According to a story from PR Newswire, the pharmaceutical company Saol Therapeutics has recently announced that it has completed its enrollment goals for its phase 3 clinical trial. This trial is evaluating Sodium Dichloroacetate (DCA) as a treatment for pyruvate dehydrogenase complex deficiency (PDCD), a rare type of mitochondrial disease. Finding patients for rare disease trials can be difficult, but over 30 patients are now signed up to participate. Saol is focused on developing treatments for CNS disorders and orphan illnesses.

About Pyruvate Dehydrogenase Complex Deficiency (PDCD)

Pyrvuate dehydrogenase deficiency, also called pyruvate dehydrogenase complex deficiency (PDCD), is an uncommon neurodegenerative disorder linked to abnormal metabolism of the mitochondria. The disease is the result of genetic mutations in a component of the pyruvate dehydrogenase complex. Commonly affected genes include PDHA1, DLAT, PDHX, PDP1, and PDHB. Symptoms present in two primary forms: lactic acidosis, which is the metabolic form, and a neurological form. Symptoms include intellectual disability, brain abnormalities, microcephaly, blindness, hypotonia, lethargy, difficulty feeding, breathing problems, thin cerebral tissue, and facial deformities. Males tend to have more severe symptoms. There is no cure, but a ketogenic diet can help slow progression in less severe cases. More effective therapies are desperately needed for PDCD. To learn more about PDCD, click here. 

About The Study

The patients participating in this study will receive daily treatment with DCA. The double-blind study will compare this treatment against placebo for a period of nine months. The treatment will be administered as an oral solution and dosing will be individualized based on a genetic test that determines the drug metabolic rate of each patient.

If the results of this trial are positive, DCA has the potential to be the first medication approved for the treatment of PDCD.