5 results
Primary objectives:To assess the bioequivalence of a single-dose TRI as a whole tablet (reference) compared to a crushed and suspended tablet (intervention I).To assess the bioequivalence of a single-dose TRI as a whole tablet (reference) compared…
-To assess the safety and tolerability of CLDN6 CAR-T +/- CLDN6 RNALPX and to assess the comparability of CLDN6 CAR-T from the manualand automated processes-To identify the maximum tolerated dose (MTD)/RP2D for each IMP (i.e. CLDN6 CAR-T +/- CLDN6…
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-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…