Clinical trials
Paroxysmal Nocturnal Hemoglobinuria clinical trials
Below are recruiting paroxysmal nocturnal hemoglobinuria clinical trials, each written for real people, not researchers. We’re tracking 30 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06411626Recruiting
sharing PNH experiences from home
This study asks people with PNH to share their symptoms and experiences from home using a phone or computer. The goal is to learn more about how living with PNH affects everyday life.
East Hanover, New JerseyAges 18–99 - NCT06449001RecruitingPhase 3
Danicopan plus standard therapy for pediatric PNH
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.
Saskatoon, SaskatchewanAges 12–17 - NCT06412497RecruitingPhase 2
Stem cell transplant for aplastic anemia and bone marrow failure
This trial tests a stem cell transplant using a milder conditioning regimen and a drug called post-transplant cyclophosphamide to treat severe aplastic anemia and other acquired bone marrow failure conditions. The goal is to help patients achieve a functioning donor bone marrow with fewer side effects.
Minneapolis, MinnesotaAges birth–75 years - NCT06312644Recruiting
Study of Ultomiris safety during pregnancy
This study looks at the safety of the medication Ultomiris when taken during pregnancy or breastfeeding. If you are pregnant and currently taking Ultomiris for an approved condition, this research will help gather information on how it affects you and your baby.
Boston, MassachusettsAges Any age - NCT04645199Recruiting
Long-term follow-up study for blood and bone marrow diseases
This study is collecting long-term follow-up information from people diagnosed with certain blood and bone marrow conditions. It may help doctors understand what happens over time and improve future care, but you’re mainly contributing data rather than receiving a new treatment.
Tianjin, Tianjin MunicipalityAges Any age - NCT03520647RecruitingPhase 2
Haplo transplant for severe bone marrow failure using donor cells
This trial tests a stem cell transplant from a closely matched but not fully matched family donor (haploidentical), with chemotherapy after the transplant to help prevent graft-versus-host disease (GVHD). It may help people with severe aplastic anemia, certain MDS after transformation, or PNH when other treatments have not worked or are not available.
Bethesda, MarylandAges 4–75
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Common questions
- Are there clinical trials for paroxysmal nocturnal hemoglobinuria?
- Yes. Clin2 currently lists 30 recruiting paroxysmal nocturnal hemoglobinuria studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a paroxysmal nocturnal hemoglobinuria trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a paroxysmal nocturnal hemoglobinuria trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.