Trial Outcomes & Findings for Confocal Endoscopic Microscopy for Detection of Early Stage Gastric Cancer in Subjects With Hereditary Diffuse Gastric Cancer Syndrome (NCT NCT03648879)

Last Updated: 2021-07-12

Results Overview

Sensitivity for detection of SRC foci in CDH1 germline mutation carriers was assessed by confocal endoscopic microscopy (CEM) compared to the current method of and standard white light endoscopy. Sensitivity in CEM and WLE is defined as the percentage of participants with detectable cancer on endoscopic biopsy.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

37 participants

Primary outcome timeframe

14 days

Results posted on

2021-07-12

Participant Flow

Participant milestones

Participant milestones
Measure	1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones.
Overall Study STARTED	37
Overall Study COMPLETED	36
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones.
Overall Study Subject determined to have Cadherin-1 (CDH1) variant of uncertain significance.	1

Baseline Characteristics

Confocal Endoscopic Microscopy for Detection of Early Stage Gastric Cancer in Subjects With Hereditary Diffuse Gastric Cancer Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy n=37 Participants Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	30 Participants n=5 Participants
Age, Categorical >=65 years	7 Participants n=5 Participants
Age, Continuous	50.51 years STANDARD_DEVIATION 14.02 • n=5 Participants
Sex: Female, Male Female	28 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	4 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	31 Participants n=5 Participants
Race/Ethnicity, Customized Ethnicity Unknown or Not Reported	2 Participants n=5 Participants
Race/Ethnicity, Customized White	33 Participants n=5 Participants
Race/Ethnicity, Customized Race Other	2 Participants n=5 Participants
Race/Ethnicity, Customized Race Unknown	2 Participants n=5 Participants
Region of Enrollment United States	37 participants n=5 Participants

PRIMARY outcome

Timeframe: 14 days

Population: 36/37 were evaluable for this outcome measure. One participant was determined to have Cadherin-1 (CDH1) variant of uncertain significance.

Outcome measures

Outcome measures
Measure	1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy n=36 Participants Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones.
Percentage of Participants With Detectable Confocal Endoscopic Microscopy (CEM) w/Greater Sensitivity for Detection of Signet Ring Cells (SRC)Foci in Cadherin-1 (CDH1) Germline Mutation Carriers Compared to Current Method of Standard White Light Endoscopy CEM	16.7 Percentage of participants
Percentage of Participants With Detectable Confocal Endoscopic Microscopy (CEM) w/Greater Sensitivity for Detection of Signet Ring Cells (SRC)Foci in Cadherin-1 (CDH1) Germline Mutation Carriers Compared to Current Method of Standard White Light Endoscopy White Light Endoscopy	11.1 Percentage of participants

SECONDARY outcome

Timeframe: Date of enrollment to date of prophylactic gastrectomy, approximately 6 months or an average of 1 month up to 12 months.

Population: 36/37 were evaluable for this outcome measure. One participant was determined to have Cadherin-1 (CDH1) variant of uncertain significance.

In participants who choose to undergo prophylactic total gastrectomy with permanent pathologic analysis, the false negative rate (the fraction of participants who have SRC foci not identified by CEM and White Light Endoscopy techniques) will be determined and reported. The fraction percentage of patients who underwent prophylactic total gastrectomy with findings of SRC foci in the gastrectomy specimen will represent the denominator (number) and the number of patients with negative findings by CEM and White Light Endoscopy (WLE) will represent the numerator (number) to generate the false negative detection rate (fraction) for CEM and WLE, respectively.

Outcome measures

Outcome measures
Measure	1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy n=36 Participants Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones.
Percentage of Participants Who Have Signet Ring Cells (SRC) Foci Not Identified by Confocal Endoscopic Microscopy (CEM) CEM	67 Percentage of participants
Percentage of Participants Who Have Signet Ring Cells (SRC) Foci Not Identified by Confocal Endoscopic Microscopy (CEM) White Light Endoscopy	87 Percentage of participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Date of enrollment to date off study, approximately 11 months and 19 days.

Population: 36/37 were evaluable for this outcome measure. One participant was determined to have Cadherin-1 (CDH1) variant of uncertain significance.

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Outcome measures
Measure	1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy n=36 Participants Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones.
Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)	1 Participants

Adverse Events

1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy n=36 participants at risk Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones.
General disorders Fever	2.8% 1/36 • Number of events 1 • Date of enrollment to date off study, approximately 11 months and 19 days. 36/37 were evaluable for adverse events. One participant was determined to have Cadherin-1 (CDH1) variant of uncertain significance.
Infections and infestations Urinary Tract Infection	2.8% 1/36 • Number of events 1 • Date of enrollment to date off study, approximately 11 months and 19 days. 36/37 were evaluable for adverse events. One participant was determined to have Cadherin-1 (CDH1) variant of uncertain significance.

Additional Information

Dr. Jeremy L. Davis

National Cancer Institute

Phone: 240-760-6229

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place