Advanced Solid TumorPhase 2RAGNARNCT04083976
A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations
A Phase 2 study testing Erdafitinib in people with Advanced Solid Tumor, run by Janssen Research & Development, LLC at 179 sites in 15 countries.
Trial summary
The purpose of this study is to evaluate the efficacy of erdafitinib in terms of overall response rate (ORR) in adult and pediatric participants with advanced solid tumors with fibroblast growth factor receptor (FGFR) alterations (mutations or gene fusions). Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Advanced Solid Tumor
- What is being tested?
- Erdafitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 316 participants
- Where
- 179 sitesin 15 countries
- Study dates
- 20 Nov 2019to 28 Feb 2026
- Phase
- Phase 2
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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Histologic demonstration of an unresectable, locally advanced, or metastatic solid tumor malignancy with an fibroblast growth factor receptor (FGFR) mutation or FGFR gene fusion
- Measurable disease
- Participant must have received at least one prior line of systemic therapy in the advanced, unresectable, or metastatic setting; or is a child or adolescent participant with a newly-diagnosed solid tumor and no acceptable standard therapies
- Documented progression of disease, defined as any progression that requires a change in treatment, prior to full study screening
Who cannot join
- Has had prior chemotherapy, targeted therapy, or treatment with an investigational anticancer agent within 15 days or less than or equal to (<=) 5 half-lives of the agent (whichever is longer) and up to a maximum of 30 days before the first dose of erdafitinib
- The presence of FGFR gatekeeper and resistance mutations
- Histologic demonstration of urothelial carcinoma
- Hematologic malignancy (i.e., myeloid and lymphoid neoplasms
- For non-small cell lung cancer participants only: pathogenic somatic mutations or gene fusions in the following genes: EGFR, ALK, ROS1, NTRK, BRAF V600E and KRAS
- Active malignancies other than for disease requiring therapy
Trial locations and contacts
179 sites in 15 countries.
179 sites in 15 countries
United States27 sites
Hawaii Cancer Care
‘Aiea, Hawaii, 96701
Emory University
Atlanta, Georgia, 30022
Dana Farber Cancer Institute
Boston, Massachusetts, 02115
Levine Cancer Institute, Carolinas HealthCare System
Charlotte, North Carolina, 28204
Tennessee Oncology, PLLC
Chattanooga, Tennessee, 37404
Oncology Hematology Care
Cincinnati, Ohio, 45242
Rocky Mountain Cancer Centers
Colorado Springs, Colorado, 80907
Nationwide Children's Hospital
Columbus, Ohio, 43205
Texas Oncology Baylor Charles A Sammons Cancer Center
Dallas, Texas, 75246
Henry Ford Hospital
Detroit, Michigan, 48202
Texas Oncology-Fort Worth Cancer Center
Fort Worth, Texas, 76104
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Memorial Cancer Institute
Hollywood, Florida, 33021
MD Anderson
Houston, Texas, 77030
Oncology Consultants Texas
Houston, Texas, 77030
Cancer Specialists of North Florida
Jacksonville, Florida, 32256
Tennessee Oncology
Nashville, Tennessee, 37203
Mount Sinai
New York, New York, 10029
Memorial Sloan Kettering Cancer Center
New York, New York, 10128
Texas Oncology Odessa-West Texas Cancer Center
Odessa, Texas, 79761
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15232
Texas Oncology San Antonio Northeast
San Antonio, Texas, 78217
Maine Medical Center
Scarborough, Maine, 04074
NorthWest Medical Specialties, PLLC
Tacoma, Washington, 98405
Arizona Oncology Associates, PC - HOPE
Tucson, Arizona, 85711
Aurora Research Institute
