4 results
The objective is to compare the efficacy and safety of intravenous high-dose penicillin G and hydrocortisone versus placebo in combination with riluzole in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS).
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…
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.*…