Condition: Pyruvate Dehydrogenase Complex Deficiency · Sponsor: Jirair Krikor Bedoyan
This is a medical research study to test a medication in patients with a disease called Pyruvate Dehydrogenase Complex (PDC) Deficiency. The medication is triheptanoin, which is currently FDA approved for the treatment of Long-Chain Fatty Acid Oxidation Disorders. Previous research suggests that triheptanoin may also be effective in the treatment PDC Deficiency. This study will investigate the safety and efficacy (how well it works) of triheptanoin in patients with PDC Deficiency.
This description comes directly from the study's public registry record.
Angela Riemenschneider · 412-692-5232 · angela.riemenschneider@chp.edu
Jirair Bedoyan, MD, PhD · bedoyanjk@upmc.edu
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| UPMC Children's Hospital of Pittsburgh | Pittsburgh, Pennsylvania, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06340685