Promising new drug shows hope for rare anemia patients

NCT ID NCT02476916

Completed Disease control Sponsor: Agios Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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 help control the disease. Participants took the drug by mouth, and researchers monitored side effects and blood health over time.

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Conditions

The condition(s) this trial relates to.

hemolytic anemia pyruvate kinase deficiency of red cells

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PYRUVATE KINASE DEFICIENCY

Contacts and locations

Locations

Boston Children's Hospital

Boston, Massachusetts, 02215, United States
Central Pennsylvania Clinic

Belleville, Pennsylvania, 17004, United States
Children Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, 19104, United States
Hammersmith Hospital

London, W12 0NN, United Kingdom
Hôpital Henri Mondor

Créteil, Île-de-France Region, 94010, France
Hôpital Saint-Vincent de Paul

Lille, Nord, 59000, France
New York Presbyterian Hospital- Weil Cornell Medical College

New York, New York, 10065, United States
Stanford University

Palo Alto, California, 94304, United States
UOC Oncoematologia Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy
Universitair Medisch Centrum Utrecht

Utrecht, 3584 CX, Netherlands
University Health Network

Toronto, Ontario, M5G 2C4, Canada
University of Utah

Salt Lake City, Utah, 84113, United States
Wayne State University School of Medicine - Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

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