- recruitingPHASE1, PHASE2
A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
This study looks at the long-term safety and effects of a treatment called mRNA-3927 for propionic acidemia (a rare condition). It's for people who have already taken part in a previous study of this treatment. Researchers want to see how the treatment works over a longer time.
United States · Canada · France - recruiting
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
This study looks at how a medicine called Carglumic Acid helps people with rare conditions called Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) over a long time. It gathers information from patient records and talks to patients and their families.
France · Germany · Italy - recruitingPHASE1, PHASE2
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
This study is testing a new medicine, mRNA-3927, for Propionic Acidemia (PA). We want to understand if it's safe, how well it works, and find the best dose for people with PA, including children and infants.
United States · Canada · France - recruiting
A Retrospective Study to Characterize Participants With Propionic Acidemia
This study looks back at medical records of people with propionic acidemia (PA) to understand their health journey. It gathers information on how the disease affects them, aiming to learn more about this rare condition.
United States · Australia · Canada - authorisedPhase I and Phase II (Integrated)- Other
A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
This study is an extension of a previous trial for Propionic Acidemia. It aims to see how safe and effective a new medicine, mRNA-3927, is over a longer period. Doctors will look closely at any side effects and how patients are doing.
Spain · Netherlands · France