Capturing patient experiences in 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

This observational study aims to longitudinally gather data on symptoms, treatment usage, and overall health-related quality of life (HRQoL) from patients diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH). Participants will use the Folia mobile app to report their experiences, including symptom burden and treatment changes, over a six-month period. The study will employ a hybrid recruitment strategy, combining clinic and community referrals, and will include monthly survey check-ins to enhance data collection. The goal is to create a real-world data source that reflects the variability of symptoms and HRQoL in PNH patients, including those on specific therapies like iptacopan.

Why it matters

Who can join: the full rules

Inclusion Criteria:

Study participants eligible for inclusion in this study must meet all of the following criteria:

* Aged 18 or older
* US-based with a proficient understanding of and ability to read the English language
* Any patient with a diagnosis of PNH, regardless of symptom or treatment history

Exclusion Criteria:

Study participants who do not fit all inclusion criteria listed above are unable to participate in this study. Outside of required inclusion criteria, there are no other exclusion criteria in order to meet the exploratory nature of the primary endpoint.

Where it's running

• Novartis Investigative Site
  East Hanover, New Jersey, United States Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: +41613241111

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.