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NCT Number: NCT07416162

A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy

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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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria Anemia Anemia, Hemolytic Bone Marrow Diseases C3 Glomerulopathy Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Glomerulonephritis Glomerulonephritis, Membranoproliferative Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Hemolysis Immune System Diseases Kidney Diseases Male Urogenital Diseases Myelodysplastic Syndromes Nephritis Pathologic Processes Pathological Conditions, Signs and Symptoms Proteinuria Signs and Symptoms Urination Disorders Urogenital Diseases Urologic Diseases Urological Manifestations

Age range

18 year–99 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis Investigative Site, Jeollanam, South Korea

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. 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

  1. Hemoglobin Levels

    Time frame: Baseline and Week 24

  2. Lactate Dehydrogenase (LDH) levels

    Time frame: Baseline and Week 24

  3. White Blood Cell Count (WBC)

    Time frame: Baseline and Week 24

  4. Red Blood Cell Count (RBC)

    Time frame: Baseline and Week 24

  5. Hematocrit Levels

    Time frame: Baseline and Week 24

  6. Platelet Count

    Time frame: Baseline and Week 24

  7. Reticulocyte Count

    Time frame: Baseline and Week 24

  8. Ferritin Levels

    Time frame: Baseline and Week 24

  9. Total Bilirubin Levels

    Time frame: Baseline and Week 24

  10. Blood Urea Nitrogen (BUN) Levels

    Time frame: Baseline and Week 24

  11. Creatinine Levels

    Time frame: Baseline and Week 24

  12. Estimated Glomerular Filtration Rate (eGFR)

    Time frame: Baseline and Week 24

  13. Log-transformed Ratio to Baseline in Urine Protein Creatinine Ratio (UPCR) [Random Spot Urine Test]

    Time frame: 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.

  14. Change From Baseline in Serum Creatinine

    Time frame: Baseline and Week 24

  15. Change From Baseline in eGFR

    Time frame: Baseline and Week 24

  16. Overall Improvement Assessed by Investigator

    Time frame: 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".

Study contacts

Contact information is provided by the study sponsor or research team.

Novartis Pharmaceuticals

CONTACT

[email protected]

+41613241111

Novartis Pharmaceuticals

CONTACT

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

A Post-marketing Surveillance of Fabhalta® (Iptacopan) in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) or C3 Glomerulopathy (C3G)

Important dates

Study start
2026
Primary completion
2029
Study completion
2029
First posted
Feb 18, 2026
Registry last updated
Jun 26, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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