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

NCT07416162 | Not Yet Recruiting

• 1 other identifier: CLNP023C1KR01
• Study Type: observational
• Target: 21
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria; C3 Glomerulopathy

Keywords

Iptacopan; Factor B inhibitor; Post-Marketing Surveillance; Complement inhibition; Hemolysis; Proteinuria; Dense Deposit Disease; Paroxysmal Nocturnal Hemoglobinuria; C3 Glomerulopathy

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (16)

• 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

Study Arms (2)

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.

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adult patients in South Korea who receive iptacopan for the treatment of PNH or C3G during routine clinical practice.

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal; Hemolysis; Proteinuria; Glomerulonephritis, Membranoproliferative

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Urination Disorders; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Urological Manifestations; Signs and Symptoms; Glomerulonephritis; Nephritis; Kidney Diseases; Immune System Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 11, 2026
• First Posted: February 18, 2026
• Study Start (Estimated): July 31, 2026
• Primary Completion (Estimated): February 1, 2029
• Study Completion (Estimated): February 1, 2029
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share