Propionic Acidemia clinical trials at UCLA
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, 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.
Los Angeles, California and other locations
Our lead scientists for Propionic Acidemia research studies include Gerald Lipshutz.