Propionic Acidemia clinical trials at UCLA

This First-in-Human (FIH) Phase 1/2 study will evaluate the safety, pharmacological activity, and efficacy of mRNA-3927 in participants 1 year of age and older with genetically confirmed propionic acidemia (PA). This study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the optimal dose of mRNA-3927 in participants with PA. After establishing a dose with acceptable safety and pharmacodynamic (PD) response in a Dose Optimization Part (Part 1), additional participants will be enrolled into the study in a Dose Expansion Part (Part 2) to allow for further characterization of the efficacy, safety, and PD of mRNA-3927.