pyruvate (lac/pyr) ratio detected within target liver or other intra-abdominal metastasis in patients with advanced solid tumor malignancies enrolled in the feasibility cohort will be determined

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Up to 24 months

Population: Data was not collected for this endpoint for participants in Cohort B

Descriptive statistics will be used to characterize the mean change from baseline in intra-tumoral HP pyruvate/lactate ratio after initiation of treatment with PI3K/mTOR pathway inhibitor for participants enrolled in the biomarker cohort, along with 95% confidence interval.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 24 months

Number of participants with documented treatment-related adverse events will be reported for all patients having received a single dose of HP C-13 pyruvate. The study will use the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4 for reporting of adverse events that occur within 3 days of each imaging procedure over the course of the study and any biopsy-related adverse events.

Outcome measures

Outcome measures
Measure
Part A: Feasibility Run-In
n=18 Participants
Patients with advanced solid tumor malignancies with at least one liver metastasis will be enrolled with iterative adjustment of coil design to optimize imaging parameters including spatial resolution and signal-to-noise ratio (SNR) of hyperpolarized pyruvate / lactate within the target metastatic lesion(s).
Part B: Biomarker Cohort
n=5 Participants
Patients with advanced solid tumor malignancies and the presence of at least one liver metastasis amenable to hyperpolarized C-13 pyruvate metabolic MR imaging who are planning on being treated with agent targeting PI3K/mTOR pathway will be enrolled.
Number of Participants With Reported Treatment-related Adverse Events
0 Participants
0 Participants

SECONDARY outcome

Timeframe: Up to 24 months

Population: Data was not collected for this endpoint

For the purposes of analyzing the association between percent change from baseline in intra-tumoral peak lactate/pyruvate ratio on HP MRI with subsequent clinical outcomes, the cohort will be dichotomized by the median percent change. The objective response rate by RECIST 1.1 criteria and clinical benefit rate (response or stable disease for \> 24 weeks) will be compared between dichotomized groups using the chi-squared test.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 24 months

Population: Data was not collected for this endpoint

The cohort will be dichotomized by the median percent change. The log-rank test statistic will then be used to test the association of estimates of the hazard functions of the two groups at each observed event time by computing the observed and expected number of events in one of the groups at each observed event time and then adding these to obtain an overall summary across all-time points where there is an event.

Outcome measures

Outcome data not reported

Adverse Events

Part A: Feasibility Run-In

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Part B: Biomarker Cohort

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Rahul Aggarwal, MD

University of California, San Francisco

Phone: (415) 353-9278

Results disclosure agreements

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