Study of a new RNA drug for PNH anemia
Part of Blood & lymphatic clinical trials.
This trial tests an experimental RNA drug (ALN-CFB) for people with PNH who still have low red blood cell counts despite taking a C5 inhibitor. The drug aims to block a protein (complement factor B) to improve anemia.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have been diagnosed with PNH (a rare blood disorder that destroys red blood cells).
- You have been on a stable dose of a C5 inhibitor (like eculizumab or ravulizumab) for at least 6 months.
- Your hemoglobin level is 10.5 g/dL or lower, meaning you have ongoing anemia.
- Your reticulocyte count (young red blood cells) is 100 or higher, showing your body is trying to make new cells.
- You have not had a bone marrow or organ transplant, and you do not have active infections or recent serious infections.
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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