Propionic Acidemia clinical trials at University of California Health
1 research study open to eligible people
open to eligible people ages 1 year and up
This First-in-Human (FIH) Phase 1/2 study will evaluate the safety and pharmacological activity of mRNA-3927 in participants 1 year of age and older with genetically confirmed propionic acidemia (PA). The study is designed to characterize baseline biomarker levels followed by assessment of safety, pharmacokinetics (PK), and pharmacodynamics (PD) of different doses of mRNA-3927 in participants affected by PA as part of the Dose Optimization phase.