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About this data Sourced primarily from ClinicalTrials.gov — a listing isn't proof of safety or FDA approval.

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.

Not yet reported

Mitapivat

Company
Agios Pharmaceuticals, Inc.
Mechanism
Mechanism not yet reported
Route
Route not yet reported
Recruiting trials
4 recruiting

Efficacy results: Not yet reported

Find a Clinical Trial Near Me

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.

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Treatment type

"Added to GLP-1" means the investigational treatment is being studied on top of an existing GLP-1-based therapy.

Your ZIP code is only used to calculate distance to trial sites — it isn't stored.

Trial details
  • 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-alone
    Other conditions: Anemia
    View 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-alone
    Other conditions: Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
    View 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-alone
    Other conditions: Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
    View 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-alone
    Other conditions: Sickle Cell Disease
    View 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-alone
    Other conditions: Healthy Volunteer
    View 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-alone
    Other conditions: Healthy Volunteer
    View 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-alone
    Other conditions: Pyruvate Kinase Deficiency
    View 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-alone
    Other conditions: Healthy, Japanese, Non-Asian
    View 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 site
    Other conditions: Healthy Volunteers
    View 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-alone
    Other conditions: Pyruvate Kinase Deficiency, Anemia, Hemolytic
    View 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-alone
    Other conditions: Pyruvate Kinase Deficiency, Anemia, Hemolytic
    View 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-alone
    Other conditions: Thalassemia
    View 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-alone
    Other conditions: Healthy Volunteers
    View 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-alone
    Other conditions: Pyruvate Kinase Deficiency
    View 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-alone
    Other conditions: Healthy Volunteers
    View 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-alone
    Other conditions: Sickle Cell Disease
    View 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-alone
    Other conditions: Healthy Volunteers
    View 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-alone
    Other conditions: Healthy Volunteers
    View 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-alone
    Other conditions: Sickle Cell Disease, Hemolytic Anemia
    View 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-alone
    Other conditions: Healthy Volunteers
    View 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-alone
    Other conditions: Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
    View 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-alone
    Other conditions: Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
    View 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 sites
    Other conditions: Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Anemia, Anemia, Hemolytic
    View 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 sites
    Other conditions: Pyruvate Kinase Deficiency
    View 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-alone
    Other conditions: Sickle Cell Disease
    View 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-alone
    Other conditions: Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
    View 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-alone
    Other conditions: Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
    View 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-alone
    Other conditions: Moderate Hepatic Impairment
    View 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 site
    Other conditions: Sickle Cell Anemia, Sickle Cell Thalassemia, Sickle Cell Pain, Hbss, Hbsc, Sickle Beta Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Syndrome Variant
    View 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-alone
    Other conditions: Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, Thalassemia
    View 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-alone
    Other 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-alone
    Other conditions: Sickle Cell Disease, Nephropathy
    View 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-alone
    Other conditions: Healthy Participants
    View 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.