Trial Outcomes & Findings for A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations (NCT NCT04083976)

Last Updated: 2025-11-12

Results Overview

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.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

316 participants

Primary outcome timeframe

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

Results posted on

2025-11-12

Participant Flow

As pre-specified in the protocol, adolescent participants enrolled in the Broad Panel Cohort who met the pediatric cohort definition were planned to be analyzed as part of the Broad Panel Cohort and the Pediatric Cohort. 2 adolescent participants from the broad panel cohort were included in the pediatric cohort. Hence, these 2 participants are counted as enrolled twice (once in Broad Panel Cohort and once in Pediatric Cohort).

Participant milestones

Participant milestones
Measure	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.	Cholangiocarcinoma (CCA) Expansion Cohort Adolescent and adult participants with target FGFR mutations or any FGFR gene fusion once the broad panel cohort has reached the cap of approximately 30 participants for cholangiocarcinoma, 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 (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.	Exploratory Cohort Adolescent and adult participants with any other FGFR mutations that are not captured in the broad panel cohort, 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 (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.	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.
Overall Study STARTED	217	35	53	11
Overall Study Adolescent Participants From Broad Panel Cohort	0	0	0	2
Overall Study Core Panel Cohort	109	0	0	0
Overall Study COMPLETED	183	34	43	3
Overall Study NOT COMPLETED	34	1	10	8

Reasons for withdrawal

Reasons for withdrawal
Measure	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.	Cholangiocarcinoma (CCA) Expansion Cohort Adolescent and adult participants with target FGFR mutations or any FGFR gene fusion once the broad panel cohort has reached the cap of approximately 30 participants for cholangiocarcinoma, 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 (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.	Exploratory Cohort Adolescent and adult participants with any other FGFR mutations that are not captured in the broad panel cohort, 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 (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.	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.
Overall Study Lost to Follow-up	3	0	0	1
Overall Study Withdrawal by Subject	16	1	9	0
Overall Study Sponsor Decision	14	0	0	7
Overall Study Other	1	0	1	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Broad Panel Cohort n=217 Participants 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.	Cholangiocarcinoma (CCA) Expansion Cohort n=35 Participants Adolescent and adult participants with target FGFR mutations or any FGFR gene fusion once the broad panel cohort has reached the cap of approximately 30 participants for cholangiocarcinoma, 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 (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.	Exploratory Cohort n=53 Participants Adolescent and adult participants with any other FGFR mutations that are not captured in the broad panel cohort, 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 (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.	Pediatric Cohort n=11 Participants 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.	Total n=316 Participants Total of all reporting groups
Ethnicity (NIH/OMB) Hispanic or Latino	9 Participants n=10 Participants	1 Participants n=10 Participants	0 Participants n=20 Participants	5 Participants n=45 Participants	15 Participants n=44 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	173 Participants n=10 Participants	27 Participants n=10 Participants	52 Participants n=20 Participants	4 Participants n=45 Participants	256 Participants n=44 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	35 Participants n=10 Participants	7 Participants n=10 Participants	1 Participants n=20 Participants	2 Participants n=45 Participants	45 Participants n=44 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=20 Participants	0 Participants n=45 Participants	1 Participants n=44 Participants
Age, Customized 6 years to <12 years	0 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=20 Participants	4 Participants n=45 Participants	4 Participants n=44 Participants
Age, Customized 12 years to <18 years	2 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=20 Participants	7 Participants n=45 Participants	9 Participants n=44 Participants
Age, Customized 18 years to <65 years	162 Participants n=10 Participants	25 Participants n=10 Participants	31 Participants n=20 Participants	0 Participants n=45 Participants	218 Participants n=44 Participants
Age, Customized 65 years and over	53 Participants n=10 Participants	10 Participants n=10 Participants	22 Participants n=20 Participants	0 Participants n=45 Participants	85 Participants n=44 Participants
Sex: Female, Male Female	97 Participants n=10 Participants	23 Participants n=10 Participants	32 Participants n=20 Participants	7 Participants n=45 Participants	159 Participants n=44 Participants
Sex: Female, Male Male	120 Participants n=10 Participants	12 Participants n=10 Participants	21 Participants n=20 Participants	4 Participants n=45 Participants	157 Participants n=44 Participants
Race (NIH/OMB) Asian	57 Participants n=10 Participants	11 Participants n=10 Participants	24 Participants n=20 Participants	2 Participants n=45 Participants	94 Participants n=44 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	2 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=20 Participants	0 Participants n=45 Participants	2 Participants n=44 Participants
Race (NIH/OMB) Black or African American	6 Participants n=10 Participants	0 Participants n=10 Participants	4 Participants n=20 Participants	2 Participants n=45 Participants	12 Participants n=44 Participants
Race (NIH/OMB) White	112 Participants n=10 Participants	18 Participants n=10 Participants	25 Participants n=20 Participants	5 Participants n=45 Participants	160 Participants n=44 Participants
Race (NIH/OMB) More than one race	1 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=20 Participants	0 Participants n=45 Participants	1 Participants n=44 Participants
Race (NIH/OMB) Unknown or Not Reported	38 Participants n=10 Participants	6 Participants n=10 Participants	0 Participants n=20 Participants	2 Participants n=45 Participants	46 Participants n=44 Participants
Region of Enrollment ARGENTINA	2 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=20 Participants	4 Participants n=45 Participants	6 Participants n=44 Participants
Region of Enrollment AUSTRALIA	12 Participants n=10 Participants	2 Participants n=10 Participants	4 Participants n=20 Participants	0 Participants n=45 Participants	18 Participants n=44 Participants
Region of Enrollment BELGIUM	10 Participants n=10 Participants	5 Participants n=10 Participants	7 Participants n=20 Participants	0 Participants n=45 Participants	22 Participants n=44 Participants
Region of Enrollment BRAZIL	8 Participants n=10 Participants	1 Participants n=10 Participants	0 Participants n=20 Participants	1 Participants n=45 Participants	10 Participants n=44 Participants
Region of Enrollment CHINA	12 Participants n=10 Participants	3 Participants n=10 Participants	0 Participants n=20 Participants	0 Participants n=45 Participants	15 Participants n=44 Participants
Region of Enrollment FRANCE	26 Participants n=10 Participants	5 Participants n=10 Participants	0 Participants n=20 Participants	1 Participants n=45 Participants	32 Participants n=44 Participants
Region of Enrollment GERMANY	22 Participants n=10 Participants	4 Participants n=10 Participants	4 Participants n=20 Participants	0 Participants n=45 Participants	30 Participants n=44 Participants
Region of Enrollment ITALY	3 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=20 Participants	0 Participants n=45 Participants	3 Participants n=44 Participants
Region of Enrollment JAPAN	23 Participants n=10 Participants	3 Participants n=10 Participants	2 Participants n=20 Participants	2 Participants n=45 Participants	30 Participants n=44 Participants
Region of Enrollment POLAND	3 Participants n=10 Participants	0 Participants n=10 Participants	3 Participants n=20 Participants	0 Participants n=45 Participants	6 Participants n=44 Participants
Region of Enrollment SOUTH KOREA	6 Participants n=10 Participants	1 Participants n=10 Participants	7 Participants n=20 Participants	0 Participants n=45 Participants	14 Participants n=44 Participants
Region of Enrollment SPAIN	16 Participants n=10 Participants	3 Participants n=10 Participants	6 Participants n=20 Participants	1 Participants n=45 Participants	26 Participants n=44 Participants
Region of Enrollment TAIWAN	12 Participants n=10 Participants	4 Participants n=10 Participants	15 Participants n=20 Participants	0 Participants n=45 Participants	31 Participants n=44 Participants
Region of Enrollment UNITED KINGDOM	14 Participants n=10 Participants	1 Participants n=10 Participants	4 Participants n=20 Participants	0 Participants n=45 Participants	19 Participants n=44 Participants
Region of Enrollment UNITED STATES	48 Participants n=10 Participants	3 Participants n=10 Participants	1 Participants n=20 Participants	2 Participants n=45 Participants	54 Participants n=44 Participants
AgeContinuous	55.6 years STANDARD_DEVIATION 13.51 • n=10 Participants	57 years STANDARD_DEVIATION 9.94 • n=10 Participants	60 years STANDARD_DEVIATION 11.74 • n=20 Participants	12.4 years STANDARD_DEVIATION 2.98 • n=45 Participants	55 years STANDARD_DEVIATION 15.07 • n=44 Participants

