Mitapivat Clinical Trials

Hipa.ai Research · Source: ClinicalTrials.gov / AACT

Synced daily from ClinicalTrials.gov via AACT. Last sync: June 18, 2026.

Total Trials

Recruiting

Completed

1,219

Total Enrollment

States

Mitapivat Clinical Trials

Sortable list of all 19 Mitapivat trials — recruiting status, pivotal acronyms, indication grouping, NCT links.

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Mitapivat History and Updates

Every FDA approval, label revision, recall, trial milestone, and pivotal publication for Mitapivat — sourced from openFDA, ClinicalTrials.gov, and PubMed.

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Recent Mitapivat updates

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Aqvesme (mitapivat) Gains FDA Supplemental Approval for Efficacy· Dec 23, 2025
Mitapivat Pyruvate Kinase Deficiency Extension Study Posts Safety Results· Nov 18, 2025
Mitapivat Shows Significant Transfusion Reduction in Thalassemia Trial· May 25, 2025
Mitapivat Phase 3 Trial for Pediatric PKD Completes Primary Study· Dec 13, 2024
Mitapivat Phase 3 Trial for Pediatric PKD Completes Primary Study· May 3, 2024
Mitapivat Phase 3 for Transfusion-Dependent Thalassemia Reduces Transfusion…· Apr 11, 2024
Mitapivat Phase 3 for Thalassemia Hits Primary Hemoglobin Response Endpoint· Nov 13, 2023

What Is Mitapivat?

Mitapivat is a medication being investigated for its role in treating certain blood disorders. It works by activating pyruvate kinase, an enzyme crucial for the energy metabolism of red blood cells. By enhancing the function of this enzyme, Mitapivat aims to improve red blood cell health and reduce symptoms associated with conditions where these cells are not functioning properly.

Mitapivat is currently under study for various conditions, primarily those affecting red blood cells, such as Pyruvate Kinase Deficiency, Sickle Cell Disease, and different forms of Thalassemia. Clinical trials are exploring its potential to address the underlying issues in these disorders, which can lead to anemia and other complications. A total of 19 trials involving 1,219 participants have been conducted or are ongoing, with the first trial starting in 2019.

Uses and Conditions Under Study

Mitapivat is being investigated across a range of conditions, primarily focusing on inherited red blood cell disorders. These conditions often involve issues with red blood cell production, function, or lifespan, leading to anemia and other health problems.

Pyruvate Kinase Deficiency (PKD): This rare genetic disorder affects red blood cells, causing them to break down prematurely (hemolytic anemia). Mitapivat, as a pyruvate kinase activator, is being studied to improve red blood cell function and reduce anemia in both adult and pediatric patients. A total of 4 trials are investigating Mitapivat for PKD.
Thalassemia: This group of inherited blood disorders results in abnormal hemoglobin production, leading to anemia. Mitapivat is being explored for its potential to improve red blood cell health in various forms, including Transfusion-dependent Beta-Thalassemia, Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia, and Transfusion-dependent Alpha-Thalassemia. There are 8 trials studying Mitapivat for different types of Thalassemia.
Sickle Cell Disease (SCD): SCD is a genetic blood disorder characterized by abnormally shaped red blood cells that can block blood flow, causing pain and organ damage. Mitapivat is being studied in 3 trials to see if it can improve red blood cell function and reduce the severity of symptoms in patients with SCD.
Hemolytic Anemia: This condition occurs when red blood cells are destroyed faster than they can be made, leading to anemia. Mitapivat is being investigated for its potential to treat general hemolytic anemia and pediatric hemolytic anemia, with 3 trials focusing on these conditions.
Healthy Volunteers: Mitapivat has also been studied in 4 trials involving healthy volunteers. These studies are typically conducted to understand how the drug is absorbed, distributed, metabolized, and excreted by the body, as well as to assess its safety profile.

Dosing

Mitapivat is available in tablets or granules for oral administration. The specific dosage and form depend on the study and condition being investigated. In clinical trials, Mitapivat has been administered as oral tablets.

For adult patients, investigational dosing often starts at 50 mg taken twice daily (BD or BID). The dose may be increased to a maximum of 100 mg twice daily, depending on the patient's response and tolerability. Some studies have involved patients receiving Mitapivat for an extended period, such as during 56 weeks.

While specific pediatric doses are not detailed, Mitapivat is being studied in pediatric populations for conditions like Pyruvate Kinase Deficiency and Hemolytic Anemia. The dosing regimens for children are carefully determined within clinical trials to ensure safety and efficacy for younger patients.

