A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan on Disease Management, Treatment-Related Outcomes and Healthcare Resource Utilization for Adult Patients With Paroxysmal Nocturnal Hemoglobinuria in China

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Observational
SUMMARY

The implementation of new standards for the management of PNH and the use of iptacopan in patients with PNH are expected to change the treatment landscape and improve the overall prognosis of patients. Based on these backgrounds, we plan to conduct a real-world study of iptacopan to further evaluate its impact on treatment-related outcomes, disease management, and healthcare resource utilization in Chinese patients with PNH.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 80
Healthy Volunteers: f
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⁃ Patient who meets all the following criteria can be included in this study:

• Age ≥ 18 years at the time of signing the ICF;

• Patient with a documented diagnosis of PNH;

• Patient who has never received complement inhibitor therapy;

• Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;

• 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.

• Patient who has signed the ICF.

⁃ For Cohort 2,

⁃ Patient who meets all the following criteria can be included in this study:

• Age ≥ 18 years at the time of signing the ICF;

• 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;

• Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;

• 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.

• Patient who has signed the ICF. Exclusion criteria

Locations
Other Locations
China
Novartis Investigative Site
RECRUITING
Tianjin
Contact Information
Primary
Novartis Pharmaceuticals
novartis.email@novartis.com
+41613241111
Backup
Novartis Pharmaceuticals
Time Frame
Start Date: 2025-06-10
Estimated Completion Date: 2027-04-20
Participants
Target number of participants: 80
Treatments
Cohort1
PNH patients with a confirmed diagnosis of PNH in the medical record and who had never received complement inhibitor therapy (complement inhibitor naive patients)
Cohort 2
PNH patients with a confirmed diagnosis of PNH in the medical record who had been treated with a stable dose of a C5 complement inhibitor (e.g., eculizumab) for at least 3 months (C5i-treated patients; this cohort will be only start enrollment after the indication for iptacopan is to be approved in China).
Sponsors
Leads: Novartis Pharmaceuticals

This content was sourced from clinicaltrials.gov