Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome (MOSAIC) (MK-7075-002)

Summary

This is an open label, Phase 1/2 study of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase, catalytic subunit alpha (PIK3CA)-related Overgrowth Spectrum (PROS) and Proteus Syndrome (PS) (MOSAIC).

Timeline

Start

2017-05-16

Primary completion

2022-04-11

Completion

2022-04-11

Publications

• Eng W, Iacobas I, Perkins J, Zampino G, Leoni C, Buonuomo PS, Simonetti A, Goel H, Briones M, Huang M, Goldmacher G, Liaw D, Hammill A. Safety findings from the phase 1/2 MOSAIC study of miransertib for patients with PIK3CA-related overgrowth spectrum or Proteus syndrome. Orphanet J Rare Dis. 2025 Jul 25;20(1):375. doi: 10.1186/s13023-025-03831-z.
• Forde K, Resta N, Ranieri C, Rea D, Kubassova O, Hinton M, Andrews KA, Semple R, Irvine AD, Dvorakova V. Clinical experience with the AKT1 inhibitor miransertib in two children with PIK3CA-related overgrowth syndrome. Orphanet J Rare Dis. 2021 Feb 27;16(1):109. doi: 10.1186/s13023-021-01745-0.

Drugs

Indications