Danicopan plus standard therapy for pediatric PNH
Part of Blood & lymphatic clinical trials.
This trial tests adding danicopan to a standard treatment (ravulizumab or eculizumab) for children with PNH who still have anemia from extravascular hemolysis. The goal is to see if the combination helps improve red blood cell counts.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be diagnosed with PNH (paroxysmal nocturnal hemoglobinuria).
- You must have anemia (low red blood cells) and a high reticulocyte count, meaning your body is making extra red blood cells to try to keep up.
- You must already be taking ravulizumab or eculizumab for at least 12 weeks, with no planned changes in dose.
- You must be up to date on certain vaccines: meningitis, Hib, and pneumococcus.
- You cannot have a very low platelet count, a very low white blood cell count, or serious liver problems.
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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