5 results
Primary: To assess the proportion of patients with intervention failure at 12 months after dose reduction, defined as patients who have restarted their initial dose due to (expected) loss of major molecular response.
Cognitive and functional abilities will be evaluated using psychometric scales (ie, cognitive subscale of the Alzheimer*s Disease Assessment Scale [ADAS cog11]), assessment of activities of daily living (ie, Amsterdam Instrumental Activities of…
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
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…
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…