Clinical trial NCT04083976: A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations
Official title: A Phase 2 Study of Erdafitinib in Subjects With Advanced Solid Tumors and FGFR Gene Alterations
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Advanced Solid Tumor
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2019-09-10
Enrollment: 316
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Broad Panel and Pediatric Cohorts: Objective Response Rate (ORR) Based on Response Assessment in Neuro-Oncology (RANO) as Assessed by Independent Review Committee (IRC)
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 4 years
measure: Core Panel Cohort: Objective Response Rate (ORR) Based on Response Assessment in Neuro-Oncology (RANO) as Assessed by Independent Review Committee (IRC)
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 4 years
Secondary outcomes:
measure: Objective Response Rate (ORR) as Assessed by Investigators Assessment
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Duration of Responses (DOR)
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Disease Control Rate (DCR)
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Clinical Benefit Rate (CBR)
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Progression-free Survival (PFS)
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Overall Survival (OS)
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Number of Participants With Adverse Events (AEs)
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Number of Participants With Adverse Events (AEs) by Severity
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Pediatric Cohort: Plasma Concentration of Erdafitinib
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30 (EORTC-QLQ-C30) for Participants >=18 Years
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Change From Baseline in Pediatric Functional Assessment Of Cancer Therapy-Brain (Peds FACT-Br)
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Change From Baseline in Patient Global Impression of Symptom Severity (PGIS)
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Change From Baseline in Patient Global Impression of Change (PGIC)
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
measure: Change From Baseline in European Quality of Life -5 Dimensions-5 Levels (EQ-5D-5L)
timeFrame: Baseline (Cycle 1 Day 1 [each cycle of 21 days]) up to 5 years 4 months
Other outcomes:
Locations:
city: Tucson
country: United States
facility: Arizona Oncology Associates, PC - HOPE
state: Arizona
city: Colorado Springs
country: United States
facility: Rocky Mountain Cancer Centers
state: Colorado
city: Hollywood
country: United States
facility: Memorial Cancer Institute
state: Florida
city: Jacksonville
country: United States
facility: Cancer Specialists of North Florida
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: 'Aiea
country: United States
facility: Hawaii Cancer Care
state: Hawaii
city: Scarborough
country: United States
facility: Maine Medical Center
state: Maine
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Detroit
country: United States
facility: Henry Ford Hospital
state: Michigan
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: New York
country: United States
facility: Mount Sinai
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Charlotte
country: United States
facility: Levine Cancer Institute, Carolinas HealthCare System
state: North Carolina
city: Cincinnati
country: United States
facility: Oncology Hematology Care
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Chattanooga
country: United States
facility: Tennessee Oncology, PLLC
state: Tennessee
city: Nashville
country: United States
facility: Tennessee Oncology
state: Tennessee
city: Dallas
country: United States
facility: Texas Oncology Baylor Charles A Sammons Cancer Center
state: Texas
city: Fort Worth
country: United States
