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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| Measure | Description | Time Frame |
|---|---|---|
| Incidence Rate of Adverse Events (AE), Serious Adverse Events (SAE), and Adverse Drug Reactions (ADR) | 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. | Up to 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Hemoglobin Levels | Baseline and Week 24 | |
| Lactate Dehydrogenase (LDH) levels | Baseline and Week 24 | |
| White Blood Cell Count (WBC) |
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Inclusion criteria:
Exclusion criteria:
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Adult patients in South Korea who receive iptacopan for the treatment of PNH or C3G during routine clinical practice.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Novartis Pharmaceuticals | Contact | +41613241111 | novartis.email@novartis.com | |
| Novartis Pharmaceuticals | Contact |
| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Recruiting | Jeollanam | 519763 | South Korea | ||
| Novartis Investigative Site |
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| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| D006461 | Hemolysis |
| D011507 | Proteinuria |
| D015432 | Glomerulonephritis, Membranoproliferative |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| 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 |
| Estimated Glomerular Filtration Rate (eGFR) | Baseline and Week 24 |
| Log-transformed Ratio to Baseline in Urine Protein Creatinine Ratio (UPCR) [Random Spot Urine Test] | To evaluate the effect of iptacopan on proteinuria at Week 24. A random spot urine sample is a single urine sample collected at any time of the day. The level of protein in this sample is compared to the level of creatinine to calculate the UPCR, which is commonly used to assess the severity of proteinuria. | 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 | The investigator will evaluate the overall improvement of the therapeutic effect of iptacopan on PNH and C3G at the last visit on the basis of the patient's condition before the start of iptacopan and during the clinical course thereafter, as "improved", "unchanged", "aggravated", or "unable to assess". | Week 24 |
| Recruiting |
| Seoul |
| 03080 |
| South Korea |
| Novartis Investigative Site | Recruiting | Seoul | 06591 | South Korea |
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D014555 | Urination Disorders |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D020924 | Urological Manifestations |
| D012816 | Signs and Symptoms |
| D005921 | Glomerulonephritis |
| D009393 | Nephritis |
| D007674 | Kidney Diseases |
| D007154 | Immune System Diseases |