Biomarker Research Study for Patients With FGFR-Mutant Bladder Cancer Receiving Erdafitinib

The purpose of this study is to collect biomarker data from subjects receiving standard of care erdafitinib to further investigate the relationship between treatment with erdafitinib and clinical response, progression, and/or genetic alterations in cell free tumor DNA (cfDNA) from blood and urine samples.

In this study, next generation sequencing of cfDNA isolated from blood and urine will be performed by Resolution Bioscience. Blood-based cfDNA amplification and analysis is done by a CLIA-approved assay and the results of which will thus be available to physicians and patients. Urine-based cfDNA analysis from Resolution Bioscience is not yet approved and results will only be communicated if an actionable finding is identified that is not present on the blood-based test. Samples will be collected at the patients homes by a qualified phlebotomist.

xCures may provide the treating physician and the subject the biomarker results from blood-based cfDNA tests. Because the testing of urine DNA is experimental and done solely for research, those results will not be shared unless there is an important finding known to be relevant to the subject's medical care at the time of the study. The genetic tests are not approved for bladder cancer, so the information generated from biomarker research studies is preliminary in nature. Therefore, the significance and scientific validity of the results are undetermined. xCures and/or Tyra may additionally share data with the treating physician and researchers for the purposes of publication.

Patients co-enroll in an observational research study (XCELSIOR, NCT03793088) and consent to provide xCures right of access to their full medical records under HIPAA.

Patient clinical data is structured and centralized by xCures in a 21 CFR Part 11-compliant REDCap Cloud database in Case Report Forms (CRFs). The CRFs are a set of electronic forms for each patient that provides a record of clinical data generated according to the protocol. Data is abstracted directly from medical records generated in the standard practice of medicine.

xCures will request medical records directly from treating institutions and from other providers (commercial next generation sequencing providers, for example) and will work with treating physicians to ensure the completeness and accuracy of CRF content based on electronic medical records.