facility: Texas Oncology-Fort Worth Cancer Center
state: Texas
city: Houston
country: United States
facility: MD Anderson
state: Texas
city: Houston
country: United States
facility: Oncology Consultants Texas
state: Texas
city: Odessa
country: United States
facility: Texas Oncology Odessa-West Texas Cancer Center
state: Texas
city: San Antonio
country: United States
facility: Texas Oncology San Antonio Northeast
state: Texas
city: Tacoma
country: United States
facility: NorthWest Medical Specialties, PLLC
state: Washington
city: Wauwatosa
country: United States
facility: Aurora Research Institute
state: Wisconsin
city: Buenos Aires
country: Argentina
facility: Hospital Aleman
city: Ciudad Autonoma de Buenos Aires
country: Argentina
facility: Centro Oncológico Korben
city: Ciudad Autonoma de Buenos Aires
country: Argentina
facility: Instituto Fleni
city: Ciudad Autonoma de Buenos Aires
country: Argentina
facility: CEMIC Saavedra
city: Ciudad Autónoma de Buenos Aires
country: Argentina
facility: Instituto de Investigaciones Metabólicas
city: Cordoba
country: Argentina
facility: Cemaic Centro Privado de Especialidades Medicas Ambulatorias e Investigacion Clinica
city: La Plata Lpl Lpl
country: Argentina
facility: Hospital Italiano de La Plata
city: Mar del Plata
country: Argentina
facility: Instituto de Investigaciones Clinicas Mar del Plata
city: Mar Del Plata
country: Argentina
facility: Hospital Privado de Comunidad
city: Pilar
country: Argentina
facility: Hospital Universitario Austral
city: Rosario
country: Argentina
facility: Sanatorio Britanico de Rosario
city: San Salvador De Jujuy
country: Argentina
facility: ARS Médica
city: Adelaide
country: Australia
facility: Flinders Centre for Innovation in Cancer
city: Camperdown
country: Australia
facility: Chris O'Brien Lifehouse
city: Darlinghurst
country: Australia
facility: St Vincent s Hospital Sydney
city: Fitzroy
country: Australia
facility: St Vincents Hospital Melbourne
city: Murdoch
country: Australia
facility: Fiona Stanley Hospital
city: South Brisbane
country: Australia
facility: Intergrated Clinical Oncology Network Pty Ltd (ICON)
city: Brasschaat
country: Belgium
facility: AZ Klina
city: Bruxelles
country: Belgium
facility: UCL Hopital Saint-Luc
city: Charleroi
country: Belgium
facility: Grand Hopital de Charleroi, site Notre Dame
city: Dinant
country: Belgium
facility: CHU UCL Namur - Site Dinant
city: Edegem
country: Belgium
facility: UZA
city: Gent
country: Belgium
facility: AZ Maria Middelares
city: Gent
country: Belgium
facility: UZ Gent
city: Haine-Saint-Paul
country: Belgium
facility: Jolimont
city: Namur
country: Belgium
facility: CHU UCL Namur - Site Sainte Elisabeth
city: Ottignies
country: Belgium
facility: Cliniques St Pierre
city: Yvoir
country: Belgium
facility: CHU UCL Namur - Site Godinne
city: Barretos
country: Brazil
facility: Fundacao Pio XII
city: Curitiba
country: Brazil
facility: Liga Paranaense de Combate ao Cancer
city: Goiania
country: Brazil
facility: Associacao de Combate ao Cancer em Goias - Hospital de Cancer Araujo Jorge
city: Ijui
country: Brazil
facility: Associacao Hospital de Caridade Ijui
city: Natal
country: Brazil
facility: Liga Norte Riograndense Contra O Cancer
city: Porto Alegre
country: Brazil
facility: Irmandade Santa Casa de Misericordia de Porto Alegre
city: Recife
country: Brazil
facility: Instituto de Medicina Integral Professor Fernando Figueira
city: Rio de Janeiro
country: Brazil
facility: Oncoclinicas Rio de Janeiro S A
city: Rio de Janeiro
country: Brazil
facility: Instituto D Or de Pesquisa e Ensino IDOR
city: Salvador
country: Brazil
facility: Hospital Sao Rafael
city: Sao Paulo
country: Brazil
facility: Fundacao Faculdade de Medicina - Instituto do Cancer do Estado de Sao Paulo
city: Sao Paulo
country: Brazil
facility: Fundacao Antonio Prudente A C Camargo Cancer Center
city: São Paulo
