Condition

Paroxysmal Nocturnal Hemoglobinuria (PNH) clinical trials and research

• 13Clinical trials
• 1New in 30 days
• 17Countries

Newest Paroxysmal Nocturnal Hemoglobinuria (PNH) clinical trials

All recruiting trials (13)

• Understanding congenital bone marrow failure and related disorders through a national network

  This project tests whether people with rare inherited bone marrow failure syndromes or related conditions really have the underlying genetic causes, by collecting data and samples across multiple centers.

  Recruiting at 4 sites Istituto Giannina Gaslini · Avellino and 3 other locations · NCT07877727

• See if TUL321 is safe and tolerable in healthy adults

  This phase 1 study tests whether a single and multiple doses of TUL321 capsules are safe and tolerable in healthy adults, and how food affects its absorption.

  Recruiting Phase 1 Zhuhai United Laboratories Co., Ltd. · Changsha, Hunan · NCT07760467

• CMS-D017 capsules: single and multiple dose testing in healthy adults

  This trial will test CMS-D017 capsules in healthy Chinese adults to see if single and multiple oral doses are safe and how the drug behaves in the body.

  Recruiting Phase 1 Shenzhen Kangzhe Biotechnology Co., Ltd. · Beijing · NCT07462780

• Real-world use of iptacopan for PNH in Italy

  This project will see if iptacopan works and is safe for adults with paroxysmal nocturnal hemoglobinuria (PNH) who are treated in Italian clinics.

  Recruiting at 12 sites Novartis · Alessandria, AL and 11 other locations · NCT07229235

• ALN-CFB for adults with PNH who remain anemic on C5 inhibitors

  This trial will test ALN-CFB, an experimental siRNA medicine, versus placebo in adults with PNH who remain anemic despite taking a C5 inhibitor.

  Recruiting at 8 sites Phase 1/2 Regeneron Pharmaceuticals · Toronto, Ontario and 7 other locations · NCT07187401

• How HSK39297 is processed and tolerated in people with mild to moderate liver impairment

  This trial tests how the drug HSK39297 is absorbed, cleared, and tolerated in adults with mild to moderate liver impairment compared with people who have normal liver function.

  Recruiting Phase 1 Haisco Pharmaceutical Group Co., Ltd. · Beijing · NCT07152288

• Iptacopan for children and teens with PNH to see how the drug behaves in the body and whether it is safe

  This trial will try oral iptacopan in children and teens (ages 2 to under 18) with PNH to see how their bodies handle the drug and whether it is safe and well tolerated.

  Recruiting at 15 sites Phase 3 Novartis · Atlanta, Georgia and 14 other locations · NCT06934967

• Long-term safety and tolerability of MY008211A tablets for patients with PNH

  This study is testing how safe and well-tolerated MY008211A tablets are for people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have already benefited from the treatment in earlier trials.

  Recruiting Phase 2/3 Wuhan Createrna Science and Technology Co., Ltd · Tianjin, Tianjin Municipality · NCT06933914

• Safety of Ultomiris during pregnancy

  This study looks at how safe Ultomiris is for pregnant women and their babies to see if it causes any problems during pregnancy and breastfeeding.

  Recruiting at 4 sites Alexion Pharmaceuticals, Inc. · Boston, Massachusetts and 6 other locations · NCT06312644

• Evaluating ADX-038 for safety and effectiveness in healthy individuals and PNH patients

  This study is testing a new drug called ADX-038 to see if it's safe and effective for healthy people and those with paroxysmal nocturnal hemoglobinuria who still have anemia despite treatment.

  Recruiting Phase 1/2 ADARx Pharmaceuticals, Inc. · Brisbane, Queensland and 3 other locations · NCT05876312

• Pegcetacoplan treatment for adolescents (12–17) with PNH

  This trial will try pegcetacoplan given by twice-weekly home subcutaneous infusion in 12–17 year-olds with PNH to see if it is safe and helps their anemia and blood counts.

  Recruiting at 7 sites Phase 2 Apellis Pharmaceuticals, Inc. · Atlanta, Georgia and 11 other locations · NCT04901936

• Long-term study of blood diseases in patients in China

  This study is trying to gather detailed information from patients in China with blood diseases like leukemia and multiple myeloma to better understand how these conditions affect their lives and treatment options.

  Recruiting Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT04645199

• Transplantation using family member stem cells for severe blood disorders

  This study is testing if using stem cells from family members can help people with severe blood disorders feel better and have better results than traditional bone marrow transplants.

  Recruiting Phase 2 National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT03520647

Related topics

Quick answers

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

13, as of November 12, 2026. 1 of them was first registered in the last 30 days. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 17 countries. The most sites are in China (5 trials), United Kingdom (4 trials), United States (4 trials), Italy (4 trials), South Korea (2 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1: 3, Phase 1/2: 2, Phase 2: 2, Phase 2/3: 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.

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