DCA Trial in PDC Deficiency

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We are studying an investigational drug called sodium dichloroacetate (DCA). Investigational means that the study drug has not been approved by the US Food and Drug Administration (FDA) and that its use in this study is experimental. This a purpose of this research study is to determine whether DCA is a helpful treatment for patient with pyruvate dehydrogenase complex (PDC) deficiency (PDCD).

Eligibility & Criteria

IRB #:
17-014294
Official Title:
Trial of DCA in PDC Deficiency
Study Phase:
Phase III
Eligible Age Range:
6 Months - 17 Years
Gender:
All
Study Categories:
Leader
Rebecca D. Ganetzky

Rebecca D. Ganetzky, MD