We are studying whether Mitapivat can help increase hemoglobin levels in children with Pyruvate Kinase Deficiency who do not receive regular blood transfusions. This trial compares the treatment to a placebo to assess its effectiveness.
Health conditions and diseases that the clinical trial is designed to study and treat.
Pyruvate kinase deficiency anemia is a rare genetic disorder that affects the production of red blood cells, leading to chronic hemolytic anemia. This condition can cause fatigue, jaundice, and an enlarged spleen due to the breakdown of red blood cells.
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.
