C3 Glomerulopathy, Paroxysmal Nocturnal Hemoglobinuria
Conditions
Keywords
Iptacopan, Factor B inhibitor, Post-Marketing Surveillance, Complement inhibition, Hemolysis, Proteinuria, Dense Deposit Disease, Paroxysmal Nocturnal Hemoglobinuria, C3 Glomerulopathy
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.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 99 Years
Healthy volunteers
No
Inclusion criteria
1. Patients aged 18 years or older who have been diagnosed with PNH or C3G. 2. Patients who have received vaccination in accordance with the approved Korean labeling prior to initiating treatment with iptacopan. 3. Patients who are being treated or will be treated with iptacopan in accordance with the approved Korean labeling. 4. Patients who have voluntarily provided consent for study participation (written informed consent).
Exclusion criteria
1. Patients who fall under the contraindications for iptacopan administration according to approved Korean labeling. 2. Patients for whom iptacopan administration is deemed inappropriate based on the investigator's judgment.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence Rate of Adverse Events (AE), Serious Adverse Events (SAE), and Adverse Drug Reactions (ADR) | 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
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
| Estimated Glomerular Filtration Rate (eGFR) | Baseline and Week 24 | — |
| Log-transformed Ratio to Baseline in Urine Protein Creatinine Ratio (UPCR) [Random Spot Urine Test] | Week 24 | 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. |
| 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 | 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". |
Countries
South Korea
Contacts
CONTACTNovartis Pharmaceuticals
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed