Testing an experimental pill for PNH
Treatments studied
Part of Blood & lymphatic clinical trials.
This trial tests a new pill called MY008211A for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder where red blood cells are destroyed. If you have moderate anemia and high LDH (a marker of blood cell breakdown), this study may be an option.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be at least 18 years old with a body mass index (BMI) of 18 or higher.
- You must have a confirmed diagnosis of PNH with a clone size (percentage of affected cells) of at least 10%.
- Your average hemoglobin level must be below 100 g/L (indicating anemia).
- Your LDH level must be more than 1.5 times the upper limit of normal (showing active red blood cell breakdown).
- You need to have received a vaccine against meningococcus before starting the study drug. Vaccines against pneumococcus and Haemophilus influenzae are also recommended if not already given.
- You must not have used a complement inhibitor (like eculizumab) recently, or have stopped it for less than 5 half-lives or 120 days.
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
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This study checks the long-term safety of the drug MY008211A for people with paroxysmal nocturnal hemoglobinuria (PNH). It is only for those who already took the drug in an earlier study and had benefit, and who have had specific vaccines.
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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.
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