Real World Evidence of PD-L1, TMB Prevalence and Efficacy of 1st Line Chemotherapy in These High or Low Population for Stage IV Urothelial Cancer

Completed

• Conditions: Urologic Neoplasms

• Registration Number: NCT04052113
• Lead Sponsor: AstraZeneca

Brief Summary

This study is a multi-center, non-interventional study. Patients' background, treatment pattern, treatment outcome, efficacy will be collected from medical records in Stage IV urotherial (UC) patients. Archived patient's formalin-fixed paraffin-embedded (FFPE) primary tumor samples will be collected to assay PD-L1 expression and next generation sequencer (NGS) assay for tumor mutation burden (TMB).

Detailed Description

This study is a multi-center, non-interventional study. Patients' background, treatment pattern, treatment outcome, efficacy will be collected from medical records in Stage IV urotherial (UC) patients. Archived patient's formalin-fixed paraffin-embedded (FFPE) primary tumor samples will be collected from each site and conduct programmed cell death 1 ligand 1 (PD-L1) assay and next generation sequencer (NGS) assay for tumor mutation burden (TMB). Based on these data, prevalence of PD-L1, TMB and overall survival (OS), progression free survival (PFS) from start of 1st line treatment in stage IV will be assessed.

In this study, 150 patients will be enrolled from approximately 30 sites in Japan. The patients should have received at least 1 cycle of chemotherapy and never receive immune oncology drug as 1st line treatment in stage IV. The patients will be enrolled continuously from the 1st patient who is enrolled in this study until target number of patients in each site.

Study Timeline

• Study First Submitted: 2019-07-22
• Study First Submitted QC: 2019-08-08
• Study First Posted: 2019-08-09
• Study Start: 2019-10-25
• Primary Completion: 2020-03-23
• Study Completion: 2020-03-23
• Status Verified: 2021-02
• Last Update Submitted: 2021-02-25
• Last Update Posted: 2021-03-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 152

Inclusion Criteria

• Age > 20, Japanese men and women.
• Patients who have started at least 1 cycle of chemotherapy.
• Patients who provided informed consent by appropriate methods. In dead case, optout will be applicable.
• Patients who are diagnosed as stage IV (T4b, any N or any T, N2-3 or M1) UC between January 1st in 2017 and December 31st in 2018.
• Patients who have FFPE primary tumor sample collected after January 1st in 2017. The sample should be collected before any therapies including 1st line treatment in stage IV and therapies in stage III and other stages. It is possible to send sliced undyed section of primary tumor including 1 HE-stained section of same sample in case that patients cannot send FFPE primary tumor block.

Exclusion Criteria

-Patients who are prior exposure to immune-mediated therapy as 1st line treatment in stage IV.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Prevalence of PD-L1 in stage IV UC patients in real world setting.	Baseline	Summarize number and calculate ratio of PD-L1 high or low/negative patients, respectively.

Secondary Outcome Measures

Name	Time	Method
Prevalence of TMB in stage IV UC patients in real world setting.	Baseline	Calculate median and average of TMB levels. Summarize number and calculate ratio of TMB high or low/negative patients, respectively.
OS from start of 1st line treatment in stage IV	2 years	Median OS and OS rate at 12 months, 18 months and 24 months will be analysed based on Kaplan-Meier method and presented along with its 95% confidence interval.
PFS from start of 1st line treatment in stage IV	18 months	Median PFS and PFS rate at 6months, 12months and 18 months will be analysed based on Kaplan-Meier method and presented along with its 95% confidence interval.

Trial Locations

Locations (1)

Research Site; 🇯🇵 Kyoto-shi, Japan