Condition

Paroxysmal Nocturnal Hemoglobinuria clinical trials and research

• 13Clinical trials
• 1NIH research projects
• 24Countries

Newest Paroxysmal Nocturnal Hemoglobinuria clinical trials

All recruiting trials (13)

• Oral HS-10542 for paroxysmal nocturnal hemoglobinuria

  This study will test whether the oral drug HS-10542 can reduce hemolysis and raise hemoglobin in adults with PNH, including those not fully helped by C5 inhibitor therapy.

  Recruiting Phase 1/2 Jiangsu Hansoh Pharmaceutical Co., Ltd. · Hangzhou, Zhejiang · NCT07470762

• Danicopan added to ravulizumab or eculizumab for PNH in Korea

  This will see if adding Danicopan to ravulizumab or eculizumab is safe and helps people with PNH who are treated in routine care in Korea.

  Recruiting at 3 sites AstraZeneca · Busan and 3 other locations · NCT07457151

• Iptacopan use in Korean patients with PNH or C3G

  This post-marketing surveillance study tests how well iptacopan works and how safe it is for Korean adults with PNH or C3G in real-world care.

  Recruiting at 3 sites Novartis · Jeollanam and 2 other locations · NCT07416162

• Pozelimab + cemdisiran for adults with PNH whose current treatment isn't working

  This trial tests whether combining pozelimab and cemdisiran can better reduce hemolysis in adults with PNH whose C5 inhibitor treatment isn’t controlling their disease.

  Recruiting at 20 sites Phase 3 Regeneron Pharmaceuticals · Ribeirão Preto, São Paulo and 19 other locations · NCT07154745

• Evaluating MY008211A for treating anemia in PNH patients on anti-C5 therapy

  This study is testing a new treatment called MY008211A to see if it can help people with PNH who still have anemia even while on anti-C5 therapy.

  Recruiting Phase 3 Wuhan Createrna Science and Technology Co., Ltd · Beijing, Beijing Municipality · NCT06932471

• Iptacopan treatment for adults with PNH in China

  This study will try iptacopan (LNP023) in adults with PNH in China to see if it improves clinical and laboratory outcomes and changes healthcare use.

  Recruiting at 14 sites Novartis · Hefei, Anhui and 13 other locations · NCT06931691

• Long-term safety and tolerability of HSK39297 for patients with PNH

  This study is testing if continuing treatment with HSK39297 is safe and effective for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have already used this medication.

  Recruiting Phase 2 Haisco Pharmaceutical Group Co., Ltd. · Nanjing, Jiangsu · NCT06745622

• International registry for patients with Paroxysmal Nocturnal Hemoglobinuria

  This study is collecting health information from people with Paroxysmal Nocturnal Hemoglobinuria to see how they are doing over time and how well their treatments are working.

  Recruiting International PNH Interest Group · Altamonte Springs, Florida · NCT06524726

• Evaluating Danicopan with Ravulizumab or Eculizumab for Pediatric PNH Patients

  This study is testing if adding danicopan to the current treatments of ravulizumab or eculizumab can help kids with PNH have better hemoglobin levels.

  Recruiting at 4 sites Phase 3 Alexion Pharmaceuticals, Inc. · Saskatoon, Saskatchewan and 3 other locations · NCT06449001

• Hematopoietic cell transplant for severe blood disorders

  This study is testing a new way to prepare patients with severe blood disorders for a stem cell transplant to see if it can improve their recovery and reduce complications.

  Recruiting Phase 2 Masonic Cancer Center, University of Minnesota · Minneapolis, Minnesota · NCT06412497

• Capturing patient experiences in Paroxysmal Nocturnal Hemoglobinuria

  This study is trying to gather information from people with Paroxysmal Nocturnal Hemoglobinuria about their symptoms and treatment experiences using a mobile app over six months to better understand their quality of life.

  Recruiting Novartis · East Hanover, New Jersey · NCT06411626

• Global registry for patients with Paroxysmal Nocturnal Hemoglobinuria

  This study is collecting information from people with Paroxysmal Nocturnal Hemoglobinuria to better understand the condition and improve care for patients.

  Recruiting Aplastic Anemia and MDS International Foundation · Bethesda, Maryland · NCT05755867

• Long-term treatment for patients with paroxysmal nocturnal hemoglobinuria using pozelimab and cemdisiran

  This study is testing a new combination of two experimental drugs, pozelimab and cemdisiran, to see if they can safely help adults with paroxysmal nocturnal hemoglobinuria over a long period of time.

  Recruiting at 48 sites Phase 3 Regeneron Pharmaceuticals · Curitiba, Jardim Santo Andre and 49 other locations · NCT05744921

NIH-funded research (1)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Genetic and immune clues in pediatric paroxysmal nocturnal hemoglobinuria (PNH)

  A multi-institution study in children with PNH looks for genetic and immune markers that explain how the disease presents and progresses.

  NIH research Boston Children's Hospital · Boston, United States · NIH-11453343

Related topics

Quick answers

How many Paroxysmal Nocturnal Hemoglobinuria clinical trials are recruiting right now?

13, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 24 countries. The most sites are in United States (4 trials), South Korea (4 trials), China (4 trials), Canada (3 trials), Brazil (2 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 4, Phase 2: 2, Phase 1/2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.

Describe your situation →