4 results
The primary objective of this study is to assess the efficacy of Ecopipam to reduce self-injurious behaviors (SIB) in adults and children with Lesch-Nyhan Disease (LND) in an outpatient setting. The secondary objectives of this study are to assess…
This trial is, along with establishment of the safety, tolerability and pharmacokinetics and -dynamics of CDZ173 in the target population, designed to select the optimal dose to normalize the function of the PI3K protein in patients with APDS/PASLI…
In this study we will compare leniolisib as capsules with leniolisib as tablets. We compare this in terms of how quickly and to what extent each form of leniolisib is absorbed, transported, and eliminated from the body (this is called…
In this study we will investigate how safe the new compound leniolisib is and how well it is tolerated by healthy male participants.We also investigate how quickly and to what extent leniolisib is absorbed, transported, and eliminated from the body…