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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04172675
Registration number
NCT04172675
Ethics application status
Date submitted
20/11/2019
Date registered
21/11/2019
Titles & IDs
Public title
A Study of Erdafitinib Versus Investigator Choice of Intravesical Chemotherapy in Participants Who Received Bacillus Calmette-Guérin (BCG) and Recurred With High Risk Non-Muscle-Invasive Bladder Cancer (NMIBC)
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Scientific title
A Randomized Phase 2 Study of Erdafitinib Versus Investigator Choice of Intravesical Chemotherapy in Subjects Who Received Bacillus Calmette-Guérin (BCG) and Recurred With High Risk Non-Muscle-Invasive Bladder Cancer (NMIBC) and FGFR Mutations or Fusions
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Secondary ID [1]
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2019-002449-39
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Secondary ID [2]
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CR108699
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Urinary Bladder Neoplasms
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Condition category
Condition code
Cancer
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Bladder - transitional cell cancer
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Renal and Urogenital
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0
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Other renal and urogenital disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Erdafitinib
Treatment: Drugs - Investigator Choice (Gemcitabine)
Treatment: Drugs - Investigator Choice (Mitomycin C)
Experimental: Cohort 1: Erdafitinib - Participants with high-risk non-muscle-invasive bladder cancer (NMIBC) presenting as papillary tumor only (carcinoma in situ \[CIS\], absent), with disease recurrence after bacillus Calmette- Guerin (BCG) therapy will receive treatment with erdafitinib.
Active comparator: Cohort 1: Investigators Choice - Participants with high-risk NMIBC presenting as papillary tumor only (CIS, absent), with disease recurrence after BCG therapy will receive the investigator's choice of either intravesical gemcitabine or intravesical mitomycin C (MMC) or hyperthermic MMC. Participants who are randomized to gemcitabine or MMC or hyperthermic MMC in Cohort 1 and demonstrate a recurrence via investigator disease assessment will have the opportunity to cross over to treatment with erdafitinib.
Experimental: Cohort 2 - Participants with high-risk, BCG- unresponsive NMIBC presenting as CIS with or without concurrent papillary tumor will receive treatment with erdafitinib.
Experimental: Cohort 3 - Marker lesion study in intermediate-risk NMIBC presenting as papillary disease only. All enrolled participants will receive treatment with erdafitinib.
Treatment: Drugs: Erdafitinib
Participants will receive erdafitinib orally beginning on Cycle 1 Day 1 until 2 years of treatment have been completed, disease recurrence, intolerable toxicity, withdrawal of consent, a decision by the investigator to discontinue treatment, or study termination, whichever occurs first. Each cycle is of 28 days.
Treatment: Drugs: Investigator Choice (Gemcitabine)
Investigator's Choice treatment will be given once weekly for at least 4 doses of induction followed by monthly maintenance for at least 6 months.
Treatment: Drugs: Investigator Choice (Mitomycin C)
Investigator's Choice treatment will be given once weekly for at least 4 doses of induction followed by monthly maintenance for at least 6 months.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Recurrence-Free Survival (RFS)
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Assessment method [1]
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RFS is defined as the time from the date of randomization until the date of the reappearance of high-risk disease, or death, whichever is reported first.
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Timepoint [1]
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Up to 4 years
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Secondary outcome [1]
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Time to Progression
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Time from the date of randomization until the date of first documented evidence of any of progression or death. Participants who are progression -free and alive or have unknown status will be censored at the date of the last tumor assessment.
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Timepoint [1]
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Up to 4 years
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Secondary outcome [2]
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Overall Survival
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Assessment method [2]
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The time from the date of randomization to the date of the participant's death resulting from any cause. Participants who are alive or have unknown vital status will be censored at the date the participant was last known to be alive.
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Timepoint [2]
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Up to 4 years
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Secondary outcome [3]
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Recurrence-Free Survival
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Assessment method [3]
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RFS is defined as the time from the date of randomization until the date of the reappearance of high-risk disease, or death, whichever is reported first. Participants who are recurrence-free and alive or have unknown status will be censored at the last tumor assessment.
