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

Exploring Iptacopan's safety for Paroxysmal Nocturnal Hemoglobinuria patients.

Recruiting
18 years and older
All
Phase 3

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare blood disease where red blood cells break apart too early. The study is about a medicine called iptacopan, which is being tested for long-term safety and how well it works for people with PNH. This study is an open-label, meaning both doctors and patients know what treatment is being given. It is a single-arm study, so everyone gets the same treatment, and it will happen in many different places (multicenter). Only patients who have been part of earlier studies with iptacopan and have seen positive effects can join. They also need certain vaccinations. This study does not include people with serious infections or who have had certain past medical treatments.

  • The study will last long-term but no exact length is given.
  • Participants must have been in previous studies with iptacopan.
  • There are certain health requirements to join and risks to consider, like possible infections.
Study details
    Paroxysmal Nocturnal Hemoglobinuria

NCT04747613

Novartis Pharmaceuticals

4 January 2025

Step 1 Get in touch with the nearest study center
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