Wauwatosa, Wisconsin, 53226
China18 sites
Cancer Hospital Chinese Academy of Medical Sciences
Beijing, 100021
Beijing Tiantan Hospital, Capital Medical University
Beijing, 100070
Peking University Third Hospital
Beijing, 100191
Chinese PLA General Hospital
Beijing, 100853
Beijing Luhe Hospital, Capital Medical University
Beijing, 101100
Jilin cancer hospital
Changchun, 130012
The First Hospital of Jilin University
Changchun, 130021
West China Hospital,Sichuan University
Chengdu, 610041
Chongqing Southwest Hospital
Chongqing, 400038
Third Military Medical University Daping Hospital Cancer Center
Chongqing, 400042
Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University
Guangzhou, 510289
First affiliated Hospital of Zhejiang University
Hangzhou, 310003
Zhejiang Cancer Hospital
Hangzhou, 310022
Eastern Theater General Hospital, Qinhuai District Medical Area
Nanjing, 210002
Ruijin Hospital Shanghai Jiao Tong University
Shanghai, 200025
Cancer Hospital, FuDan University
Shanghai, 200032
Shanghai Zhongshan Hospital
Shanghai, 200032
Huashan Hospital Fudan University
Shanghai, 200040
Brazil16 sites
Fundacao Pio XII
Barretos, 14784-400
Liga Paranaense de Combate ao Cancer
Curitiba, 81520 060
Associacao de Combate ao Cancer em Goias - Hospital de Cancer Araujo Jorge
Goiânia, 74605-070
Associacao Hospital de Caridade Ijui
Ijuí, 98700-000
Liga Norte Riograndense Contra O Cancer
Natal, 59075-740
Irmandade Santa Casa de Misericordia de Porto Alegre
Porto Alegre, 90050-170
Instituto de Medicina Integral Professor Fernando Figueira
Recife, 50070-550
Oncoclinicas Rio de Janeiro S A
Rio de Janeiro, 22250 905
Instituto D Or de Pesquisa e Ensino IDOR
Rio de Janeiro, 22281 100
Hospital Sao Rafael
Salvador, 41253 190
Fundacao Faculdade de Medicina - Instituto do Cancer do Estado de Sao Paulo
São Paulo, 01246 000
Sociedade Beneficente de Senhoras Hospital Sirio Libanes
São Paulo, 01308 901
Real e Benemerita Associacao Portuguesa de Beneficencia
São Paulo, 01321-001
Fundacao Antonio Prudente A C Camargo Cancer Center
São Paulo, 01509 900
Hospital Das Clinicas Da Faculdade De Medicina Da USP
São Paulo, 05410-030
GRAACC
São Paulo, 4039001
Spain13 sites
Hosp. Univ. Quiron Dexeus
Barcelona, 08028
Hosp Univ Vall D Hebron
Barcelona, 08035
Hosp Clinic de Barcelona
Barcelona, 08036
Hosp. Sant Joan de Deu
Barcelona, 08950
Inst. Cat. Doncologia-H Duran I Reynals
L'Hospitalet de Llobregat, 08908
Hosp. Infantil Univ. Nino Jesus
Madrid, 28009
Clinica Univ. de Navarra
Madrid, 28027
Hosp. Univ. Ramon Y Cajal
Madrid, 28034
Hosp Univ Fund Jimenez Diaz
Madrid, 28040
Hosp. Univ. 12 de Octubre
Madrid, 28041
Hosp. Univ. La Paz
Madrid, 28046
Hosp. Virgen Del Rocio
Seville, 41013
Hosp. Clinico Univ. de Valencia
Valencia, 46010
Argentina12 sites
Instituto de Investigaciones Metabólicas
Buenos Aires, C1012AAR
Hospital Aleman
Buenos Aires, C1118AAT
Centro Oncológico Korben
Buenos Aires, C1426AGE
Instituto Fleni
Buenos Aires, C1428AQK
CEMIC Saavedra
Buenos Aires, C1431FWN
Cemaic Centro Privado de Especialidades Medicas Ambulatorias e Investigacion Clinica
Córdoba, 5000
Hospital Italiano de La Plata
La Plata Lpl Lpl, 1900
Instituto de Investigaciones Clinicas Mar del Plata
Mar del Plata, B7600FZN
Hospital Privado de Comunidad
Mar del Plata, B7602CBM
Hospital Universitario Austral
Pilar, 1629
Sanatorio Britanico de Rosario
Rosario, 2000
ARS Médica
San Salvador de Jujuy, Y4600AFW
Germany12 sites
Charité
Berlin, 10115
Universitaetsklinikum Koeln
Cologne, 50937
Medizinische Fakultaet Carl Gustav Carus Technische Universitaet Dresden
Dresden, 01307
Universitaetsklinikum Essen
Essen, 45147
Universitaetsklinikum Frankfurt
Frankfurt, 60385
Asklepios Klinik Altona
Hamburg, 22763
Universitaetsklinikum Heidelberg 1
Heidelberg, 69120
Universitaetsklinikum Heidelberg
Heidelberg, 69120
Universitaetsklinikum Leipzig
Leipzig, 04103
Universitatsmedizin der Johannes Gutenberg Universitat Mainz
Mainz, 55131
Klinikum rechts der Isar der TU Munchen
München, 81675
Universitatsklinikum Tubingen
Tübingen, 72076
South Korea12 sites
Severance Hospital Yonsei University Health System 1
Seoul, 03722
Severance Hospital Yonsei University Health System 2
Seoul, 03722
Severance Hospital Yonsei University Health System
Seoul, 03722
Asan Medical Center 1
Seoul, 05505
Asan Medical Center 2
Seoul, 05505
Asan Medical Center
Seoul, 05505
Samsung Medical Center 1
Seoul, 06351
Samsung Medical Center 2
Seoul, 06351
Samsung Medical Center
Seoul, 06351
The Catholic University of Korea Seoul St Marys Hospital 1
Seoul, 06591
The Catholic University of Korea Seoul St Marys Hospital
Seoul, 06591
Seoul National University Hospital
Seoul, 3080
Taiwan12 sites
Changhua Christian Hospital
Changhua, 50006
Kaohsiung Medical University Chung Ho Memorial Hospital
Kaohsiung City, 807
E-Da Cancer Hospital
Kaohsiung City, 82445
Chang Gung Medical Foundation
Kaohsiung City, 833
China Medical University Hospital
Taichung, 403
Taichung Veterans General Hospital
Taichung, 40705
Chi Mei Medical Center Yong Kang
Tainan, 710
National Cheng Kung University Hospital
Tainan, 710
Chi Mei Medical Center Liu Ying
Tainan, 736
National Taiwan University Hospital
Taipei, 10002
Taipei Veterans General Hospital
Taipei, 11217
Chang Gung Memorial Hospital Linkou Branch
Taoyuan City, 333
Belgium11 sites
AZ Klina
Brasschaat, 2930
UCL Hopital Saint-Luc
Brussels, 1200
Grand Hopital de Charleroi, site Notre Dame
Charleroi, 6000
CHU UCL Namur - Site Dinant
Dinant, 5500
UZA
Edegem, 2650
AZ Maria Middelares
Ghent, 9000
UZ Gent
Ghent, 9000
Jolimont
Haine-Saint-Paul, 7100
CHU UCL Namur - Site Sainte Elisabeth
Namur, 5000
Cliniques St Pierre
Ottignies, 1340
CHU UCL Namur - Site Godinne
Yvoir, 5530
France11 sites
Hopital Saint André
Bordeaux, 33075
Centre Francois Baclesse
Caen, 14000
Centre Jean Perrin
Clermont-Ferrand, 63011
Centre Georges Francois Leclerc
Dijon, 21000
Centre Oscar Lambret
Lille, 59000
Centre Leon Berard
Lyon, 69008
Hopital de la Timone
Marseille, 130005
Institut Curie
Paris, 75005
CHU De Poitiers
Poitiers, 86021
Institut de Cancerologie de Lorraine
Vandœuvre-lès-Nancy, 54519
Institut Gustave Roussy
Villejuif, 94800
Italy9 sites
Istituto Ortopedico Rizzoli
Bologna, 40136
ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162
ASST di Monza
Monza, 20900
Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
Naples, 80138
Fondazione G Pascale Istituto Nazionale Tumori IRCCS
Naples, 80138
Ospedale S. Maria Della Misericordia
Perugia, 06132
Ospedale Santa Chiara AO Universitaria Pisana
Pisa, 56126
Azienda Ospedaliera S. Maria Terni
Terni, 5100
Ospedale Borgo Roma
Verona, 37134
Poland8 sites
Centrum Onkologii im Prof F Lukaszczyka w Bydgoszczy
Bydgoszcz, 85 796
Przychodnia Lekarska KOMED Roman Karaszewski
Konin, 62-500
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki w Krakowie
Krakow, 31 501
Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im M Kopernika w Lodzi
Lodz, 93 513
Centrum Medyczne Pratia Poznan
Skorzewo, 60 185
Instytut Matki i Dziecka
Warsaw, 01 211
Centralny Szpital Kliniczny MSWiA w Warszawie
Warsaw, 02-507
Narodowy Instytut Onkologii im Marii Sklodowskiej Curie Panstwowy Instytut Badawczy
Warsaw, 02-781
United Kingdom7 sites
Bristol Royal Hospital for Children
Bristol, BS2 8AE
Bristol Haematology and Oncology Centre
Bristol, BS2 8ED
The Christie Nhs Foundation Trust
Manchester, M20 4BX