PRIMARY outcome

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

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

Outcome measures

Outcome measures
Measure	Broad Panel Cohort n=217 Participants 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.	Pediatric Cohort n=3 Participants 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.
Broad Panel and Pediatric Cohorts: Objective Response Rate (ORR) Based on Response Assessment in Neuro-Oncology (RANO) as Assessed by Independent Review Committee (IRC)	29.5 Percentage of participants Interval 23.5 to 36.0	66.7 Percentage of participants Interval 9.4 to 99.2

PRIMARY outcome

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

Population: 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.

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).

Outcome measures

Outcome measures
Measure	Broad Panel Cohort n=109 Participants 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.	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.
Core Panel Cohort: Objective Response Rate (ORR) Based on Response Assessment in Neuro-Oncology (RANO) as Assessed by Independent Review Committee (IRC)	26.6 Percentage of participants Interval 18.6 to 35.9	—

Adverse Events

Broad Panel Cohort

Serious events: 86 serious events

Other events: 215 other events

Deaths: 8 deaths

Cholangiocarcinoma (CCA) Expansion Cohort

Serious events: 15 serious events

Other events: 35 other events

Deaths: 1 deaths

Exploratory Cohort

Serious events: 26 serious events

Other events: 53 other events

Deaths: 1 deaths

Pediatric Cohort

Serious events: 8 serious events

Other events: 11 other events

Deaths: 1 deaths

Serious adverse events

Other adverse events

Additional Information

Executive Medical Director Oncology

Janssen Research & Development, LLC

Phone: 844-434-4210

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee If an investigator wishes to publish information from the study, a copy of the manuscript must be provided to the sponsor for review at least 60 days before submission for publication or presentation. If requested by the sponsor in writing, the investigator will withhold such publication for up to an additional 60 days to allow for filing of a patent application.
Publication restrictions are in place

Restriction type: OTHER