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Timepoint [3]
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Months 6 and 12
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Secondary outcome [4]
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Plasma Concentration of Erdafitinib
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Assessment method [4]
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Plasma concentration of erdafitinib will be reported.
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Timepoint [4]
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Cycle 1 Day 14, Cycle 2 Day 1 (each cycle is of 28 days)
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Secondary outcome [5]
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Number of Participants with Adverse events
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Assessment method [5]
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An adverse event is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product
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Timepoint [5]
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Up to 4 years
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Eligibility
Key inclusion criteria
* Histologically confirmed, recurrent, non-muscle-invasive urothelial carcinoma of the bladder. Variant pathology are allowed
* Tumor with specified fibroblast growth factor receptor (FGFR) mutations or fusions
* Bacillus Calmette- Guerin (BCG)-unresponsive after adequate BCG therapy or BCG experienced participants
* Refuses or is not eligible for cystectomy (Cohort 1 and Cohort 2 only)
* Eastern Cooperative Oncology Group (ECOG) performance status Grade 0-1
* Must sign an informed consent form (ICF) (or their legally acceptable representative must sign) indicating that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study
* A woman of childbearing potential must have a negative pregnancy test (beta-human chorionic gonadotropin [beta-hCG]) (urine or serum) within 7 days before randomization (Cohort 1) or the first dose of study drug (Cohort 2 and Cohort 3)
* Adequate bone marrow, liver, and renal function as specified in the protocol
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Minimum age
18
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Histologically confirmed, muscle-invasive (T2 or higher stage) urothelial carcinoma of the bladder
* Histopathology demonstrating any small cell component, pure adenocarcinoma, pure squamous cell carcinoma, or pure squamous CIS of the bladder
* Prior treatment with an FGFR inhibitor
* Active malignancies other than the disease being treated under study. The only allowed exceptions are: (a) skin cancer treated within the last 24 months that is considered completely cured (b) adequately treated lobular carcinoma in situ (LCIS) and ductal CIS (c) history of localized breast cancer and receiving antihormonal agents, or history of localized prostate cancer (N0M0) and receiving androgen deprivation therapy
* Current central serous retinopathy or retinal pigment epithelial detachment of any grade
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
28/02/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/03/2025
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Actual
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Sample size
Target
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Accrual to date
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Final
107
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Flinders Medical Centre - Bedford Park
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St Vincent s Hospital Sydney - Darlinghurst
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Macquarie University - Macquarie University
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Peter MacCallum Cancer Centre - Melbourne
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5042 - Bedford Park
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2010 - Darlinghurst
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2109 - Macquarie University
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3000 - Melbourne
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Stevenage
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Janssen Research & Development, LLC
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Ethics approval
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Summary
Brief summary
The purpose of this study is to evaluate recurrence-free survival (RFS) in participants treated with erdafitinib vs Investigator's Choice, for participants with high-risk non-muscle-invasive bladder cancer (NMIBC) who harbor fibroblast growth factor receptor (FGFR) mutations or fusions, and who recurred after bacillus calmette-guerin (BCG) therapy.
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Trial website
https://clinicaltrials.gov/study/NCT04172675
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Trial related presentations / publications
Han MA, Maisch P, Jung JH, Hwang JE, Narayan V, Cleves A, Hwang EC, Dahm P. Intravesical gemcitabine for non-muscle invasive bladder cancer. Cochrane Database Syst Rev. 2021 Jun 14;6(6):CD009294. doi: 10.1002/14651858.CD009294.pub3.
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Public notes
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Contacts
Principal investigator
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Janssen Research & Development, LLC Clinical Trial
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Janssen Research & Development, LLC
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Contact person for public queries
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
The data sharing policy of the Janssen Pharmaceutical Companies of Johnson \& Johnson is available at www.janssen.com/clinical trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale open Data Access (YODA) Project site at yoda.yale.edu
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://www.janssen.com/clinical-trials/transparency
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT04172675