New combination therapy for PNH when current treatment isn't working
Treatments studied
Part of Blood & lymphatic clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have been diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) through a blood test.
- You have been on eculizumab, ravulizumab, or crovalimab for at least 6 months.
- Your LDH levels have been high (above 1.5 times the normal limit) for the past 6 months, and your doctor thinks it's due to red blood cell breakdown.
- You are willing to get the meningococcal vaccine series (if not already up to date) and keep it current during the study.
- You have not had an organ transplant, bone marrow transplant, or other blood cell transplant.
- You do not have an active infection or have had one within the last 2 weeks before screening.
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 Phase 3 study tests whether long-term treatment with pozeli(z)umab plus cemdisiran is safe and works well in adults with paroxysmal nocturnal hemoglobinuria (PNH). It is mainly for people whose PNH does not respond to certain standard medicines or who have genetic changes that make them refractory.
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 is testing a new pill (FXS6837) compared to the standard IV medicine eculizumab for people with PNH. The study lasts 24 weeks and aims to see if the new pill works better or as well and has fewer side effects.
This trial tests a new drug, HS-10542, for people with PNH who are already on a C5 inhibitor like eculizumab or covalizumab but still have low hemoglobin or need transfusions. It aims to see if this additional treatment can improve blood counts and reduce transfusion needs.
This Phase 2 trial studies NM8074 in adults with paroxysmal nocturnal hemoglobinuria (PNH) whose blood counts and breakdown of red blood cells (hemolysis) are not controlled enough on Soliris. NM8074 is meant to reduce the abnormal immune “complement” activity driving the disease and its symptoms like anemia.
This study tests a new oral medication called NTQ5082 for people with PNH who have not been treated with a complement inhibitor before. It aims to see if the drug can reduce the breakdown of red blood cells and improve anemia.
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