Extension Study to Evaluate Long-Term Safety and Clinical Effects of mRNA-3927 in Participants with Propionic Acidemia Previously in the mRNA-3927-P101 Trial

What this Trial Is About

This research aims to evaluate the long-term safety of mRNA-3927 in people with propionic acidemia PA who previously took part in the mRNA-3927-P101 study. It is a phase 12, open-label extension study focusing on monitoring safety and clinical activity over an extended period. Participants will continue receiving mRNA-3927 at the same dose and schedule they had in the earlier study, with dose adjustments possible based on the sponsors recommendation. The study includes two main periods a treatment period where participants receive the study drug, followed by a 90-day follow-up period after the end of treatment. During the study, researchers will monitor participants for adverse events, serious side effects, and any events leading to stopping treatment. They will also track metabolic decompensation events and hospitalizations related to PA over up to eight years. Participants will have regular assessments to evaluate these outcomes and ensure ongoing safety throughout the study duration.

A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study