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

NCT04083976 | Completed Phase 2 Results FDA Drug

• 4 other identifiers: CR10866142756493CAN20022019-002113-192023-510301-18-00
• Study Type: interventional
• Target: 316
• Locations: 15 countries
• Sites: 179

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.

Conditions

Advanced Solid Tumor

Outcome Measures

Primary Outcomes (2)

• 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.

  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).

  Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 4 years

Secondary Outcomes (14)

• Objective Response Rate (ORR) as Assessed by Investigators Assessment

  Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months

• Duration of Responses (DOR)

  Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months

• Disease Control Rate (DCR)

  Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months

• Clinical Benefit Rate (CBR)

  Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months

• Progression-free Survival (PFS)

  Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months

Study Arms (1)

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.

Drug: Erdafitinib

Interventions

Erdafitinib DRUG

Participants will receive erdafitinib oral tablets.

Also known as: JNJ-42756493

Erdafitinib

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (179)

Arizona Oncology Associates, PC - HOPE

Tucson, Arizona, 85711, United States

Location

Rocky Mountain Cancer Centers

Colorado Springs, Colorado, 80907, United States

Location

Memorial Cancer Institute

Hollywood, Florida, 33021, United States

Location

Cancer Specialists of North Florida

Jacksonville, Florida, 32256, United States

Location

Emory University

Atlanta, Georgia, 30022, United States

Location

Hawaii Cancer Care

‘Aiea, Hawaii, 96701, United States

Location

Maine Medical Center

Scarborough, Maine, 04074, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

Henry Ford Hospital

Detroit, Michigan, 48202, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Mount Sinai

New York, New York, 10029, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10128, United States

Location

Levine Cancer Institute, Carolinas HealthCare System

Charlotte, North Carolina, 28204, United States

Location

Oncology Hematology Care

Cincinnati, Ohio, 45242, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15232, United States

Location

Tennessee Oncology, PLLC

Chattanooga, Tennessee, 37404, United States

Location

Tennessee Oncology

Nashville, Tennessee, 37203, United States

Location

Texas Oncology Baylor Charles A Sammons Cancer Center

Dallas, Texas, 75246, United States

Location

Texas Oncology-Fort Worth Cancer Center

Fort Worth, Texas, 76104, United States

Location

MD Anderson

Houston, Texas, 77030, United States

Location

Oncology Consultants Texas

Houston, Texas, 77030, United States

Location

Texas Oncology Odessa-West Texas Cancer Center

Odessa, Texas, 79761, United States

Location

Texas Oncology San Antonio Northeast

San Antonio, Texas, 78217, United States

Location

NorthWest Medical Specialties, PLLC

Tacoma, Washington, 98405, United States

Location

Aurora Research Institute

Wauwatosa, Wisconsin, 53226, United States

Location

Instituto de Investigaciones Metabólicas

Buenos Aires, C1012AAR, Argentina

Location

Hospital Aleman

Buenos Aires, C1118AAT, Argentina

Location

Centro Oncológico Korben

Buenos Aires, C1426AGE, Argentina

Location

Instituto Fleni

Buenos Aires, C1428AQK, Argentina

Location

CEMIC Saavedra

Buenos Aires, C1431FWN, Argentina

Location

Cemaic Centro Privado de Especialidades Medicas Ambulatorias e Investigacion Clinica

Córdoba, 5000, Argentina

Location

Hospital Italiano de La Plata

La Plata Lpl Lpl, 1900, Argentina

Location

Instituto de Investigaciones Clinicas Mar del Plata

Mar del Plata, B7600FZN, Argentina

Location

Hospital Privado de Comunidad

Mar del Plata, B7602CBM, Argentina

Location

Hospital Universitario Austral

Pilar, 1629, Argentina

Location

Sanatorio Britanico de Rosario

Rosario, 2000, Argentina

Location

ARS Médica

San Salvador de Jujuy, Y4600AFW, Argentina

Location

Flinders Centre for Innovation in Cancer

Adelaide, 5042, Australia

Location

Chris O'Brien Lifehouse

Camperdown, 2050, Australia

Location

St Vincent s Hospital Sydney

Darlinghurst, 2010, Australia

Location

St Vincents Hospital Melbourne

Fitzroy, 3065, Australia

Location

Fiona Stanley Hospital

Murdoch, 6150, Australia

Location

Intergrated Clinical Oncology Network Pty Ltd (ICON)

