Pozelizumab plus cemdisiran for adult PNH over the long term
Treatments studied
Part of Blood & lymphatic clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have been in the related PNH parent study and completed it (or are eligible to enter from it) without permanently stopping the treatment.
- If you are entering for genetic reasons: your PNH is linked to a specific C5 gene change that makes eculizumab or ravulizumab not work well.
- Your PNH diagnosis is confirmed by a specialized blood test, and you currently have PNH symptoms based on the study rules.
- Your screening lab marker (LDH) is at least 2 times the lab’s upper normal limit.
- You can and will follow the visit schedule and get meningococcal vaccines required by the study.
- You do not have certain major exclusions, like a recent organ/bone marrow transplant, certain infections (hepatitis B/C or specific HIV history), or serious uncontrolled autoimmune or 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.
Similar studies
Other trials that look related to this one.
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.
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 study is for people with paroxysmal nocturnal hemoglobinuria (PNH) who have already taken the experimental drug HSK39297. It checks the long-term safety of continuing the treatment and makes sure it is still helpful.
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 an experimental pill called NTQ5082 for people with PNH who haven't been treated with complement inhibitors before. The goal is to see if it can improve anemia and control PNH symptoms like fatigue and blood clots.
This study tests a new pill (FXS6837) against a standard treatment (eculizumab) for people with PNH. The goal is to see if the pill is safe and effective in improving anemia and reducing the need for transfusions.
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