Study drug mitapivat for rare blood membrane anemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This Phase 2 trial tests mitapivat in adults with rare inherited red-blood-cell membrane problems, including congenital dyserythropoietic anemia type II (CDA II). It looks at whether the medicine is safe and improves red-blood-cell function, aiming to help people with anemia and active red-blood-cell breakdown.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You’re an adult (18+) with a confirmed red-blood-cell membrane disorder or CDA II
- Your DNA test must match a specific group of “likely disease-causing” genetic variants
- Your average hemoglobin is below the trial’s cutoff (or above it, you must have signs like enlarged spleen or ongoing hemolysis)
- You take folic acid daily (at least the equivalent of 0.8 mg by mouth) during the study
- Your kidney and liver blood tests are within allowed ranges, and kidney function is not too low
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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