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A Phase 1, Randomized, Double Blinded, Placebo Controlled Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Single and Multiple Ascending Doses of ARGX 119 in Healthy Participants

In this study we will investigate how safe the new compound ARGX-119 is and how well it is tolerated when it is used by healthy subjects.We also investigate how quickly and to what extent ARGX-119 is distributed and eliminated from the body. In…

An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single Blind, Dose-Evaluation Phase and a Phase 3 Double-Blind, Placebo-controlled Phase to Assess the Efficacy and Safety of Setrusumab in Subjects with Osteogenesis Imperfecta

This study has been transitioned to CTIS with ID 2024-510919-29-00 check the CTIS register for the current data. OBJECTIVES - Phase 2 (not to be conducted in NL):Phase 2 Primary Objective:- Identify a setrusumab dosing strategy in subjects with OI…

The CanISleepinMS Study:
Effect of cannabidiol (CBD) on sleep quality in patients with multiple sclerosis, a series of 15 randomised, placebo-controlled N-of-1 trials

This study has been transitioned to CTIS with ID 2024-518280-35-00 check the CTIS register for the current data. To investigate the effect of CBD on MS patients with impaired sleep quality.

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

This study has been transitioned to CTIS with ID 2024-515025-28-00 check the CTIS register for the current data. To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in…