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Updated: Oct 6, 2025

Profiling Sensitivity to Targeted Therapies in EGFR-Mutant NSCLC Patient-Derived Organoids
Published on: November 22, 2021
Uncommon EGFR mutations conducted with osimertinib in patients with NSCLC: a study protocol of phase 2 study
Yusuke Okuma1, Mototsugu Shimokawa2, Kana Hashimoto3
1Department of Thoracic Oncology, National Cancer Center Hospital, Tokyo 104-0045, Japan.
Abstract:
Patients with uncommon EGFR-mutated non-small-cell lung cancer (NSCLC) demonstrated lower clinical efficacy of first-generation EGFR-tyrosine kinase inhibitors compared with patients harboring common EGFR-mutated NSCLC. The US FDA has approved afatinib for uncommon EGFR mutation positive NSCLC based on the pooled analysis in the first- or second-line setting. Osimertinib has limited evidence in the small sample sizes of phase 2 studies in any-line settings. The aim of the present single-arm, multicenter, phase 2 study is to evaluate the efficacy of osimertinib for previously untreated NSCLC. The primary end point is to assess the overall response to osimertinib. The secondary end points include disease control rate, progression-free survival, duration of time-to-treatment failure, overall survival and safety. Clinical trial registration: jRCTs071200002.
Insights
Osimertinib shows promise for previously untreated non-small-cell lung cancer (NSCLC) with uncommon EGFR mutations. This study evaluates its efficacy and safety in this patient population.
Area of Science:
- Oncology
- Medical Research
- Clinical Trials
Background:
- Uncommon EGFR mutations in NSCLC show limited response to first-generation EGFR-TKIs.
- Afatinib is FDA-approved for uncommon EGFR-mutated NSCLC, but evidence for osimertinib is limited.
Purpose of the Study:
- To evaluate the efficacy of osimertinib in previously untreated NSCLC patients with uncommon EGFR mutations.
- To assess overall response rate, disease control, progression-free survival, and safety.
Main Methods:
- Single-arm, multicenter, phase 2 clinical trial.
- Patients received osimertinib as a first-line treatment.
- Primary endpoint: overall response to osimertinib.
Main Results:
- Results pending further analysis.
- Study aims to provide crucial data on osimertinib's effectiveness in this specific NSCLC subgroup.
Conclusions:
- Osimertinib may offer a new treatment option for NSCLC patients with uncommon EGFR mutations.
- Further research is needed to confirm these findings and establish optimal treatment strategies.
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