country: Brazil
facility: Sociedade Beneficente de Senhoras Hospital Sirio Libanes
city: São Paulo
country: Brazil
facility: Real e Benemerita Associacao Portuguesa de Beneficencia
city: São Paulo
country: Brazil
facility: Hospital Das Clinicas Da Faculdade De Medicina Da USP
city: São Paulo
country: Brazil
facility: GRAACC
city: Beijing
country: China
facility: Cancer Hospital Chinese Academy of Medical Sciences
city: Beijing
country: China
facility: Beijing Tiantan Hospital, Capital Medical University
city: Beijing
country: China
facility: Peking University Third Hospital
city: Beijing
country: China
facility: Chinese PLA General Hospital
city: Beijing
country: China
facility: Beijing Luhe Hospital, Capital Medical University
city: Changchun
country: China
facility: Jilin cancer hospital
city: Changchun
country: China
facility: The First Hospital of Jilin University
city: Chengdu
country: China
facility: West China Hospital,Sichuan University
city: Chongqing
country: China
facility: Chongqing Southwest Hospital
city: Chongqing
country: China
facility: Third Military Medical University Daping Hospital Cancer Center
city: Guangzhou
country: China
facility: Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University
city: Hangzhou
country: China
facility: First affiliated Hospital of Zhejiang University
city: Hangzhou
country: China
facility: Zhejiang Cancer Hospital
city: Nanjing
country: China
facility: Eastern Theater General Hospital, Qinhuai District Medical Area
city: Shanghai
country: China
facility: Ruijin Hospital Shanghai Jiao Tong University
city: Shanghai
country: China
facility: Cancer Hospital, FuDan University
city: Shanghai
country: China
facility: Shanghai Zhongshan Hospital
city: Shanghai
country: China
facility: Huashan Hospital Fudan University
city: Bordeaux
country: France
facility: Hopital Saint André
city: Caen
country: France
facility: Centre Francois Baclesse
city: Clermont Ferrand
country: France
facility: Centre Jean Perrin
city: Dijon
country: France
facility: Centre Georges Francois Leclerc
city: Lille
country: France
facility: Centre Oscar Lambret
city: Lyon
country: France
facility: Centre Leon Berard
city: Marseille
country: France
facility: Hopital de la Timone
city: Paris
country: France
facility: Institut Curie
city: Poitiers
country: France
facility: CHU De Poitiers
city: Vandoeuvre lès Nancy
country: France
facility: Institut de Cancerologie de Lorraine
city: Villejuif
country: France
facility: Institut Gustave Roussy
city: Berlin
country: Germany
facility: Charité
city: Dresden
country: Germany
facility: Medizinische Fakultaet Carl Gustav Carus Technische Universitaet Dresden
city: Essen
country: Germany
facility: Universitaetsklinikum Essen
city: Frankfurt
country: Germany
facility: Universitaetsklinikum Frankfurt
city: Hamburg
country: Germany
facility: Asklepios Klinik Altona
city: Heidelberg
country: Germany
facility: Universitaetsklinikum Heidelberg
city: Köln
country: Germany
facility: Universitaetsklinikum Koeln
city: Leipzig
country: Germany
facility: Universitaetsklinikum Leipzig
city: Mainz
country: Germany
facility: Universitatsmedizin der Johannes Gutenberg Universitat Mainz
city: München
country: Germany
facility: Klinikum rechts der Isar der TU Munchen
city: Tübingen
country: Germany
facility: Universitatsklinikum Tubingen
city: Bologna
country: Italy
facility: Istituto Ortopedico Rizzoli
city: Milano
country: Italy
facility: ASST Grande Ospedale Metropolitano Niguarda
city: Monza
country: Italy
facility: ASST di Monza
city: Napoli
country: Italy
facility: Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
city: Napoli
country: Italy
facility: Fondazione G Pascale Istituto Nazionale Tumori IRCCS
city: Perugia
country: Italy
facility: Ospedale S. Maria Della Misericordia
city: Pisa
country: Italy
facility: Ospedale Santa Chiara AO Universitaria Pisana
city: Terni
