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

June 19, 2026 updated by: Novartis Pharmaceuticals

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

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
200

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

  • Name: Novartis Pharmaceuticals

Study Locations

  • Switzerland
      • Basel, Switzerland
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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).
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
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).
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
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).
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
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).
From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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.
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)
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.
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
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.
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
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.
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
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.
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)
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.
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)
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.
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)
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.
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
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.
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)
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.
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)
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.
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
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.
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
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.
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
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.
From the Last Menstrual Period to pregnancy outcome (in case of live birth, up to 12 months post delivery)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 31, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 1, 2029

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 1, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 24, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 24, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 30, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 23, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 19, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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