Agios Pharmaceuticals, Inc.

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 tested an oral medication called AG-348 in 20 adults with a form of thalassemia that doesn't require regular blood transfusions. The main goal was to see if the pill could safely and effectively raise hemoglobin levels, which carry oxygen in the blood. Participants too…

This clinical trial is testing whether an oral medication called mitapivat can help people with sickle cell disease. Researchers want to see if the drug increases healthy hemoglobin levels in the blood and reduces painful crises compared to a placebo. The study involves 286 parti…

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 tested whether an oral medication called mitapivat could improve anemia and reduce fatigue in adults with non-transfusion-dependent thalassemia, a genetic blood disorder. About 194 participants were randomly assigned to receive either mitapivat or a placebo pill for 24…

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 testing whether a drug called mitapivat can help children with pyruvate kinase deficiency (PK deficiency), a rare genetic disorder that destroys red blood cells, rely less on regular blood transfusions. About 49 children, aged 1 to 18, who currently need frequent tr…

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…