SMO or PTCH-1 (Hedgehog)

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All lines of therapy

Vismodegib monotherapy

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Study Evidence
Hainsworth et al. 2018 (MyPathway) Phase 2a

Eligibility criteria

  • SMO p.W535L or other nonsynonymous mutations, or PTCH-1 loss-of-function mutations

Targeted therapy

Continued indefinitely


  1. MyPathway: Hainsworth JD, Meric-Bernstam F, Swanton C, Hurwitz H, Spigel DR, Sweeney C, Burris H, Bose R, Yoo B, Stein A, Beattie M, Kurzrock R. Targeted therapy for advanced solid tumors on the basis of molecular profiles: results from MyPathway, an open-label, phase IIa multiple basket study. J Clin Oncol. 2018 Feb 20;36(6):536-542. Epub 2018 Jan 10. link to original article contains verified protocol PubMed