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Article

Safety findings from the phase 1/2 MOSAIC study of miransertib for patients with PIK3CA-related overgrowth spectrum or Proteus syndrome.

Orphanet journal of rare diseases - 25 Jul 2025

Eng Whitney, Iacobas Ionela, Perkins Jonathan, Zampino Giuseppe, Leoni Chiara, Buonuomo Paola Sabrina, Simonetti Alessandra, Goel Himanshu, Briones Michael, Huang Mo, Goldmacher Gregory, Liaw Danny, Hammill Adrienne

Abstract excerpt

BACKGROUND: PIK3CA-related overgrowth spectrum (PROS) and Proteus syndrome are associated with mosaic tissue overgrowth of varying severity that commonly presents in childhood. The multicenter, open-label, phase 1/2 MOSAIC study (NCT03094832) was designed to evaluate the clinical efficacy and safety of the selective pan-AKT inhibitor miransertib for participants with PROS or Proteus syndrome. METHODS:...

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Identifiers

PMID: 40713644
DOI: 10.1186/s13023-025-03831-z
PMCID: PMC12296598

Genes

PIK3CA
AKT1

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