Agios Pharmaceuticals, Inc.

This study tests an experimental drug called mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic disorder that causes red blood cells to break down too quickly. The goal is to see if mitapivat can safely reduce the number of blood transfusions these…

This study tested an experimental drug called AG-348 in 20 adults with a form of thalassemia that does not require regular blood transfusions. The goal was to see if the drug could safely raise hemoglobin levels, which carry oxygen in the blood. Participants took the pill twice d…

This study offers ongoing access to the drug mitapivat for 6 adults with pyruvate kinase deficiency, a rare genetic blood condition causing anemia. Participants must have completed a prior Agios-sponsored mitapivat study and be unable to get the drug commercially. The main goal i…

This study aimed to test the drug mitapivat in people with sickle cell disease who also have kidney damage (nephropathy). The goal was to see if mitapivat could reduce protein in the urine, a sign of kidney stress. The trial was withdrawn before any participants were enrolled, so…

This study tests an oral drug called mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic anemia that causes red blood cells to break down too quickly. The goal is to see if the drug can raise hemoglobin levels and reduce the need for blood transfusi…

This study tests a drug called mitapivat in 286 people with sickle cell disease to see if it can raise hemoglobin levels and reduce painful sickle cell crises. Participants will receive either the drug or a placebo, and researchers will monitor safety and effectiveness over time.…

This study tested a drug called mitapivat in 194 people with alpha- or beta-thalassemia who do not need regular blood transfusions. The goal was to see if the drug could raise hemoglobin levels and reduce fatigue compared to a placebo. Participants took the drug or placebo daily …

This study tests an experimental drug called AG-946 in about 87 adults with anemia due to lower-risk myelodysplastic syndromes (MDS). The goal is to see if the drug can raise hemoglobin levels or help people go without blood transfusions for at least 8 weeks. Participants receive…

This study tests a new drug, tebapivat, to see if it can improve anemia in people with sickle cell disease. About 59 adults will receive either tebapivat or a placebo pill. The main goal is to measure increases in hemoglobin and check for side effects.

This phase 3 study tests whether mitapivat, an oral drug, can safely reduce the need for blood transfusions in adults with transfusion-dependent alpha- or beta-thalassemia. Participants receive either mitapivat or a placebo for 48 weeks. The main goal is to see if more people on …

This study is a long-term registry for people with pyruvate kinase deficiency, a rare red blood cell disorder. It will collect health data from about 500 participants worldwide over up to 9 years. The goal is to better understand how the disease progresses, how it is treated, and…

This early-stage study is testing a single injection of a new drug called AG-236 in 24 healthy adults aged 18 to 55. The main goal is to check for side effects and see how the drug moves through the body. No treatment for any disease is being tested here.