4 results
CORE PERIODPrimary:* Evaluate the safety and tolerability of up to 24 weeks of AG-348 administration in patients with pyruvate kinase deficiency (PK deficiency).Secondary:* Evaluate the pharmacokinetics (PK) of AG-348 and the metabolite AGI-8702.*…
The purpose of this study is to compare the effectiveness of treatment Arm A and Arm B by measuring the event free survival (EFS) and pathological complete response (pCR). EFS is defined as the time from the first treatment dose that the patient…
The primary objective of the study is to evaluate the efficacy of treatment with AG-348, as assessed by the reduction in transfusion burden.Secondary: The secondary objective of this study is to evaluate the safety of treatment with AG 348.
Primary:• To evaluate the efficacy of treatment with AG-348 compared with placebo in increasing hemoglobin (Hb) concentrationsSecondary:• To evaluate the safety of AG-348• To determine the effect of the study treatment regimens on markers of…