Levocarnitine for vismodegib-associated muscle spasms: a pilot randomized, double-blind, placebo-controlled, investigator initiated trial

Abstract

Smoothened inhibitors (SIs) are a new, targeted therapy for the treatment of advanced basal cell carcinoma (BCC), but their usage is significantly limited by the most common side effect, muscle spasms. Muscle spasms affect up to 80% of users, and are the most common reason for drug discontinuation despite tumor response. Currently, no placebo-controlled trials exist to guide the management of vismodegib-related muscle spasms. The medical literature suggests that the naturally occurring substance, levocarnitine (LC), can improve contractile function of muscle and reduce markers of exercise-induced stress. Hence, we explored the effect of oral LC on vismodegib-related muscle spasms via a double-blind, randomized, placebo-controlled, two-period crossover design.

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