Drug research

Mitapivat

Open a linked trial to see its study details and recorded changes, or check the regulatory events below and follow their sources.

Drug identity checked against registry records

Also recorded as AG-348; AG-348 sulfate hydrate; Mitapivat Sulfate; Mitapivat sulfate

A drug appearing in a clinical trial does not mean it is FDA-approved. Check the original regulatory source for any approval claim.

Included trials

Trial	Sponsor	Phase	Status	Primary completion
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)NCT07656415	AGIO	Phase 3	Recruiting	2029-08Estimated
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 PeriodNCT05144256	AGIO	Phase 3	Active not recruiting	2024-05-03Actual
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 PeriodNCT05175105	AGIO	Phase 3	Active not recruiting	2024-12-13Actual
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)NCT07506863	AGIO	Phase 3	Recruiting	2029-06Estimated
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)NCT04770753	AGIO	Phase 3	Active not recruiting	2023-11-13Actual
Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007NCT03853798	AGIO	Phase 3	Completed	2024-07-03Actual
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent ThalassemiaNCT07517133	AGIO	Phase 3	Recruiting	2029-03Estimated
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)NCT05031780	AGIO	Phase 2, Phase 3	Active not recruiting	2025-10-30Actual
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)NCT04770779	AGIO	Phase 3	Active not recruiting	2024-04-11Actual

A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)

AGIORecruitingPrimary completion: 2029-08 · Estimated

Trial details

Registry ID: NCT07656415
Phase: Phase 3

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

AGIOActive not recruitingPrimary completion: 2024-05-03 · Actual

Trial details

Registry ID: NCT05144256
Phase: Phase 3

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

AGIOActive not recruitingPrimary completion: 2024-12-13 · Actual

Trial details

Registry ID: NCT05175105
Phase: Phase 3

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)

AGIORecruitingPrimary completion: 2029-06 · Estimated

Trial details

Registry ID: NCT07506863
Phase: Phase 3

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

AGIOActive not recruitingPrimary completion: 2023-11-13 · Actual

Trial details

Registry ID: NCT04770753
Phase: Phase 3

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

AGIOCompletedPrimary completion: 2024-07-03 · Actual

Trial details

Registry ID: NCT03853798
Phase: Phase 3

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

AGIORecruitingPrimary completion: 2029-03 · Estimated

Trial details

Registry ID: NCT07517133
Phase: Phase 3

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

AGIOActive not recruitingPrimary completion: 2025-10-30 · Actual

Trial details

Registry ID: NCT05031780
Phase: Phase 2, Phase 3

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

AGIOActive not recruitingPrimary completion: 2024-04-11 · Actual

Trial details

Registry ID: NCT04770779
Phase: Phase 3

Regulatory dates and decisions

No regulatory events match this view.Try another date range or view. Events appear after automated source checks. This does not mean no FDA events have occurred. Dates that a source does not disclose stay undisclosed.