Clinical trial NCT05052372: Biomarker Research Study for Patients With FGFR-Mutant Bladder Cancer Receiving Erdafitinib
Official title: Biomarker Research Study for Patients With FGFR-Mutant Bladder Cancer Receiving Erdafitinib
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Bladder Cancer
Urothelial Carcinoma
Transitional Cell Carcinoma
Platinum-Resistant Urothelial Carcinoma
Bladder Urothelial Carcinoma
FGFR Mutation
FGFR2 Gene Mutation
FGFR3 Gene Mutation
FGFR2 Amplification
Locally Advanced Urothelial Carcinoma
Metastatic Urothelial Carcinoma
Refractory Bladder Carcinoma
Refractory Bladder Urothelial Carcinoma
Lead sponsor: xCures
Sponsor class: INDUSTRY
First posted: 2021-09-22
Enrollment: 2
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients in the United States with a diagnosis of locally advanced or metastatic bladder cancer (TCC/UC) may be screened for this study. In order to enroll on the study, subjects must have documented proof of an FGFR alteration according to a CLIA-based test with planned treatment with erdafitinib, or currently undergoing treatment with erdafitinib.

Brief summary: Bladder cancers are associated with genetic mutations that are present in the patient's bladder or urothelium, the lining of the lower urinary tract. Fibroblast growth factor (FGFR) alterations are present in approximately one in five patients with recurrent and refractory bladder cancer.

This study will collect biomarker data from subjects receiving erdafitinib to further investigate the relationship between treatment with erdafitinib and clinical response, progression, and/or genetic alterations.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* Subjects with a diagnosis of locally advanced or metastatic bladder cancer
* Documented proof of an FGFR alteration according to a CLIA-based test
* Subjects who are receiving or will receive erdafitinib as their standard medical therapy as a monotherapy or in combination
* Age greater than or equal to 18 years.
* Subject is a resident of or seeking care in the United States
* Able and willing to provide informed consent to this biomarker study
* Informed consent obtained for the XCELSIOR longitudinal outcomes registry (NCT03793088).

Exclusion Criteria:

* Subjects younger than 18 years of age
* Subjects unwilling or unable to provide informed consent
* Subjects that are not receiving treatment in the United States or US Territories

Primary outcomes:
measure: Relationship between treatment with erdafitinib and clinical response
timeFrame: Time to progression will vary for each patient. We expect most patients to progress on erdafitinib within 2 to 15 months from baseline
Secondary outcomes:
Other outcomes:
Locations:
city: Raleigh
country: United States
facility: xCures Virtual Site
state: North Carolina
Study officials:
affiliation: Duke Health
name: Daniel J. George, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Facchinetti F, Hollebecque A, Bahleda R, Loriot Y, Olaussen KA, Massard C, Friboulet L. Facts and New Hopes on Selective FGFR Inhibitors in Solid Tumors. Clin Cancer Res. 2020 Feb 15;26(4):764-774. doi: 10.1158/1078-0432.CCR-19-2035. Epub 2019 Oct 4.