Trial Outcomes & Findings for Metabolomic and BH3 Profiling of Esophageal Cancers: Identification of Novel Assessment Methods of Treatment Response for Precision Therapy (NCT NCT03223662)
NCT ID: NCT03223662
Last Updated: 2018-10-30
Results Overview
Specimens of normal esophagus, esophageal tumors, blood, or urine will be analyzed to determine specific signatures and outcome of pathological complete response after neoadjuvant chemoradiotherapy in patients with esophageal adenocarcinoma or squamous cell carcinoma. Response will be defined by the Mandard Score. Major response with no viable tumor (Grade 1) and \<10% viable tumor (Grade 2) versus non major response of \>10% viable tumor (Grade 3-5) as assessed by final pathology. Grade 1-2 is better survival than Grade 3-5.
TERMINATED
PHASE2
2 participants
After neoadjuvant therapy >4 weeks but prior to surgery
2018-10-30
Participant Flow
Participant milestones
| Measure |
Neoadjuvant Chemoradiotherapy and Esophagectomy
Standard of care neoadjuvant chemoradiotherapy (nCRT) and esophagectomy
Chemoradiotherapy: Chemotherapy: Carboplatin (AUC=2)x5 and Paclitaxel (50 mg/m(2))x5 for two cycles. Radiation: a total dose of 40.4 Gray (Gy) will be given in 23 fractions of 1.8 Gy, 5 fractions per week, starting the first day of the first cycle of chemotherapy.
Esophagectomy: Minimally-invasive esophagectomy (RAMIE) or traditional open approach if necessary.
|
|---|---|
|
Overall Study
STARTED
|
2
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Neoadjuvant Chemoradiotherapy and Esophagectomy
Standard of care neoadjuvant chemoradiotherapy (nCRT) and esophagectomy
Chemoradiotherapy: Chemotherapy: Carboplatin (AUC=2)x5 and Paclitaxel (50 mg/m(2))x5 for two cycles. Radiation: a total dose of 40.4 Gray (Gy) will be given in 23 fractions of 1.8 Gy, 5 fractions per week, starting the first day of the first cycle of chemotherapy.
Esophagectomy: Minimally-invasive esophagectomy (RAMIE) or traditional open approach if necessary.
|
|---|---|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Study closure
|
1
|
Baseline Characteristics
Metabolomic and BH3 Profiling of Esophageal Cancers: Identification of Novel Assessment Methods of Treatment Response for Precision Therapy
Baseline characteristics by cohort
| Measure |
Neoadjuvant Chemoradiotherapy and Esophagectomy
n=2 Participants
Standard of care neoadjuvant chemoradiotherapy (nCRT) and esophagectomy
Chemoradiotherapy: Chemotherapy: Carboplatin (AUC=2)x5 and Paclitaxel (50 mg/m(2))x5 for two cycles. Radiation: a total dose of 40.4 Gray (Gy) will be given in 23 fractions of 1.8 Gy, 5 fractions per week, starting the first day of the first cycle of chemotherapy.
Esophagectomy: Minimally-invasive esophagectomy (RAMIE) or traditional open approach if necessary.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Age, Continuous
|
58.55 years
STANDARD_DEVIATION 5.3 • n=99 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
2 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: After neoadjuvant therapy >4 weeks but prior to surgeryPopulation: Neither patient enrolled in the trial underwent resection based on post neoadjuvant therapy assessment of resectability. These patients were initially deemed potentially resectable prior to neoadjuvant therapy, but their course deviated from the initial plans. Therefore, the primary endpoint cannot be assessed.
Specimens of normal esophagus, esophageal tumors, blood, or urine will be analyzed to determine specific signatures and outcome of pathological complete response after neoadjuvant chemoradiotherapy in patients with esophageal adenocarcinoma or squamous cell carcinoma. Response will be defined by the Mandard Score. Major response with no viable tumor (Grade 1) and \<10% viable tumor (Grade 2) versus non major response of \>10% viable tumor (Grade 3-5) as assessed by final pathology. Grade 1-2 is better survival than Grade 3-5.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Pre-neoadjuvant therapy within 4 weeks, and after neoadjuvant therapy >4 weeks but prior to surgeryPopulation: Neither patient enrolled in the trial underwent resection based on post neoadjuvant therapy assessment of resectability. These patients were initially deemed potentially resectable prior to neoadjuvant therapy, but their course deviated from the initial plans. Therefore, the primary endpoint cannot be assessed.
Two tumor samples and two normal esophagus samples will be obtained for BH3 profiling of pre-neoadjuvant tumor biopsy and outcome of pathological complete response after neoadjuvant chemoradiotherapy in patients with esophageal adenocarcinoma or squamous cell carcinoma. Response will be defined by the Mandard Score. Major response with no viable tumor (Grade 1) and \<10% viable tumor (Grade 2) versus non major response of \>10% viable tumor (Grade 3-5) as assessed by final pathology. Grade 1-2 is better survival than Grade 3-5.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Pre-neoadjuvant therapy within 4 weeks, and after neoadjuvant therapy >4 weeks but prior to surgeryPopulation: This outcome measure was not done. Neither patient enrolled in the trial underwent resection based on post neoadjuvant therapy assessment of resectability. These patients were initially deemed potentially resectable prior to neoadjuvant therapy, but their course deviated from the initial plans.
Evaluation of metabolomic profiles and Bcl-2 homology domain-3 (BH-3) profiling in resectable esophageal adenocarcinomas (EAC) and esophageal squamous cell carcinoma (ESCC) treated with neoadjuvant chemoradiotherapy (nCRT)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Pre-neoadjuvant therapy within 4 weeks, and after neoadjuvant therapy >4 weeks but prior to surgeryPopulation: This outcome measure was not done. Neither patient enrolled in the trial underwent resection based on post neoadjuvant therapy assessment of resectability. These patients were initially deemed potentially resectable prior to neoadjuvant therapy, but their course deviated from the initial plans.
Specimens of tumor, blood, or urine will be obtained to determine metabolomic signatures or BH3 profiling in tumor, blood, or urine of EAC and ESCC with major responses (Mandard score of 1 and 2) versus minimal response (Mandard score 3-5). Grade 1-2 is better survival than Grade 3-5.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Pre-neoadjuvant therapy within 4 weeks, and after neoadjuvant therapy >4 weeks but prior to surgeryPopulation: This outcome measure was not done. Neither patient enrolled in the trial underwent resection based on post neoadjuvant therapy assessment of resectability. These patients were initially deemed potentially resectable prior to neoadjuvant therapy, but their course deviated from the initial plans.
One tumor sample and one normal esophagus sample will be obtained. p53 mutational analysis will be performed to determine mutational status and the metabolomic profiles .
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From the time of esophagectomy until development of metastatic disease or death, whichever comes firstPopulation: This outcome measure was not done because one patient was deemed to be unsafe/unfit for surgery by the doctor. And the second patient was taken off-study prior to surgery because the principal investigator left the National Institutes of Health and closed the trial.
Disease-free survival is defined as the appearance of any new lesion that is likely metastatic or locally-recurrent esophageal cancer and will be assessed from the time of esophagectomy until development of metastatic disease or death, whichever comes first
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From treatment start date until date of death or date last known alive.Population: This outcome measure was not done because one patient was deemed to be unsafe/unfit for surgery by the doctor. And the second patient was taken off-study prior to surgery because the principal investigator left the National Institutes of Health and closed the trial.
Overall survival is defined as the time from treatment start date until date of death or date last known alive.
Outcome measures
Outcome data not reported
Adverse Events
Neoadjuvant Chemoradiotherapy and Esophagectomy
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place