A Study of Erdafitinib in Participants With Advanced Soli... | NCT04083976

ID	Type	Description
42756493CAN2002	Other Identifier	Janssen Research & Development, LLC
2019-002113-19	EudraCT Number
2023-510301-18-00	Registry Identifier	EUCT number

Label	Type	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
Erdafitinib	Drug	Participants will receive erdafitinib oral tablets.	Erdafitinib

Measure	Description	Time Frame
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

Measure	Description	Time Frame
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

Name	Affiliation	Role
Janssen Research & Development, LLC Clinical Trial	Janssen Research & Development, LLC	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Arizona Oncology Associates, PC - HOPE		Tucson	Arizona	85711	United States
Rocky Mountain Cancer Centers

PubMed Identifier	Type	Citation	Retractions
37541273	Derived	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.

ID	Title	Description
FG000	Broad Panel Cohort	Adolescent and adult participants with target fibroblast growth factor receptor (FGFR) mutations or any FGFR gene fusions, were enrolled in this cohort. Adolescent participants aged greater than or equal to (>=)12 to less than (<)15 years received erdafitinib 5 milligrams (mg) once daily orally, with possible up-titration to 6 mg or further to 8 mg based on Cycle 1 Day 14 and Cycle 2 Day 7 (each cycle of 21 days) serum phosphate levels. Adolescent participants aged >=15 to <18 years and adults participants aged 18 years and older received erdafitinib 8 mg once daily orally, with possible up-titration to 9 mg or maintenance at 8 mg daily based on Cycle 1 Day 14 serum phosphate levels.

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Feb 22, 2023	Dec 3, 2024

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Primary

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.

The treated population consisted of all participants in broad panel and pediatric cohorts who received at least 1 dose of study drug.

Posted

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95% Confidence Interval

Percentage of participants

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

ID	Title	Description
OG000	Broad Panel Cohort	Adolescent and adult participants with target fibroblast growth factor receptor (FGFR) mutations or any FGFR gene fusions, were enrolled in this cohort. Adolescent participants aged greater than or equal to (>=)12 to less than (<)15 years received erdafitinib 5 milligrams (mg) once daily orally, with possible up-titration to 6 mg or further to 8 mg based on Cycle 1 Day 14 and Cycle 2 Day 7 (each cycle of 21 days) serum phosphate levels. Adolescent participants aged >=15 to <18 years and adults participants aged 18 years and older received erdafitinib 8 mg once daily orally, with possible up-titration to 9 mg or maintenance at 8 mg daily based on Cycle 1 Day 14 serum phosphate levels.
OG001	Pediatric Cohort	Children and adolescent (from Broad Panel cohort) participants with locally advanced or metastatic solid tumors harboring FGFR alterations, any gene fusions or FGFR internal tandem duplication who had either progressed on prior therapies and who had no acceptable standard therapies, or who had a newly diagnosed solid tumor and who had no acceptable standard therapies, were enrolled in this cohort. Children aged >=6 to <12 years received erdafitinib 3 mg once daily orally, with possible up-titration to 4 mg or further to 5 mg based on Cycle 1 Day 14 and Cycle 2 Day 7 (each cycle of 21 days) serum phosphate levels. Adolescent participants aged >=12 to <15 years received erdafitinib 5 mg once daily orally, with possible up-titration to 6 mg or further to 8 mg based on Cycle 1 Day 14 and Cycle 2 Day 7 serum phosphate levels, Adolescent participants aged >=15 to <18 years received erdafitinib 8 mg once daily orally, with possible up-titration to 9 mg or maintenance at 8 mg daily based on Cycle 1 Day 14 serum phosphate levels.

Units	Counts
Participants	OG000217 OG0013

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Title	Measurements
	OG00029.5(23.5 to 36.0) OG00166.7(9.4 to 99.2)

Primary

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).

The treated population (core panel) consisted of a subgroup of participants in the broad panel cohort (fibroblast growth factor receptor [FGFR+]) with a select panel of pre-specified FGFR markers who received at least 1 dose of study drug.

Posted

Number

95% Confidence Interval

Percentage of participants

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

ID	Title	Description
OG000	Core Panel Cohort	Core panel cohort is a sub-group of broad panel cohort. Adolescent and adult participants with target FGFR mutations or any FGFR gene fusions, were enrolled in this cohort. Adolescent participants aged >=12 to <15 years received erdafitinib 5 mg once daily orally, with possible up-titration to 6 mg or further to 8 mg based on Cycle 1 Day 14 and Cycle 2 Day 7 serum phosphate levels. Adolescent participants aged >=15 to <18 years and adults participants aged 18 years and older received erdafitinib 8 mg once daily orally, with possible up-titration to 9 mg or maintenance at 8 mg daily based on Cycle 1 Day 14 serum phosphate levels.

Secondary

Objective Response Rate (ORR) as Assessed by Investigators Assessment

Not Posted

Mar 2026