A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations

NCT ID: NCT04083976

Last Updated: 2025-11-12

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 316 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-11-20
• Study Completion Date: 2026-12-31

Brief 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). It will also evaluate ORR in pediatric participants with advanced solid tumors and FGFR alterations.

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Erdafitinib

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.

Group Type: EXPERIMENTAL

Erdafitinib

Intervention Type: DRUG

Participants will receive erdafitinib oral tablets.

Interventions

Erdafitinib

Participants will receive erdafitinib oral tablets.

Intervention Type: DRUG

Other Intervention Names

JNJ-42756493

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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

• Minimum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Janssen Research & Development, LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Janssen Research & Development, LLC Clinical Trial

Role: STUDY_DIRECTOR

Janssen Research & Development, LLC

Locations

Arizona Oncology Associates, PC - HOPE

Tucson, Arizona, United States

Rocky Mountain Cancer Centers

Colorado Springs, Colorado, United States

Memorial Cancer Institute

Hollywood, Florida, United States

Cancer Specialists of North Florida

Jacksonville, Florida, United States

Emory University

Atlanta, Georgia, United States

Hawaii Cancer Care

‘Aiea, Hawaii, United States

Maine Medical Center

Scarborough, Maine, United States

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Henry Ford Hospital

Detroit, Michigan, United States

Hackensack University Medical Center

Hackensack, New Jersey, United States

Mount Sinai

New York, New York, United States

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Levine Cancer Institute, Carolinas HealthCare System

Charlotte, North Carolina, United States

Oncology Hematology Care

Cincinnati, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Thomas Jefferson University

Philadelphia, Pennsylvania, United States

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Tennessee Oncology, PLLC

Chattanooga, Tennessee, United States

Tennessee Oncology

Nashville, Tennessee, United States

Texas Oncology Baylor Charles A Sammons Cancer Center

Dallas, Texas, United States

Texas Oncology-Fort Worth Cancer Center

Fort Worth, Texas, United States

MD Anderson

Houston, Texas, United States

Oncology Consultants Texas

Houston, Texas, United States

Texas Oncology Odessa-West Texas Cancer Center

Odessa, Texas, United States

Texas Oncology San Antonio Northeast

San Antonio, Texas, United States

NorthWest Medical Specialties, PLLC

Tacoma, Washington, United States

Aurora Research Institute

Wauwatosa, Wisconsin, United States

Instituto de Investigaciones Metabólicas

Buenos Aires, , Argentina

Hospital Aleman

Buenos Aires, , Argentina

Centro Oncológico Korben

Buenos Aires, , Argentina

Instituto Fleni

Buenos Aires, , Argentina

CEMIC Saavedra

Buenos Aires, , Argentina

Cemaic Centro Privado de Especialidades Medicas Ambulatorias e Investigacion Clinica

Córdoba, , Argentina

Hospital Italiano de La Plata

La Plata Lpl Lpl, , Argentina

Instituto de Investigaciones Clinicas Mar del Plata

Mar del Plata, , Argentina

Hospital Privado de Comunidad

Mar del Plata, , Argentina

Hospital Universitario Austral

Pilar, , Argentina

Sanatorio Britanico de Rosario

Rosario, , Argentina

ARS Médica

San Salvador de Jujuy, , Argentina

Flinders Centre for Innovation in Cancer

Adelaide, , Australia

Chris O'Brien Lifehouse

Camperdown, , Australia

St Vincent s Hospital Sydney

Darlinghurst, , Australia

St Vincents Hospital Melbourne

Fitzroy, , Australia

Fiona Stanley Hospital

Murdoch, , Australia

Intergrated Clinical Oncology Network Pty Ltd (ICON)