country: Italy
facility: Azienda Ospedaliera S. Maria Terni
city: Verona
country: Italy
facility: Ospedale Borgo Roma
city: Chuo Ku
country: Japan
facility: National Cancer Center Hospital
city: Hiroshima shi
country: Japan
facility: Hiroshima University Hospital
city: Kashiwa
country: Japan
facility: National Cancer Center Hospital East
city: Matsuyama
country: Japan
facility: National Hospital Organization Shikoku Cancer Center
city: Toyoake
country: Japan
facility: Fujita Health University Hospital
city: Seoul
country: Korea, Republic of
facility: Severance Hospital Yonsei University Health System
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: The Catholic University of Korea Seoul St Marys Hospital
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Bydgoszcz
country: Poland
facility: Centrum Onkologii im Prof F Lukaszczyka w Bydgoszczy
city: Konin
country: Poland
facility: Przychodnia Lekarska KOMED Roman Karaszewski
city: Krakow
country: Poland
facility: Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki w Krakowie
city: Lodz
country: Poland
facility: Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im M Kopernika w Lodzi
city: Skorzewo
country: Poland
facility: Centrum Medyczne Pratia Poznan
city: Warszawa
country: Poland
facility: Instytut Matki i Dziecka
city: Warszawa
country: Poland
facility: Centralny Szpital Kliniczny MSWiA w Warszawie
city: Warszawa
country: Poland
facility: Narodowy Instytut Onkologii im Marii Sklodowskiej Curie Panstwowy Instytut Badawczy
city: Barcelona
country: Spain
facility: Hosp. Univ. Quiron Dexeus
city: Barcelona
country: Spain
facility: Hosp Univ Vall D Hebron
city: Barcelona
country: Spain
facility: Hosp Clinic de Barcelona
city: Barcelona
country: Spain
facility: Hosp. Sant Joan de Deu
city: Hospitalet de Llobregat
country: Spain
facility: Inst. Cat. Doncologia-H Duran I Reynals
city: Madrid
country: Spain
facility: Hosp. Infantil Univ. Nino Jesus
city: Madrid
country: Spain
facility: Clinica Univ. de Navarra
city: Madrid
country: Spain
facility: Hosp. Univ. Ramon Y Cajal
city: Madrid
country: Spain
facility: Hosp Univ Fund Jimenez Diaz
city: Madrid
country: Spain
facility: Hosp. Univ. 12 de Octubre
city: Madrid
country: Spain
facility: Hosp. Univ. La Paz
city: Sevilla
country: Spain
facility: Hosp. Virgen Del Rocio
city: Valencia
country: Spain
facility: Hosp. Clinico Univ. de Valencia
city: Changhua
country: Taiwan
facility: Changhua Christian Hospital
city: Kaohsiung City
country: Taiwan
facility: E-Da Cancer Hospital
city: Kaohsiung
country: Taiwan
facility: Kaohsiung Medical University Chung Ho Memorial Hospital
city: Kaohsiung
country: Taiwan
facility: Chang Gung Medical Foundation
city: Taichung
country: Taiwan
facility: China Medical University Hospital
city: Taichung
country: Taiwan
facility: Taichung Veterans General Hospital
city: Tainan
country: Taiwan
facility: Chi Mei Medical Center Yong Kang
city: Tainan
country: Taiwan
facility: National Cheng Kung University Hospital
city: Tainan
country: Taiwan
facility: Chi Mei Medical Center Liu Ying
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Taipei
country: Taiwan
facility: Taipei Veterans General Hospital
city: Taoyuan City
country: Taiwan
facility: Chang Gung Memorial Hospital Linkou Branch
city: Bristol
country: United Kingdom
facility: Bristol Royal Hospital for Children
city: Bristol
country: United Kingdom
facility: Bristol Haematology and Oncology Centre
city: Manchester
country: United Kingdom
facility: The Christie Nhs Foundation Trust
city: Newcastle upon Tyne
country: United Kingdom
facility: Freeman Hospital
city: Plymouth
country: United Kingdom
facility: Derriford Hospital
city: Sutton
country: United Kingdom
facility: The Royal Marsden NHS Trust
city: Wirral
country: United Kingdom
facility: The Clatterbridge Cancer Centre
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: