- Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humans
A Global, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants with Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency
This study is testing a new medicine, mRNA-3705, for a rare genetic condition called methylmalonic acidemia (MMA). Doctors want to see if the medicine is safe, how the body handles it, and if it helps reduce harmful acid levels in the blood.
For: Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase DeficiencyFrance · Netherlands · Spain - RecruitingPHASE1, PHASE2
An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705
This study looks at the long-term safety and effects of a medicine called mRNA-3705 for people with Methylmalonic Acidemia (MMA). It's for those who have already taken part in earlier studies of this same medicine. We want to see how it works and if it's safe over a longer time.
For: Methylmalonic AcidemiaUnited States · Canada · France