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

Official Title

A Phase 2 Study of Erdafitinib in Subjects With Advanced Solid Tumours and FGFR Gene Alterations

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 tumours with fibroblast growth factor receptor (FGFR) alterations (mutations or gene fusions). It will also evaluate ORR in pediatric participants with advanced solid tumours and FGFR alterations.

Trial Description

Primary Outcome:

• Overall Response Rate (ORR) as Assessed by Independent Review Committee (IRC)

Secondary Outcome:

• Overall Response Rate as Assessed by Investigator
• Duration of Response (DOR)
• Disease Control Rate (DCR)
• Clinical Benefit Rate (CBR)
• Progression Free Survival (PFS)
• Overall Survival (OS)
• Plasma Concentrations of Erdafitinib
• Number of Participants with Adverse Events (AEs)
• Number of Participants with Adverse Events by Severity
• 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
• Change from Baseline in Health-Related Quality of Life as Assessed by Patient Global Impression of Symptom Severity (PGIS) Scale Score
• Change from Baseline in Health-Related Quality of Life as Assessed by Patient Global Impression of Change (PGIC) Scale
• 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
• Change from Baseline in Pediatric Functional Assessment of Cancer Therapy - Brain (Peds FACT-Br) Total Score

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 tumours irrespective of solid tumour 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 participants has died, withdraws consent, is lost to follow-up, or the end of study, whichever comes first). End of study is defined as 4 years from enrollment of last participant into study or last follow-up visit of last participant, whichever occurs first.

View this trial on ClinicalTrials.gov