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Mitapivat-matching placebo

DRUG3 trials

Sponsors

Agios Pharmaceuticals, Inc.

Conditions

Pediatric Hemolytic AnemiaPediatric Pyruvate Kinase DeficiencySickle Cell Disease

Phase 2

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
Active, not recruitingNCT05031780
Agios Pharmaceuticals, Inc.Sickle Cell Disease
Start: 2022-02-11End: 2030-02-01Updated: 2026-06-23

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
Active, not recruitingNCT05144256
Agios Pharmaceuticals, Inc.Pediatric Hemolytic Anemia, Pediatric Pyruvate Kinase Deficiency
Start: 2022-06-08End: 2029-06-01Updated: 2026-08-27
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 recruitingNCT05175105
Agios Pharmaceuticals, Inc.Pediatric Hemolytic Anemia, Pediatric Pyruvate Kinase Deficiency
Start: 2022-06-06End: 2030-01-01Updated: 2026-08-27

Related Papers

Efficacy and safety of mitapivat in pediatric patients with pyruvate kinase deficiency who are not regularly transfused: Results from the Phase 3, global, randomized, double-blind, placebo-controlled ACTIVATE-Kids trial
Blood2025-11-03
Safety and efficacy of mitapivat in sickle cell disease (RISE UP): results from the phase 2 portion of a global, double-blind, randomised, placebo-controlled trial.
2024-12-044 citations
Dual Activation of PKR and PKM2 Reduced the Development of Fibrosis and Iron Deposition in a Sickle Cell Disease Nephropathy Mouse Model
Blood2024-11-05
One-year safety and efficacy of mitapivat in sickle cell disease: follow-up results of a phase 2, open-label study
Blood Advances2023-11-0720 citations
A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study of Mitapivat in Patients with Sickle Cell Disease: RISE UP Phase 2 Results
Blood2023-11-025 citations
A Patient-Centric Approach to Sickle Cell Disease Clinical Trials: Integrating Patient Perspectives in the RISE UP Phase 2/3 Trial of Mitapivat for Informed Protocol Design and Associated Patient Community Benefit
Blood2023-11-021 citations
One-Year Safety and Efficacy of Mitapivat in Sickle Cell Disease: Follow-up Results of a Phase 2, Open-Label Study
Blood2023-11-02
Pyruvate Kinase Thermostability Is Associated with Red Blood Cell Adhesion, Deformability and Oxygen Affinity in Patients with Sickle Cell Disease
Blood2023-11-02
<i>Ex Vivo</i> Treatment By Mitapivat, an Allosteric Pyruvate Kinase Activator, Reduced Oxidative Stress and Promoted Terminal Erythropoiesis in a Severe Hemolytic Anemia Patient Due to Krϋppel-like Factor 1 Mutations
Blood2023-11-02
ACTIVATE-Kids: Mitapivat in Children with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused
Blood2022-11-153 citations
ACTIVATE-KidsT: Mitapivat in Children with Pyruvate Kinase Deficiency Who Are Regularly Transfused
Blood2022-11-152 citations
A PHASE 2/3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF MITAPIVAT IN PATIENTS WITH SICKLE CELL DISEASE
Hematology Transfusion and Cell Therapy2022-10-01
PI-05: PHARMACODYNAMIC EFFECTS OF AG-946, A HIGHLY POTENT NOVEL ACTIVATOR OF PYRUVATE KINASE, IN EX VIVO TREATMENT OF RED BLOOD CELLS FROM SICKLE CELL DISEASE PATIENTS
HemaSphere2022-08-01
P1547: ACTIVATE-KIDS: MITAPIVAT IN CHILDREN WITH PYRUVATE KINASE DEFICIENCY WHO ARE NOT REGULARLY TRANSFUSED
HemaSphere2022-06-012 citations
P1546: ACTIVATE-KIDST: MITAPIVAT IN CHILDREN WITH PYRUVATE KINASE DEFICIENCY WHO ARE REGULARLY TRANSFUSED
HemaSphere2022-06-012 citations
P1492: PHARMACODYNAMIC EFFECTS OF AG-946, A HIGHLY POTENT NOVEL ACTIVATOR OF PYRUVATE KINASE, IN EX VIVO TREATMENT OF RED BLOOD CELLS FROM SICKLE CELL DISEASE PATIENTS
HemaSphere2022-06-011 citations
PB2247: PHARMACOKINETIC MODELING AND SIMULATION TO SUPPORT MITAPIVAT DOSE SELECTION USED IN PEDIATRIC PHASE III STUDIES
HemaSphere2022-06-01
P107: A PHASE 2/3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF MITAPIVAT IN PATIENTS WITH SICKLE CELL DISEASE
HemaSphere2022-01-01