NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice

Launched by NOVARTIS PHARMACEUTICALS · Jun 17, 2025

Keywords

ClinConnect Summary

This clinical trial is looking at how well a medicine called iptacopan works and how safe it is for adults with a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH causes red blood cells to break down too early, which can lead to fatigue and other health problems. The study will be done in real-world medical settings in Russia, meaning it will observe patients who are already being treated with iptacopan as part of their regular care.

Adults aged 18 and older who have been prescribed iptacopan by their doctor can take part, as long as they agree to join the study and don’t have any reasons that would make the medicine unsafe for them. Participants won’t have to change their treatment—doctors will decide to prescribe iptacopan based on usual practice, and the study will simply follow how patients do over time. This study is not yet open for enrollment, but it aims to better understand how iptacopan helps people with PNH outside of tightly controlled clinical trials.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Age ≥18 years at the start of iptacopan therapy.
• • 2. The patient is undergoing treatment with iptacopan.
• • 3. The treating physician decided to prescribe iptacopan based on the Summary of Product Characteristics during routine clinical practice, regardless of study participation.
• • 4. Provision of written informed consent.
• Exclusion Criteria:
• • 1. Any situations where iptacopan is contraindicated in accordance with the Summary of Product Characteristics.