Trial Outcomes & Findings for Molecular Phenotyping in Predicting Response in Patients With Stage IB-III Esophageal Cancer Receiving Combination Chemotherapy (NCT NCT02392377)

NCT ID: NCT02392377

Last Updated: 2018-02-13

Results Overview

Baseline gene profiles will be compared for 10 (18F) FDG-PET responders vs. 15 (18F) FDG-PET non-responders per treatment regimen using a t-test and p-values will be corrected for multiple comparisons by calculating the false discovery rate (FDR) p-value (filtering on a FDR p-value \< 0.05). Top genes identified will be tested in combination for their sensitivity and specificity using logistic regression models.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

Baseline

Results posted on

2018-02-13

Participant Flow

Participant milestones

Participant milestones
Measure
Arm I (Paclitaxel, Carboplatin, Radiation Therapy, Surgery)
INDUCTION: Patients receive chemotherapy with carboplatin and paclitaxel. Patients receive carboplatin (AUC=2) and paclitaxel (90 mg/m2) intravenously on days 1 and 8 of a 21 day treatment cycle for two cycles (total of 6 weeks). CHEMORADIATION THERAPY: Patients receive carboplatin (AUC=2) and paclitaxel (50 mg/m2) intravenously once weekly for five weeks throughout the duration of their radiation which is daily (Monday through Friday). SURGERY: Approximately 4-10 weeks after completion of chemoradiation therapy, patients undergo esophagectomy at the discretion of the treating team.
Arm II (Combination Chemotherapy, Radiation Therapy, Surgery)
INDUCTION: Patients receive mFOLFOX6 where they get oxaliplatin 85 mg/m2 intravenously on day 1, leucovorin 400 mg/m2 IV on day 1, 5-FU 400 mg/m2 IV on day 1 and then 5FU at 2400 mg/m2 IV to be administered over a 46 hour period. This is repeated every 2 weeks for 3 cycles (total of 6 weeks). CHEMORADIATION THERAPY: Patients receive oxaliplatin 85 mg/m2 IV on day 1 every 2 weeks for a total of 3 cycles (6 weeks) as well as 5FU 300 mg/m2/day over 96 hours via continuous infusion each week of radiation for a total of 6 weeks. SURGERY: Approximately 4-10 weeks after completion of chemoradiation therapy, patients undergo esophagectomy at the discretion of the treating team.
Overall Study
STARTED
0
1
Overall Study
COMPLETED
0
0
Overall Study
NOT COMPLETED
0
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Arm I (Paclitaxel, Carboplatin, Radiation Therapy, Surgery)
INDUCTION: Patients receive chemotherapy with carboplatin and paclitaxel. Patients receive carboplatin (AUC=2) and paclitaxel (90 mg/m2) intravenously on days 1 and 8 of a 21 day treatment cycle for two cycles (total of 6 weeks). CHEMORADIATION THERAPY: Patients receive carboplatin (AUC=2) and paclitaxel (50 mg/m2) intravenously once weekly for five weeks throughout the duration of their radiation which is daily (Monday through Friday). SURGERY: Approximately 4-10 weeks after completion of chemoradiation therapy, patients undergo esophagectomy at the discretion of the treating team.
Arm II (Combination Chemotherapy, Radiation Therapy, Surgery)
INDUCTION: Patients receive mFOLFOX6 where they get oxaliplatin 85 mg/m2 intravenously on day 1, leucovorin 400 mg/m2 IV on day 1, 5-FU 400 mg/m2 IV on day 1 and then 5FU at 2400 mg/m2 IV to be administered over a 46 hour period. This is repeated every 2 weeks for 3 cycles (total of 6 weeks). CHEMORADIATION THERAPY: Patients receive oxaliplatin 85 mg/m2 IV on day 1 every 2 weeks for a total of 3 cycles (6 weeks) as well as 5FU 300 mg/m2/day over 96 hours via continuous infusion each week of radiation for a total of 6 weeks. SURGERY: Approximately 4-10 weeks after completion of chemoradiation therapy, patients undergo esophagectomy at the discretion of the treating team.
Overall Study
Withdrawal by Subject
0
1

