Mitapivat (AG-348) Clinical Trials

Size	Price	Qty
5mg	USD 147
25mg	USD 390

Clinical Trials

Numerous clinical trials spanning Phase 1 through Phase 3 evaluate Mitapivat across various hereditary erythrocyte disorders and associated complications, including pyruvate kinase deficiency, pediatric hemolytic anemia, sickle cell disease, alpha- and beta-thalassemia, and moderate hepatic impairment. Sponsored by industry entities such as Agios Pharmaceuticals Inc. alongside major research institutions like the National Heart, Lung, and Blood Institute and EuroBloodNet Association, these protocols demonstrate recruitment statuses ranging from actively recruiting and completed to active, not recruiting and withdrawn.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT07656415	RECRUITING	Sickle Cell Disease	Agios Pharmaceuticals, Inc.	2026-07-21	PHASE3
NCT05777993	ENROLLING_BY_INVITATION	Pyruvate Kinase Deficiency; Anemia, Hemolytic; Sickle Cell Disease; Thalassemia	Agios Pharmaceuticals, Inc.	2023-07-06	PHASE4
NCT07506863	RECRUITING	Transfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia	Agios Pharmaceuticals, Inc.	2026-09	PHASE3
NCT07517133	RECRUITING	Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia	Agios Pharmaceuticals, Inc.	2026-09	PHASE3
NCT04610866	ACTIVE_NOT_RECRUITING	Sickle Cell Disease; Hemolytic Anemia	National Heart, Lung, and Blood Institute (NHLBI)	2020-12-09	PHASE1; PHASE2
NCT06286046	WITHDRAWN	Sickle Cell Disease; Nephropathy	Agios Pharmaceuticals, Inc.	2026-03	PHASE2
NCT07055243	RECRUITING	Anemia	University Health Network, Toronto	2025-06-26	PHASE2
NCT05031780	ACTIVE_NOT_RECRUITING	Sickle Cell Disease	Agios Pharmaceuticals, Inc.	2022-02-11	PHASE2; PHASE3
NCT05935202	NOT_YET_RECRUITING	Hereditary Red Blood Cell Disorder (Disorder)	EuroBloodNet Association	2023-12-21	PHASE2
NCT05175105	ACTIVE_NOT_RECRUITING	Pediatric Pyruvate Kinase Deficiency; Pediatric Hemolytic Anemia	Agios Pharmaceuticals, Inc.	2022-06-06	PHASE3
NCT06648824	COMPLETED	Healthy Participants	Agios Pharmaceuticals, Inc.	2024-10-17	PHASE1
NCT03853798	COMPLETED	Pyruvate Kinase Deficiency	Agios Pharmaceuticals, Inc.	2019-03-21	PHASE3
NCT05144256	ACTIVE_NOT_RECRUITING	Pediatric Pyruvate Kinase Deficiency; Pediatric Hemolytic Anemia	Agios Pharmaceuticals, Inc.	2022-06-08	PHASE3
NCT05675436	Enrolling by invitation	Sickle Cell Anemia\|Sickle Cell Thalassemia\|Sickle Cell Pain\|Hbss\|Hbsc\|Sickle Beta Thalassemia\|Sickle Beta Zero Thalassemia\|Sickle Cell Syndrome Variant	National Heart Lung and Blood Institute (NHLBI)\|National Institutes of Health Clinical Center (CC)	2024-05-15	--
NCT04770779	ACTIVE_NOT_RECRUITING	Transfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia	Agios Pharmaceuticals, Inc.	2021-11-30	PHASE3
NCT05935202	Not yet recruiting	Hereditary Red Blood Cell Disorder (Disorder)	EuroBloodNet Association	2023-12-21	Phase 2
NCT04770753	ACTIVE_NOT_RECRUITING	Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia	Agios Pharmaceuticals, Inc.	2021-12-20	PHASE3
NCT05610657	COMPLETED	Moderate Hepatic Impairment	Agios Pharmaceuticals, Inc.	2023-01-10	PHASE1
NCT05610657	Completed	Moderate Hepatic Impairment	Agios Pharmaceuticals Inc.	2023-01-10	Phase 1
NCT05175105	Recruiting	Pediatric Pyruvate Kinase Deficiency\|Pediatric Hemolytic Anemia	Agios Pharmaceuticals Inc.	2022-06-06	Phase 3
NCT05144256	Active not recruiting	Pediatric Pyruvate Kinase Deficiency\|Pediatric Hemolytic Anemia	Agios Pharmaceuticals Inc.	2022-06-08	Phase 3
NCT05031780	Recruiting	Sickle Cell Disease	Agios Pharmaceuticals Inc.	2022-02-11	Phase 2\|Phase 3
NCT04696393	COMPLETED	Healthy Volunteers	Agios Pharmaceuticals, Inc.	2021-01-06	PHASE1
NCT04565678	COMPLETED	Healthy Volunteers	Agios Pharmaceuticals, Inc.	2020-09-21	PHASE1
NCT04472832	COMPLETED	Healthy Volunteers	Agios Pharmaceuticals, Inc.	2020-06-17	PHASE1
NCT03991312	COMPLETED	Healthy Volunteers	Agios Pharmaceuticals, Inc.	2019-06-20	PHASE1

(data from https://clinicaltrials.gov, updated on 2026-08-04)

Check the Mitapivat (AG-348) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Mitapivat functions as a small-molecule allosteric activator of pyruvate kinase, binding to the enzyme to enhance glycolytic pathway flux, restore adenosine triphosphate production, and reduce toxic upstream metabolite accumulation. This restoration of erythrocyte energy metabolism enhances red blood cell integrity and survival, directly addressing the underlying pathology in clinical trial indications such as pyruvate kinase deficiency, sickle cell disease, and thalassemia.

Appendix RUO and cGMP Quality Standards

Quality Dimension	RUO (Research Use Only)	cGMP (Current Good Manufacturing Practice)
Clinical Applicability	Prohibited in human clinical trials or medical diagnostics.	Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status	Non-regulated grade; exempt from drug manufacturing laws.	Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment	Unclassified analytical or research laboratories.	Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control	Basic purity and activity testing.	Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation	Basic equipment calibration; no process validation required.	Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance	Vendor self-declared without required formal QMS.	Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact	High risk of IND rejection if used as a critical raw material.	Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).