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Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

Studying Iptacopan's safety in patients with Paroxysmal Nocturnal Hemoglobinuria.

Recruiting
18 years and older
All
Phase 3

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare blood disease where red blood cells break apart too easily. This study is testing a drug called iptacopan, to see if it's safe and works well over a long time. The study will give patients who finished earlier iptacopan studies a chance to continue using it. It is called open-label because both the researchers and participants know what treatment is being given. A single-arm study means all participants get the same treatment without comparing to another group.

  • Participants must be 18 or older, with PNH, and have completed specific earlier studies.
  • Prior vaccinations for certain infections are required.
  • Study duration is long-term and no tapering down of medication is allowed.

To join, you should be healthy except for PNH and have no history of certain infections or stem cell transplants. The study aims to ensure iptacopan is safe and continues to be helpful for PNH patients.

Study details
    Paroxysmal Nocturnal Hemoglobinuria

NCT04747613

Novartis Pharmaceuticals

4 January 2025

Step 1 Get in touch with the nearest study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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