International registry for patients with Paroxysmal Nocturnal Hemoglobinuria

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

The International PNH Interest Group PNH Registry is an observational database designed to collect real-world health information from patients diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH). This registry aims to gather data on clinical outcomes, patient-reported outcomes, and health resource utilization over time, while also ensuring long-term safety data for patients receiving anti-complement therapies. The registry is managed by a network of international PNH experts and is supported by pharmaceutical partners to fulfill post-approval regulatory commitments. Data collection occurs at enrollment and every six months thereafter.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Patients with PNH confirmed by flow cytometry.
* Patient and/or parent/legally authorized representative provide written informed consent/assent to participate in the registry in a manner approved by the Institutional Review Board/Independent Ethics Committee and local regulations.

Exclusion Criteria:

* Participating in an interventional PNH clinical trial. Note: A patient included in the registry, who enrolls in an interventional PNH clinical trial during the course of the registry, will be kept in the registry but data collection will be paused in the registry during their involvement in the clinical trial/extension study. Data collection in the registry will continue after patient involvement in the clinical trial/extension study has ended or trial protocol mandated data collection ceases.

Where it's running

• International PNH Interest Group
  Altamonte Springs, Florida, United States Recruiting

Study contacts

• Principal investigator: Richard Kelly, MBChB PhD — International PNH Interest Group
• Study coordinator: IPIG Registry Coordinator
• Email: registry@pnhinterestgroup.org
• Phone: Please email

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• 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

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.