Trial Outcomes & Findings for F-18 16 Alpha-Fluoroestradiol-Labeled Positron Emission Tomography in Predicting Response to First-Line Hormone Therapy in Patients With Stage IV Breast Cancer (NCT NCT00602043)

Results Overview

Patients were expected to start endocrine therapy within 2 weeks of the FES PET scan. Response assessment was evaluated at 3 and 6 months. For patients with at least one site of measurable disease \[per response evaluation criteria in solid tumors (RECIST, version 1.1)\], size-based response criteria were used to assess response. For patients without disease evaluable by RECIST 1.1, largely patients with bone-dominant metastatic breast cancer, serial FDG PET scanning was used to determine response. A decline in the FDG PET SUV (standard uptake value) of 30% or more was considered as response and an increase of 20% or more was considered to be progressive disease (PD). The initial (baseline) FES uptake was compared to clinical benefit (PD versus other outcome at 6 months).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

20 participants

Primary outcome timeframe

Up to 6 months

Results posted on

2020-02-19

Participant Flow

Participant milestones

Participant milestones
Measure	Diagnostic (FES) Patients undergo \[\^18F\] FES PET scan. Patients also undergo standard clinical fludeoxyglucose F 18 (FDG)-PET or FDG-PET/CT scan up to 14 days prior to \[\^18F\] FES PET scan. Patients begin clinically indicated endocrine therapy. Patients are followed-up to determine response on the therapy for 6 months using clinical exams, tumor marker assays, conventional imaging and standard clinical FDG PET/CT. laboratory biomarker analysis: Correlative studies
Overall Study STARTED	20
Overall Study COMPLETED	15
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

F-18 16 Alpha-Fluoroestradiol-Labeled Positron Emission Tomography in Predicting Response to First-Line Hormone Therapy in Patients With Stage IV Breast Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	First Line Endocrine Therapy for a Stage IV Disease n=20 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	13 Participants n=5 Participants
Age, Categorical >=65 years	7 Participants n=5 Participants
Sex: Female, Male Female	20 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Region of Enrollment United States	20 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 6 months

Patients were expected to start endocrine therapy within 2 weeks of the FES PET scan. Response assessment was evaluated at 3 and 6 months. For patients with at least one site of measurable disease \[per response evaluation criteria in solid tumors (RECIST, version 1.1)\], size-based response criteria were used to assess response. For patients without disease evaluable by RECIST 1.1, largely patients with bone-dominant metastatic breast cancer, serial FDG PET scanning was used to determine response. A decline in the FDG PET SUV (standard uptake value) of 30% or more was considered as response and an increase of 20% or more was considered to be progressive disease (PD). The initial (baseline) FES uptake was compared to clinical benefit (PD versus other outcome at 6 months).

Outcome measures

Outcome measures
Measure	Diagnostic FES: Average FES SUVmean >1.5, no Negative Sites n=13 Participants Patients undergo \[\^18F\] FES PET scan. Patients also undergo standard clinical fludeoxyglucose F 18 (FDG)-PET or FDG-PET/CT scan up to 14 days prior to \[\^18F\] FES PET scan. Patients begin clinically indicated endocrine therapy. Patients are followed-up to determine response on the therapy for 6 months using clinical exams, tumor marker assays, conventional imaging and standard clinical FDG PET/CT. This group represents patients who had positive FES uptake at all disease sites on the baseline diagnostic FES PET scan. laboratory biomarker analysis: Correlative studies	Diagnostic FES: Patients With FES Negative Sites of Disease n=3 Participants Patients undergo \[\^18F\] FES PET scan. Patients also undergo standard clinical fludeoxyglucose F 18 (FDG)-PET or FDG-PET/CT scan up to 14 days prior to \[\^18F\] FES PET scan. Patients begin clinically indicated endocrine therapy. Patients are followed-up to determine response on the therapy for 6 months using clinical exams, tumor marker assays, conventional imaging and standard clinical FDG PET/CT. This group represents patients who had some or all disease sites negative for FES uptake on the baseline diagnostic FES PET scan.
Best Overall Response	5 patients with progressive disease	2 patients with progressive disease

SECONDARY outcome

Timeframe: Up to 6 months

FES SUV prior to endocrine treatment (dichotomized and as a continuous predictor) will also be tested as predictor of clinical benefit. Patients were expected to start endocrine therapy within 2 weeks of the FES PET scan. Response assessment was evaluated at 3 and 6 months. The initial (baseline) FES uptake was compared to clinical benefit (PD versus other outcome at 6 months).

