A Phase 2 Study of Erdafitinib in Subjects with Advanced Solid Tumors and FGFR Gene Alterations

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-002113-19-DE

Enrollment

336

Registered

2019-10-11

Start date

Unknown

Completion date

Unknown

Last updated

2024-03-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Advanced solid tumors (other than Urothelial tumors), and FGFR gene alterations. MedDRA version: 21.1 Level: LLT Classification code 10065143 Term: Malignant solid tumour System Organ Class: 100000004864

Interventions

Product Name: Erdafitinib Product Code: JNJ-42756493 Pharmaceutical Form: Film-coated tablet INN or Proposed INN: Erdafitinib CAS Number: 1346242-81-6 Current Sponsor code: JNJ-42756493 Other descript

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Criterion modified per Amendment 3 1.1 =6 years of age 2. Criterion modified per Amendment 1 2.1 Criterion modified per Amendment 2 2.2 Criterion modified per Amendment 3 2.3 Histologic demonstration of an unresectable, locally advanced, or metastatic solid tumor malignancy bearing an FGFR mutation or fusion, as determined by local* or central laboratory screening (Section 8.1.1.1). Molecular Criteria for Broad Panel Cohort and Cholangiocarcinoma Expansion Cohort: *Subjects with target FGFR mutations or any** FGFR gene fusions are eligible for enrollment in the Broad Panel Cohort (The List of Target FGFR Mutations is provided in Section 10.11) *Subjects with other FGFR mutations*** not captured in the Broad Panel Cohort are eligible for enrollment in the Exploratory Cohort. Molecular Criteria for Pediatric Cohort: *Subjects with any FGFR mutation*** (exclusive of FGFR valine gatekeeper and resistance alterations defined in the Exclusion Criteria) or any** FGFR gene fusions, or FGFR internal tandem duplication**** are eligible for enrolment in the Pediatric Cohort **FGFR Fusion Specifications: - Have a report suggesting the presence of an intact FGFR kinase domain. - FGFR fusion with a 3-prime partner (FGFR gene is listed first, eg FGFR-GENE or FGFR3-TACC3): -The FGFR portion of the fusion must involve exon 17 or greater (=17) - FGFR fusion with a 5-prime partner (Partner gene is listed first and FGFR gene is second, eg GENE-FGFR or KLK2-FGFR2): - The FGFR portion of the fusion must involve less than or equal to exon 11 (=11) - Have a named FGFR fusion partner gene (self-fusions or rearrangements, eg FGFR-FGFR, are not eligible) (Broad Panel Cohort only) -FGFR gene identifiers, canonical transcript identifiers, and kinase domain positions are provided, see Section 5.1. 3. Measurable disease according to RECIST v1.1 or RANO for primary brain tumors. 4. Criterion modified per Amendment 2 4.1 Criterion modified per Amendment 3 4.2 Subject must have received at least one prior line of systemic therapy in the advanced, unresectable, or metastatic setting; or is a child or adolescent subject with a newly-diagnosed solid tumor and no acceptable standard therapies. 5. Subject does not have standard of care options that have shown meaningful clinical benefit for the relevant underlying histology and line of therapy or the subject is unable to tolerate the therapy 6. Criterion modified per Amendment 4 6.1 Documented progression of disease, defined as any progression that requires a change in treatment, prior to full study screening. Note: Not applicable for treatment naïve pediatric subjects with no standard of care therapies. 7. Toxicities from previous anticancer therapies should have resolved to baseline levels or to Grade 1 or less except for alopecia, peripheral neuropathy, and Grade 2 laboratory values eligible per Inclusion Criterion 9. 8. Criterion modified per Amendment 3 8.1 Criterion modified per Amendment 4 8.2 For adults (=18 years of age), ECOG performance status Grade 0 or 1 (Section 10.5) For children and adolescents (=6 to <16 years of age), Lansky Score of =70 (Section 10.6). For adolescents (=16 to <18 years of age), Karnofsky Score of =70 (Section 10.6). 9. Criterion modified per Amendment 1 9.1 Criterion modified per Amendment 2 9.2 Criterion modified per Amendment 3 9.3 Adequate bone marrow, liver, and renal function: a. Bone marrow function (without the support of cytokines or erythropoiesis-stimulating agent transfu

