Malignant solid neoplasm, SMO-mutated or PTCH-1-mutated (Hedgehog)

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All lines of therapy

Vismodegib monotherapy

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Regimen

Study Evidence
Hainsworth et al. 2018 (MyPathway) Phase IIa

Eligibility criteria

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

Targeted therapy

Continued indefinitely

References

  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