Early access to danicopan for adults with PNH
Part of Blood & lymphatic clinical trials.
This program gives adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition, access to danicopan, an investigational pill that may help improve anemia. It is for people who have been on Soliris or Ultomiris for at least 6 months and still have significant anemia.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 18 years or older
- You have a diagnosis of PNH with anemia (hemoglobin level 9.5 g/dL or lower) and high reticulocyte count
- You have been on Soliris or Ultomiris for at least 6 months
- Your platelet count is 30,000 or higher and your neutrophil count is 500 or higher
- You have been vaccinated against meningococcal disease (N meningitidis)
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
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.
This trial is studying danicopan as an add-on therapy for people with paroxysmal nocturnal hemoglobinuria (PNH) who are already taking a C5 inhibitor like eculizumab or ravulizumab. It aims to see how well danicopan works when used together with these standard treatments.
This trial tests a new pill (MY008211A) for people with paroxysmal nocturnal hemoglobinuria (PNH) who still have low red blood cell counts (anemia) even after taking anti-C5 antibody therapy. The goal is to see if this new drug can raise hemoglobin levels and improve energy.
This trial tests an investigational drug called NM8074 for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have not yet been treated with complement inhibitors. The goal is to see if NM8074 can reduce red blood cell destruction and improve anemia, potentially reducing the need for blood transfusions.
This study looks at how well iptacopan works for people with PNH (a condition where red blood cells break down too quickly). It is for adults who have never used certain treatments or who have been on other similar medicines.
This trial tests a new combination of two drugs (pozelimab and cemdisiran) for adults with PNH whose current treatment (eculizumab, ravulizumab, or crovalimab) isn't controlling their symptoms well. It aims to see if the combination reduces red blood cell breakdown and improves blood counts.
Hear when a new Paroxysmal Nocturnal Hemoglobinuria trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.