NCT02435173 The purpose of this study is to assess the effectiveness, tolerability and pharmacokinetics of CDZ173 in patients with APDS/PASLI (activated phosphoinositide 3-kinase delta syndrome/ p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency).
NCT02859727 This study is an open-label extension and enrollment is open to patients who have completed study CCDZ173X2201 (NCT02435173). The purpose of this study is to obtain long term safety, tolerability, efficacy and pharmacokinetic data.
NCT03138811 The purpose of this study is to investigate the tolerability, pharmacokinetics and pharmacodynamics of CSJ117. The study will determine how patients’ bodies respond to the study drug (the way the body absorbs, distributes, and eliminates the drug).
NCT03334812 The purpose of this study is to investigate the tolerability and preliminary effectiveness of LNA043 administered by intra-articular injection in regenerating the articular cartilage of the knee in patients undergoing autologous chondrocyte implantation.
NCT03205150 The purpose of the study is to investigate whether LIK066 is tolerated and has beneficial effects in reducing liver fat and inflammation in patients with non-alcoholic steatohepatitis (NASH) and increased body weight. The study will measure blood levels of alanine aminotransferase (ALT), which is a test used to detect liver injury.
NCT03374657 The purpose of the study is to investigate whether CPK850 is tolerated in patients with RLBP1 retinitis pigmentosa to explore dose levels throughout the study, and determine if it is effective in improving vision in patients with impaired vision.