South Brisbane, 4101, Australia

Location

AZ Klina

Brasschaat, 2930, Belgium

Location

UCL Hopital Saint-Luc

Brussels, 1200, Belgium

Location

Grand Hopital de Charleroi, site Notre Dame

Charleroi, 6000, Belgium

Location

CHU UCL Namur - Site Dinant

Dinant, 5500, Belgium

Location

UZA

Edegem, 2650, Belgium

Location

AZ Maria Middelares

Ghent, 9000, Belgium

Location

UZ Gent

Ghent, 9000, Belgium

Location

Jolimont

Haine-Saint-Paul, 7100, Belgium

Location

CHU UCL Namur - Site Sainte Elisabeth

Namur, 5000, Belgium

Location

Cliniques St Pierre

Ottignies, 1340, Belgium

Location

CHU UCL Namur - Site Godinne

Yvoir, 5530, Belgium

Location

Fundacao Pio XII

Barretos, 14784-400, Brazil

Location

Liga Paranaense de Combate ao Cancer

Curitiba, 81520 060, Brazil

Location

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

Goiânia, 74605-070, Brazil

Location

Associacao Hospital de Caridade Ijui

Ijuí, 98700-000, Brazil

Location

Liga Norte Riograndense Contra O Cancer

Natal, 59075-740, Brazil

Location

Irmandade Santa Casa de Misericordia de Porto Alegre

Porto Alegre, 90050-170, Brazil

Location

Instituto de Medicina Integral Professor Fernando Figueira

Recife, 50070-550, Brazil

Location

Oncoclinicas Rio de Janeiro S A

Rio de Janeiro, 22250 905, Brazil

Location

Instituto D Or de Pesquisa e Ensino IDOR

Rio de Janeiro, 22281 100, Brazil

Location

Hospital Sao Rafael

Salvador, 41253 190, Brazil

Location

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

São Paulo, 01246 000, Brazil

Location

Sociedade Beneficente de Senhoras Hospital Sirio Libanes

São Paulo, 01308 901, Brazil

Location

Real e Benemerita Associacao Portuguesa de Beneficencia

São Paulo, 01321-001, Brazil

Location

Fundacao Antonio Prudente A C Camargo Cancer Center

São Paulo, 01509 900, Brazil

Location

Hospital Das Clinicas Da Faculdade De Medicina Da USP

São Paulo, 05410-030, Brazil

Location

GRAACC

São Paulo, 4039001, Brazil

Location

Cancer Hospital Chinese Academy of Medical Sciences

Beijing, 100021, China

Location

Beijing Tiantan Hospital, Capital Medical University

Beijing, 100070, China

Location

Peking University Third Hospital

Beijing, 100191, China

Location

Chinese PLA General Hospital

Beijing, 100853, China

Location

Beijing Luhe Hospital, Capital Medical University

Beijing, 101100, China

Location

Jilin cancer hospital

Changchun, 130012, China

Location

The First Hospital of Jilin University

Changchun, 130021, China

Location

West China Hospital,Sichuan University

Chengdu, 610041, China

Location

Chongqing Southwest Hospital

Chongqing, 400038, China

Location

Third Military Medical University Daping Hospital Cancer Center

Chongqing, 400042, China

Location

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

Guangzhou, 510289, China

Location

First affiliated Hospital of Zhejiang University

Hangzhou, 310003, China

Location

Zhejiang Cancer Hospital

Hangzhou, 310022, China

Location

Eastern Theater General Hospital, Qinhuai District Medical Area

Nanjing, 210002, China

Location

Ruijin Hospital Shanghai Jiao Tong University

Shanghai, 200025, China

Location

Cancer Hospital, FuDan University

Shanghai, 200032, China

Location

Shanghai Zhongshan Hospital

