- Ongoing, recruitingPhase I and Phase II (Integrated)- Other
An open-label, multicenter Phase 1/2 dose escalation and expansion study of THOR-707 as a single agent and as a combination therapy in adult subjects with advanced or metastatic solid tumors
This study is testing a new medicine, THOR-707, for people with advanced cancer. It aims to find the safest and most effective dose, and see how well it works alone or with other treatments to shrink tumours and improve health.
For: CancerSpain - Ongoing, recruitingTherapeutic use (Phase IV)
FAMODREP : Interest of Famotidine in Reducing Endothelial Expression of P-selectin in Children With Sickle Cell Disease: Pilot Study, Single-center, Prospective, Non-comparative
This study looks at whether a medicine called famotidine can help children with sickle cell disease. Researchers want to see if famotidine can reduce a specific marker in the blood that is linked to this condition. It's a small, early study to learn more.
For: Sickle Cell DiseaseFrance - 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, recruitingTherapeutic exploratory (Phase II)
A Phase I-II, First-in-Human Study of SKB264 in Patients with Locally Advanced Unresectable /Metastatic Solid Tumors Who are Refractory to Available Standard Therapies
This study is testing a new medicine called SKB264 for people with advanced solid tumours that haven't responded to other treatments. It's a first step to see how safe and effective SKB264 is, and to understand its effects on the body.
For: Metastatic or locally advanced unresectable solid tumors progressing after available standard therapiesSpain · Italy - 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 - RecruitingPHASE1, PHASE2
A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease
This study is testing a new medicine, BMS-986470, for people with sickle cell disease and healthy volunteers. Researchers want to see if it's safe, how it works in the body, and if it helps with sickle cell symptoms. It's an early-stage study.
For: Anemia, Sickle Cell · Healthy VolunteersUnited States · France · United Kingdom