Mitapivat for inherited red blood cell membrane disorders
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a drug called mitapivat (taken as a pill) to see if it can safely improve red blood cell health and reduce anemia symptoms in people with inherited red blood cell membrane disorders. It may help increase hemoglobin levels and relieve fatigue.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 or older and have a red blood cell membrane disorder (like hereditary spherocytosis or elliptocytosis) or a rare type of anemia called CDA type II, confirmed by genetic testing.
- Your average hemoglobin (the protein in red blood cells that carries oxygen) must be below 13.0 g/dL (for men) or 11.0 g/dL (for women). If it's above 10.0, you also need an enlarged spleen, fatigue from red blood cell breakdown, or lab signs of active breakdown.
- You must be willing to take folic acid (a vitamin) every day during the study.
- You must have adequate liver and kidney function.
- If you can become pregnant, you must have a negative pregnancy test and agree to use two effective birth control methods during the trial.
- You cannot have had a bone marrow or stem cell transplant, or be currently taking certain medications or blood-stimulating agents (like erythropoietin).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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