7 results
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…
The main objective of this trial is to investigate safety of long term use of inhaled molgramostim by collecting information on the side effects.
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…
The main purpose of this study is to investigate how molgramostim improves the flow of oxygen from the lungs to the blood in patients with aPAP. The effect on lung function as a whole will also be studied, along with any improvement in the patients…
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.*…
To study the safety and efficacy of mitapivat in the treatment of adult subjects with sickle cell disease.