Propionic Acidemia clinical trials at UCLA
2 research studies open to eligible people
mRNA-3927 in Participants With Propionic Acidemia
open to all eligible people
This First-in-Human (FIH) Phase 1/2 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 genetically confirmed propionic acidemia (PA). After establishing a dose with acceptable safety and pharmacodynamic (PD) response in a Dose Optimization Group (Part 1) in participants ≥1 year of age, additional participants will be enrolled into the study in a Dose Expansion Group (Part 2) to allow for further characterization of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (<1 year of age).
Los Angeles, California and other locations
Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
open to eligible people ages 1 year and up
The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).
Los Angeles, California and other locations
Our lead scientists for Propionic Acidemia research studies include Gerald S. Lipshutz, MD.
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