Exclusion criteria

Exclusion criteria: 1. Criterion modified per Amendment 2 1.1 Criterion modified per Amendment 3 1.2 Has had prior chemotherapy, targeted therapy, or treatment with an investigational anticancer agent within 15 days or 480 milliseconds; or for children and adolescent subjects, Bazett: QTc >440 milliseconds) 12. Known history of AIDS (human immunodeficiency virus [HIV] infection), unless the subject has been on a stable anti-retroviral therapy regimen for the last 6 months or more, has had no opportunistic infections in the last 6 months, and has CD4 count >350. 13. Criterion modified per Amendment 1 13.1 Criterion modified per Amendment 2 13.2. Evidence of active hepatitis B or C infection (for example, subjects with history of hepatitis C infection but normal hepatitis C virus polymerase chain reaction [PCR] test and subjects with inactive hepatitis B with positive HBsAg antibody or normal PCR are allowed) 14. Not recovered from reversible toxicity of prior anticancer therapy (except toxicities which are not clinically significant such as alopecia, skin discoloration, neuropathy, hearing loss). 15. Impaired woun

Design outcomes

Primary

Measure	Time frame
Main Objective: - Broad Panel Cohort and Core Panel Cohort: To evaluate the efficacy of erdafitinib in terms of overall response rate (ORR) as assessed by the Independent review Committee (IRC) in subjects with advanced solid tumors with target FGFR mutations and any gene fusions (Broad Panel Cohort), or in a pre-specified subgroup of subjects with a selected panel of FGFR markers (Core Panel Cohort), or in both cohorts - Pediatric Cohort: To evaluate the efficacy of erdafitinib in terms of ORR as assessed by the IRC in pediatric subjects with advanced solid tumors with FGFR mutations, any gene fusions, or FGFR internal tandem duplication (Pediatric Cohort), including adolescent subjects with target FGFR mutations and any gene fusions;Secondary Objective: - To evaluate the efficacy of erdafitinib, in terms of the ORR, as assessed by investigator - To evaluate the efficacy of erdafitinib in terms of DOR - To evaluate other measures of efficacy including DCR, clinical benefit rate (CBR), PFS, and overall survival (OS) - To evaluate erdafitinib pharmacokinetics (PK) - To evaluate safety and tolerability of erdafitinib - To evaluate Health-Related Quality of Life (HRQoL) Cholangiocarcinoma Expansion Cohort -To evaluate the efficacy and safety of erdafitinib in subjects with cholangiocarcinoma with target FGFR mutations and any gene fusions;Primary end point(s): - Broad Panel Cohort and Core Panel Cohort: The proportion of subjects who achieve a complete response (CR) or partial response (PR) based on Response Evaluation Criteria in Solid Tumors (RECIST, version 1.1) (RECIST v1.1.) or Response Assessment in Neuro-Oncology (RANO) as assessed by IRC - Pediatric Cohort: The proportion of subjects who achieve a CR or PR based on RECIST v1.1. or RANO as assessed by IRC;Timepoint(s) of evaluation of this end point: Throughout the study	—

Measure

Time frame

Main Objective: - Broad Panel Cohort and Core Panel Cohort: To evaluate the efficacy of erdafitinib in terms of overall response rate (ORR) as assessed by the Independent review Committee (IRC) in subjects with advanced solid tumors with target FGFR mutations and any gene fusions (Broad Panel Cohort), or in a pre-specified subgroup of subjects with a selected panel of FGFR markers (Core Panel Cohort), or in both cohorts - Pediatric Cohort: To evaluate the efficacy of erdafitinib in terms of ORR as assessed by the IRC in pediatric subjects with advanced solid tumors with FGFR mutations, any gene fusions, or FGFR internal tandem duplication (Pediatric Cohort), including adolescent subjects with target FGFR mutations and any gene fusions;Secondary Objective: - To evaluate the efficacy of erdafitinib, in terms of the ORR, as assessed by investigator - To evaluate the efficacy of erdafitinib in terms of DOR - To evaluate other measures of efficacy including DCR, clinical benefit rate (CBR), PFS, and overall survival (OS) - To evaluate erdafitinib pharmacokinetics (PK) - To evaluate safety and tolerability of erdafitinib - To evaluate Health-Related Quality of Life (HRQoL) Cholangiocarcinoma Expansion Cohort -To evaluate the efficacy and safety of erdafitinib in subjects with cholangiocarcinoma with target FGFR mutations and any gene fusions;Primary end point(s): - Broad Panel Cohort and Core Panel Cohort: The proportion of subjects who achieve a complete response (CR) or partial response (PR) based on Response Evaluation Criteria in Solid Tumors (RECIST, version 1.1) (RECIST v1.1.) or Response Assessment in Neuro-Oncology (RANO) as assessed by IRC - Pediatric Cohort: The proportion of subjects who achieve a CR or PR based on RECIST v1.1. or RANO as assessed by IRC;Timepoint(s) of evaluation of this end point: Throughout the study

