A Post-marketing Surveillance of Fabhalta® (Iptacopan) in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) or C3 Glomerulopathy (C3G)
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Novartis Pharmaceuticals
- Enrollment
- 21
- Locations
- 3
- Primary Endpoint
- Incidence Rate of Adverse Events (AE), Serious Adverse Events (SAE), and Adverse Drug Reactions (ADR)
Study Overview
Brief Summary
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.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 18 Years to 99 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
PNH Cohort
Patients in South Korea who receive at least one dose of iptacopan for the treatment of PNH.
C3G Cohort
Patients in South Korea who receive at least one dose of iptacopan for the treatment of C3G.
Outcomes
Primary Outcomes
Incidence Rate of Adverse Events (AE), Serious Adverse Events (SAE), and Adverse Drug Reactions (ADR)
Time Frame: Up to 2 years
Incidence rate of all AE/ADR, SAE/serious adverse drug reactions (SADR), unexpected AE/ADR, and unexpected SAE/SADR that occur after the administration of iptacopan.
Secondary Outcomes
- Estimated Glomerular Filtration Rate (eGFR)(Baseline and Week 24)
- Hemoglobin Levels(Baseline and Week 24)
- Lactate Dehydrogenase (LDH) levels(Baseline and Week 24)
- White Blood Cell Count (WBC)(Baseline and Week 24)
- Red Blood Cell Count (RBC)(Baseline and Week 24)
- Hematocrit Levels(Baseline and Week 24)
- Platelet Count(Baseline and Week 24)
- Reticulocyte Count(Baseline and Week 24)
- Ferritin Levels(Baseline and Week 24)
- Total Bilirubin Levels(Baseline and Week 24)
- Blood Urea Nitrogen (BUN) Levels(Baseline and Week 24)
- Creatinine Levels(Baseline and Week 24)
- Log-transformed Ratio to Baseline in Urine Protein Creatinine Ratio (UPCR) [Random Spot Urine Test](Week 24)
- Change From Baseline in Serum Creatinine(Baseline and Week 24)
- Change From Baseline in eGFR(Baseline and Week 24)
- Overall Improvement Assessed by Investigator(Week 24)