37 completed, 12 active not recruiting, 4 recruiting, 3 terminated. 31 Phase 1, 12 Phase 3, 11 Phase 2, 1 Early Phase 1, 1 Phase 4.
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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 AG-236 in Subjects ≥18 Years of Age With Polycythemia Vera AG-236, Placebo - Polycythemia Vera | Phase 2 / Phase 3 | Not yet recruiting | Agios Pharmaceuticals, Inc. | 245 | 2026-11-01 | 2026-10-13 |
| 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 | 2026-09-28 |
| 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 | 2026-09-01 | 2026-09-28 |
| 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 | 2026-09-28 |
| A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria AG-181 - Phenylketonuria | Phase 1 | Recruiting | Agios Pharmaceuticals, Inc. | 20 | 2026-04-17 | 2026-09-28 |
| 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in Participants With Sickle Cell Disease and Nephropathy Mitapivat - Sickle Cell Disease, Nephropathy | Phase 2 | Withdrawn | Agios Pharmaceuticals, Inc. | 2026-03-01 | 2026-04-03 | |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 |
| A Study to Determine the Safety and Tolerability of AG-236 and How it is Absorbed, Broken Down, and Eliminated From the Body in Healthy Participants AG-236, Placebo - Healthy Participants | Phase 1 | Active not recruiting | Agios Pharmaceuticals, Inc. | 24 | 2025-07-07 | 2026-07-30 |
| A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD) Tebapivat, Tebapivat Matched Placebo - Sickle Cell Disease | Phase 2 | Active not recruiting | Agios Pharmaceuticals, Inc. | 59 | 2025-05-01 | 2026-09-28 |
| A Study to Determine How Tebapivat is Absorbed, Broken Down, and Removed From the Body and the Extent to Which Tebapivat is Made Available in the Body in Healthy Participants [14C]-tebapivat, [13C2,15N3]-tebapivat - Healthy Participants | Phase 1 | Completed | Agios Pharmaceuticals, Inc. | 8 | 2024-12-20 | 2025-03-18 |
| 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 |
| Single and Multiple Ascending Dose Study and Food Effect Study for AG181 AG-181, Placebo - Healthy Volunteers | Phase 1 | Completed | Agios Pharmaceuticals, Inc. | 88 | 2024-02-20 | 2025-12-23 |
| 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. | 59 | 2023-07-06 | 2026-09-29 |
| 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 of Tebapivat (AG-946) in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS) Tebapivat - Myelodysplastic Syndromes | Phase 2 | Active not recruiting | Agios Pharmaceuticals, Inc. | 87 | 2022-11-07 | 2026-09-28 |
| 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 | 2026-09-28 |
| 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 | 2026-08-27 |
| 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 |
| Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms Blood Draw - Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia | Completed | Massachusetts General Hospital | 18 | 2022-02-01 | 2026-05-19 | |
| 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 | 2026-09-28 |
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Pyruvate Kinase Deficiency of Red Cells (12) · Anemia (7) · Anemia, Sickle Cell (7) · Anemia, Hemolytic (6) · Leukemia, Myeloid, Acute (4) · Myelodysplastic Syndromes (4) · Anemia, Hemolytic, Congenital Nonspherocytic (3) · Hematologic Diseases (3) · Hematologic Neoplasms (2) · Lymphoma (2) · Metabolic Diseases (2) · Thalassemia (2) · Anemia, Hemolytic, Congenital (1) · Chondrosarcoma (1) · Glioma (1)
mitapivat (27) · ivosidenib (8) · Itraconazole (2) · Blood Specimen Collection (1) · Lamotrigine (1) · Midazolam (1) · Nivolumab (1) · Omeprazole (1) · Rifampin (1) · enasidenib (1) · vorasidenib (1)
United States (54) · Canada (15) · United Kingdom (15) · France (14) · Netherlands (14) · Italy (12) · Spain (11) · Germany (9) · Denmark (8) · Turkey (Türkiye) (8) · Brazil (6) · Thailand (6) · Czechia (5) · Switzerland (5) · Ireland (4)
Samuel Agresta, MD, MPH & TM (2) · Amir T Fathi, MD (1) · Ciara M Kelly, MBBCh, BAO (1) · Eva Gallagher, VP, Medical Affairs (1) · Gary A Connor, RN (1) · Hanny Al-Samkari, MD (1) · Jason J Luke, MD, PhD (1) · Sam Agresta, MD (1) · Samuel Agresta, MD, MPH (1) · Samuel Agresta, MD, MPH &TM (1)
PMID 31974203, PMID 36594181, PMID 30091852, PMID 38330179, PMID 41809202, PMID 29243965, PMID 29950729, PMID 31483964, PMID 32145771, PMID 32648303, PMID 33394722, PMID 34427990, PMID 34470054, PMID 35417638, PMID 35576529, PMID 35964609, PMID 35988546, PMID 36958777, PMID 37254256, PMID 37354069, PMID 37934880, PMID 37943362, PMID 38446419, PMID 38507688, PMID 39265169, PMID 39644907, PMID 40544857, PMID 40569673, PMID 40778139, PMID 41138165, PMID 42053456, PMID 42721980, PMID 42913682
2025 (3), 2018 (2), 2015 (1), 2017 (1), 2023 (1)