Real-world clinical outcomes associated with gene mutation patterns in patients with biliary tract cancer (BTC) in Japan

UMIN ID: UMIN000055174

Registered date:01/10/2024

Basic Information

• Recruitment status: Complete: follow-up continuing
• Health condition(s) or Problem(s) studied: Biliary tract cancer (BTC)
• Date of first enrollment: 2024/07/16
• Target sample size: 4700
• Countries of recruitment: Japan
• Study type: Observational

Outcome(s)

• Primary Outcome: The following outcomes will be described among subgroups categorized by gene mutation patterns (HER2/MDM2/TP53), first-line treatment types, as well as BTC subtype-based cohorts. 1. HER2 alterations, MDM2 amplification, TP53 mutation 2. First-line treatment types 3. Baseline demographics and clinical characteristics 4. Overall response rate (ORR) 5. Disease control rate (DCR) 6. Time on treatment (ToT) 7. Overall survival (OS)
• Secondary Outcome: The following outcomes will be examined among subgroups categorized by BTC subtype-based cohorts. 1. To describe the OS following the administration of each 1L treatment type by thirty genes with the highest frequency of gene mutations 2. To examine association between co-mutations identified by the clustering analysis and the drug response to 1L treatment (ORR and DCR) 3. To examine association between identify mutations in the gene sets by the molecular-interaction pathway analysis and the drug response to 1L treatment (ORR and DCR)

Key inclusion & exclusion criteria

• Age minimum: 18years-old
• Age maximum: Not applicable
• Gender: Male and Female
• Exclude criteria: None.

Related Information

• Primary Sponsor: Nippon Boehringer Ingelheim Co., Ltd.
• Source(s) of Monetary Support: Nippon Boehringer Ingelheim Co., Ltd.