South Brisbane, , Australia

AZ Klina

Brasschaat, , Belgium

UCL Hopital Saint-Luc

Brussels, , Belgium

Grand Hopital de Charleroi, site Notre Dame

Charleroi, , Belgium

CHU UCL Namur - Site Dinant

Dinant, , Belgium

UZA

Edegem, , Belgium

AZ Maria Middelares

Ghent, , Belgium

UZ Gent

Ghent, , Belgium

Jolimont

Haine-Saint-Paul, , Belgium

CHU UCL Namur - Site Sainte Elisabeth

Namur, , Belgium

Cliniques St Pierre

Ottignies, , Belgium

CHU UCL Namur - Site Godinne

Yvoir, , Belgium

Fundacao Pio XII

Barretos, , Brazil

Liga Paranaense de Combate ao Cancer

Curitiba, , Brazil

Associacao de Combate ao Cancer em Goias - Hospital de Cancer Araujo Jorge

Goiânia, , Brazil

Associacao Hospital de Caridade Ijui

Ijuí, , Brazil

Liga Norte Riograndense Contra O Cancer

Natal, , Brazil

Irmandade Santa Casa de Misericordia de Porto Alegre

Porto Alegre, , Brazil

Instituto de Medicina Integral Professor Fernando Figueira

Recife, , Brazil

Oncoclinicas Rio de Janeiro S A

Rio de Janeiro, , Brazil

Instituto D Or de Pesquisa e Ensino IDOR

Rio de Janeiro, , Brazil

Hospital Sao Rafael

Salvador, , Brazil

Fundacao Faculdade de Medicina - Instituto do Cancer do Estado de Sao Paulo

São Paulo, , Brazil

Sociedade Beneficente de Senhoras Hospital Sirio Libanes

São Paulo, , Brazil

Real e Benemerita Associacao Portuguesa de Beneficencia

São Paulo, , Brazil

Fundacao Antonio Prudente A C Camargo Cancer Center

São Paulo, , Brazil

Hospital Das Clinicas Da Faculdade De Medicina Da USP

São Paulo, , Brazil

GRAACC

São Paulo, , Brazil

Cancer Hospital Chinese Academy of Medical Sciences

Beijing, , China

Beijing Tiantan Hospital, Capital Medical University

Beijing, , China

Peking University Third Hospital

Beijing, , China

Chinese PLA General Hospital

Beijing, , China

Beijing Luhe Hospital, Capital Medical University

Beijing, , China

Jilin cancer hospital

Changchun, , China

The First Hospital of Jilin University

Changchun, , China

West China Hospital,Sichuan University

Chengdu, , China

Chongqing Southwest Hospital

Chongqing, , China

Third Military Medical University Daping Hospital Cancer Center

Chongqing, , China

Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University

Guangzhou, , China

First affiliated Hospital of Zhejiang University

Hangzhou, , China

Zhejiang Cancer Hospital

Hangzhou, , China

Eastern Theater General Hospital, Qinhuai District Medical Area

Nanjing, , China

Ruijin Hospital Shanghai Jiao Tong University

Shanghai, , China

Cancer Hospital, FuDan University

Shanghai, , China

Shanghai Zhongshan Hospital

Shanghai, , China

Huashan Hospital Fudan University

Shanghai, , China

Hopital Saint André

Bordeaux, , France

Centre Francois Baclesse

Caen, , France

Centre Jean Perrin

Clermont-Ferrand, , France

Centre Georges Francois Leclerc

Dijon, , France

Centre Oscar Lambret

Lille, , France

Centre Leon Berard

Lyon, , France

Hopital de la Timone

Marseille, , France

Institut Curie

Paris, , France

CHU De Poitiers

Poitiers, , France

Institut de Cancerologie de Lorraine

Vandœuvre-lès-Nancy, , France

Institut Gustave Roussy

Villejuif, , France

Charité

Berlin, , Germany

Universitaetsklinikum Koeln

Cologne, , Germany

Medizinische Fakultaet Carl Gustav Carus Technische Universitaet Dresden

Dresden, , Germany

Universitaetsklinikum Essen

Essen, , Germany

Universitaetsklinikum Frankfurt

Frankfurt, , Germany

Asklepios Klinik Altona

Hamburg, , Germany

Universitaetsklinikum Heidelberg

Heidelberg, , Germany

Universitaetsklinikum Leipzig

Leipzig, , Germany

Universitatsmedizin der Johannes Gutenberg Universitat Mainz

Mainz, , Germany

Klinikum rechts der Isar der TU Munchen

München, , Germany

Universitatsklinikum Tubingen

Tübingen, , Germany

Istituto Ortopedico Rizzoli

Bologna, , Italy

ASST Grande Ospedale Metropolitano Niguarda

Milan, , Italy

ASST di Monza

