drugset / Trial / NCT07643844
AAVrh10-PCCA Gene Therapy for Propionic Acidemia
Non-randomizedSequentialOpen-labelTreatment
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.
Timeline
- Start
- 2026-06
- Primary completion
- 2032-12
- Completion
- 2033-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAVrh10-PCCA | Gene therapy (AAV / viral vector) | 2e+12 vg/kg | Intravenous |
| Subject | AAVrh10-PCCA | Gene therapy (AAV / viral vector) | 8e+12 vg/kg | Intravenous |
| Subject | AAVrh10-PCCA | Gene therapy (AAV / viral vector) | 3.2e+13 vg/kg | Intravenous |