Agios Pharmaceuticals, Inc.

This study tests a drug called tebapivat in 59 people with sickle cell disease to see if it can improve anemia (low red blood cells) better than a placebo. Participants take either tebapivat or a dummy pill, and researchers check for changes in hemoglobin levels and any side effe…

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

This study offers continued access to the drug mitapivat for 6 adults with pyruvate kinase deficiency, a rare genetic blood condition that causes anemia. Participants must have completed a prior mitapivat study and be unable to get the drug commercially. The main goal is to monit…

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 a drug called AG-348 in 20 adults with a type of thalassemia that doesn't 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 daily for up to …

This study tests a drug called mitapivat in 194 adults with alpha- or beta-thalassemia who do not need regular blood transfusions. The goal is to see if mitapivat can raise hemoglobin levels and reduce fatigue compared to a placebo. Participants take the drug or placebo daily for…

This study tests a drug called mitapivat in people with sickle cell disease. The goal is to see if it can raise hemoglobin levels and reduce painful sickle cell crises compared to a placebo. About 286 people aged 16 and older will take part. The trial has two phases: first to fin…

This phase 3 study tests whether mitapivat, an oral medication, can reduce the number of blood transfusions needed by adults with transfusion-dependent alpha- or beta-thalassemia. Participants receive either mitapivat or a placebo, and researchers measure if there is at least a 5…

This study aimed to see if the drug mitapivat could improve kidney function in people with sickle cell disease and kidney damage. It planned to enroll about 0 participants aged 16 and older with specific types of sickle cell disease. The trial was withdrawn before any participant…

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

This study is a long-term registry for people of all ages with pyruvate kinase deficiency, a rare red blood cell disorder. It does not test any new treatments. Instead, researchers will collect health information from doctors and patients over up to 9 years to better understand h…

This early-stage study is testing a single dose of a new drug called AG-236 in 24 healthy adults aged 18 to 55. The main goal is to see if it is safe and how the body processes it. No treatment for any disease is being tested here.