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 29 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07154745RecruitingPhase 3
New combination therapy for PNH when current treatment isn't working
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.
Ribeirão Preto, São PauloAges 18 years+ - NCT05744921RecruitingPhase 3
Pozelizumab plus cemdisiran for adult PNH over the long term
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.
Toronto, OntarioAges 18 years+ - NCT04901936RecruitingPhase 2
Study of pegcetacoplan for kids with PNH blood problems
This trial tests pegcetacoplan in children (ages 12–17) with paroxysmal nocturnal hemoglobinuria (PNH), a condition that can cause ongoing blood cell breakdown. The goal is to see if the medicine can improve anemia related to PNH.
Atlanta, GeorgiaAges 12–17 - NCT05876312RecruitingPhase 1/Phase 2
Study drug ADX-038 in healthy people and PNH patients
This early-stage study tests how safe and tolerable ADX-038 is, and how the body processes it, in healthy volunteers and in people with paroxysmal nocturnal hemoglobinuria (PNH). It may help researchers understand whether the drug works safely for PNH patients and identify the right dosing.
Brisbane, QueenslandAges 18 years+ - NCT06745622RecruitingPhase 2
Long-term safety of HSK39297 for PNH patients
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.
Nanjing, JiangsuAges 18 years+ - NCT06932744RecruitingPhase 3
Testing a new PNH pill for people who haven't had complement therapy
This study tests a new oral medication called MY008211A for people with PNH who have not yet tried complement inhibitors. It aims to improve anemia and reduce the need for blood transfusions by targeting the part of the immune system that destroys red blood cells.
Beijing, Beijing MunicipalityAges 18–75 - NCT06933914RecruitingPhase 2/Phase 3
Long-term safety of MY008211A for PNH
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.
Tianjin, Tianjin MunicipalityAges 18 years+ - NCT06978699RecruitingPhase 2
Testing XH-S003 for people with PNH
This trial tests a new medicine, XH-S003, for people with paroxysmal nocturnal hemoglobinuria (PNH) who have not tried complement inhibitors before. The goal is to see if it can help manage symptoms like low red blood cells and fatigue.
TianjinAges 18 years+ - NCT07187401RecruitingPhase 1/Phase 2
Study of a new RNA drug for PNH anemia
This trial tests an experimental RNA drug (ALN-CFB) for people with PNH who still have low red blood cell counts despite taking a C5 inhibitor. The drug aims to block a protein (complement factor B) to improve anemia.
Toronto, OntarioAges 18 years+ - NCT07229235Recruiting
Real-world study of iptacopan for PNH in Italy
This study looks at how well iptacopan works in everyday medical practice for people with paroxysmal nocturnal hemoglobinuria (PNH) being treated in Italy. It gathers information from routine care to understand real-world effectiveness.
Alessandria, ALAges 18 years+ - NCT07256301RecruitingPhase 1
Testing EA5 in adults with paroxysmal nocturnal hemoglobinuria (PNH)
This clinical trial tests an investigational drug called EA5 for people with PNH, a rare blood disease where red blood cells break down too quickly. It is for adults who either have never taken a complement inhibitor or stopped taking one recently, and who still have signs or symptoms of PNH.
Tianjin, Tianjin MunicipalityAges 18 years+ - NCT05776472Recruiting
Study real-world pegcetacoplan effects in adults with PNH
This study looks at how the medicine pegcetacoplan works in real life for adults with paroxysmal nocturnal hemoglobinuria (PNH). It may help doctors understand expected benefits and everyday use patterns.
Liverpool, New South WalesAges 18 years+ - NCT07416162Recruiting
Study of iptacopan for PNH or C3G in Korea
This trial tests a medicine called iptacopan for people with paroxysmal nocturnal hemoglobinuria (a rare blood disorder) or C3 glomerulopathy (a kidney disease). It aims to see if the drug is safe and effective for Korean patients.
JeollanamAges 18–99 - NCT07470762RecruitingPhase 1/Phase 2
Study of HS-10542 for paroxysmal nocturnal hemoglobinuria (PNH)
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.
Hangzhou, ZhejiangAges 18–75 - NCT07657052Enrolling by invitation
Long-term safety and effectiveness of XH-S003 for PNH
This study tests if the drug XH-S003 is safe and works well over a long period for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease. If you've already taken XH-S003 in a past study and your doctor thinks it helped, you may join to keep getting the treatment.
Tianjin, JinanAges 18 years+ - NCT06934967RecruitingPhase 3
Iptacopan study for children with PNH
This trial tests an oral medication, iptacopan, for children with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition. It aims to see how well the drug works in kids who have not tried anti-C5 therapy or who are switching from it.
Atlanta, GeorgiaAges 2–18 - NCT07457151Recruiting
Danicopan study for PNH in Korea
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.
BusanAges 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 - 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 - NCT05755867Recruiting
Global registry for people with paroxysmal nocturnal hemoglobinuria
This trial is a global patient registry that collects health information from people diagnosed with PNH (a blood condition caused by a genetic change in the PIG-A gene). It may help researchers better understand the condition and what care people receive.
Bethesda, MarylandAges Any age - 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 - 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 - 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 - 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
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Common questions
- Are there clinical trials for paroxysmal nocturnal hemoglobinuria?
- Yes. Clin2 currently lists 29 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.