Bladder CancerNCT05052372
Biomarker Research Study for Patients With FGFR-Mutant Bladder Cancer Receiving Erdafitinib
An observational study of Bladder Cancer and Urothelial Carcinoma, run by xCures at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Bladder Cancer, Urothelial Carcinoma, Transitional Cell Carcinoma
- What is being tested?
- Balversa
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Nov 2021to 20 Dec 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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).
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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.
- Conditions
- Bladder Cancer, Urothelial Carcinoma, Transitional Cell Carcinoma, Platinum-Resistant Urothelial Carcinoma, Bladder Urothelial Carcinoma, FGFR Mutation
- Being tested
- Balversa
- Medical subject headings
- Urinary Bladder Neoplasms, Carcinoma, Transitional Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BalversaDrug
BALVERSA® is a prescription medicine used to treat adults with bladder cancer (urothelial cancer) that has spread or cannot be removed by surgery
What is being measured
The primary outcome is the main question the study is designed to answer.
Relationship between treatment with erdafitinib and clinical response
Specimen collection will be performed at the following timepoints to assess clinical response while being treated with erdafitinib:
- Baseline (prior to or within 7 days of starting erdafitinib)
- Suspicion of progression (optional)
- Post-progression
Measured: Time to progression will vary for each patient. We expect most patients to progress on erdafitinib within 2 to 15 months from baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2021
- Study start21 Nov 2021
- Primary completion20 Dec 2022
- Study completion20 Dec 2022
Why it stopped: Sponsor request to terminate study due to enrollment numbers
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Bladder CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- xCures
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Daniel J. George, MD, Principal investigator, Duke Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05052372
- Sponsor's protocol ID
- TYRA-001-BC-2021
- Status last verified
- Sep 2021
- First submitted
- 10 Sep 2021
- Last updated
- 22 Dec 2022
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 31585937
Study at a glance
From the registry record, last updated by the sponsor on 22 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Biomarker Research Study for Patients With FGFR-Mutant Bladder Cancer Receiving Erdafitinib
- ClinicalTrials.gov ID
- NCT05052372
- Acronym / sponsor ID
- TYRA-001-BC-2021
- Conditions
- Bladder Cancer, Urothelial Carcinoma, Transitional Cell Carcinoma, Platinum-Resistant Urothelial Carcinoma, Bladder Urothelial Carcinoma, FGFR Mutation
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Sponsor request to terminate study due to enrollment numbers
- Enrolment
- 2 participants (actual)
- Lead sponsor
- xCuresIndustry (pharma, biotech, devices)
- Study start
- 21 Nov 2021 (actual)
- Primary completion
- 20 Dec 2022 (actual)
- Study completion
- 20 Dec 2022 (actual)
- First posted
- 22 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BalversaBALVERSA® is a prescription medicine used to treat adults with bladder cancer (urothelial cancer) that has spread or cannot be removed by surgery | Drug | – |
Primary outcome
Relationship between treatment with erdafitinib and clinical response
Time frame: Time to progression will vary for each patient. We expect most patients to progress on erdafitinib within 2 to 15 months from baseline
Specimen collection will be performed at the following timepoints to assess clinical response while being treated with erdafitinib: 1. Baseline (prior to or within 7 days of starting erdafitinib) 2. Suspicion of progression (optional) 3. Post-progression
Results and safety
The study was stopped early (reason given: Sponsor request to terminate study due to enrollment numbers). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Dec 2022.
- Background: PubMed 31585937Facchinetti 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Daniel J. George, MD principal investigator
Duke Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Bladder Cancer and Urothelial Carcinoma, run by xCures at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Sponsor request to terminate study due to enrollment numbers). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Relationship between treatment with erdafitinib and clinical response (time frame: Time to progression will vary for each patient. We expect most patients to progress on erdafitinib within 2 to 15 months from baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05052372, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Dec 2022.