Mitapivat for red blood cell membrane disorders or CDAII
Treatments studied
Part of Blood & lymphatic clinical trials.
This trial tests if mitapivat, an experimental pill, can safely improve anemia and reduce symptoms like fatigue in people with inherited red blood cell membrane disorders or congenital dyserythropoietic anemia type II (CDAII). It is a proof-of-concept study to see if the drug works.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a red blood cell membrane disorder or CDAII, confirmed by genetic testing showing a variant of uncertain significance, likely pathogenic, or pathogenic variant.
- You are 18 years or older.
- Your average hemoglobin is less than 13.0 g/dL (men) or 11.0 g/dL (women). If your average is above 10.0 g/dL, you must also have an enlarged spleen, fatigue from red blood cell breakdown, or signs of active hemolysis.
- You are willing to take folic acid (0.8 mg daily) during the study.
- Your liver and kidney function must be adequate as determined by blood tests.
- You are not regularly receiving blood transfusions (more than 5 in the last year or any in the last 3 months).
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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