Nilotinib in KIT-driven advanced melanoma: Results from the phase II single-arm NICAM trial.
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Authors
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
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
Document Type
Journal Article
Date
2024-03-19
Date Accepted
2024-01-26
Abstract
Mucosal (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).
Citation
Cell Reports Medicine, 2024, 5 (3), pp. 101435 -
Source Title
Cell Reports Medicine
Publisher
Elsevier BV
ISSN
2666-3791
eISSN
2666-3791
2666-3791
2666-3791
Collections
Research Team
Clin Trials & Stats Unit