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)
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
4 other identifiers
interventional
107
17 countries
150
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Feb 2020
Longer than P75 for phase_2
150 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 20, 2019
CompletedFirst Posted
Study publicly available on registry
November 21, 2019
CompletedStudy Start
First participant enrolled
February 28, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 12, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
February 27, 2025
CompletedResults Posted
Study results publicly available
August 1, 2025
CompletedFebruary 24, 2026
February 1, 2026
4.4 years
November 20, 2019
July 11, 2025
February 12, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Cohort 1: Recurrence-Free Survival (RFS)
RFS was defined as the time from the date of randomization until the date of the reappearance of high-risk disease (high-grade Ta, T1 or carcinoma in situ \[CIS\]), or death, whichever was reported first. Recurrence was assessed using cystoscopy, bladder mapping, urine cytology, and computed tomography (CT)/ magnetic resonance imaging (MRI) urogram. Participants who were recurrence-free and alive or had unknown status were censored at the last tumor assessment. The Kaplan-Meier method was used to estimate the distribution of overall RFS for each treatment group.
From randomization (Cycle 1 Day 1, pre-dose) up to 48.2 months
Secondary Outcomes (5)
Cohort 1: Recurrence-Free Survival Rate at 6 Months and 12 Months
At Month 6 and Month 12
Cohort 1: Time to Progression
From randomization (Cycle 1 Day 1, pre-dose) up to 48.2 months
Cohort 1: Overall Survival
From randomization (Cycle 1 Day 1, pre-dose) up to 48.2 months
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
From start of treatment (Day 1) up to 25.2 months
Plasma Concentrations of Erdafitinib
All cohorts: Pre-dose on Cycle 1 Day 14, pre-dose and 3 hours post-dose on Cycle 2 Day 1 (each cycle was of 28 days)
Study Arms (4)
Cohort 1: Erdafitinib
EXPERIMENTALParticipants 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.
Cohort 1: Investigators Choice
ACTIVE COMPARATORParticipants 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.
Cohort 2
EXPERIMENTALParticipants with high-risk, BCG- unresponsive NMIBC presenting as CIS with or without concurrent papillary tumor will receive treatment with erdafitinib.
Cohort 3
EXPERIMENTALMarker lesion study in intermediate-risk NMIBC presenting as papillary disease only. All enrolled participants will receive treatment with erdafitinib.
Interventions
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.
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.
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.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (150)
Urological Associates of Southern Arizona, P.C.
Tucson, Arizona, 85741, United States
USC Institute of Urology
Los Angeles, California, 90033, United States
The Urology Center of Colorado
Denver, Colorado, 80211, United States
Urological Research Network
Hialeah, Florida, 33016, United States
Emory University Winship Cancer Institute
Atlanta, Georgia, 30322, United States
Northwestern University
Chicago, Illinois, 60611, United States
Simmons Cancer Institute
Springfield, Illinois, 62702, United States
University of Kansas
Westwood, Kansas, 66205, United States
Albany Medical College
Albany, New York, 12208, United States
NYU Langone Health
New York, New York, 10016, United States
Great Lakes Physician PC d/b/a Western New York Urology Associates
Sanborn, New York, 14132, United States
Montefiore Medical Center
The Bronx, New York, 10461, United States
Levine Cancer Institute
Charlotte, North Carolina, 28204, United States
The Urology Group
Cincinnati, Ohio, 45212, United States
The Ohio State University- James Cancer Hospital
Columbus, Ohio, 43221, United States
Oregon Health And Science University
Portland, Oregon, 97239, United States
MidLantic Urology
Bala-Cynwyd, Pennsylvania, 19004, United States
University of Pittsburgh Medical Center - Cancer Centers
Pittsburgh, Pennsylvania, 15232, United States
Urology Associates
Nashville, Tennessee, 37209, United States
UT Southwestern Medical Center
Dallas, Texas, 75390, United States
University of Washington
Seattle, Washington, 98008, United States
Medical College of Wisconsin Froedtert Hospital
