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?
Everyone