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

NCT ID: NCT06903234

Last Updated: 2025-05-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

200 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-03-31

Study Completion Date

2029-10-01

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Hemoglobinuria, Paroxysmal

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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Iptacopan

Adult patients with PNH treated with iptacopan in routine care.

Iptacopan

Intervention Type DRUG

Adult patients with PNH treated with iptacopan

Interventions

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Iptacopan

Adult patients with PNH treated with iptacopan

Intervention Type DRUG

Other Intervention Names

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Fabhalta

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Novartis Investigative Site

Basel, , Switzerland

Site Status

Countries

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Switzerland

Other Identifiers

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CLNP023C12003

Identifier Type: -

Identifier Source: org_study_id

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