Milwaukee, Wisconsin, 53226, United States
Sociedade Beneficente de Senhoras Hospital Sirio Libanes
Buenos Aires, C1419AHN, Argentina
CEMIC Saavedra
Buenos Aires, C1431FWN, Argentina
Hospital Britanico de Buenos Aires
Buenos Aires, Argentina
Hospital Italiano de Buenos Aires
Ciudad Autonoma de, C1199ABB, Argentina
Cemaic Centro Privado de Especialidades Medicas Ambulatorias e Investigacion Clinica
Córdoba, 5000, Argentina
Centro Urologico Profesor Bengio
Córdoba, X5000KPH, Argentina
Hospital Privado de Cordoba
Córdoba, XX5016KEH, Argentina
Flinders Medical Centre
Bedford Park, 5042, Australia
St Vincent s Hospital Sydney
Darlinghurst, 2010, Australia
Macquarie University
Macquarie University, 2109, Australia
Peter MacCallum Cancer Centre
Melbourne, 3000, Australia
AZ Maria Middelares
Ghent, 9000, Belgium
UZ Leuven
Leuven, 3000, Belgium
CHU Sart-Tilman
Liège, 4000, Belgium
Algemeen Ziekenhuis Delta
Roeselare, 8800, Belgium
Liga Paranaense de Combate ao Cancer
Curitiba, 81520 060, Brazil
Oncocentro Ceará
Fortaleza, 60135-237, Brazil
Associacao de Combate ao Cancer em Goias - Hospital de Cancer Araujo Jorge
Goiânia, 74605-070, Brazil
Liga Norte Riograndense Contra O Cancer
Natal, 59075-740, Brazil
Irmandade Santa Casa de Misericordia de Porto Alegre
Porto Alegre, 90050-170, Brazil
Hospital Nossa Senhora da Conceicao S A
Porto Alegre, 91350 200, Brazil
Instituto de Medicina Integral Professor Fernando Figueira
Recife, 50070-550, Brazil
Hospital Das Clinicas Da Faculdade De Medicina De RPUSP HCRP
Ribeirão Preto, 14048-900, Brazil
Oncoclinicas Rio de Janeiro S A
Rio de Janeiro, 22250 905, Brazil
Instituto de Educacao, Pesquisa e Gestao em Saude Instituto Americas (COI)
Rio de Janeiro, 22775 001, Brazil
CEPHO Centro de Estudos e Pesquisa de Hematologia e Oncologia
Santo André, 09060-650, Brazil
Fundacao Faculdade de Medicina Instituto do Cancer do Estado de Sao Paulo
São Paulo, 01246 000, Brazil
Sociedade Beneficente de Senhoras Hospital Sirio Libanes
São Paulo, 01308 901, Brazil
Instituto de Assistencia Medica ao Servidor Publico Estadual IAMSPE
São Paulo, 04039-004, Brazil
Nanjing Drum Tower Hospital
Nanjing, 210008, China
Fudan University Shanghai Cancer Center
Shanghai, 200032, China
Renji Hospital, Shanghai Jiaotong University School of Medicine
Shanghai, 200240, China
Tongji Hospital Tongji Medical College of Huazhong University of Science and Technology
Wuhan, 430030, China
Fakultni nemocnice u sv. Anny v Brne
Brno, 656 91, Czechia
Krajská nemocnice Liberec
Liberec, 460 63, Czechia
Vseobecna fakultni nemocnice v Praze
Prague, 128 08, Czechia
Fakultni nemocnice v Motole
Prague, 150 06, Czechia
Hopital Pellegrin CHU Bordeaux
Bordeaux, 33076, France
CHU Gabriel-Montpied
Clermont-Ferrand, 63000, France
Hopital Huriez
Lille, 59037, France
Hôpital Edouard Herriot
Lyon, 69437, France
Institut Paoli Calmettes
Marseille, 13273, France
Hôpital Universitaire Pitié-Salpêtrière
Paris, 75013, France
Groupe Hospitalier Diaconesses Croix Saint Simon
Paris, 75020, France
Hopital Bichat Claude Bernard
Paris, 75877, France
CHU De Poitiers
Poitiers, 86021, France
Chu Rennes Hopital Pontchaillou
Rennes, 35033, France
Hopital Charles Nicolle
Rouen, 76031, France
CHP Saint Gregoire
Saint-Grégoire, 35760, France
Institut Universitaire du Cancer Toulouse Oncopole
Toulouse, 31000, France
Centre Hospitalier Universitaire de Nancy - Hôpital Central
Vandœuvre-lès-Nancy, 54519, France
Gustave Roussy
Villejuif, 94800, France
Urologicum Duisburg
Duisburg, 47169, Germany
Klinikum Herne - Urologie
Herne, 44625, Germany
Universitatsklinikum Schleswig Holstein Campus Lubeck
Lübeck, 23538, Germany
Praxisklinik Urologie Rhein/Ruhr - Germany
Mülheim, 45468, Germany
Klinikum rechts der Isar - III. Med. Klinik und Poliklinik
München, 81675, Germany
Universitaetsklinikum Muenster
Münster, 48149, Germany
MVZ Urologie 24 gGmbH
Nuremberg, 90491, Germany
Studienpraxis Urologie Drs. Feyerabend
Nürtingen, 72622, Germany
CUROS - Uberörtliche urologische Gemeinschaftspraxis
Wesseling, 50389, Germany
Health Care Global Enterprises pvt Ltd
Bangalore, 560027, India
Rajiv Gandhi Cancer Institute & Research Centre
Delhi, 110085, India
King Georges Medical University
Lucknow, 226003, India
Meenakshi Mission Hospital and Research Center
Madurai, 625107, India
Muljibhai Patel Urological Hospital
Nadiād, 387001, India
CIMET s Inamdar Multispeciality Hospital