Side Effects

In clinical trials involving 301 patients taking Mitapivat, the most common side effects included:

Headache: 24.9% of patients taking Mitapivat experienced headache, compared to 10.8% on placebo.
Initial insomnia: 14.0% of patients on Mitapivat experienced initial insomnia (difficulty falling asleep), compared to 4.7% on placebo.
Upper respiratory tract infection: 13.3% of patients on Mitapivat experienced an upper respiratory tract infection, compared to 12.2% on placebo.
Fatigue: 10.0% of patients on Mitapivat experienced fatigue, compared to 4.1% on placebo.
Diarrhea: 10.0% of patients on Mitapivat experienced diarrhea, compared to 8.8% on placebo.
Nausea: 9.3% of patients on Mitapivat experienced nausea, compared to 6.8% on placebo.
Middle insomnia: 6.0% of patients on Mitapivat experienced middle insomnia (difficulty staying asleep), compared to 2.7% on placebo.

Some side effects occurred less frequently in patients taking Mitapivat compared to placebo. For example, pyrexia (fever) was reported by 7.0% of patients on Mitapivat versus

Currently Recruiting Trials

Patients interested in contributing to medical research for Mitapivat have a few opportunities to participate in ongoing clinical trials. These studies aim to further understand the safety and effectiveness of Mitapivat for various conditions. One such opportunity is presented by NCT07055243, a Phase 2 exploratory study. This trial is designed to evaluate the safety and initial efficacy of Mitapivat in patients diagnosed with red blood cell membranopathies or Congenital Dyserythropoietic Anemia Type II (CDAII), which is a rare subtype of anemia. Sponsored by the University Health Network, Toronto, this study plans to enroll 9 patients from Princess Margaret Hospital who have CDAII. Participants will receive Mitapivat and be monitored for a period of 57 days to assess the drug's impact on their condition. Another important study is NCT05777993, a Phase 4 trial sponsored by Agios Pharmaceuticals, Inc. This study serves a unique purpose: to provide continued access to Mitapivat for participants who have previously completed an Agios-sponsored Mitapivat study and currently do not have commercial access to the drug. This trial focuses on conditions such as Pyruvate Kinase Deficiency and hemolytic anemia. It is designed for a small group of 6 participants, ensuring they can continue to benefit from the treatment.

Where to Participate

Currently, there are no specific physical locations, cities, or states publicly listed for the recruiting Mitapivat trials. This suggests that participation might be managed through specific research centers or potentially through arrangements that do not require a broad geographic listing. For those interested in participating, the eligibility criteria for these studies are specific. Participants must be between 18 and 18 years of age, and individuals of all genders are welcome to join. It is important to note that these trials are not seeking healthy volunteers; they are specifically designed for individuals with particular medical conditions. Children are also not eligible for these particular studies.

Development Timeline

The clinical development journey for Mitapivat began on February 26, 2019, with the initiation of its first clinical trial. Since then, the drug has undergone extensive research, with the latest trial projected to conclude by April 8, 2026. In total, 19 clinical trials have been conducted or are currently underway, involving a significant number of participants, with an overall enrollment of 1,219 individuals across all studies. Agios Pharmaceuticals, Inc. has been the primary driver of Mitapivat's development, sponsoring 16 of these trials. Other institutions, including EuroBloodNet Association, the National Heart, Lung, and Blood Institute (NHLBI), and the University Health Network, Toronto, have also contributed to the research, each sponsoring one study. Mitapivat's research initially explored conditions such as IBS-C and hyperphosphatemia. Over time, the development pipeline significantly expanded, shifting focus to a range of blood disorders. This expansion included various forms of thalassemia, such as Transfusion-dependent Beta-Thalassemia, Non-Transfusion-dependent Alpha-Thalassemia, and Transfusion-dependent Alpha-Thalassemia. The drug's potential was also investigated for Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia, and other hereditary red blood cell disorders, demonstrating a broad and evolving scope of research into its therapeutic applications. The trials have progressed through various phases, including 6 Phase 1 studies, 3 Phase 2 studies, 7 Phase 3 studies, and a Phase 4 study, alongside combined Phase 1/Phase 2 and Phase 2/Phase 3 trials.

Mitapivat Development Timeline

Clinical trial activity from 2019 to 2026.

2026

NCT07517133PHASE3not yet recruiting

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia

45 enrolled

NCT07506863PHASE3not yet recruiting

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)

54 enrolled

NCT06286046PHASE2withdrawn

A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy

0 enrolled

2025

NCT07055243PHASE2recruiting

To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.

