A Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia
mRNA-3927 P101
What is the goal of the study?
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 with genetically confirmed propionic acidemia (PA) by identification of variants in the genes encoding for propionyl-CoA carboxylase (PCC) subunit. (PCCA) or subunit (PCCB). There are no approved therapies that address this underlying enzymatic deficiency. mRNA-3927 contains 2 messenger ribonucleic acids (mRNAs) encoding human PCCA and PCCB proteins and is designed to restore functional PCC enzyme by delivering both PCCA and PCCB subunits in a single drug product. The rationale for conducting this FIH study in adult and pediatric participants with PA is based on several tenets: Early intervention is critical to reduce the risk of the devastating sequelae of PA. Few patients with PA have a normal lifespan. Death is often related to complications of end-organ injury that accumulate during the lifetime of patients with PA. Older patients with PA also develop complications due to deficient PCC activity.
Who can participate in the study?
Please contact the study team listed below to learn more.