---
title: Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry
nct_id: NCT06903234
overall_status: ACTIVE_NOT_RECRUITING
sponsor: Novartis Pharmaceuticals
study_type: OBSERVATIONAL
primary_condition: Hemoglobinuria, Paroxysmal
countries: Switzerland
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT06903234.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT06903234"
ct_last_update_post_date: 2025-05-15
last_seen_at: "2026-05-12T06:35:35.485Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# 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

**NCT ID:** [NCT06903234](https://clinicaltrials.gov/study/NCT06903234)

## Key Facts

- **Status:** ACTIVE_NOT_RECRUITING
- **Study Type:** OBSERVATIONAL
- **Target Enrollment:** 200
- **Lead Sponsor:** Novartis Pharmaceuticals
- **Conditions:** Hemoglobinuria, Paroxysmal
- **Start Date:** 2025-03-31
- **Completion Date:** 2029-10-01
- **CT.gov Last Update:** 2025-05-15

## 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

- **Minimum age:** 18 Years
- **Maximum age:** 100 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
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
```

## Arms

- **Iptacopan** — Adult patients with PNH treated with iptacopan in routine care.

## Interventions

- **Iptacopan** (DRUG) — Adult patients with PNH treated with iptacopan

## Primary Outcomes

- **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).
- **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).
- **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).
- **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

- **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.)_
- **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.)_
- **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.)_
- **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.)_
- **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.)_
- **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.)_
- **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.)_
- **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.)_
- **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.)_
- **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.)_
- **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.)_
- **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.)_
- **Number of patients with serious hemolysis following discontinuation of iptacopan** _(time frame: 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))_

## Locations (1)

- Novartis Investigative Site, Basel, Switzerland

## Recent Field Changes (last 30 days)

- `eligibility.sex` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.maxAge` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.novartis investigative site|basel||switzerland` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT06903234.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT06903234*  
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