Erdafitinib in patients with advanced solid tumours with FGFR alterations (RAGNAR): an international, single-arm, phase 2 study

Iyer, Gopa; Witt, Olaf; Doi, Toshihiko; Massard, Christophe; Stuyckens, Kim; Crow, Lauren; Najmi, Saltanat; Hammond, Constance; Thomas, Shibu; Santiago-Walker, Ademi; Triantos, Spyros; Sweiti, Hussein; Carranza, Omar; Greco, Maria Raffaella; Coward, Jermaine; Joshua, Anthony M.; Karapetis, Christos Stelios; Hart, Christopher; Zhang, Alison; Prenen, Hans; Goeminne, Jean-Charles; Machiels, Jean‐Pascal; Rottey, Sylvie; Corassa, Marcelo; Molin, Graziela Zibetti Dal; Tiscoski, Katsuki Arima; Jardim, Denis Leonardo Fontes; Mak, Milena Perez; Fu, Wenbin; Yao, Herui; Huang, Jing; Jiang, Haiping; Qin, Shukui; Chen, Baoshi; Dong, Yujuan; Yang, Yaewon; Loriot, Yohann; Tourneau, Christophe Le; Penel, Nicolas; Salas, Sébastien; Blay, Jean‐Yves; Brachet, Pierre-Emmanuel; Durando, Xavier; Emambux, Sheik; Ravaud, Alain; Folprecht, Gunnar; Arnold, Dirk; Schüler, Martin; Ahrens, Marit; Golf, Alexander; Haag, Georg Martin; Lordick, Florian; Desuki, Alexander; Cazzaniga, M.; Ciardiello, Fortunato; Milella, Michèle; Koyama, Takafumi; Hirooka, Yoshiki; Okamoto, Wataru; Aogi, Kenjiro; Kuboki, Yasutoshi; Lee, Jungyun; Kim, Sung‐Bae; Ahn, Myung‐Ju; Chang, Jong Hee; Kim, Yong‐Man; Nam, Do‐Hyun; Park, Jaesung; Ługowska, Iwona; Paz‐Ares, Luis; Moreno, Víctor; Cervantes, Alexis; Calvo, Mariona; Falcón, Alejandro; González, Antonio G.; Tabernero, Josep; Bueno, Alejandro Martínez; García-Corbacho, Javier; Longo, Federico; Yen, Chia‐Jui; Chen, Jen‐Shi; Hou, Ming‐Feng; Chao, Yee; Rau, Kun‐Ming; Chiu, Tai‐Jan; Feng, Yin‐Hsun; Hsu, Chih‐Hung; Huang, Wen-Tsung; Lai, Kuan-Ming; Yeh, Su‐Peng; Palmer, Daniel H.; Minchom, Anna; Winter, Helen; Welsh, Liam; Plummer, Ruth; Iyer, Gopakumar V.; Gutierrez, Martin; Bilen, Mehmet Asım; Arrowsmith, Edward; Pant, Shubham; Spigel, David R.; Zandberg, Dan P.; Doroshow, Deborah Blythe; Lu-Emerson, Christine; Moezi, Mehdi M.; Paulson, Scott; Reardon, David A.; Ward, Patrick; Chaves, Jorge; Grigg, Claud; Hussein, Atif M.; Manda, Sudhir; Monticelli, M; Qamar, Rubina; Richey, Stephen; Tamura, David; Wilks, Sharon

doi:10.1016/s1470-2045(23)00275-9

Cited by 51 publications

(23 citation statements)

References 16 publications

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“…The ORR was 55.6% (95% CI: 30.8-78.5%), and median OS was 19.7 months. Safety data were consistent with the known profile of erdafitinib [8,9].…”

Section: Ragnarsupporting

confidence: 73%

Pancreatic, Hepatic, and Biliary Tract Oncology Highlights from the European Society for Medical Oncology Annual Meeting 2023

Heise,

Nieto,

Scheck

et al. 2024

Oncol Res Treat

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“…The ORR was 55.6% (95% CI: 30.8-78.5%), and median OS was 19.7 months. Safety data were consistent with the known profile of erdafitinib [8,9].…”