Shanghai, 200032, China

Location

Huashan Hospital Fudan University

Shanghai, 200040, China

Location

Hopital Saint André

Bordeaux, 33075, France

Location

Centre Francois Baclesse

Caen, 14000, France

Location

Centre Jean Perrin

Clermont-Ferrand, 63011, France

Location

Centre Georges Francois Leclerc

Dijon, 21000, France

Location

Centre Oscar Lambret

Lille, 59000, France

Location

Centre Leon Berard

Lyon, 69008, France

Location

Hopital de la Timone

Marseille, 130005, France

Location

Institut Curie

Paris, 75005, France

Location

CHU De Poitiers

Poitiers, 86021, France

Location

Institut de Cancerologie de Lorraine

Vandœuvre-lès-Nancy, 54519, France

Location

Institut Gustave Roussy

Villejuif, 94800, France

Location

Charité

Berlin, 10115, Germany

Location

Universitaetsklinikum Koeln

Cologne, 50937, Germany

Location

Medizinische Fakultaet Carl Gustav Carus Technische Universitaet Dresden

Dresden, 01307, Germany

Location

Universitaetsklinikum Essen

Essen, 45147, Germany

Location

Universitaetsklinikum Frankfurt

Frankfurt, 60385, Germany

Location

Asklepios Klinik Altona

Hamburg, 22763, Germany

Location

Universitaetsklinikum Heidelberg 1

Heidelberg, 69120, Germany

Location

Universitaetsklinikum Heidelberg

Heidelberg, 69120, Germany

Location

Universitaetsklinikum Leipzig

Leipzig, 04103, Germany

Location

Universitatsmedizin der Johannes Gutenberg Universitat Mainz

Mainz, 55131, Germany

Location

Klinikum rechts der Isar der TU Munchen

München, 81675, Germany

Location

Universitatsklinikum Tubingen

Tübingen, 72076, Germany

Location

Istituto Ortopedico Rizzoli

Bologna, 40136, Italy

Location

ASST Grande Ospedale Metropolitano Niguarda

Milan, 20162, Italy

Location

ASST di Monza

Monza, 20900, Italy

Location

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

Naples, 80138, Italy

Location

Fondazione G Pascale Istituto Nazionale Tumori IRCCS

Naples, 80138, Italy

Location

Ospedale S. Maria Della Misericordia

Perugia, 06132, Italy

Location

Ospedale Santa Chiara AO Universitaria Pisana

Pisa, 56126, Italy

Location

Azienda Ospedaliera S. Maria Terni

Terni, 5100, Italy

Location

Ospedale Borgo Roma

Verona, 37134, Italy

Location

National Cancer Center Hospital

Chūōku, 104 0045, Japan

Location

Hiroshima University Hospital

Hiroshima, 734 8551, Japan

Location

National Cancer Center Hospital East

Kashiwa, 277 8577, Japan

Location

National Hospital Organization Shikoku Cancer Center

Matsuyama, 791-0280, Japan

Location

Fujita Health University Hospital

Toyoake, 470-1192, Japan

Location

Centrum Onkologii im Prof F Lukaszczyka w Bydgoszczy

Bydgoszcz, 85 796, Poland

Location

Przychodnia Lekarska KOMED Roman Karaszewski

Konin, 62-500, Poland

Location

Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki w Krakowie

Krakow, 31 501, Poland

Location

Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im M Kopernika w Lodzi

Lodz, 93 513, Poland

Location

Centrum Medyczne Pratia Poznan

Skorzewo, 60 185, Poland

Location

Instytut Matki i Dziecka

Warsaw, 01 211, Poland

Location

Centralny Szpital Kliniczny MSWiA w Warszawie

Warsaw, 02-507, Poland

Location

Narodowy Instytut Onkologii im Marii Sklodowskiej Curie Panstwowy Instytut Badawczy

