Clinical trial NCT06903234: Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry
Official title: Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the Non-interventional IPIG PNH Registry
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Hemoglobinuria, Paroxysmal
Keywords: Paroxysmal nocturnal hemoglobinuria
Iptacopan
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2025-03-30
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult patients with PNH who are enrolled in the IPIG PNH Registry, newly treated with iptacopan.

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of patients with infections caused by encapsulated bacteria
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Cumulative incidence of infections (event probability as a function of time), caused by encapsulated bacteria
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Number of patients with infections events per 100 participants -years (incidence rates) caused by encapsulated bacteria
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Number of infections episodes per 100 patients -years (occurrence rates) caused by encapsulated bacteria
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Secondary outcomes:
measure: Number of patients with serious infections caused by encapsulated bacteria and all serious infection
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Cumulative incidence of serious infections, caused by encapsulated bacteria and all serious infection (event probability as a function of time)
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Number of patients with serious infections events per 100 patients -years (incidence rates) caused by encapsulated bacteria and all serious infection
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Number of serious infections episodes per 100 patients -years (occurrence rates) caused by encapsulated bacteria and all serious infection
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Number of patients with potential breakthrough hemolysis, solid tumors, hematological malignancies, Major adverse vascular events (MAVEs), serious adverse events (SAEs), hyperlipidemia and thrombocytopenia
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Cumulative incidence of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia (event probability as a function of time)
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Number of patients with potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia events per 100 patients -years (incidence rates)
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Number of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia episodes per 100 patients -years (occurrence rates)
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Number of patients with death due to any cause
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Cumulative incidence of death due to any cause (event probability as a function of time)
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Number of patients with death due to any cause events per 100 patients -years (incidence rates)
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Number of patients vaccinated against Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae at each study visit
timeFrame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
measure: Number of patients with serious hemolysis following discontinuation of iptacopan
timeFrame: From the iptacopan discontinuation up to 14 days
measure: Number of patients who became pregnant during treatment with iptacopan, exposure characteristics (e.g. trimester of exposure) and birth outcomes
timeFrame: From the Last Menstrual Period to pregnancy outcome (in case of live birth, up to 12 months post delivery)
Other outcomes:
Locations:
city: Basel
country: Switzerland
facility: Novartis Investigative Site
Study officials:
References: