The purpose of this study is to evaluate the efficacy of erdafitinib in terms of overall response rate (ORR) in participants with advanced solid tumors with fibroblast growth factor receptor (FGFR) mutations and gene fusions.
Name: Erdafitinib
Description: ORR as assessed by IRC is defined as the percentage of participants who achieve a complete response (CR) or partial response (PR).
Measure: Overall Response Rate (ORR) as Assessed by Independent Review Committee (IRC) Time: Up to 6 YearsDescription: ORR as assessed by investigator is defined as the percentage of participants who achieve a CR or PR.
Measure: Overall Response Rate as Assessed by Investigator Time: Up to 6 YearsDescription: DOR is the duration from the date of initial documentation of a response to the date of first documented evidence of progressive disease (or relapse for participants who experience CR during the study), or death, whichever comes first.
Measure: Duration of Response (DOR) Time: Up to 6 YearsDescription: DCR is defined as the percentage of participants with CR, PR or stable disease (SD).
Measure: Disease Control Rate (DCR) Time: Up to 6 YearsDescription: PFS is 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 participants who experience CR during the study) or death, whichever comes first.
Measure: Progression Free Survival (PFS) Time: Up to 6 YearsDescription: OS will be measured from the date of first dose of study drug to the date of the participant's death.
Measure: Overall Survival (OS) Time: Up to 6 YearsDescription: Plasma concentrations of erdafitinib will be reported.
Measure: Plasma Concentrations of Erdafitinib Time: Predose and 2-4 hours postdoseDescription: An AE is any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. An adverse event does not necessarily have a causal relationship with the relevant investigational product.
Measure: Number of Participants with Adverse Events (AEs) Time: Up to 6 YearsDescription: An AE is any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. Adverse event severity is a clinical determination of the intensity of an adverse event.
Measure: Number of Participants with Adverse Events by Severity Time: Up to 6 YearsDescription: The EORTC QLQ-C30 includes 30 items in 5 functional scales, 1 global health status scale, 3 symptom scales, and 6 single symptom items. The responses are reported using a verbal rating scale. The item and scale scores are transformed to a 0 to 100 scale. A higher score represents greater HRQoL, better functioning, and more (worse) symptoms.
Measure: Change from Baseline in Health-Related Quality of Life (HRQoL) as Assessed by European Organisation for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30 (EORTC-QLQ-C30) Scale Score Time: Baseline up to 6 YearsDescription: The PGIS is a single question regarding the patient report of disease severity: considering all aspects of your cancer symptoms right now would you say your cancer symptoms are none, mild, moderate, severe, or very severe?
Measure: Change from Baseline in Health-Related Quality of Life as Assessed by Patient Global Impression of Symptom Severity (PGIS) Scale Score Time: Baseline up to 6 YearsDescription: The PGIC is the patient-reported outcome (PRO) counterpart to the clinical global impressions (CGI) scale. The PGIC is a single verbal rating scale ranging from 1 = a lot better now to 7 = a lot worse now.
Measure: Change from Baseline in Health-Related Quality of Life as Assessed by Patient Global Impression of Change (PGIC) Scale Time: Baseline up to 6 YearsDescription: The EQ-5D-5L is a generic measure of health status. The EQ-5D-5L is a 5-item questionnaire that assesses 5 domains including mobility, self-care, usual activities, pain/discomfort and anxiety/depression plus a visual analog scale rating "health today" with anchors ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).
Measure: Change from Baseline in Health-Related Quality of Life as Assessed by European Quality of Life - 5 Dimensions-5 Levels (EQ-5D-5L) Scale Score Time: Baseline up to 6 YearsAllocation: N/A
Single Group Assignment
There is one SNP
The EQ-5D-5L is a 5-item questionnaire that assesses 5 domains including mobility, self-care, usual activities, pain/discomfort and anxiety/depression plus a visual analog scale rating "health today" with anchors ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).. 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 - 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 greater 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, and BRAF V600E - Active malignancies other than for disease requiring therapy 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 - 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 greater 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, and BRAF V600E - Active malignancies other than for disease requiring therapy Advanced Solid Tumor Neoplasms 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. --- V600E ---
The EQ-5D-5L is a 5-item questionnaire that assesses 5 domains including mobility, self-care, usual activities, pain/discomfort and anxiety/depression plus a visual analog scale rating "health today" with anchors ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).. 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 - 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 greater 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, and BRAF V600E - Active malignancies other than for disease requiring therapy 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 - 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 greater 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, and BRAF V600E - Active malignancies other than for disease requiring therapy Advanced Solid Tumor Neoplasms 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. --- V600E --- --- V600E ---