Patients & Caregivers

Agios Clinical Trials

Agios is committed to developing innovative treatment options that make a meaningful difference in patients’ lives and fundamentally change the way genetically defined diseases are treated. Our leading clinical programs leverage our leadership in PK activation to explore investigational, oral treatment approaches for pyruvate kinase (PK) deficiency, thalassemia and sickle cell disease.

Our ongoing clinical trials are listed below. More information about participating in clinical trials is available here.

DISEASE PROGRAM STUDY PHASE STATUS LEARN MORE
Pyruvate kinase deficiency Mitapivat Phase 3 Study (ACTIVATE) to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) Completed clinicaltrials.gov
Pyruvate kinase deficiency Mitapivat Phase 3 Study (ACTIVATE-T) Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) Completed clinicaltrials.gov
Pyruvate kinase deficiency Mitapivat Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007 Active, not recruiting clinicaltrials.gov
Pyruvate kinase deficiency Mitapivat Phase 2 DRIVE PK Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency Active, not recruiting clinicaltrials.gov
Pyruvate kinase deficiency Pyruvate Kinase Deficiency Global Longitudinal Registry (PEAK Registry) Recruiting clinicaltrials.gov
Thalassemia (NTD) Mitapivat A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion Dependent Alpha- or Beta-Thalassemia (ENERGIZE) Recruiting clinicaltrials.gov
Thalassemia (TD) Mitapivat A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T) Recruiting clinicaltrials.gov
Thalassemia Mitapivat A Phase 2, Open-label, Multicenter Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Subjects With Non-transfusion-dependent Thalassemia Active, not recruiting clinicaltrials.gov
Sickle cell disease Mitapivat A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease Not yet recruiting clinicaltrials.gov
Sickle cell disease Healthy volunteers AG-946 A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease Recruiting clinicaltrials.gov

The safety and efficacy of the agents and uses under investigation have not been established. There is no guarantee that the agents will receive health authority approval or become commercially available in any country for the uses being investigated.