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Poster Discussion session -Gastrointestinal, non-colorectal

5302 - Preliminary Results of a Ph2a Study to Evaluate the Clinical Efficacy and Safety of Erdafitinib in Asian Patients with Biomarker-Selected Advanced Cholangiocarcinoma (CCA)


19 Oct 2018


Poster Discussion session -Gastrointestinal, non-colorectal


Cytotoxic Therapy

Tumour Site

Hepatobiliary Cancers


Yen-Yang Chen


Annals of Oncology (2018) 29 (suppl_8): viii205-viii270. 10.1093/annonc/mdy282


Y. Chen1, J.O. Park2, W. Su3, D. Oh4, K. Kim5, Y. Feng6, L. Shen7, H. Liao8, J. Nie9, M. Qing10, J. Li11, P. De Porre12

Author affiliations

  • 1 Department Of Haemato-oncology, Chang Gung Memorial Hospital-Kaohsiung, 833 - Kaohsiung/TW
  • 2 Division Of Hematology-oncology, Department Of Medicine, Samsung Medical Center Sungkyunkwan University School of Medicine, 135-710 - Seoul/KR
  • 3 Department Of Internal Medicine, Department of Internal Medicine, National Cheng Kung University Hospital, Tainan/TW
  • 4 Department Of Internal Medicine, Seoul National University Hospital, 110-744 - Seoul/KR
  • 5 Oncology, Asan Medical Center, University of Ulsan College of Medicine, 138-931 - Seoul/KR
  • 6 Division Of Hematology And Oncology , Chi-Mei Medical Center, 710 - Tainan/TW
  • 7 .department Of Gi Oncology, Peking University Cancer Hospital & Institute, Beijing/CN
  • 8 Statistics & Decision Sciences, Janssen China R & D Center, Shanghai/CN
  • 9 Clinical Pharmacology, Janssen China R & D Center, Beijing/CN
  • 10 Department Of Biomarker, Janssen China R & D Center, Shanghai/CN
  • 11 Early Development, Janssen China R & D Center, Shanghai/CN
  • 12 Be Clinical Oncology, Janssen R&D BE, Beerse/BE


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Abstract 5302


Advanced CCA patients (pts) who progressed after first-line chemotherapy have limited treatment options and poor prognosis. Aberrant fibroblast growth factor receptor (FGFR) signalling is a driver of the pathogenesis of CCA and is observed in 14-17% of pts. Erdafitinib, a potent oral pan-FGFR tyrosine kinase inhibitor, demonstrated encouraging clinical activity with manageable adverse events (AEs) in the Europe (EU)/United States (US) phase 1 study in solid tumours, including subjects with CCA.


LUC2001 is an open-label, multicenter, Ph2a study in advanced CCA pts with FGFR alterations, based on FoundationOne testing, who failed at least 1 prior systemic treatment. The primary endpoint is objective response rate (ORR; by RECIST 1.1). The secondary endpoints are disease control rate (DCR), safety and pharmacokinetics. Disease is evaluated every 8 weeks until disease progression (PD).


As of 20 March 2018, 150 CCA pts are molecularly screened; 25 have FGFR alterations, of whom 11 (with FGFR2 fusions [7] or FGFR 2 [2] or 3 [2] mutations) are dosed with continuous 8 mg once daily erdafitinib, all response evaluable. Median age is 53.0 years, ECOG score 0 and 1 in 6 and 5 pts, respectively, 8 males and 3 females. Median number of treatment cycles is 4.0 and median treatment duration is 3.5 months. There are 3 confirmed partial response (PR), 2 unconfirmed PR (uPR), 4 stable disease (SD), and 2 PD (both with FGFR3 mutations). The ORR (CR+PR+uCR+uPR) is 45.5%. The DCR (CR+PR+uCR+uPR+SD) is 81.8%. Six pts are still on treatment. The most common AEs (>30%) are hyperphosphatemia, dry mouth, stomatitis, diarrhea, nail disorder, and palmar-plantar erythrodysaesthesia syndrome. Seven pts experienced Grade 3 or higher AEs and 3 subjects had AEs leading to dose reduction. Three pts experienced non-drug related serious AEs (SAEs), while no AE led to treatment discontinuation or death. PK characteristics are consistent with data from other studies.


Erdafitinib shows encouraging clinical activity and tolerable safety profile in Asian and EU/US pts with FGFR-altered advanced CCA with high unmet medical need.

Clinical trial identification


Legal entity responsible for the study

Janssen (China) Research & Development Center, a division of Johnson & Johnson (China) Investment Ltd.


Janssen APMS China Research & Development.

Editorial Acknowledgement


H. Liao, J. Nie, M. Qing, J. Li, P. De Porre: Employee: Janssen Research and Development; Stock ownership: Johnson&Johnson. All other authors have declared no conflicts of interest.

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