Agios Pharmaceuticals, Inc.

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. Participan…

This phase 3 trial tests the drug 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. These children need regular blood transfusions. The study compares mitapivat to a placebo to see if…

This study offers continued access to the drug mitapivat for adults with pyruvate kinase deficiency who completed an earlier Agios-sponsored trial and cannot get the drug commercially. Only 6 participants are enrolled, and the main goal is to monitor side effects. The study does …

This phase 3 study tests whether mitapivat, an oral medication, can safely reduce the number of blood transfusions needed by 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 m…

This phase 3 trial tests whether the drug mitapivat can improve anemia and reduce fatigue in people with alpha- or beta-thalassemia who do not need regular blood transfusions. About 194 participants received either mitapivat or a placebo daily for 24 weeks. The main goal is to se…

This study tests a 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 trial compares mitapivat to a placebo to see if it can raise hemoglobin levels…

This study tests a drug called mitapivat in 286 people with sickle cell disease, ages 16 and older. The goal is to see if mitapivat can raise hemoglobin levels and reduce painful sickle cell crises compared to a placebo. The trial has two phases: first to find the best dose, then…

This Phase 2 trial tests whether tebapivat, an oral tablet, can improve anemia in people with sickle cell disease compared to a placebo. About 59 participants will take the drug or a dummy pill for several weeks. The main goal is to see if hemoglobin levels rise, which could mean…

This study aimed to see if the drug mitapivat could help people with sickle cell disease who also have kidney damage. It planned to measure changes in a key kidney marker over six months. However, the trial was withdrawn before enrolling any participants, so no results are availa…

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 a day for up to …

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 see if it is safe and how the body processes it. No treatment for any disease is being tested here.

This study is a global registry that will follow about 500 people with pyruvate kinase (PK) deficiency, a rare inherited anemia, for up to 9 years. Researchers will collect medical data to better understand the disease's natural history, treatments, and complications. No new drug…