Agios Pharmaceuticals, Inc.

This study tested whether the drug AG-348 could safely increase hemoglobin levels in adults with a less severe form of thalassemia who don't regularly need blood transfusions. Twenty participants took the drug twice daily for up to 24 weeks, with the option to continue in a long-…

This Phase 3 trial tested whether an oral medication called mitapivat could improve anemia in adults with non-transfusion-dependent thalassemia. The study involved 194 participants who were randomly assigned to receive either mitapivat or a placebo pill for 24 weeks. The main goa…

This study is testing an oral medication called mitapivat for people with sickle cell disease. The goal is to see if the drug can safely increase healthy hemoglobin levels in the blood and reduce the frequency of painful sickle cell crises. The trial involves about 286 participan…

This study is testing whether an oral medication called mitapivat can help children with pyruvate kinase deficiency (PK deficiency), a rare genetic disorder that destroys red blood cells. Children in the trial, who currently need regular blood transfusions to survive, will take e…

This Phase 3 trial tested whether an oral medication called mitapivat could reduce the need for regular blood transfusions in adults with severe forms of thalassemia. 258 participants who required frequent transfusions were randomly assigned to receive either mitapivat or a place…

This study is testing whether an oral drug called mitapivat is safe and effective for children with pyruvate kinase deficiency (PKD), a rare genetic disorder that causes chronic anemia. About 30 children, aged 1 to 18, who do not regularly need blood transfusions will take either…

This study is testing an investigational drug called AG-946 (tebapivat) for adults with a lower-risk form of myelodysplastic syndromes (MDS), a bone marrow disorder that causes anemia. The main goal is to see if the drug can help patients become independent from regular blood tra…

This study provides continued access to the drug mitapivat for people with pyruvate kinase deficiency who completed earlier research studies. It's for participants who benefited from the drug but can't get it through regular commercial channels. The main goal is to monitor safety…

This study is creating a long-term registry to track people with pyruvate kinase deficiency, a rare inherited anemia. It will follow up to 500 patients for 2 to 9 years to collect information on their health, treatments, and daily life. The goal is to better understand how the di…