Paroxysmal Nocturnal HemoglobinuriaPNH-NISNCT06931691
A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Novartis Pharmaceuticals at 14 sites in China.
Trial summary
The implementation of new standards for the management of PNH and the use of iptacopan in patients with PNH are expected to change the treatment landscape and improve the overall prognosis of patients. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- LNP023
- Type of treatment
- Pill or oral medicine
- How many people
- 80 participants
- Where
- 14 sitesChina
- Study dates
- 10 Jun 2025to 21 Apr 2027
- 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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult PNH patients receiving iptacopan for the first time, including those who are complement inhibitor naive and treated
Who may be able to join
- Patient who meets all the following criteria can be included in this study:
- Age ≥ 18 years at the time of signing the ICF;
- Patient with a documented diagnosis of PNH;
- Patient who has never received complement inhibitor therapy;
- Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
- Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation; If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible.
Show all 23Show fewer
- Patient who has signed the ICF.
- For Cohort 2,
- Patient who meets all the following criteria can be included in this study:
- Age ≥ 18 years at the time of signing the ICF;
- Patient with a documented diagnosis of PNH; Patients who have been receiving stable treatment with C5 complement inhibitors for at least three months prior to enrollment;
- Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
- Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation;
- If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible.
- Patient who has signed the ICF. Exclusion criteria
- For Cohort 1 and Cohort 2, patients who meet any of the following criteria will meet the exclusion criteria for this study:
- Participating in an interventional PNH clinical study;
- Have an active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to first dose;
- Documented with a history of recurrent invasive infections, e.g. active systemic bacterial, viral or fungal infection within 14 days prior to first dose;
- Documented with a history of HIV infection;
- Women who are pregnant or breastfeeding or intending to conceive during the study period;
- Existence of bone marrow failure (reticulocytes < 100 × 109/L, platelets < 30 × 109/L, and neutrophils < 0.5 × 109/L) determined by the investigator;
- Other conditions that are not suitable for participating in the study, in the judgment of the investigator.
Who cannot join
None listed.
Trial locations and contacts
14 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
14 sites in 1 country
China14 sites, 14 recruiting
- Recruiting
Novartis Investigative Site
Changchun, Jilin, 130021
- Recruiting
Novartis Investigative Site
Guangzhou, Guangdong, 510000
- Recruiting
Novartis Investigative Site
Guangzhou, Guangdong, 510080
- Recruiting
Novartis Investigative Site
Hefei, Anhui, 230022
- Recruiting
Novartis Investigative Site
Jinan, 250012
- Recruiting
Novartis Investigative Site
Nanchang, Jiangxi, 330006
- Recruiting
Novartis Investigative Site
Nantong, Jiangsu, 226000
- Recruiting
Novartis Investigative Site
Shijiazhuang, Hebei, 050000
- Recruiting
Novartis Investigative Site
Tianjin, 300020
- Recruiting
Novartis Investigative Site
Tianjin, 300052
- Recruiting
Novartis Investigative Site
Wuhan, Hubei, 430030
- Recruiting
Novartis Investigative Site
Wuhan, 430022
- Recruiting
Novartis Investigative Site
Zhengzhou, Henan, 450003
- Recruiting
Novartis Investigative Site
Zhengzhou, Henan, 450008
About this study
Official title: A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan on Disease Management, Treatment-Related Outcomes and Healthcare Resource Utilization for Adult Patients With Paroxysmal Nocturnal Hemoglobinuria in China
The implementation of new standards for the management of PNH and the use of iptacopan in patients with PNH are expected to change the treatment landscape and improve the overall prognosis of patients.
Based on these backgrounds, we plan to conduct a real-world study of iptacopan to further evaluate its impact on treatment-related outcomes, disease management, and healthcare resource utilization in Chinese patients with PNH.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- LNP023
- Keywords
- Phase IV, Adult Patients, Paroxysmal Nocturnal Hemoglobinuria, LNP023
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LNP023Drug
Also called Iptacopan Hydrochloride
Capsules for oral administration
| Study group | Role | What they receive |
|---|---|---|
| Cohort1 | PNH patients with a confirmed diagnosis of PNH in the medical record and who had never received complement inhibitor therapy (complement inhibitor naive patients) Receives: Drug: LNP023 | |
| Cohort 2 | PNH patients with a confirmed diagnosis of PNH in the medical record who had been treated with a stable dose of a C5 complement inhibitor (e.g., eculizumab) for at least 3 months (C5i-treated patients; this cohort will be only start enrollment after the indication for iptacopan is to be approved in China). Receives: Drug: LNP023 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in hemoglobin (Hb) levels at designated time points
Assessment of the Hematological Response to iptacopan.
Measured: Baseline, 12 months
Secondary outcomes (23)
Number of participants having Hb normalization (Hb level ≥ 12 g/dL) after iptacopan initiation.
