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Lookup NU author(s): Professor Ruth Plummer
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© 2024 The AuthorsMucosal (MM) and acral melanomas (AM) are rare melanoma subtypes of unmet clinical need; 15%–20% harbor KIT mutations potentially targeted by small-molecule inhibitors, but none yet approved in melanoma. This multicenter, single-arm Phase II trial (NICAM) investigates nilotinib safety and activity in KIT mutated metastatic MM and AM. KIT mutations are identified in 39/219 screened patients (18%); of 29/39 treated, 26 are evaluable for primary analysis. Six patients were alive and progression free at 6 months (local radiology review, 25%); 5/26 (19%) had objective response at 12 weeks; median OS was 7.7 months; ddPCR assay correctly identifies KIT alterations in circulating tumor DNA (ctDNA) in 16/17 patients. Nilotinib is active in KIT-mutant AM and MM, comparable to other KIT inhibitors, with demonstrable activity in nonhotspot KIT mutations, supporting broadening of KIT evaluation in AM and MM. Our results endorse further investigations of nilotinib for the treatment of KIT-mutated melanoma. This clinical trial was registered with ISRCTN (ISRCTN39058880) and EudraCT (2009-012945-49).
Author(s): Larkin J, Marais R, Porta N, Gonzalez de Castro D, Parsons L, Messiou C, Stamp G, Thompson L, Edmonds K, Sarker S, Banerji J, Lorigan P, Evans TRJ, Corrie P, Marshall E, Middleton MR, Nathan P, Nicholson S, Ottensmeier C, Plummer R, Bliss J, Valpione S, Turajlic S
Publication type: Article
Publication status: Published
Journal: Cell Reports Medicine
Year: 2024
Volume: 5
Issue: 3
Print publication date: 19/03/2024
Online publication date: 24/02/2024
Acceptance date: 26/01/2024
Date deposited: 26/03/2024
ISSN (electronic): 2666-3791
Publisher: Cell Press
URL: https://doi.org/10.1016/j.xcrm.2024.101435
DOI: 10.1016/j.xcrm.2024.101435
Data Access Statement: The ddPCR primer sequences are available from BioRad Assay Design Tool. De-identified data reported in this paper will be shared upon request; applicants can contact the Lead applicant of the Clinical Trials and Statistics Unit at the Institute of Cancer Research (ICR-CTU), who coordinated this study. Trial data are collected, managed, stored, shared, and archived according to ICR-CTSU Standard Operating Procedures to ensure the enduring quality, integrity, and utility of the data. Formal re quests for data sharing are considered in line with ICR-CTSU procedures with due regard given to funder and sponsor guidelines. Requests are via a standard proforma describing the nature of the proposed research and extent of data requirements. The rest of the statement is available in the published paper
PubMed id: 38417447
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