Paroxysmal Nocturnal HemoglobinuriaNCT07416162
A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy
An observational study of Paroxysmal Nocturnal Hemoglobinuria and C3 Glomerulopathy, run by Novartis Pharmaceuticals at 3 sites in South Korea.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria, C3 Glomerulopathy
- How many people
- 21 participants
- Where
- 3 sitesSouth Korea
- Study dates
- 15 Jun 2026to 1 Feb 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult patients in South Korea who receive iptacopan for the treatment of PNH or C3G during routine clinical practice.
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
South Korea3 sites, 3 recruiting
- Recruiting
Novartis Investigative Site
Jeollanam, 519763
- Recruiting
Novartis Investigative Site
Seoul, 03080
- Recruiting
Novartis Investigative Site
Seoul, 06591
About this study
Official title: A Post-marketing Surveillance of Fabhalta® (Iptacopan) in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) or C3 Glomerulopathy (C3G)
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.
- Keywords
- Iptacopan, Factor B inhibitor, Post-Marketing Surveillance, Complement inhibition, Hemolysis, Proteinuria, Dense Deposit Disease, Paroxysmal Nocturnal Hemoglobinuria, C3 Glomerulopathy
- Medical subject headings
- Hemoglobinuria, Paroxysmal, Hemolysis, Proteinuria, Glomerulonephritis, Membranoproliferative
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 2 years
Secondary outcomes (16)
Hemoglobin Levels
Measured: Baseline and Week 24
Lactate Dehydrogenase (LDH) levels
Measured: Baseline and Week 24
White Blood Cell Count (WBC)
Measured: Baseline and Week 24
Red Blood Cell Count (RBC)
Measured: Baseline and Week 24
Hematocrit Levels
Measured: Baseline and Week 24
Platelet Count
Measured: Baseline and Week 24
Reticulocyte Count
Measured: Baseline and Week 24
Ferritin Levels
Measured: Baseline and Week 24
Total Bilirubin Levels
Measured: Baseline and Week 24
Blood Urea Nitrogen (BUN) Levels
Measured: Baseline and Week 24
Creatinine Levels
Measured: Baseline and Week 24
Estimated Glomerular Filtration Rate (eGFR)
Measured: 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.
Measured: Week 24
Change From Baseline in Serum Creatinine
Measured: Baseline and Week 24
Change From Baseline in eGFR
Measured: 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".
Measured: Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Feb 2026
- Study start15 Jun 2026
- Primary completion1 Feb 2029 (estimated)
- Study completion1 Feb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07416162
- Sponsor's protocol ID
- CLNP023C1KR01
- Status last verified
- Jun 2026
- First submitted
- 11 Feb 2026
- Last updated
- 26 Jun 2026
- Results posted
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Post-marketing Surveillance of Fabhalta® (Iptacopan) in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) or C3 Glomerulopathy (C3G)
- ClinicalTrials.gov ID
- NCT07416162
- Acronym / sponsor ID
- CLNP023C1KR01
- Keywords
- Iptacopan, Factor B inhibitor, Post-Marketing Surveillance, Complement inhibition, Hemolysis, Proteinuria, Dense Deposit Disease, Paroxysmal Nocturnal Hemoglobinuria
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 15 Jun 2026 (actual)
- Primary completion
- 1 Feb 2029 (estimated)
- Study completion
- 1 Feb 2029 (estimated)
- First posted
- 18 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult patients in South Korea who receive iptacopan for the treatment of PNH or C3G during routine clinical practice.
Key inclusion criteria (4 listed)
- 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).
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
16 secondary outcomes
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]
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
Time frame: Week 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2026.
- Individual participant dataSharing planned: No
Related trials for Paroxysmal Nocturnal Hemoglobinuria
Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Novartis Pharmaceuticals contact
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Paroxysmal Nocturnal Hemoglobinuria and C3 Glomerulopathy, run by Novartis Pharmaceuticals at 3 sites in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (+41613241111, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence Rate of Adverse Events (AE), Serious Adverse Events (SAE), and Adverse Drug Reactions (ADR) (time frame: Up to 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jun 2026.