Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
Status:
Recruiting
Trial end date:
2023-05-31
Target enrollment:
Participant gender:
Summary
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
Phase:
Phase 3
Details
Lead Sponsor:
University of Florida
Collaborators:
Columbia University Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) International Pyruvate Dehydrogenase Complex Deficiency Organization Medosome Biotec LLC Saol Therapeutics