An observational study in Paroxysmal Nocturnal Hemoglobinuria and C3 Glomerulopathy, sponsored by Novartis Pharmaceuticals. Recruiting at 3 sites in South Korea. Open to participants aged 18 Years to 99 Years. Per ClinicalTrials.gov, last updated 2026-06-26.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Observational
Among 16 studies in Hemoglobinuria, Paroxysmal by Novartis Pharmaceuticals — 6 open to participants now, 9 under way, 2 of them phase 3. Hemoglobinuria, Paroxysmal · Novartis Pharmaceuticals.
Among the 16 studies in Hemoglobinuria, Paroxysmal by Novartis Pharmaceuticals, this one is one of 2 that started in 2026, is one of 5 observational ones, and was the 16th registered.
191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.
This study's planned enrollment of 21 is below the median of 90 across 37 observational studies indexed under Hemoglobinuria, Paroxysmal.
Browse Hemoglobinuria, Paroxysmal studies →6 of the 16 studies in Hemoglobinuria, Paroxysmal by Novartis Pharmaceuticals are open now, in 8 countries. Each count lists the studies.
Of the 30 Hemoglobinuria, Paroxysmal studies recruiting now, 14 (47%) set no upper age limit and 12 (40%) stop at 75 or younger; 12 take children under 18; 4 take healthy volunteers.
This study: 18 Years to 99 Years · all sexes · no healthy volunteers.
The full table on the hub →16 studies; the count open now beside each. This study's row is marked.
First in 2015; this study’s year is marked. 2 started in 2026.
Median enrollment 28 across the 8 interventional studies, 80 across the 7 observational. Of the 7 that completed or were terminated, 6 (86%) have results posted.
Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
2 of the studies in Hemoglobinuria, Paroxysmal by Novartis Pharmaceuticals that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04747613Phase 3Novartis PharmaceuticalsLong-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria | 2027-10-18 |
| NCT06931691Novartis PharmaceuticalsA Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China | 2027-04-21 |
Among the 16 studies in Hemoglobinuria, Paroxysmal by Novartis Pharmaceuticals, from their registry updates:
Nothing recorded among them since 2026-09-25.
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.
Adult patients in South Korea who receive iptacopan for the treatment of PNH or C3G during routine clinical practice.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients in South Korea who receive at least one dose of iptacopan for the treatment of PNH.
Patients in South Korea who receive at least one dose of iptacopan for the treatment of C3G.
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.
Time frame: Up to 2 years
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
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.
Time frame: Week 24
Change From Baseline in Serum Creatinine
Time frame: Baseline and Week 24
Change From Baseline in eGFR
Time frame: 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".
Time frame: Week 24
Plan to share: No
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