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
- 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+ - 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+ - 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 - 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 - 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+ - 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+ - 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+ - 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+ - 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+ - 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 - 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+ - 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+ - 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.
São PauloAges 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 - NCT07877727Recruiting
Rare blood disease network for better care
This study creates a national network to collect health information and samples from people with certain rare blood diseases. The goal is to improve treatment and understand these conditions better.
AvellinoAges 4 weeks–65 years - NCT06524726Recruiting
PNH registry for people with confirmed PNH
This is a long-term registry study for people with a confirmed diagnosis of paroxysmal nocturnal hemoglobinuria (PNH). It simply collects information over time to better understand the condition and how it's managed. There is no experimental treatment involved.
Altamonte Springs, FloridaAges Any age - NCT07760467RecruitingPhase 1
Study of an oral medication for safety in healthy adults
This study tests an investigational oral medication called TUL321 in healthy volunteers to see if it is safe and how the body handles it. It may help understand the drug's effects and is an early step in developing a new treatment.
Changsha, HunanAges 18–45 - NCT07462780RecruitingPhase 1
Testing CMS-D017 in Healthy Volunteers
This trial tests a new investigational drug called CMS-D017 in healthy volunteers. Researchers will give single and multiple doses to see how the body processes it and if it's safe. You could help find an important new treatment.
BeijingAges 18–55 - 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 - 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 - NCT07152288RecruitingPhase 1
Study of HSK39297 in people with liver problems
This trial tests how a new drug, HSK39297, works in people with mild to moderate liver impairment (Child-Pugh A or B). It compares them to healthy volunteers to see how the liver affects the drug's safety and how the body processes it.
BeijingAges 18–70 - NCT06932471RecruitingPhase 3
Study of a new drug for PNH patients with anemia despite current treatment
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.
Beijing, Beijing MunicipalityAges 18–75 - 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 - NCT06931691Recruiting
Study of Iptacopan for adults with PNH in China
This study looks at how well iptacopan works for people with PNH (a condition where red blood cells break down too quickly). It is for adults who have never used certain treatments or who have been on other similar medicines.
Hefei, AnhuiAges 18–80
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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.