- Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humans
An open-label, first-in-human, dose-escalation/expansion study of SAR443579 administered as single agent by intravenous infusion in adult and pediatric participants with relapsed or refractory acute myeloid leukemia (R/R AML), B-cell acute lymphoblastic leukemia (B-ALL), high risk-myelodysplasia (HR-MDS), or blastic plasmacytoid dendritic cell neoplasm (BPDCN)
This study is testing a new medicine called SAR443579 for adults and children with certain types of blood cancers that haven't responded to other treatments. It aims to find the right dose and see how well it works and if it's safe.
For: Acute lymphocytic leukaemia- Acute myeloid leukaemia refractory - Myelodysplastic syndrome - Blastic plasmacytoid dendritic cell neoplasiaFrance · Netherlands - 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 - AuthorisedTherapeutic exploratory (Phase II)
The HIt HArd and hiT early in multiple sclerosis trial – HiHat trial
This study, called HiHat, is looking into new treatments for relapsing-remitting multiple sclerosis (MS) in people who have had the condition for less than 10 years. It's investigating if certain medicines can stop MS from getting worse, while making sure they are safe.
For: Patients with relapsing-remitting multiple sclerosis with less than 10 years disease duration.Sweden