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Not Yet Recruiting
NCT07643844
AAVrh10-PCCA Gene Therapy for Propionic Acidemia
Conditions: Propionic Acidemia
Sex: All
Ages: 6 Months – 2 Years
Healthy volunteers: No
Phase: PHASE1
Enrollment: 9
Sponsor: Mayo Clinic
Location: Mayo Clinic Rochester Minnesota
Summary
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.
Eligibility Criteria
Inclusion Criteria:
* Age six months to 2 years of age at day of vector infusion. For those \
Source: ClinicalTrials.gov (NCT07643844). StuddyBuddy aggregates publicly available trial information.
