A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Paroxysmal Nocturnal Hemoglobinuria, C3 Glomerulopathy. Basic parameters: 18 years — 99 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- South Korea
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Post-marketing Surveillance of Fabhalta® (Iptacopan) in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) or C3 Glomerulopathy (C3G)
Overview
This is a post-marketing surveillance study conducted as part of the Risk Management Plan (RMP) for South Korea, to evaluate the safety and effectiveness of iptacopan in real-world clinical settings for the treatment of either PNH or C3G in Korean patients. Prospective data will be collected from patient medical records to address the objectives for all eligible populations.
Primary outcome measures
- Incidence Rate of Adverse Events (AE), Serious Adverse Events (SAE), and Adverse Drug Reactions (ADR) [Time frame: Up to 2 years]
Secondary outcome measures (12)
- Hemoglobin Levels [Time frame: Baseline and Week 24]
- Lactate Dehydrogenase (LDH) levels [Time frame: Baseline and Week 24]
- White Blood Cell Count (WBC) [Time frame: Baseline and Week 24]
- Red Blood Cell Count (RBC) [Time frame: Baseline and Week 24]
- Hematocrit Levels [Time frame: Baseline and Week 24]
- Platelet Count [Time frame: Baseline and Week 24]
- Reticulocyte Count [Time frame: Baseline and Week 24]
- Ferritin Levels [Time frame: Baseline and Week 24]
- Total Bilirubin Levels [Time frame: Baseline and Week 24]
- Blood Urea Nitrogen (BUN) Levels [Time frame: Baseline and Week 24]
- Creatinine Levels [Time frame: Baseline and Week 24]
- Estimated Glomerular Filtration Rate (eGFR) [Time frame: Baseline and Week 24]
Eligibility criteria
Inclusion criteria
- Patients aged 18 years or older who have been diagnosed with PNH or C3G.
- Patients who have received vaccination in accordance with the approved Korean labeling prior to initiating treatment with iptacopan.
- Patients who are being treated or will be treated with iptacopan in accordance with the approved Korean labeling.
- Patients who have voluntarily provided consent for study participation (written informed consent).
Exclusion criteria
- Patients who fall under the contraindications for iptacopan administration according to approved Korean labeling.
- Patients for whom iptacopan administration is deemed inappropriate based on the investigator's judgment.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
South Korea · 3 centers
- Novartis Investigative Site — Jeollanam
- Novartis Investigative Site — Seoul
- Novartis Investigative Site — Seoul
Identifiers
NCT: NCT07416162 · CLNP023C1KR01