Warsaw, 02-781, Poland

Location

Severance Hospital Yonsei University Health System 1

Seoul, 03722, South Korea

Location

Severance Hospital Yonsei University Health System 2

Seoul, 03722, South Korea

Location

Severance Hospital Yonsei University Health System

Seoul, 03722, South Korea

Location

Asan Medical Center 1

Seoul, 05505, South Korea

Location

Asan Medical Center 2

Seoul, 05505, South Korea

Location

Asan Medical Center

Seoul, 05505, South Korea

Location

Samsung Medical Center 1

Seoul, 06351, South Korea

Location

Samsung Medical Center 2

Seoul, 06351, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

The Catholic University of Korea Seoul St Marys Hospital 1

Seoul, 06591, South Korea

Location

The Catholic University of Korea Seoul St Marys Hospital

Seoul, 06591, South Korea

Location

Seoul National University Hospital

Seoul, 3080, South Korea

Location

Hosp. Univ. Quiron Dexeus

Barcelona, 08028, Spain

Location

Hosp Univ Vall D Hebron

Barcelona, 08035, Spain

Location

Hosp Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hosp. Sant Joan de Deu

Barcelona, 08950, Spain

Location

Inst. Cat. Doncologia-H Duran I Reynals

L'Hospitalet de Llobregat, 08908, Spain

Location

Hosp. Infantil Univ. Nino Jesus

Madrid, 28009, Spain

Location

Clinica Univ. de Navarra

Madrid, 28027, Spain

Location

Hosp. Univ. Ramon Y Cajal

Madrid, 28034, Spain

Location

Hosp Univ Fund Jimenez Diaz

Madrid, 28040, Spain

Location

Hosp. Univ. 12 de Octubre

Madrid, 28041, Spain

Location

Hosp. Univ. La Paz

Madrid, 28046, Spain

Location

Hosp. Virgen Del Rocio

Seville, 41013, Spain

Location

Hosp. Clinico Univ. de Valencia

Valencia, 46010, Spain

Location

Changhua Christian Hospital

Changhua, 50006, Taiwan

Location

Kaohsiung Medical University Chung Ho Memorial Hospital

Kaohsiung City, 807, Taiwan

Location

E-Da Cancer Hospital

Kaohsiung City, 82445, Taiwan

Location

Chang Gung Medical Foundation

Kaohsiung City, 833, Taiwan

Location

China Medical University Hospital

Taichung, 403, Taiwan

Location

Taichung Veterans General Hospital

Taichung, 40705, Taiwan

Location

Chi Mei Medical Center Yong Kang

Tainan, 710, Taiwan

Location

National Cheng Kung University Hospital

Tainan, 710, Taiwan

Location

Chi Mei Medical Center Liu Ying

Tainan, 736, Taiwan

Location

National Taiwan University Hospital

Taipei, 10002, Taiwan

Location

Taipei Veterans General Hospital

Taipei, 11217, Taiwan

Location

Chang Gung Memorial Hospital Linkou Branch

Taoyuan City, 333, Taiwan

Location

Bristol Royal Hospital for Children

Bristol, BS2 8AE, United Kingdom

Location

Bristol Haematology and Oncology Centre

Bristol, BS2 8ED, United Kingdom

Location

The Christie Nhs Foundation Trust

Manchester, M20 4BX, United Kingdom

Location

The Clatterbridge Cancer Centre

Metropolitan Borough of Wirral, CH63 4JY, United Kingdom

Location

Freeman Hospital

Newcastle upon Tyne, NE7 7DN, United Kingdom

Location

Derriford Hospital

Plymouth, PL6 8DH, United Kingdom

Location

The Royal Marsden NHS Trust

Sutton, SM2 5PT, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 37541273 DERIVED

MeSH Terms

Interventions

erdafitinib

Results Point of Contact

• Title: Executive Medical Director Oncology
• Organization: Janssen Research & Development, LLC

ClinicalTrialDisclosure@its.jnj.com

844-434-4210

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 6, 2019
• First Posted: September 10, 2019
• Study Start: November 20, 2019
• Primary Completion: December 4, 2023
• Study Completion: February 28, 2026
• Last Updated: May 4, 2026
• Results First Posted: February 24, 2025

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

US (27); TW (12); GB (7); CN (18); AR (12); ES (13); FR (11); AU (6); JP (5); KR (12); PL (8); IT (9); BE (11); DE (12); BR (16)