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Phase 1 Study of AAVrh10-PCCA Gene Therapy in Propionic Acidemia
NCT07643844Phase 1Recruiting
- Sponsor
- Mayo Clinic
- Start
- Jul 2026
- Primary completion
- Dec 2032
- Countries
- 1
01
Overview
Phase 1, open-label trial of AAVrh10-PCCA in propionic acidemia (PA) (3 cohorts), sponsored by Mayo Clinic. Primary endpoint: Incidence of treatment-emergent adverse events at 7 years. Started Jul 2026; status: recruiting.
02
Drugs
AAVrh10-PCCA
03
Conditions
propionic acidemia (PA)
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