8 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 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…
To characterize the safety and tolerability of ascending single oral doses (part A) and ascending repeated oral doses (part B) of GM-1020 and the effect of food on single dose GM-1020 (part C) in healthy volunteers.
Objectives: Primary:• To evaluate the long-term safety and tolerability of AG-348Secondary:• To evaluate the long-term efficacy of AG-348• To evaluate the efficacy of AG-348 in increasing hemoglobin (Hb) concentrations in subjects who previously…
To study the safety and efficacy of mitapivat in the treatment of adult subjects with sickle cell disease.
The objective of this study is to investigate the safety and effectiveness of different combinations of cancer immunotherapies compared to either Nivolumab or Ipilimumab, as determined by comparing the Overall Response Rate, at 24 weeks in patients…
To compare the 120 mg once daily (QD) dose and 200 mg QD dose of MK*6482with respect to objective response rate (ORR) based on Response Criteria in Solid Tumors (RECIST) 1.1 as assessed by blinded independent central review (BICR).