Baseline Characteristics

Molecular Phenotyping in Predicting Response in Patients With Stage IB-III Esophageal Cancer Receiving Combination Chemotherapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Arm I (Paclitaxel, Carboplatin, Radiation Therapy, Surgery)
INDUCTION: Patients receive chemotherapy with carboplatin and paclitaxel. Patients receive carboplatin (AUC=2) and paclitaxel (90 mg/m2) intravenously on days 1 and 8 of a 21 day treatment cycle for two cycles (total of 6 weeks). CHEMORADIATION THERAPY: Patients receive carboplatin (AUC=2) and paclitaxel (50 mg/m2) intravenously once weekly for five weeks throughout the duration of their radiation which is daily (Monday through Friday). SURGERY: Approximately 4-10 weeks after completion of chemoradiation therapy, patients undergo esophagectomy at the discretion of the treating team.
Arm II (Combination Chemotherapy, Radiation Therapy, Surgery)
n=1 Participants
INDUCTION: Patients receive mFOLFOX6 where they get oxaliplatin 85 mg/m2 intravenously on day 1, leucovorin 400 mg/m2 IV on day 1, 5-FU 400 mg/m2 IV on day 1 and then 5FU at 2400 mg/m2 IV to be administered over a 46 hour period. This is repeated every 2 weeks for 3 cycles (total of 6 weeks). CHEMORADIATION THERAPY: Patients receive oxaliplatin 85 mg/m2 IV on day 1 every 2 weeks for a total of 3 cycles (6 weeks) as well as 5FU 300 mg/m2/day over 96 hours via continuous infusion each week of radiation for a total of 6 weeks. SURGERY: Approximately 4-10 weeks after completion of chemoradiation therapy, patients undergo esophagectomy at the discretion of the treating team.
Total
n=1 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Age, Categorical
Between 18 and 65 years
0 Participants
n=39 Participants
1 Participants
n=41 Participants
1 Participants
n=35 Participants
Age, Categorical
>=65 years
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Sex: Female, Male
Female
0 Participants
n=39 Participants
1 Participants
n=41 Participants
1 Participants
n=35 Participants
Sex: Female, Male
Male
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
0 Participants
n=39 Participants
1 Participants
n=41 Participants
1 Participants
n=35 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Race (NIH/OMB)
Asian
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Race (NIH/OMB)
White
0 Participants
n=39 Participants
1 Participants
n=41 Participants
1 Participants
n=35 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=39 Participants
0 Participants
n=41 Participants
0 Participants
n=35 Participants
Region of Enrollment
United States
1 Participants
n=41 Participants
1 Participants
n=35 Participants

PRIMARY outcome

Timeframe: Baseline

Population: No patients completed study and no patients were enrolled on the comparison group arm. No analysis could be completed.

Baseline gene profiles will be compared for 10 (18F) FDG-PET responders vs. 15 (18F) FDG-PET non-responders per treatment regimen using a t-test and p-values will be corrected for multiple comparisons by calculating the false discovery rate (FDR) p-value (filtering on a FDR p-value \< 0.05). Top genes identified will be tested in combination for their sensitivity and specificity using logistic regression models.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Baseline

Population: No patients completed study and no patients were enrolled on the comparison group arm. No analysis could be completed.

Baseline microRNA profiles will be compared for 10 (18F) FDG-PET responders vs. 15 (18F) FDG-PET non-responders per treatment regimen using a t-test and p-values will be corrected for multiple comparisons by calculating the FDR p-value (filtering on a FDR p-value \< 0.05). Top microRNAs identified will be tested in combination for their sensitivity and specificity using logistic regression models.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Up to 6 weeks

Population: No patients completed study and no patients were enrolled on the comparison group arm. No analysis could be completed.

