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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Hemoglobinuria, Paroxysmal
• Intervention / Treatment: Drug: Iptacopan

Detailed 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.

• Study Type: Observational
• Enrollment (Estimated): 200

Contacts and Locations

Study Locations

• Switzerland
  • Basel, Switzerland
    • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult patients with PNH who are enrolled in the IPIG PNH Registry, newly treated with iptacopan.

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Iptacopan; Adult patients with PNH treated with iptacopan in routine care.	Drug: Iptacopan; Adult patients with PNH treated with iptacopan; Other Names: Fabhalta

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients with infections caused by encapsulated bacteria	To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Cumulative incidence of infections (event probability as a function of time), caused by encapsulated bacteria	To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with infections events per 100 participants -years (incidence rates) caused by encapsulated bacteria	To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of infections episodes per 100 patients -years (occurrence rates) caused by encapsulated bacteria	To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients with serious infections caused by encapsulated bacteria and all serious infection	To describe the short- and long-term risk of the serious infections in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae) and all serious infection.	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Cumulative incidence of serious infections, caused by encapsulated bacteria and all serious infection (event probability as a function of time)	To describe the short- and long-term risk of the serious infections in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae) and all serious infection.	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with serious infections events per 100 patients -years (incidence rates) caused by encapsulated bacteria and all serious infection	To describe the short- and long-term risk of the serious infections in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae) and all serious infection.	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of serious infections episodes per 100 patients -years (occurrence rates) caused by encapsulated bacteria and all serious infection	To describe the short- and long-term risk of the serious infections in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae) and all serious infection.	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with potential breakthrough hemolysis, solid tumors, hematological malignancies, Major adverse vascular events (MAVEs), serious adverse events (SAEs), hyperlipidemia and thrombocytopenia	To describe the short- and long-term risk of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia in patients with PNH treated with iptacopan in routine clinical practice.	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Cumulative incidence of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia (event probability as a function of time)	To describe the short- and long-term risk of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia in patients with PNH treated with iptacopan in routine clinical practice.	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia events per 100 patients -years (incidence rates)	To describe the short- and long-term risk of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia in patients with PNH treated with iptacopan in routine clinical practice.	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia episodes per 100 patients -years (occurrence rates)	To describe the short- and long-term risk of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia in patients with PNH treated with iptacopan in routine clinical practice.	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with death due to any cause	To describe the short- and long-term risk of all-cause mortality in patients with PNH treated with iptacopan in routine clinical practice.	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Cumulative incidence of death due to any cause (event probability as a function of time)	To describe the short- and long-term risk of all-cause mortality in patients with PNH treated with iptacopan in routine clinical practice.	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with death due to any cause events per 100 patients -years (incidence rates)	To describe the short- and long-term risk of all-cause mortality in patients with PNH treated with iptacopan in routine clinical practice.	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients vaccinated against Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae at each study visit	To describe the number of patients receiving mandatory and recommended vaccinations against encapsulated bacteria.	From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with serious hemolysis following discontinuation of iptacopan	To describe the risk of serious hemolysis following discontinuation of iptacopan in patients with PNH treated with iptacopan in routine clinical practice.	From the iptacopan discontinuation up to 14 days
Number of patients who became pregnant during treatment with iptacopan, exposure characteristics (e.g. trimester of exposure) and birth outcomes	To describe the frequency of use of iptacopan during pregnancy in PNH patients, characteristics of pregnancies exposed to iptacopan and frequency of selected pregnancy and birth outcomes.	From the Last Menstrual Period to pregnancy outcome (in case of live birth, up to 12 months post delivery)

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): March 31, 2025
• Primary Completion (Estimated): October 1, 2029
• Study Completion (Estimated): October 1, 2029

Study Registration Dates

• First Submitted: March 24, 2025
• First Submitted That Met QC Criteria: March 24, 2025
• First Posted (Actual): March 30, 2025

Study Record Updates

• Last Update Posted (Actual): June 23, 2026
• Last Update Submitted That Met QC Criteria: June 19, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: Paroxysmal nocturnal hemoglobinuria; Iptacopan
• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Hemic and Lymphatic Diseases; Hemoglobinuria, Paroxysmal; iptacopan
• Other Study ID Numbers: CLNP023C12003; EUPAS1000000457 (Other Identifier: EU PAS number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No