4 results
See protocol P5:The primary objective of this study is to evaluate the efficacy of 12 months of oral ACH-0144471 in participants with C3G or ICMPGNbased on histologic scoring and proteinuriaThe secondary objectives of this study are: * To evaluate…
The Primary objective is to determine whether the administration of ACH 0144471 can increase C3 levels in patients with low C3 levels due to either C3G or IC-MPGN.The secondary objectives are:* To evaluate the safety and tolerability of ACH 0144471…
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the reduction in transfusion burden in pediatric subjects with pyruvate kinase deficiency (PK deficiency) who are regularly transfused.
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