15 completed, 8 active not recruiting, 3 recruiting, 1 enrolling by invitation. 11 Phase 1, 11 Phase 3, 7 Phase 2, 1 Early Phase 1, 1 Phase 4.
Follow mitapivat: new trials by email Literature Review of mitapivat trials Research Report on mitapivat
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) Mitapivat Matched Placebo, Mitapivat - Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia | Phase 3 | Recruiting | Agios Pharmaceuticals, Inc. | 54 | 2026-09-24 | 2026-10-29 |
| A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia Mitapivat, Placebo Matching Mitapivat - Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia | Phase 3 | Recruiting | Agios Pharmaceuticals, Inc. | 45 | 2026-09-01 | 2026-10-28 |
| A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD) Mitapivat Matched Placebo, Mitapivat - Sickle Cell Disease | Phase 3 | Recruiting | Agios Pharmaceuticals, Inc. | 159 | 2026-07-21 | 2026-10-28 |
| A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy Mitapivat - Sickle Cell Disease, Nephropathy | Phase 2 | Withdrawn | Agios Pharmaceuticals, Inc. | 2026-03-01 | 2026-04-03 | |
| To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII. Mitapivat - Anemia | Phase 2 | Active not recruiting | University Health Network, Toronto | 9 | 2025-10-20 | 2026-10-15 |
| Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants Mitapivat, Midazolam - Healthy Participants | Phase 1 | Completed | Agios Pharmaceuticals, Inc. | 20 | 2024-10-17 | 2025-01-28 |
| Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies Mitapivat sulfate - Hereditary Red Blood Cell Disorder (Disorder) | Phase 2 | Not yet recruiting | EuroBloodNet Association | 25 | 2023-12-21 | 2023-12-26 |
| A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study Mitapivat - Pyruvate Kinase Deficiency, Anemia, Hemolytic | Phase 4 | Enrolling by invitation | Agios Pharmaceuticals, Inc. | 55 | 2023-07-06 | 2026-10-28 |
| A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With Normal Hepatic Function Mitapivat - Moderate Hepatic Impairment | Phase 1 | Completed | Agios Pharmaceuticals, Inc. | 20 | 2023-01-10 | 2023-09-21 |
| A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period Mitapivat, Mitapivat-matching placebo - Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia | Phase 3 | Active not recruiting | Agios Pharmaceuticals, Inc. | 49 | 2022-06-08 | 2026-10-28 |
| A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period Mitapivat, Mitapivat-matching placebo - Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia | Phase 3 | Active not recruiting | Agios Pharmaceuticals, Inc. | 30 | 2022-06-06 | 2026-10-28 |
| A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP) Mitapivat, Mitapivat-matching placebo - Sickle Cell Disease | Phase 2 / Phase 3 | Active not recruiting | Agios Pharmaceuticals, Inc. | 286 | 2022-02-11 | 2026-10-26 |
| A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) Placebo Matching Mitapivat, Mitapivat - Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia | Phase 3 | Active not recruiting | Agios Pharmaceuticals, Inc. | 194 | 2021-12-20 | 2026-10-30 |
| A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) Placebo Matching Mitapivat, Mitapivat - Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia | Phase 3 | Active not recruiting | Agios Pharmaceuticals, Inc. | 258 | 2021-11-30 | 2026-10-28 |
| A Study to Compare the Pharmacokinetics and Safety of Mitapivat 100 mg Tablet Formulation With Mitapivat 2 × 50 mg Tablet Formulation in Healthy Adult Participants Mitapivat tablet - Healthy Volunteers | Phase 1 | Completed | Agios Pharmaceuticals, Inc. | 26 | 2021-01-06 | 2021-04-13 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat Mitapivat - Sickle Cell Disease, Hemolytic Anemia | Phase 1 / Phase 2 | Active not recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 15 | 2020-12-09 | 2026-11-12 |
| A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Participants Mitapivat tablets, Mitapivat coated granules - Healthy Volunteers | Phase 1 | Completed | Agios Pharmaceuticals, Inc. | 32 | 2020-09-21 | 2020-12-10 |
| A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants Placebo for Treatment A, Mitapivat 100 mg, Placebo for Treatment B - Healthy Volunteers | Phase 1 | Completed | Agios Pharmaceuticals, Inc. | 32 | 2020-06-17 | 2021-02-18 |
| A Dose-Finding Study of AG-348 in Sickle Cell Disease AG-348 - Sickle Cell Disease | Early Phase 1 | Completed | National Institutes of Health Clinical Center (CC) | 17 | 2019-07-11 | 2022-07-12 |
| Study to Evaluate the Effect of Multiple Doses of Itraconazole and Rifampin on the Single-Dose Pharmacokinetics of Mitapivat Sulfate (AG-348) in Healthy Adult Participants itraconazole, rifampin, mitapivat sulfate - Healthy Volunteers | Phase 1 | Completed | Agios Pharmaceuticals, Inc. | 28 | 2019-06-20 | 2019-10-23 |
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Pyruvate Kinase Deficiency of Red Cells (7) · Anemia, Sickle Cell (6) · Anemia, Hemolytic (5) · Anemia (3) · Hematologic Diseases (2) · Metabolic Diseases (2) · Thalassemia (2) · Kidney Diseases (1)
Agios Pharmaceuticals, Inc. (25) · EuroBloodNet Association (1) · National Heart, Lung, and Blood Institute (NHLBI) (1) · National Institutes of Health Clinical Center (CC) (1) · University Health Network, Toronto (1)
United States (25) · Canada (12) · Netherlands (10) · United Kingdom (10) · France (9) · Denmark (7) · Italy (7) · Turkey (Türkiye) (7) · Brazil (6) · Germany (6) · Spain (6) · Thailand (5) · Switzerland (4) · Japan (3) · Lebanon (3)
Samuel Agresta, MD, MPH & TM (2) · Swee Lay Thein, M.D. (2)
PMID 31974203, PMID 36594181, PMID 41809202, PMID 30091852, PMID 38330179, PMID 31483964, PMID 35417638, PMID 35576529, PMID 35964609, PMID 35988546, PMID 37254256, PMID 37934880, PMID 37943362, PMID 38450513, PMID 39079928, PMID 39265169, PMID 39644907, PMID 40544857, PMID 40569673, PMID 42721980, PMID 42913682
2018 (2), 2025 (2), 2015 (1), 2017 (1), 2023 (1)