Pune, 411040, India
Ente Ecclesiastico Ospedale Generale Regionale F. Miulli
Acquaviva delle Fonti, 70021, Italy
Ospedale Regionale Umberto Parini
Aosta, 11100, Italy
Fondazione Istituto G. Giglio
Cefalù, 90015, Italy
Ospedale Civile di Guastalla
Guastalla, 42016, Italy
Azienda Ospedaliera ''Vito Fazzi''
Lecce, 73100, Italy
UOC Oncologia Ospedale Provinciale di Macerata
Macerata, 62100, Italy
IRCCS Ospedale San Raffaele
Milan, 20132, Italy
Fondazione IRCCS Istituto Nazionale dei Tumori
Milan, 20133, Italy
Fondazione G Pascale Istituto Nazionale Tumori IRCCS
Naples, 80138, Italy
Azienda Ospedaliera Sant Andrea
Roma, 00189, Italy
Azienda Ospedaliera Universitaria Integrata Verona
Verona, 37126, Italy
Juntendo University Hospital
Bunkyō City, 113 8431, Japan
Tokyo Medical and Dental University Hospital
Bunkyō City, 113 8519, Japan
Asahi General Hospital
Chiba, 289-2511, Japan
Hakodate Goryoukaku Hospital
Hakodate, 040 8611, Japan
Saitama Medical University International Medical Center
Hidaka, 350-1298, Japan
Hitachi General Hospital
Hitachi, 317-0077, Japan
St Marianna University Hospital
Kanagawa, 216 8511, Japan
Nagoya University Hospital
Nagoya, 466 8560, Japan
JOHAS Osaka Rosai Hospital
Osaka, 591-8025, Japan
Gunma Prefectural Cancer Center
Ōta-ku, 373 8550, Japan
Osaka Medical and Pharmaceutical University Hospital
Takatsuki, 569-8686, Japan
Toranomon Hospital
Tokyo, 105-8470, Japan
The Cancer Institute Hospital of JFCR
Tokyo, 135 8550, Japan
Fujita Health University Hospital
Toyoake, 470-1192, Japan
University of Tsukuba Hospital
Tsukuba, 305 8576, Japan
Yokohama City University Medical Center
Yokohama, 232 0024, Japan
Uniwersyteckie Centrum Kliniczne
Gdansk, 80 214, Poland
Pratia MCM Krakow
Krakow, 30-727, Poland
Uniwersytecki Szpital Kliniczny im. Fryderyka Chopina w Rzeszowie
Rzeszów, 35-055, Poland
Samodzielny Publiczny Szpital Kliniczny nr 2 PUM
Szczecin, 70-111, Poland
Medical Concierge Centrum Medyczne
Warsaw, 02 798, Poland
City Clinic Sp. z o.o.
Warsaw, 02-473, Poland
Uniwersytecki Szpital Kliniczny
Wroclaw, 50-556, Poland
Pusan National University Hospital
Busan, 49241, South Korea
Chungbuk National University Hospital
Cheongju-si, 361-711, South Korea
Kyungpook National University Chilgok Hospital
Daegu, 41404, South Korea
National Cancer Center
Goyang-si, 10408, South Korea
Chonnam National University Hwasun Hospital
Jeollanam-do, 58128, South Korea
Korea University Anam Hospital
Seoul, 02841, South Korea
Seoul National University Hospital
Seoul, 03080, South Korea
Kangbuk Samsung Hospital
Seoul, 03181, South Korea
Severance Hospital Yonsei University Health System
Seoul, 03722, South Korea
Samsung Medical Center
Seoul, 06351, South Korea
Fund. Puigvert
Barcelona, 08025, Spain
Hosp Clinic de Barcelona
Barcelona, 8036, Spain
Hosp. Univ. Ramon Y Cajal
Madrid, 28034, Spain
Hosp. Univ. 12 de Octubre
Madrid, 28041, Spain
Hosp. Univ. La Paz
Madrid, 28046, Spain
Corporacio Sanitari Parc Tauli
Sabadell, 08208, Spain
Hosp. Univ. Marques de Valdecilla
Santander, 39008, Spain
Instituto Valenciano de Oncologia
Valencia, 46009, Spain
Kaohsiung Medical University Chung Ho Memorial Hospital
Kaohsiung City, 80756, Taiwan
China Medical University Hospital
Taichung, 40447, Taiwan
Taichung Veterans General Hospital
Taichung, 40705, Taiwan
National Taiwan University Hospital
Taipei, 10002, Taiwan
Taipei Veterans General Hospital
Taipei, 11217, Taiwan
Chang Gung Memorial Hospital- Linkou
Taoyuan, 33305, Taiwan
The Christie NHS Foundation Trust Christie Hospital
Manchester, M20 4BX, United Kingdom
Universirty of Sheffield Teaching Hospitals NHS Trust
Sheffield, S10 2JF, United Kingdom
Lister Hospital
Stevenage, SG1 4AB, United Kingdom
Related Publications (1)
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.
PMID: 34125951DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Clinical Leader
- Organization
- Janssen Research & Development, LLC
Study Officials
- STUDY DIRECTOR
Janssen Research & Development, LLC Clinical Trial
Janssen Research & Development, LLC
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 20, 2019
First Posted
November 21, 2019
Study Start
February 28, 2020
Primary Completion
July 12, 2024
Study Completion
February 27, 2025
Last Updated
February 24, 2026
Results First Posted
August 1, 2025
Record last verified: 2026-02
Data Sharing
- IPD Sharing
- Will share
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