Condition: Propionic Acidemia · Sponsor: Mayo Clinic
Propionic acidemia is a genetic metabolic disorder characterized by metabolic acidosis, ketosis, vomiting, lethargy, cognitive impairment, and risk of death. It results from loss of function of the mitochondrial enzyme propionyl-CoA carboxylase and can be due to disease-causing variants in the PCCA gene, leading to accumulation of propionyl-CoA and its toxic metabolites. The purpose of this trial is to evaluate the safety and potential therapeutic benefit of an AAV-based gene therapy for propionic acidemia in patients with genetically confirmed biallelic variants in PCCA.
This description comes directly from the study's public registry record.
Clinical Genomics Clinical Research Team · 507-538-6151 · rstcgresearch@mayo.edu
Wyatt Anians, M.S., CCRP · anians.wyatt@mayo.edu
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| Mayo Clinic | Rochester, Minnesota, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT07643844