A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China

Launched by NOVARTIS PHARMACEUTICALS · Apr 16, 2025

Keywords

ClinConnect Summary

This clinical trial is studying the effects of a new treatment called iptacopan for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. Researchers want to see how well iptacopan works in real-life situations, focusing on how it affects patients' health, their management of the disease, and how it impacts healthcare resources in China. This research is important because it aims to improve treatment options and outcomes for people living with PNH.

To be eligible for the study, participants must be at least 18 years old and have a confirmed diagnosis of PNH. There are two groups in the study: one group includes patients who have never received previous treatment for PNH, while the other includes those who have been stable on a specific type of treatment for at least three months. Participants will need to be vaccinated against certain infections before starting iptacopan. The trial is currently not recruiting participants, but it will provide important insights into a new way to manage PNH, potentially changing how patients are treated in the future.

Gender

ALL

Eligibility criteria

• • Inclusion criteria For Cohort 1,
• Patient who meets all the following criteria can be included in this study:
• • 1. Age ≥ 18 years at the time of signing the ICF;
• • 2. Patient with a documented diagnosis of PNH;
• • 3. Patient who has never received complement inhibitor therapy;
• • 4. Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
• • 5. Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation; If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible.
• • 6. Patient who has signed the ICF.
• • For Cohort 2,
• Patient who meets all the following criteria can be included in this study:
• • 1. Age ≥ 18 years at the time of signing the ICF;
• • 2. Patient with a documented diagnosis of PNH; Patients who have been receiving stable treatment with C5 complement inhibitors for at least three months prior to enrollment;
• • 3. Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
• • 4. Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation;
• • 5. If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible.
• • 6. Patient who has signed the ICF. Exclusion criteria
• For Cohort 1 and Cohort 2, patients who meet any of the following criteria will meet the exclusion criteria for this study:
• • 1. Participating in an interventional PNH clinical study;
• • 2. Have an active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to first dose;
• • 3. Documented with a history of recurrent invasive infections, e.g. active systemic bacterial, viral or fungal infection within 14 days prior to first dose;
• • 4. Documented with a history of HIV infection;
• • 5. Women who are pregnant or breastfeeding or intending to conceive during the study period;
• • 6. Existence of bone marrow failure (reticulocytes \< 100 × 109/L, platelets \< 30 × 109/L, and neutrophils \< 0.5 × 109/L) determined by the investigator;
• • 7. Other conditions that are not suitable for participating in the study, in the judgment of the investigator.