Monza, , Italy

Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli

Napoli, , Italy

Fondazione G Pascale Istituto Nazionale Tumori IRCCS

Napoli, , Italy

Ospedale S. Maria Della Misericordia

Perugia, , Italy

Ospedale Santa Chiara AO Universitaria Pisana

Pisa, , Italy

Azienda Ospedaliera S. Maria Terni

Terni, , Italy

Ospedale Borgo Roma

Verona, , Italy

National Cancer Center Hospital

Chūōku, , Japan

Hiroshima University Hospital

Hiroshima, , Japan

National Cancer Center Hospital East

Kashiwa, , Japan

National Hospital Organization Shikoku Cancer Center

Matsuyama, , Japan

Fujita Health University Hospital

Toyoake, , Japan

Centrum Onkologii im Prof F Lukaszczyka w Bydgoszczy

Bydgoszcz, , Poland

Przychodnia Lekarska KOMED Roman Karaszewski

Konin, , Poland

Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki w Krakowie

Krakow, , Poland

Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im M Kopernika w Lodzi

Lodz, , Poland

Centrum Medyczne Pratia Poznan

Skorzewo, , Poland

Instytut Matki i Dziecka

Warsaw, , Poland

Centralny Szpital Kliniczny MSWiA w Warszawie

Warsaw, , Poland

Narodowy Instytut Onkologii im Marii Sklodowskiej Curie Panstwowy Instytut Badawczy

Warsaw, , Poland

Severance Hospital Yonsei University Health System

Seoul, , South Korea

Asan Medical Center

Seoul, , South Korea

Samsung Medical Center

Seoul, , South Korea

The Catholic University of Korea Seoul St Marys Hospital

Seoul, , South Korea

Seoul National University Hospital

Seoul, , South Korea

Hosp. Univ. Quiron Dexeus

Barcelona, , Spain

Hosp Univ Vall D Hebron

Barcelona, , Spain

Hosp Clinic de Barcelona

Barcelona, , Spain

Hosp. Sant Joan de Deu

Barcelona, , Spain

Inst. Cat. Doncologia-H Duran I Reynals

L'Hospitalet de Llobregat, , Spain

Hosp. Infantil Univ. Nino Jesus

Madrid, , Spain

Clinica Univ. de Navarra

Madrid, , Spain

Hosp. Univ. Ramon Y Cajal

Madrid, , Spain

Hosp Univ Fund Jimenez Diaz

Madrid, , Spain

Hosp. Univ. 12 de Octubre

Madrid, , Spain

Hosp. Univ. La Paz

Madrid, , Spain

Hosp. Virgen Del Rocio

Seville, , Spain

Hosp. Clinico Univ. de Valencia

Valencia, , Spain

Changhua Christian Hospital

Changhua, , Taiwan

Kaohsiung Medical University Chung Ho Memorial Hospital

Kaohsiung City, , Taiwan

E-Da Cancer Hospital

Kaohsiung City, , Taiwan

Chang Gung Medical Foundation

Kaohsiung City, , Taiwan

China Medical University Hospital

Taichung, , Taiwan

Taichung Veterans General Hospital

Taichung, , Taiwan

Chi Mei Medical Center Yong Kang

Tainan, , Taiwan

National Cheng Kung University Hospital

Tainan, , Taiwan

Chi Mei Medical Center Liu Ying

Tainan, , Taiwan

National Taiwan University Hospital

Taipei, , Taiwan

Taipei Veterans General Hospital

Taipei, , Taiwan

Chang Gung Memorial Hospital Linkou Branch

Taoyuan, , Taiwan

Bristol Royal Hospital for Children

Bristol, , United Kingdom

Bristol Haematology and Oncology Centre

Bristol, , United Kingdom

The Christie Nhs Foundation Trust

Manchester, , United Kingdom

The Clatterbridge Cancer Centre

Metropolitan Borough of Wirral, , United Kingdom

Freeman Hospital

Newcastle upon Tyne, , United Kingdom

Derriford Hospital

Plymouth, , United Kingdom

The Royal Marsden NHS Trust

Sutton, , United Kingdom

Countries

Canada; Denmark; Sweden; United States; Argentina; Australia; Belgium; Brazil; China; France; Germany; Italy; Japan; Poland; South Korea; Spain; Taiwan; United Kingdom

References

Pant 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.

Reference Type: DERIVED

PMID: 37541273 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

42756493CAN2002

Identifier Type: OTHER

Identifier Source: secondary_id

2019-002113-19

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2023-510301-18-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

CR108661

Identifier Type: -

Identifier Source: org_study_id