Outcome measures

Outcome measures
Measure	Diagnostic FES: Average FES SUVmean >1.5, no Negative Sites n=13 Participants Patients undergo \[\^18F\] FES PET scan. Patients also undergo standard clinical fludeoxyglucose F 18 (FDG)-PET or FDG-PET/CT scan up to 14 days prior to \[\^18F\] FES PET scan. Patients begin clinically indicated endocrine therapy. Patients are followed-up to determine response on the therapy for 6 months using clinical exams, tumor marker assays, conventional imaging and standard clinical FDG PET/CT. This group represents patients who had positive FES uptake at all disease sites on the baseline diagnostic FES PET scan. laboratory biomarker analysis: Correlative studies	Diagnostic FES: Patients With FES Negative Sites of Disease n=3 Participants Patients undergo \[\^18F\] FES PET scan. Patients also undergo standard clinical fludeoxyglucose F 18 (FDG)-PET or FDG-PET/CT scan up to 14 days prior to \[\^18F\] FES PET scan. Patients begin clinically indicated endocrine therapy. Patients are followed-up to determine response on the therapy for 6 months using clinical exams, tumor marker assays, conventional imaging and standard clinical FDG PET/CT. This group represents patients who had some or all disease sites negative for FES uptake on the baseline diagnostic FES PET scan.
Number of Participants With Clinical Benefit	8 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 6 months

FES SUV prior to endocrine treatment (dichotomized and as a continuous predictor) will also be tested as predictor of time to progression. Analysis will be conducted using (respectively) logistic regression and Cox proportional hazards regression. This will include univariate analysis of FES and other predictive measures (ER/PgR expression, serum sex steroid levels), followed by an exploratory multivariate analysis combining FES SUV with other measures showing predictive capability univariate analysis.

Outcome measures

Outcome measures
Measure	Diagnostic FES: Average FES SUVmean >1.5, no Negative Sites n=5 Participants Patients undergo \[\^18F\] FES PET scan. Patients also undergo standard clinical fludeoxyglucose F 18 (FDG)-PET or FDG-PET/CT scan up to 14 days prior to \[\^18F\] FES PET scan. Patients begin clinically indicated endocrine therapy. Patients are followed-up to determine response on the therapy for 6 months using clinical exams, tumor marker assays, conventional imaging and standard clinical FDG PET/CT. This group represents patients who had positive FES uptake at all disease sites on the baseline diagnostic FES PET scan. laboratory biomarker analysis: Correlative studies	Diagnostic FES: Patients With FES Negative Sites of Disease n=2 Participants Patients undergo \[\^18F\] FES PET scan. Patients also undergo standard clinical fludeoxyglucose F 18 (FDG)-PET or FDG-PET/CT scan up to 14 days prior to \[\^18F\] FES PET scan. Patients begin clinically indicated endocrine therapy. Patients are followed-up to determine response on the therapy for 6 months using clinical exams, tumor marker assays, conventional imaging and standard clinical FDG PET/CT. This group represents patients who had some or all disease sites negative for FES uptake on the baseline diagnostic FES PET scan.
Time to Progression	4.9 months Interval 2.1 to 6.0	2.2 months Interval 1.2 to 3.3

SECONDARY outcome

Timeframe: Up to 6 months

Population: Data were not collected due to variations in reporting methods for ER assays.

Graphical and numerical studies of bivariate relationships will be examined, as well as factors (i.e., tumor size, tumor location, patient age) to explain concurrence, lack of concurrence, and sources of measurement error for measurements of ER function.

Outcome measures

Outcome data not reported

Adverse Events

Diagnostic (FES)

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. David Mankoff

University of Pennsylvania

Phone: (2015) 615-3687

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60