Predefined signatures of biological pathway activities will be evaluated in order to identify pathway perturbation upon exposure to chemotherapy. Significant pathway modulations upon brief exposure will then be evaluated for association with clinical response using non-parametric tests such as the Wilcoxon signed-rank test. In all cases, statistical correction to account for multiple hypothesis testing will be employed and pathways with a false discovery rate of 10% or lower will be considered as significant.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to post-induction (36-43 days)

Population: No patients completed study and no patients were enrolled on the comparison group arm. No analysis could be completed.

Changes in baseline and post-treatment gene expression levels will be calculated between (18F) FDG-PET responders and non-responders, filtering for expression level changes of \>= 0.20. Differences in expression level change per treatment regimen will be calculated using a t-test and p-values will be corrected for multiple comparisons by calculating FDR p-value (filtering on a FDR p-value \< 0.05). Top genes identified will be tested in combination for their sensitivity and specificity using logistic regression models to predict responsiveness or resistance to each individual treatment regimen.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to post-induction (36-43 days)

Population: No patients completed study and no patients were enrolled on the comparison group arm. No analysis could be completed.

Changes in baseline and post-treatment microRNA levels will be calculated between (18F) FDG-PET responders and non-responders, filtering for expression level changes of \>= 0.20. Differences in expression level change per treatment regimen will be calculated using a t-test and p-values will be corrected for multiple comparisons by calculating FDR p-value (filtering on a FDR p-value \< 0.05). Top microRNAs identified will be tested in combination for their sensitivity and specificity using logistic regression models to predict responsiveness or resistance to each individual treatment regimen.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline

Population: No patients completed study and no patients were enrolled on the comparison group arm. No analysis could be completed.

Baseline gene expression profiles for 8 pathologic complete responders vs. 17 patients not achieving a pathologic complete response per treatment regimen using a t-test will be compared. Top genes will be tested in combination for their sensitivity and specificity using logistic regression models to predict achievement of a pathologic complete response vs. patients not achieving a pathologic complete response.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline

Population: No patients completed study and no patients were enrolled on the comparison group arm. No analysis could be completed.

Baseline microRNA profiles for 8 pathologic complete responders vs. 17 patients not achieving a pathologic complete response per treatment regimen using a t-test will be compared. Top microRNAs will be tested in combination for their sensitivity and specificity using logistic regression models to predict achievement of a pathologic complete response vs. patients not achieving a pathologic complete response.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to post-induction (36-43 days)

Population: No patients completed study and no patients were enrolled on the comparison group arm. No analysis could be completed.

Changes in baseline and post-treatment gene expression level/profiles will be compared between 8 pathologic responders vs. 17 patients not achieving a pathologic complete response per treatment regimen, filtering for expression levels changes of 0.20 and above. Differences in expression level changes per treatment regimen will be calculated using a t-test. Top genes will be tested in combination for their sensitivity and specificity using logistic regression models to predict achievement of a pathologic complete response vs. patients not achieving a pathologic complete response.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to post-induction (36-43 days)

Population: No patients completed study and no patients were enrolled on the comparison group arm. No analysis could be completed.

Changes in baseline and post-treatment microRNA level/profiles will be compared between 8 pathologic responders vs. 17 patients not achieving a pathologic complete response per treatment regimen, filtering for expression levels changes of 0.20 and above. Differences in expression level changes per treatment regimen will be calculated using a t-test. Top microRNAs will be tested in combination for their sensitivity and specificity using logistic regression models to predict achievement of a pathologic complete response vs. patients not achieving a pathologic complete response.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 30 days after the end of treatment

Population: No patients completed study and no patients were enrolled on the comparison group arm. No analysis could be completed.

Toxicities will be summarized as the percentage of patients experiencing each type and grade of event according to treatment group. Patients who receive at least one dose of treatment will be included in the analysis.

Outcome measures

Outcome data not reported

Adverse Events

Arm I (Paclitaxel, Carboplatin, Radiation Therapy, Surgery)

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

Arm II (Combination Chemotherapy, Radiation Therapy, Surgery)

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 Jennifer Eads

University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center

Phone: (216) 844-6031

Results disclosure agreements

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