Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

The objective of this research study is to conduct a pivotal phase 3 trial of treatment with the investigational drug dichloroacetate (DCA) in young children with deficiency of the pyruvate dehydrogenase complex (PDC). PDC deficiency (PDCD) is the most common cause of congenital lactic acidosis and is a frequently fatal metabolic disease of childhood for which no proven treatment exists. The investigators predict that DCA represents targeted potential therapy for PDCD because of its ability to increase both the catalytic activity and stability of the enzyme complex. The conclusions of numerous laboratory and clinical investigations are consistent with this postulate and have led to the designation of DCA as an Orphan Product for congenital lactic acidosis by the Food and Drug Administration. A novel Observer reported outcome (ObsRO) survey that is completed by study participant's parent/caregiver, is the efficacy outcome measure. Funding Source - FDA OOPD

NCT Number	NCT02616484
Lead Sponsor	Saol Therapeutics Inc Collaborators: Medosome Biotec LLC
Conditions	Pyruvate Dehydrogenase Complex Deficiency
Enrollment	34 participants
Start Date	2020-07-14
Primary Completion	2025-08-27 (estimated)
Study Completion	2025-08-27 (estimated)
Updated on ClinicalTrials.gov	2025-05-04