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

NCT06903234 | Active Not Recruiting FDA Drug

• 1 other identifier: CLNP023C12003
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Hemoglobinuria, Paroxysmal

Keywords

Paroxysmal nocturnal hemoglobinuria; Iptacopan

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (14)

• Number of patients with serious infections caused by encapsulated bacteria 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)

  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

  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

  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

  From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.

Study Arms (1)

Iptacopan

Adult patients with PNH treated with iptacopan in routine care.

Drug: Iptacopan

Interventions

Iptacopan DRUG

Adult patients with PNH treated with iptacopan

Also known as: Fabhalta

Iptacopan

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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

You may not qualify if:

• Participation in an interventional clinical trial

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis Investigative Site

Basel, Switzerland

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

iptacopan

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 24, 2025
• First Posted: March 30, 2025
• Study Start: March 31, 2025
• Primary Completion (Estimated): October 1, 2029
• Study Completion (Estimated): October 1, 2029
• Last Updated: May 15, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

CH (1)