Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry

Novartis

Status: Active, not recruiting

Conditions: Hemoglobinuria, Paroxysmal

Treatments: Drug: Iptacopan

Study type: Observational

Funder types: Industry

Identifiers

NCT06903234

CLNP023C12003

Details and patient eligibility

About

This is an observational single-arm descriptive cohort study based on the secondary use of data collected on iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry.

Full description

This multinational, non-interventional, descriptive single-arm cohort study is based on secondary analysis of data collected within the iptacopan silo of the IPIG PNH Registry (data on iptacopan-treated patients made available to Novartis). This is a non-interventional study utilizing secondary data and is considered a "registry-based study." The IPIG PNH Registry (CT.gov NCT06524726), the parent registry, includes a dedicated drug silo to collect data from patients using iptacopan in routine care.

Enrollment: 200 estimated patients

Sex: All

Ages: 18 to 100 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Signed informed consent to participate in the IPIG PNH Registry
• PNH confirmed by flow cytometry
• Incident users of iptacopan
• Aged at least 18 years at the iptacopan initiation

Exclusion criteria

• Participation in an interventional clinical trial

Trial design

200 participants in 1 patient group

Iptacopan

Description:

Adult patients with PNH treated with iptacopan in routine care.

Treatment:

Drug: Iptacopan