- Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humans
A Phase 1/2, adaptive, open-label, single ascending dose to multiple ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of mRNA-3745 in participants with glycogen storage disease type 1a (GSD1a), followed by an open-label extension
This study is testing a new medicine, mRNA-3745, for Glycogen Storage Disease Type 1a (GSD1a). It's looking at how safe it is, how well people tolerate it, and how it works in the body, starting with low doses and gradually increasing them.
For: Glycogen storage disease type 1a (GSD1a)Spain · Poland · Netherlands - Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humans
A Phase 1/2, First-in-Human, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of mRNA 3210 in Participants with Phenylketonuria
This study is testing a new messenger RNA (mRNA) medicine called mRNA-3210 for people with Phenylketonuria (PKU). Researchers want to see if it's safe, how much of it stays in the body, and how it affects PKU. It's an early-stage study, meaning it's one of the first times it's being given to humans.
For: Participants with PhenylketonuriaFrance · Spain · Italy