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Secondary

Measure	Time frame
Secondary end point(s): - The proportion of subjects who achieve a CR or PR based on RECIST v1.1. or RANO as assessed by investigator - DOR: the duration from the date of initial documentation of a response to the date of first documented evidence of progressive disease (or relapse for subjects who experience CR during the study) or death, whichever comes first. Data from subjects who are progression-free and alive or have unknown status will be censored at the last tumor assessment - DCR: the proportion of subjects with CR, PR or stable disease (SD) - CBR: the proportion of subjects with CR, PR or durable SD (defined as a duration of at least 4 months) - PFS: the duration from the date of the first dose of study drug until the date of first documented evidence of progressive disease (or relapse for subjects who experience CR during the study) or death, whichever comes first. Data from subjects who are progression-free and alive or have unknown status will be censored at the last tumor assessment - OS: measured from the date of first dose of study drug to the date of the subject’s death. If the subject is alive or the vital status is unknown, the subject’s data will be censored at the date the subject was last known to be alive - PK exposure parameters.This endpoint includes pediatrics. - Incidence and severity of AEs - Change from baseline in patient-reported health status and physical functioning scales of the European Organisation for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30 (EORTC-QLQ-C30; for subjects =18 years of age) or Pediatric Functional Assessment Of Cancer Therapy - Brain (Peds FACT-Br for subjects <18 years of age), Patient Global Impression of Symptom Severity (PGIS), Patient Global Impression of Change (PGIC), and European Quality of Life – 5 Dimensions-5 Levels (EQ-5D-5L). Cholangiocarcinoma Expansion Cohort - Key efficacy endpoints will be evaluated including ORR assessed by IRC/investigator, DOR, PFS, and OS - Inciden	—

Measure

Time frame

Secondary end point(s): - The proportion of subjects who achieve a CR or PR based on RECIST v1.1. or RANO as assessed by investigator - DOR: the duration from the date of initial documentation of a response to the date of first documented evidence of progressive disease (or relapse for subjects who experience CR during the study) or death, whichever comes first. Data from subjects who are progression-free and alive or have unknown status will be censored at the last tumor assessment - DCR: the proportion of subjects with CR, PR or stable disease (SD) - CBR: the proportion of subjects with CR, PR or durable SD (defined as a duration of at least 4 months) - PFS: the duration from the date of the first dose of study drug until the date of first documented evidence of progressive disease (or relapse for subjects who experience CR during the study) or death, whichever comes first. Data from subjects who are progression-free and alive or have unknown status will be censored at the last tumor assessment - OS: measured from the date of first dose of study drug to the date of the subject’s death. If the subject is alive or the vital status is unknown, the subject’s data will be censored at the date the subject was last known to be alive - PK exposure parameters.This endpoint includes pediatrics. - Incidence and severity of AEs - Change from baseline in patient-reported health status and physical functioning scales of the European Organisation for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30 (EORTC-QLQ-C30; for subjects =18 years of age) or Pediatric Functional Assessment Of Cancer Therapy - Brain (Peds FACT-Br for subjects <18 years of age), Patient Global Impression of Symptom Severity (PGIS), Patient Global Impression of Change (PGIC), and European Quality of Life – 5 Dimensions-5 Levels (EQ-5D-5L). Cholangiocarcinoma Expansion Cohort - Key efficacy endpoints will be evaluated including ORR assessed by IRC/investigator, DOR, PFS, and OS - Inciden

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Countries

Argentina, Australia, Belgium, Brazil, China, France, Germany, Italy, Japan, Korea, Republic of, Poland, Spain, Sweden, Taiwan, United Kingdom, United States

Contacts

Public ContactClinical Registry Group

Janssen-Cilag International NV

ClinicalTrialsEU@its.jnj.com+3171524 21 66

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026