5 results
To assess which treatment leads to the best patient outcome on the short term (1 year) and the long term (5 years)
In this study we will investigate how safe the compound leriglitazone is and how well it is tolerated when it is used by healthy participants.Apart from extensive laboratory and animal testing, leriglitazone has been used in the past both in healthy…
Study AG348-C-018 is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study designed to demonstrate the clinical efficacy and safety of mitapivat in subjects with α- or β-TDT. The primary objective of the study is to compare the…
Study AG348-C-017 is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study designed to demonstrate the clinical efficacy and safety of mitapivat in subjects with α- or β NTDT. The primary objective of the study is to compare the…
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in pediatric subjects with PK deficiency who are not regularly transfused