Clinical trial NCT04052113: 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
Official title: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Urologic Neoplasms
Keywords: Real world, PD-L1, TMB, Stage IV, urothelial cancer, Japanese
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
First posted: 2019-08-09
Enrollment: 152
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: PROBABILITY_SAMPLE
Study population: This study is a multi-center, non-interventional study, and Stage IV UC patients who have received at least 1 cycle of chemotherapy and have archived primary tumor sample collected after January 1st in 2017 will be of the interest. However, the patients who received immune oncology drug as 1st line treatment in stage IV will not be in this study.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Prevalence of PD-L1 in stage IV UC patients in real world setting.
timeFrame: Baseline
Secondary outcomes:
measure: Prevalence of TMB in stage IV UC patients in real world setting.
timeFrame: Baseline
measure: OS from start of 1st line treatment in stage IV
timeFrame: 2 years
measure: PFS from start of 1st line treatment in stage IV
timeFrame: 18 months
Other outcomes:
Locations:
city: Kyoto-shi
country: Japan
facility: Research Site
Study officials:
References: