About this data
Pipeline and clinical trial information here comes primarily from ClinicalTrials.gov. A listing on ClinicalTrials.gov means a study has been registered — not that a treatment is proven safe, effective, or FDA-approved. Investigational treatments may never be approved. Who can join a trial is decided only by that trial's own study team.
Mitapivat
- Company
- Agios Pharmaceuticals, Inc.
- Mechanism
- Mechanism not yet reported
- Route
- Route not yet reported
- Recruiting trials
- 4 recruiting
Efficacy results: Not yet reported
4 recruiting studies currently listed on ClinicalTrials.gov.
Data source: ClinicalTrials.gov · trial data as of Sep 29, 2026
Find a Clinical Trial Near Me
See recruiting studies for Mitapivat and check eligibility with the study team.
Trial details
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Recruiting NCT NCT07055243
To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.
Not yet reported University Health Network, Toronto 9 participants Est. completion 2027-06-30 1 site Stand-aloneOther conditions: AnemiaView official trial listing → -
Recruiting NCT NCT07506863
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)
Not yet reported Agios Pharmaceuticals, Inc. 54 participants Est. completion 2032-06 4 sites Stand-aloneOther conditions: Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-ThalassemiaView official trial listing → -
Recruiting NCT NCT07517133
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
Not yet reported Agios Pharmaceuticals, Inc. 45 participants Est. completion 2032-03 5 sites Stand-aloneOther conditions: Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-ThalassemiaView official trial listing → -
Recruiting NCT NCT07656415
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
Not yet reported Agios Pharmaceuticals, Inc. 159 participants Est. completion 2030-08 3 sites Stand-aloneOther conditions: Sickle Cell DiseaseView official trial listing → -
Completed NCT NCT02108106
A Phase I Study of AG-348 in Healthy Volunteers
Not yet reported Agios Pharmaceuticals, Inc. 48 participants Est. completion 2014-08 1 site Stand-aloneOther conditions: Healthy VolunteerView official trial listing → -
Completed NCT NCT02149966
A Phase I Study of AG-348 in Healthy Volunteers
Not yet reported Agios Pharmaceuticals, Inc. 48 participants Est. completion 2014-11 1 site Stand-aloneOther conditions: Healthy VolunteerView official trial listing → -
Completed NCT NCT02476916
A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency
Not yet reported Agios Pharmaceuticals, Inc. 52 participants Est. completion 2025-04-02 13 sites Stand-aloneOther conditions: Pyruvate Kinase DeficiencyView official trial listing → -
Completed NCT NCT03250598
Study to Evaluate the Pharmacokinetics, Safety, and QTc Effect of AG-348 in Healthy Subjects of Japanese Origin and Non-Asian Origin
Not yet reported Agios Pharmaceuticals, Inc. 60 participants Est. completion 2017-10-09 1 site Stand-aloneOther conditions: Healthy, Japanese, Non-AsianView official trial listing → -
Completed NCT NCT03397329
Study to Evaluate the Relative Bioavailability and Safety of the AG-348 Tablet and Capsule Formulations in Healthy Adults
Not yet reported Agios Pharmaceuticals, Inc. 26 participants Est. completion 2017-11-13 1 siteOther conditions: Healthy VolunteersView official trial listing → -
Completed NCT NCT03548220
A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
Not yet reported Agios Pharmaceuticals, Inc. 80 participants Est. completion 2020-10-09 46 sites Stand-aloneOther conditions: Pyruvate Kinase Deficiency, Anemia, HemolyticView official trial listing → -
Completed NCT NCT03559699
A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
Not yet reported Agios Pharmaceuticals, Inc. 27 participants Est. completion 2020-11-12 19 sites Stand-aloneOther conditions: Pyruvate Kinase Deficiency, Anemia, HemolyticView official trial listing → -
Active, not recruiting NCT NCT03692052
A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia
Not yet reported Agios Pharmaceuticals, Inc. 20 participants Est. completion 2030-09-30 4 sites Stand-aloneOther conditions: ThalassemiaView official trial listing → -
Completed NCT NCT03703505
A Study to Evaluate the Absorption, Distribution, Metabolism, Excretion and Absolute Bioavailability of AG-348 in Healthy Male Participants Following Administration of a Single Oral Dose of [14C]AG-348 and Concomitant Single Intravenous Microdose of [13C6]AG-348
Not yet reported Agios Pharmaceuticals, Inc. 8 participants Est. completion 2018-06-04 1 site Stand-aloneOther conditions: Healthy VolunteersView official trial listing → -
Completed NCT NCT03853798
Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007
Not yet reported Agios Pharmaceuticals, Inc. 90 participants Est. completion 2024-07-03 42 sites Stand-aloneOther conditions: Pyruvate Kinase DeficiencyView official trial listing → -
Completed NCT NCT03991312
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
Not yet reported Agios Pharmaceuticals, Inc. 28 participants Est. completion 2019-09-10 1 site Stand-aloneOther conditions: Healthy VolunteersView official trial listing → -
Completed NCT NCT04000165
A Dose-Finding Study of AG-348 in Sickle Cell Disease
Not yet reported National Institutes of Health Clinical Center (CC) 17 participants Est. completion 2021-06-21 1 site Stand-aloneOther conditions: Sickle Cell DiseaseView official trial listing → -
Completed NCT NCT04472832
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants
Not yet reported Agios Pharmaceuticals, Inc. 32 participants Est. completion 2020-11-23 1 site Stand-aloneOther conditions: Healthy VolunteersView official trial listing → -
Completed NCT NCT04565678
A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Participants
Not yet reported Agios Pharmaceuticals, Inc. 32 participants Est. completion 2020-12-03 1 site Stand-aloneOther conditions: Healthy VolunteersView official trial listing → -
Active, not recruiting NCT NCT04610866
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
Not yet reported, Not yet reported National Heart, Lung, and Blood Institute (NHLBI) 15 participants Est. completion 2028-02-28 1 site Stand-aloneOther conditions: Sickle Cell Disease, Hemolytic AnemiaView official trial listing → -
Completed NCT NCT04696393
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
Not yet reported Agios Pharmaceuticals, Inc. 26 participants Est. completion 2021-03-19 1 site Stand-aloneOther conditions: Healthy VolunteersView official trial listing → -
Active, not recruiting NCT NCT04770753
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Not yet reported Agios Pharmaceuticals, Inc. 194 participants Est. completion 2028-12 68 sites Stand-aloneOther conditions: Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-ThalassemiaView official trial listing → -
Active, not recruiting NCT NCT04770779
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Not yet reported Agios Pharmaceuticals, Inc. 258 participants Est. completion 2029-06 75 sites Stand-aloneOther conditions: Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-ThalassemiaView official trial listing → -
Terminated NCT NCT04964323
Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)
Agios Pharmaceuticals, Inc. 15 participants Est. completion 2023-06-30 5 sitesOther conditions: Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Anemia, Anemia, HemolyticView official trial listing → -
Completed NCT NCT04995315
Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008
Agios Pharmaceuticals, Inc. 13 participants Est. completion 2023-09-22 3 sitesOther conditions: Pyruvate Kinase DeficiencyView official trial listing → -
Active, not recruiting NCT NCT05031780
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
Not yet reported, Not yet reported Agios Pharmaceuticals, Inc. 286 participants Est. completion 2030-02 91 sites Stand-aloneOther conditions: Sickle Cell DiseaseView official trial listing → -
Active, not recruiting NCT NCT05144256
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
Not yet reported Agios Pharmaceuticals, Inc. 49 participants Est. completion 2029-06 22 sites Stand-aloneOther conditions: Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic AnemiaView official trial listing → -
Active, not recruiting NCT NCT05175105
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
Not yet reported Agios Pharmaceuticals, Inc. 30 participants Est. completion 2030-01 19 sites Stand-aloneOther conditions: Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic AnemiaView official trial listing → -
Completed NCT NCT05610657
A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With Normal Hepatic Function
Not yet reported Agios Pharmaceuticals, Inc. 20 participants Est. completion 2023-07-21 3 sites Stand-aloneOther conditions: Moderate Hepatic ImpairmentView official trial listing → -
Active, not recruiting NCT NCT05675436
Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
National Heart, Lung, and Blood Institute (NHLBI) 6 participants Est. completion 2027-10-27 1 siteOther conditions: Sickle Cell Anemia, Sickle Cell Thalassemia, Sickle Cell Pain, Hbss, Hbsc, Sickle Beta Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Syndrome VariantView official trial listing → -
Enrolling by invitation NCT NCT05777993
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
Not yet reported Agios Pharmaceuticals, Inc. 59 participants Est. completion 2029-08 23 sites Stand-aloneOther conditions: Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, ThalassemiaView official trial listing → -
Not yet recruiting NCT NCT05935202
Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies
Not yet reported EuroBloodNet Association 25 participants Est. completion 2026-12-15 2 sites Stand-aloneOther conditions: Hereditary Red Blood Cell Disorder (Disorder)View official trial listing → -
Withdrawn NCT NCT06286046
A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy
Not yet reported Agios Pharmaceuticals, Inc. Est. completion 2028-12 Stand-aloneOther conditions: Sickle Cell Disease, NephropathyView official trial listing → -
Completed NCT NCT06648824
Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants
Not yet reported Agios Pharmaceuticals, Inc. 20 participants Est. completion 2024-12-03 1 site Stand-aloneOther conditions: Healthy ParticipantsView official trial listing →
Efficacy history
Not yet reported.
Safety
Not yet reported.
Sources
Also known as: Mitapivat, AQVESME, PYRUKYND, AG-348, AG-348 sulfate hydrate, Mitapivat sulfate
Related On The Pen coverage
No related coverage yet.
This drug may be investigational and not yet approved for any use. Whether someone can join a clinical trial is determined only by that trial's own study team — On The Pen does not determine eligibility. Information here can change; always confirm current details on the official ClinicalTrials.gov listing or with the sponsor.
