Phase III trial of sodium dichloroacetate for pyruvate dehydrogenase complex deficiency in children.
Peter W Stacpoole, Jose E Abdenur, Jirair K Bedoyan et al.
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In 30 seconds
This Phase III randomized controlled trial evaluated sodium dichloroacetate (DCA) in 34 children with pyruvate dehydrogenase complex deficiency (PDCD). While the primary endpoint, the observer-reported outcomes motor domain score, showed no significant difference (P = 0.512), longer-term treatment indicated a significant improvement in motor function (P = 0.002) and decreased plasma lactate levels (P = 0.006).
Key findings
- No significant difference in ObsROmotor score between DCA and placebo groups (P = 0.512).
- Longer-term treatment showed significant improvement in motor function (P = 0.002).
- DCA decreased plasma lactate by -0.48 (0.82) mmol/L (-20%; P = 0.006).
- Survival was significantly greater than a natural history cohort (log-rank P = 0.027).
Why it matters
The findings suggest that while short-term treatment with DCA may not yield immediate benefits, longer-term administration could improve motor function and reduce plasma lactate levels in children with PDCD, potentially impacting clinical management strategies.