New hope for rare blood disorder: experimental drug shows promise in phase 2 trial
NCT ID NCT02476916
First seen May 13, 2026 · Last updated May 16, 2026 · Updated 1 time
Summary
This study tested a drug called mitapivat (AG-348) in 52 adults with pyruvate kinase deficiency, a rare genetic condition that causes red blood cells to break down too quickly, leading to anemia. The goal was to see if different doses of the drug are safe and can help control the disease by improving hemoglobin levels. Participants took the drug by mouth, and researchers monitored side effects and blood counts over time.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Boston Children's Hospital
Boston, Massachusetts, 02215, United States
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Central Pennsylvania Clinic
Belleville, Pennsylvania, 17004, United States
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Children Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104, United States
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Hammersmith Hospital
London, W12 0NN, United Kingdom
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Hôpital Henri Mondor
Créteil, Île-de-France Region, 94010, France
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Hôpital Saint-Vincent de Paul
Lille, Nord, 59000, France
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New York Presbyterian Hospital- Weil Cornell Medical College
New York, New York, 10065, United States
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Stanford University
Palo Alto, California, 94304, United States
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UOC Oncoematologia Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
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Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
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University Health Network
Toronto, Ontario, M5G 2C4, Canada
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University of Utah
Salt Lake City, Utah, 84113, United States
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Wayne State University School of Medicine - Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
Conditions
Explore the condition pages connected to this study.