BOLT
BOLT showed that sonidegib, a second hedgehog pathway pill, shrinks about four in ten locally advanced basal cell carcinomas at its lower dose, and that the higher dose adds side effects rather than responses.
Overview
BOLT randomised 230 patients with locally advanced or metastatic basal cell carcinoma to sonidegib 200 mg or 800 mg daily in a double-blind design with no placebo arm, the question being dose rather than efficacy against a comparator.
At the 200 mg dose the objective response rate by central review was 43 percent in locally advanced and 15 percent in metastatic disease (Lancet Oncology 2015), with more toxicity and no more responses at 800 mg. Long-term follow-up to 42 months showed durable responses and the same muscle spasm, hair loss and taste disturbance seen with vismodegib.
Sonidegib 200 mg was approved for locally advanced basal cell carcinoma in July 2015 and is used interchangeably with vismodegib; neither has been compared with the other head to head.
- 43 out of 100 people had their tumour shrink with Sonidegib 200 mg.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
- These results apply to the people the trial enrolled: Locally advanced or metastatic basal cell carcinoma: sonidegib 200 mg versus 800 mg daily. People in a different situation may not see the same effect.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
230 participants enrolled.
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Objective response rate, 200 mg (locally advanced)primary | Sonidegib 200 mg | - | 43% | - | - | link |
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