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NCT Number: NCT06903234

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

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This study is active but is not currently recruiting participants.

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Key information

Conditions

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

Age range

18 year–100 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis Investigative Site

Basel, Switzerland

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Iptacopan

Drug

Adult patients with PNH treated with iptacopan

Other names: Fabhalta

Primary outcomes

  1. Number of patients with infections caused by encapsulated bacteria

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

    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).

  2. Cumulative incidence of infections (event probability as a function of time), caused by encapsulated bacteria

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

    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).

  3. Number of patients with infections events per 100 participants -years (incidence rates) caused by encapsulated bacteria

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

    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).

  4. Number of infections episodes per 100 patients -years (occurrence rates) caused by encapsulated bacteria

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

    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).

Secondary outcomes

  1. Number of patients with serious infections caused by encapsulated bacteria and all serious infection

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

    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.

  2. Cumulative incidence of serious infections, caused by encapsulated bacteria and all serious infection (event probability as a function of time)

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

    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.

  3. Number of patients with serious infections events per 100 patients -years (incidence rates) caused by encapsulated bacteria and all serious infection

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

    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.

  4. Number of serious infections episodes per 100 patients -years (occurrence rates) caused by encapsulated bacteria and all serious infection

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

    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.

  5. Number of patients with potential breakthrough hemolysis, solid tumors, hematological malignancies, Major adverse vascular events (MAVEs), serious adverse events (SAEs), hyperlipidemia and thrombocytopenia

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

    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.

  6. Cumulative incidence of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia (event probability as a function of time)

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

    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.

  7. Number of patients with potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia events per 100 patients -years (incidence rates)

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

    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.

  8. Number of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia episodes per 100 patients -years (occurrence rates)

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

    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.

  9. Number of patients with death due to any cause

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

    To describe the short- and long-term risk of all-cause mortality in patients with PNH treated with iptacopan in routine clinical practice.

  10. Cumulative incidence of death due to any cause (event probability as a function of time)

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

    To describe the short- and long-term risk of all-cause mortality in patients with PNH treated with iptacopan in routine clinical practice.

  11. Number of patients with death due to any cause events per 100 patients -years (incidence rates)

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

    To describe the short- and long-term risk of all-cause mortality in patients with PNH treated with iptacopan in routine clinical practice.

  12. Number of patients vaccinated against Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae at each study visit

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

    To describe the number of patients receiving mandatory and recommended vaccinations against encapsulated bacteria.

  13. Number of patients with serious hemolysis following discontinuation of iptacopan

    Time frame: From the iptacopan discontinuation up to 14 days

    To describe the risk of serious hemolysis following discontinuation of iptacopan in patients with PNH treated with iptacopan in routine clinical practice.

  14. Number of patients who became pregnant during treatment with iptacopan, exposure characteristics (e.g. trimester of exposure) and birth outcomes

    Time frame: From the Last Menstrual Period to pregnancy outcome (in case of live birth, up to 12 months post delivery)

    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.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2029
Study completion
2029
First posted
Mar 30, 2025
Registry last updated
Jun 23, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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