9 enrolled

2024

NCT06648824PHASE1completed

Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants

20 enrolled

2023

NCT05935202PHASE2not yet recruiting

Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies

25 enrolled

NCT05777993PHASE4enrolling by invitation

A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study

6 enrolled

NCT05610657PHASE1completed

A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With Normal Hepatic Function

20 enrolled

2022

NCT05144256PHASE3active not recruiting

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

49 enrolled

NCT05175105PHASE3active not recruiting

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

30 enrolled

NCT05031780PHASE2/PHASE3active not recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

286 enrolled

2021

NCT04770753PHASE3active not recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

194 enrolled

NCT04770779PHASE3active not recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

258 enrolled

NCT04696393PHASE1completed

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

26 enrolled

2020

NCT04610866PHASE1/PHASE2active not recruiting

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

15 enrolled

NCT04565678PHASE1completed

A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Participants

32 enrolled

NCT04472832PHASE1completed

A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants

32 enrolled

2019

NCT03991312PHASE1completed

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

28 enrolled

NCT03853798PHASE3completed

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

90 enrolled

Conditions Under Study

Condition	NCT ID	Title	Status	Phase	Enrollment
Healthy Volunteers	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	completed	PHASE1	26
	NCT04565678	A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Participants	completed	PHASE1	32
	NCT04472832	A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants	completed	PHASE1	32
	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	completed	PHASE1	28
Sickle Cell Disease	NCT06286046	A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy	withdrawn	PHASE2	0
	NCT05031780	A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)	active not recruiting	PHASE2/PHASE3	286
	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	active not recruiting	PHASE1/PHASE2	15
Transfusion-dependent Beta-Thalassemia	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 recruiting	PHASE3	54
	NCT04770779	A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)	active not recruiting	PHASE3	258
Non-Transfusion-dependent Alpha-Thalassemia	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 recruiting	PHASE3	45
	NCT04770753	A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)	active not recruiting	PHASE3	194
Non-Transfusion-dependent Beta-Thalassemia	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 recruiting	PHASE3	45
	NCT04770753	A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)	active not recruiting	PHASE3	194
Pediatric Hemolytic Anemia	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	active not recruiting	PHASE3	49
	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	active not recruiting	PHASE3	30
Pediatric Pyruvate Kinase Deficiency	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	active not recruiting	PHASE3	49
	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	active not recruiting	PHASE3	30
Pyruvate Kinase Deficiency	NCT05777993	A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study	enrolling by invitation	PHASE4	6
	NCT03853798	Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007	completed	PHASE3	90
Transfusion-dependent Alpha-Thalassemia	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 recruiting	PHASE3	54
	NCT04770779	A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)	active not recruiting	PHASE3	258
Anemia, Hemolytic	NCT05777993	A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study	enrolling by invitation	PHASE4	6
Healthy Participants	NCT06648824	Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants	completed	PHASE1	20
Hemolytic Anemia	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	active not recruiting	PHASE1/PHASE2	15
Hereditary Red Blood Cell Disorder (Disorder)	NCT05935202	Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies	not yet recruiting	PHASE2	25
Moderate Hepatic Impairment	NCT05610657	A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With Normal Hepatic Function	completed	PHASE1	20
Nephropathy	NCT06286046	A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy	withdrawn	PHASE2	0
Anemia	NCT07055243	To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.	recruiting	PHASE2	9

All Mitapivat Clinical Trials (19)

NCT ID	Title	Status	Phase	Enrollment	Sponsor
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 recruiting	PHASE3	45	Agios Pharmaceuticals, Inc.
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 recruiting	PHASE3	54	Agios Pharmaceuticals, Inc.
NCT06286046	A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy	withdrawn	PHASE2	0	Agios Pharmaceuticals, Inc.
NCT07055243	To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.	recruiting	PHASE2	9	University Health Network, Toronto
NCT06648824	Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants	completed	PHASE1	20	Agios Pharmaceuticals, Inc.
NCT05935202	Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies	not yet recruiting	PHASE2	25	EuroBloodNet Association
NCT05777993	A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study	enrolling by invitation	PHASE4	6	Agios Pharmaceuticals, Inc.
NCT05610657	A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With Normal Hepatic Function	completed	PHASE1	20	Agios Pharmaceuticals, Inc.
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	active not recruiting	PHASE3	49	Agios Pharmaceuticals, Inc.
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	active not recruiting	PHASE3	30	Agios Pharmaceuticals, Inc.
NCT05031780	A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)	active not recruiting	PHASE2/PHASE3	286	Agios Pharmaceuticals, Inc.
NCT04770753	A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)	active not recruiting	PHASE3	194	Agios Pharmaceuticals, Inc.
NCT04770779	A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)	active not recruiting	PHASE3	258	Agios Pharmaceuticals, Inc.
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	completed	PHASE1	26	Agios Pharmaceuticals, Inc.
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	active not recruiting	PHASE1/PHASE2	15	National Heart, Lung, and Blood Institute (NHLBI)
NCT04565678	A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Participants	completed	PHASE1	32	Agios Pharmaceuticals, Inc.
NCT04472832	A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants	completed	PHASE1	32	Agios Pharmaceuticals, Inc.
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	completed	PHASE1	28	Agios Pharmaceuticals, Inc.
NCT03853798	Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007	completed	PHASE3	90	Agios Pharmaceuticals, Inc.