Neoplasm
Conditions
Keywords
Tumor, Fibroblast Growth Factor Receptor (FGFR), Non-Small Cell Lung Cancer (NSCLC), Erdafitinib, Urothelial Cancer, Esophageal Cancer, Cholangiocarcinoma
Brief summary
The primary purpose of this study is to evaluate objective response rate (ORR) as per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 of erdafitinib in a molecularly-defined subset of Asian participants with non-small-cell lung cancer (NSCLC), urothelial cancer, esophageal cancer and cholangiocarcinoma.
Detailed description
This is an open-label (all people know the identity of the intervention), multicenter, phase 2 study to evaluate the clinical efficacy, safety and pharmacokinetics of erdafitinib in Asian participants with advanced NSCLC, urothelial cancer, esophageal cancer and cholangiocarcinoma.
Interventions
DRUGErdafitinib
Participants will receive 8 mg of erdafitinib, once daily with option to up-titrate to 9 mg.
Sponsors
Janssen Research & Development, LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Pathologically or cytologically confirmed, advanced or refractory tumors (there are no restriction on the total number of lines of prior therapies, but participant should have received at least 1 line of anti-cancer therapy \[as per local standard of care\]): Squamous and non-squamous non-small-cell lung cancer (NSCLC), esophageal cancer, urothelial cancer and cholangiocarcinoma * Participants must meet the following molecular eligibility criteria (diagnosed at a central or local laboratory using either a tumor tissue based assay, which must indicate: at least one of following): a) fibroblast growth factor receptor (FGFR) gene translocations b) FGFR gene mutations c) Participants with evidence of FGFR pathway activation or other potential target/pathway inhibited by erdafitinib may also be considered and allowed for enrollment if supported by emerging biomarker data. * The presence of measurable disease according to the RECIST, Version 1.1 Criteria, and documented disease progression as defined by RECIST (Version 1.1) at baseline * Eastern Cooperative Oncology Group (ECOG) performance status score 0 or 1 * Female participants (of child bearing potential and sexually active) and male participants (with a partner of child bearing potential) must use medically acceptable methods of birth control. Male participants must use highly effective birth control measurements when sexually active and must not donate sperm * Adequate bone marrow, liver, and renal function within the 14 days prior to Day 1 of Cycle 1 up until pre-dose of Cycle 1
Exclusion criteria
* Chemotherapy, targeted therapies, immunotherapy, or treatment with an investigational anticancer agent within 2 weeks or at least 5 half-lives of the drug whichever is longer up to a maximum of 4 weeks before the first administration of study drug. Localized palliative radiation therapy (but should not include radiation to target lesions) and ongoing luteinizing hormone-releasing hormone (LHRH) agonists, bisphosphonates and denosumab, are permitted * Participants with persistent phosphate greater than (\>) upper limit of normal (ULN) during Screening (within 14 days prior to Day 1 of Cycle 1 up until pre-dose of Cycle 1) and despite medical management of phosphate levels * Participants taking medications known to have a significant risk of causing QTc prolongation and Torsades de Pointes. Participants who have discontinued any of these medications must have a wash-out period of at least 5 days or at least 5 half-lives of the drug (whichever is longer) prior to the first dose of study drug * Left ventricular ejection fraction (LVEF) less than (\<) 50% as assessed by echocardiography (or multi-gated acquisition \[MUGA\]) performed at Screening * Uncontrolled inter-current illness including, but not limited to, poorly controlled hypertension or diabetes, ongoing active infection requiring antibiotics, psychiatric illness, or at risk of gastrointestinal perforation as per investigators' assessment * Received prior selective FGFR inhibitor treatment or RET inhibitor treatment, respectively according to the biomarker prescreening result, or if the participant has known allergies, hypersensitivity, or intolerance to Erdafitinib or its excipients * Any corneal or retinal abnormality likely to increase risk of eye toxicity
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Objective Response Rate (ORR) | From the date of the first dose of study drug until disease progression or death as assessed up to the last efficacy assessment for disease progression (approximately 2 years) | Objective Response Rate (ORR) is defined as proportion of participants with best objective response of complete response (CR) or partial response (PR) based on Response Evaluation Criteria In Solid Tumors Version 1.1 (RECIST v1.1) criteria. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Duration Of Response (DOR) | From the date of the first dose of study drug until disease progression or death as assessed up to the last efficacy assessment for disease progression (approximately 2 years) | Duration of response is defined as the time from the date of initial documentation of a response (CR or PR) to the date of first documented evidence of progressive disease (or relapse for participants who experience CR during the study) or death. |
| Disease Control Rate (DCR) | From the date of the first dose of study drug until disease progression or death as assessed up to the last efficacy assessment for disease progression (approximately 2 years) | DCR defined as the proportion of participants with complete response \[CR\], partial response \[PR\], or greater than or equal to (\>=) 6 weeks stable disease \[SD\]). |
| Progression Free Survival (PFS) | From the date of the first dose of study drug until disease progression or death as assessed up to the last efficacy assessment for disease progression (approximately 2 years) | 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. |
| Number of Participants With an Adverse Event | Screening up to end of study (approximately 2 years) | — |
| Plasma Concentration of Erdafitinib | Approximately up to 16 weeks | — |
| Overall Survival (OS) | From the date of the first dose of study drug until death, or withdrawal of consent or long-term extension (LTE) phase initiates, whichever occurs first (approximately 2 years) | The OS is defined as the time from the date of first dose of study drug to date of death from any cause. If the participant is alive or the vital status is unknown, the participant will be censored at the date the participant will be last known to be alive. |
Countries
China, South Korea, Taiwan
Outcome results
None listed