Table 1 Characteristics of included studies
Trial | Phase | SMO inhibitor | Sample size | No. of MB patients | Disease stage | Endpoints |
|---|---|---|---|---|---|---|
LoRusso 2011 | I | Vismodegib | 68 | 1 | Refractory | Safety and tumor responses |
Gajjar 2013 | I | Vismodegib | 33 | 33 | Refractory or relapsed | Safety and tumor responses |
Rodon 2014 | I | Sonidegib | 103 | 9 | Relapsed | Safety and tumor responses |
Robinson 2015 | II | Vismodegib | 40 | 40 | Refractory or recurrent | Safety and tumor responses |
Kieran 2017 | I and II | Sonidegib | 76 | 55 | Phase I: Progressed Phase II: Recurrent or relapsed | Safety and tumor responses |