Section: Ragnarsupporting

confidence: 73%

Pancreatic, Hepatic, and Biliary Tract Oncology Highlights from the European Society for Medical Oncology Annual Meeting 2023

Heise,

Nieto,

Scheck

et al. 2024

Oncol Res Treat

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“…However, toxicity to Erdafitinib is a point of concern, and the selection of patients is critical. FGFR inhibitors are not effective in patients carrying certain point mutations of the FGFR gene, although there is some evidence to suggest co-therapy with Src inhibitors can help overcome the resistance caused by these mutations [53,54].…”

Section: Targeted Drugs and Drug Repurposingmentioning

confidence: 99%

Mini-Review: Current Bladder Cancer Treatment—The Need for Improvement

Gill,

Perks

2024

IJMS

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Bladder cancer is the tenth most common cancer and is a significant burden on health care services worldwide, as it is one of the most costly cancers to treat per patient. This expense is due to the extensive treatment and follow-ups that occur with costly and invasive procedures. Improvement in both treatment options and the quality of life these interventions offer has not progressed at the rates of other cancers, and new alternatives are desperately needed to ease the burden. A more modern approach needs to be taken, with urinary biomarkers being a positive step in making treatments more patient-friendly, but there is still a long way to go to make these widely available and of a comparable standard to the current treatment options. New targets to hit the major signalling pathways that are upregulated in bladder cancer, such as the PI3K/AkT/mTOR pathway, are urgently needed, with only one drug approved so far, Erdafitinib. Immune checkpoint inhibitors also hold promise, with both PD-1 and CDLA-4 antibody therapies approved for use. They effectively block ligand/receptor binding to block the immune checkpoint used by tumour cells. Other avenues must be explored, including drug repurposing and novel biomarkers, which have revolutionised this area in other cancers.

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“…FGFR inhibition has been approved for FGFR2 amplification or mutation in urinary tumor and cholangiocarcinoma. 152 , 153 Therefore, potentially beneficial tumor types may be those anatomical sites with a high degree of fibrosis caused by long-term chronic inflammatory stimulation, including pancreatic, lung, endometrial, and breast cancers, as well as head and neck squamous cell carcinoma. 153 …”

Section: Development: Excavating Therapeutic Potential According To N...mentioning

confidence: 99%

New clinical trial design in precision medicine: discovery, development and direction

Duan,

Qin,

Jiao

et al. 2024

Sig Transduct Target Ther

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In the era of precision medicine, it has been increasingly recognized that individuals with a certain disease are complex and different from each other. Due to the underestimation of the significant heterogeneity across participants in traditional “one-size-fits-all” trials, patient-centered trials that could provide optimal therapy customization to individuals with specific biomarkers were developed including the basket, umbrella, and platform trial designs under the master protocol framework. In recent years, the successive FDA approval of indications based on biomarker-guided master protocol designs has demonstrated that these new clinical trials are ushering in tremendous opportunities. Despite the rapid increase in the number of basket, umbrella, and platform trials, the current clinical and research understanding of these new trial designs, as compared with traditional trial designs, remains limited. The majority of the research focuses on methodologies, and there is a lack of in-depth insight concerning the underlying biological logic of these new clinical trial designs. Therefore, we provide this comprehensive review of the discovery and development of basket, umbrella, and platform trials and their underlying logic from the perspective of precision medicine. Meanwhile, we discuss future directions on the potential development of these new clinical design in view of the “Precision Pro”, “Dynamic Precision”, and “Intelligent Precision”. This review would assist trial-related researchers to enhance the innovation and feasibility of clinical trial designs by expounding the underlying logic, which be essential to accelerate the progression of precision medicine.

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Erdafitinib in patients with advanced solid tumours with FGFR alterations (RAGNAR): an international, single-arm, phase 2 study

Cited by 51 publications

References 16 publications

Pancreatic, Hepatic, and Biliary Tract Oncology Highlights from the European Society for Medical Oncology Annual Meeting 2023

Pancreatic, Hepatic, and Biliary Tract Oncology Highlights from the European Society for Medical Oncology Annual Meeting 2023

Mini-Review: Current Bladder Cancer Treatment—The Need for Improvement

New clinical trial design in precision medicine: discovery, development and direction

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