The Clatterbridge Cancer Centre
Metropolitan Borough of Wirral, CH63 4JY
Freeman Hospital
Newcastle upon Tyne, NE7 7DN
Derriford Hospital
Plymouth, PL6 8DH
The Royal Marsden NHS Trust
Sutton, SM2 5PT
Australia6 sites
Flinders Centre for Innovation in Cancer
Adelaide, 5042
Chris O'Brien Lifehouse
Camperdown, 2050
St Vincent s Hospital Sydney
Darlinghurst, 2010
St Vincents Hospital Melbourne
Fitzroy, 3065
Fiona Stanley Hospital
Murdoch, 6150
Intergrated Clinical Oncology Network Pty Ltd (ICON)
South Brisbane, 4101
Japan5 sites
National Cancer Center Hospital
Chūōku, 104 0045
Hiroshima University Hospital
Hiroshima, 734 8551
National Cancer Center Hospital East
Kashiwa, 277 8577
National Hospital Organization Shikoku Cancer Center
Matsuyama, 791-0280
Fujita Health University Hospital
Toyoake, 470-1192
About this study
Official title: A Phase 2 Study of Erdafitinib in Subjects With Advanced Solid Tumors and FGFR Gene Alterations
The purpose of this study is to evaluate the efficacy of erdafitinib in terms of overall response rate (ORR) in adult and pediatric participants with advanced solid tumors with fibroblast growth factor receptor (FGFR) alterations (mutations or gene fusions). It will also evaluate ORR in pediatric participants with advanced solid tumors and FGFR alterations.
Read the detailed description
Erdafitinib is a selective and potent pan FGFR 1-4 inhibitor with demonstrated clinical activity in participants with metastatic urothelial cancer and cholangiocarcinoma identified to have alterations in the FGFR pathway. This study targets the underlying altered biology of FGFR-driven tumors irrespective of solid tumor histology subtype. The study consists of screening phase, treatment phase and the post treatment follow-up phase (from the end of treatment visit until the participant has died, withdraws consent, is lost to follow-up, or the end of study, whichever comes first). End of study is considered as the time when the last participant receives the last dose of study drug on the study and either all pediatric participants are off study or until the most recently enrolled pediatric participant still participating in the study has 6 months of follow-up, whichever occurs first. Currently this study is recruiting pediatric participants only.
- Conditions
- Advanced Solid Tumor
- Being tested
- Erdafitinib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ErdafitinibDrug
Also called JNJ-42756493
Participants will receive erdafitinib oral tablets.
| Study group | Role | What they receive |
|---|---|---|
| Erdafitinib | Experimental | Participants with fibroblast growth factor receptor (FGFR) mutations and FGFR gene fusions will receive a dose of erdafitinib oral tablets until disease progression, intolerable toxicity, withdrawal of consent, decision by the investigator to discontinue treatment, or end of data collection timepoint if there is clinical benefit in the opinion of the investigator, has been achieved. Receives: Drug: Erdafitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Broad Panel and Pediatric Cohorts: Objective Response Rate (ORR) Based on Response Assessment in Neuro-Oncology (RANO) as Assessed by Independent Review Committee (IRC)
ORR is defined as the percentage of participants who achieved a complete response (CR), or partial response (PR) based on Response Assessment in Neuro-Oncology (RANO) criteria. According to RANO criteria whereby overall RANO response is based on both radiographic response (CR: disappearance of all target lesions; PR: sum of products of diameters [SPD] decreased by >=50 percent [%] from baseline value) and clinical performance status with steroid dose information.
