Mitapivat
- Generic Name
- Mitapivat
- Brand Names
- Pyrukynd 5 Mg 4-week, Pyrukynd
- Drug Type
- Small Molecule
- Chemical Formula
- C24H26N4O3S
- CAS Number
- 1260075-17-9
- Unique Ingredient Identifier
- 2WTV10SIKH
- Background
Mitapivat is a novel, first-in-class pyruvate kinase activator. It works to increase the activity of erythrocyte pyruvate kinase, a key enzyme involved in the survival of red blood cells. Defects in the pyruvate kinase enzyme in various red blood cells disorders lead to the lack of energy production for red blood cells, leading to lifelong premature destruct...
- Indication
Mitapivat is indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
- Associated Conditions
- Pyruvate Kinase Deficiency Anemia
- Associated Therapies
- -
Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants
- Conditions
- Interventions
- First Posted Date
- 2024-10-18
- Last Posted Date
- 2024-11-19
- Lead Sponsor
- Agios Pharmaceuticals, Inc.
- Target Recruit Count
- 20
- Registration Number
- NCT06648824
- Locations
- 🇺🇸
Fortrea Clinical Research Unit Inc., Dallas, Texas, United States
A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy
- First Posted Date
- 2024-02-29
- Last Posted Date
- 2024-11-14
- Lead Sponsor
- Agios Pharmaceuticals, Inc.
- Target Recruit Count
- 40
- Registration Number
- NCT06286046
A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
- First Posted Date
- 2023-03-21
- Last Posted Date
- 2024-11-18
- Lead Sponsor
- Agios Pharmaceuticals, Inc.
- Target Recruit Count
- 6
- Registration Number
- NCT05777993
- Locations
- 🇨🇦
McMaster Centre for Transfusion Medicine McMaster University, Hamilton, Ontario, Canada
🇯🇵Toho University - Omori Medical Center, Ota-Ku, Japan
🇯🇵Tohoku University Hospital, Sendai, Miyagi, Japan
A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With Normal Hepatic Function
- First Posted Date
- 2022-11-09
- Last Posted Date
- 2023-09-21
- Lead Sponsor
- Agios Pharmaceuticals, Inc.
- Target Recruit Count
- 20
- Registration Number
- NCT05610657
- Locations
- 🇺🇸
Orlando Clinical Research Center (OCRC), Orlando, Florida, United States
🇺🇸Orange County Research Center (OCRC), Tustin, California, United States
🇺🇸Clinical Pharmacology of Miami (CPMI), Miami, Florida, United States
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
- Conditions
- Interventions
- First Posted Date
- 2022-01-03
- Last Posted Date
- 2024-12-18
- Lead Sponsor
- Agios Pharmaceuticals, Inc.
- Target Recruit Count
- 30
- Registration Number
- NCT05175105
- Locations
- 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸Children's Healthcare of Atlanta - Emory, Atlanta, Georgia, United States
🇺🇸Stanford Medicine, Palo Alto, California, United States
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
- Conditions
- Interventions
- First Posted Date
- 2021-12-03
- Last Posted Date
- 2024-12-18
- Lead Sponsor
- Agios Pharmaceuticals, Inc.
- Target Recruit Count
- 49
- Registration Number
- NCT05144256
- Locations
- 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇹🇷Ege University Faculty of Medicine, Izmir, Adana, Turkey
🇺🇸Weill Cornell Medical College, New York, New York, United States
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
- Conditions
- Interventions
- First Posted Date
- 2021-09-02
- Last Posted Date
- 2024-12-20
- Lead Sponsor
- Agios Pharmaceuticals, Inc.
- Target Recruit Count
- 267
- Registration Number
- NCT05031780
- Locations
- 🇺🇸
University of California San Diego, La Jolla, California, United States
🇺🇸UCLA Health, Los Angeles, California, United States
🇺🇸Children's Hospital Oakland, Oakland, California, United States
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
- Conditions
- Interventions
- First Posted Date
- 2021-02-25
- Last Posted Date
- 2024-11-15
- Lead Sponsor
- Agios Pharmaceuticals, Inc.
- Target Recruit Count
- 258
- Registration Number
- NCT04770779
- Locations
- 🇺🇸
Penn Medicine - University of Pennsylvania Health System, Philadelphia, Pennsylvania, United States
🇺🇸Phoenix Children's Hospital, Phoenix, Arizona, United States
🇮🇹Ospedale Pediatrico Microcitemico, Cagliari, Italy
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
- Conditions
- Interventions
- First Posted Date
- 2021-02-25
- Last Posted Date
- 2024-07-11
- Lead Sponsor
- Agios Pharmaceuticals, Inc.
- Target Recruit Count
- 194
- Registration Number
- NCT04770753
- Locations
- 🇧🇬
UMHAT "Sveti Georgi" EAD, Plovdiv, Bulgaria
🇧🇬SHATHD Sofia, Sofia, Bulgaria
🇬🇷Children's Hospital Agia Sophia, National and Kapodistrian University of Athens Medical School, Athens, Greece
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
- Conditions
- Interventions
- First Posted Date
- 2020-11-02
- Last Posted Date
- 2024-12-19
- Target Recruit Count
- 15
- Registration Number
- NCT04610866
- Locations
- 🇺🇸
National Institutes of Health Clinical Center, Bethesda, Maryland, United States
Drug Development Updates
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