Sodium dichloroacetate efficacy and safety
1 evidence topic · 5 sources
Summary
The pivotal SL1009-01 trial enrolled 34 participants. The original observer-reported primary efficacy endpoint was not met. The FDA reported a treatment difference of 0.29, with a 95% confidence interval of -0.62 to 1.21 and p=0.52, favoring placebo. Conference evidence reported reduced plasma lactate with DCA (p=0.006), while the sponsor reported motor improvement during longer treatment, including the open-label extension (p=0.002). These findings do not establish success on the prespecified primary endpoint. The sponsor reported gastrointestinal disorders in 2 participants (5.9%). Reversible peripheral neuropathy is a clinically limiting toxicity of chronic DCA exposure.