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 4 years
Core Panel Cohort: Objective Response Rate (ORR) Based on Response Assessment in Neuro-Oncology (RANO) as Assessed by Independent Review Committee (IRC)
ORR is defined as the percentage of participants who achieved a CR, or PR based on RANO criteria. According to RANO criteria whereby overall RANO response is based on both radiographic response (CR: disappearance of all target lesions; PR: sum of products of diameters [SPD] decreased by >=50 percent [%] from baseline value) and clinical performance status with steroid dose information. The core panel cohort is a subgroup of the broad panel cohort with a select panel of pre-specified FGFR markers: FGFR3 mutations (S249C;Y373C; R248C; G370C); FGFR2 mutations (C382R); FGFR3 fusions (FGFR3-TACC3); FGFR2 fusions (FGFR2-BICC1; FGFR2-TACC2).
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 4 years
Secondary outcomes (14)
Objective Response Rate (ORR) as Assessed by Investigator Assessment
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Duration of Responses (DOR)
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Disease Control Rate (DCR)
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Clinical Benefit Rate (CBR)
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Progression-free Survival (PFS)
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Overall Survival (OS)
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Number of Participants With Adverse Events (AEs)
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Number of Participants With Adverse Events (AEs) by Severity
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Plasma Concentration of Erdafitinib
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Broad Panel, CCA Expansion, and Exploratory Cohorts: Change From Baseline in European Organization for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30 (EORTC-QLQ-C30)
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Pediatric Cohorts: Change From Baseline in Pediatric Functional Assessment Of Cancer Therapy-Brain (Peds FACT-Br)
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Change From Baseline in Patient Global Impression of Symptom Severity (PGIS)
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Change From Baseline in Patient Global Impression of Change (PGIC)
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Change From Baseline in European Quality of Life -5 Dimensions-5 Levels (EQ-5D-5L)
Measured: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2019
- Study start20 Nov 2019
- Primary completion4 Dec 2023
- Study completion28 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Advanced Solid TumorStudies of the same condition, open and under way.
- More trials run by Janssen Research & Development, LLCThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 316 participants (actual)
Sponsor and investigators
- Lead sponsor
- Janssen Research & Development, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Janssen Research & Development, LLC Clinical Trial, Study director, Janssen Research & Development, LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04083976
- Sponsor's protocol ID
- CR108661
- Other IDs
- 42756493CAN2002, 2019-002113-19, 2023-510301-18-00, 2023, 2019
- Acronym
- RAGNAR
- Status last verified
- Aug 2026
- First submitted
- 6 Sep 2019
- Last updated
- 17 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Publications
Carranza O, Pant S, Schuler M, Witt O, Geoerger B, Loriot Y, Farouk Sait S, Arnold D, Dal Molin GZ, Lugowska I, Winter H, Baldini C, Corassa M, Thomas S, Crow L, Xia Y, Triantos S, Sweiti H, Reardon DA. Erdafitinib in Patients With Advanced Solid Tumors With FGFR Alterations (RAGNAR): Results of Tumor-Specific Analyses and Secondary Cohorts. JCO Precis Oncol. 2026 Jul;10(7):e2501094. doi: 10.1200/PO-25-01094. Epub 2026 Jul 15.
PubMed 42456086Pant S, Schuler M, Iyer G, Witt O, Doi T, Qin S, Tabernero J, Reardon DA, Massard C, Minchom A, Lugowska I, Carranza O, Arnold D, Gutierrez M, Winter H, Stuyckens K, Crow L, Najmi S, Hammond C, Thomas S, Santiago-Walker A, Triantos S, Sweiti H, Loriot Y; RAGNAR Investigators. Erdafitinib in patients with advanced solid tumours with FGFR alterations (RAGNAR): an international, single-arm, phase 2 study. Lancet Oncol. 2023 Aug;24(8):925-935. doi: 10.1016/S1470-2045(23)00275-9.
PubMed 37541273