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: 12 months
Duration of Hb level ≥ 12 g/dL within a 12-month
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: 12 months
Change from baseline in LDH Levels after iptacopan initiation
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: Baseline, 12 Months
Number of participants with LDH levels ≤ 1.5 x ULN before vs. after iptacopan initiation
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: 12 months
Number of participants having LDH normalization before and after iptacopan initiation
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: 12 monhts
Duration of LDH level ≤ 1.5 x ULN within 12 months
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: 12 months
Change from baseline in ARC Levels after iptacopan initiation
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: Baseline, 12 monhts
Number of participants having ARC normalization before and after iptacopan initiation
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: Baseline, 12 months
Change from baseline in bilirubin after iptacopan initiation
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: Baseline, 12 months
Number of participants with hepatosplenomegaly before and after iptacopan initiation
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: Baseline, 12 months
Number of participants with a positive coomb's test after iptacopan initiation
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: 12 months
Number of participants with PNH related signs or symptoms before and after iptacopan treatment
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: Baseline, 12 months
Change from baseline in PNH clone value
Change from baseline in flow cytometry results to assess the impact of iptacopan on Treatment-Related Outcomes.
Measured: Baseline, 12 months
Rate, reasons (complement activation conditions (CAC), missed doses, etc.), duration, and interventions for breakthrough hemolysis (BTH)
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Measured: 12 months
Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale after iptacopan initiation
FACIT is a 40-item measure that assesses self-reported fatigue and its impact upon daily activities and function to assess the Impact of iptacopan on Treatment-Related Outcomes. The minimum value is 0 and maximum value is 52, and higher scores mean a worse outcome.
Measured: Baseline, 12 months
Work Productivity and Activity Impairment Questionnaire-Specific Health Problems (WPAI-SHP) at each visit, change from baseline in WPAI-SHP after iptacopan initiation
WPAI-SHP is a tool used to measure the impact of health problems on work productivity and regular activities. The scale includes several components.
Absenteeism: This measures the percentage of work time missed due to a specific health problem.
Presenteeism: This measures the percentage of impairment while working due to the health problem.
Work productivity loss: This combines absenteeism and presenteeism to measure overall work impairment.
Activity impairment: This measures the percentage of impairment in regular activities due to the health problem.
The scores are expressed as impairment percentages, with higher numbers indicating greater impairment and less productivity, which means worse outcomes.
Measured: Baseline, 12 months
Adverse events (AEs) and Serious adverse events (SAEs) occurring during treatment with iptacopan
Assessing the safety of iptacopan
Measured: 12 monhts
Treatment regimen for PNH 12 months before iptacopan treatment; reasons of discontinuation of previous complement inhibitor;
Assessing the impact of iptacopan on disease management
Measured: Baseline
Use of concomitant medications (e.g., corticosteroids, androgen, immunosuppressants, anti-coagulants, etc.) for PNH and PNH-related complications (e.g. thrombosis, renal failure, etc.) before and after iptacopan treatment;
Assessing the impact of iptacopan on disease management
Measured: Baseline, 12 months
Duration of iptacopan treatment, reasons for discontinuation, switching to other treatments, proportion of patients who switch from iptacopan to other treatments;
Assessing the impact of iptacopan on disease management
Measured: 12 monhts
Number of participants with blood transfusion before and after iptacopan treatment, duration and number of units of blood transfusion due to BTH
Assessing the impact of iptacopan on disease management
Measured: Baseline, 12 months
Number of participants hospitalized, outpatient, emergency room and intensive care unit (ICU) visits related to PNH
Assessing the impact of iptacopan on healthcare resource utilization
Measured: 12 monhts
Length of inpatient stay related to PNH
Assessing the impact of iptacopan on healthcare resource utilization
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2025
- Study start10 Jun 2025
- Primary completion21 Apr 2027 (estimated)
- Study completion21 Apr 2027 (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
- Cross-sectional
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06931691
- Sponsor's protocol ID
- CLNP023C1CN01
- Acronym
- PNH-NIS
- Status last verified
- May 2026
- First submitted
- 14 Mar 2025
- Last updated
- 1 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan on Disease Management, Treatment-Related Outcomes and Healthcare Resource Utilization for Adult Patients With Paroxysmal Nocturnal Hemoglobinuria in China
- ClinicalTrials.gov ID
- NCT06931691
- Acronym / sponsor ID
- PNH-NIS / CLNP023C1CN01
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- Phase IV, Adult Patients, Paroxysmal Nocturnal Hemoglobinuria, LNP023
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 10 Jun 2025 (actual)
- Primary completion
- 21 Apr 2027 (estimated)
- Study completion
- 21 Apr 2027 (estimated)
- First posted
- 17 Apr 2025 (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.
The implementation of new standards for the management of PNH and the use of iptacopan in patients with PNH are expected to change the treatment landscape and improve the overall prognosis of patients.
Based on these backgrounds, we plan to conduct a real-world study of iptacopan to further evaluate its impact on treatment-related outcomes, disease management, and healthcare resource utilization in Chinese patients with PNH.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort1 | – | Drug: LNP023PNH patients with a confirmed diagnosis of PNH in the medical record and who had never received complement inhibitor therapy (complement inhibitor naive patients) |
| Cohort 2 | – | Drug: LNP023PNH patients with a confirmed diagnosis of PNH in the medical record who had been treated with a stable dose of a C5 complement inhibitor (e.g., eculizumab) for at least 3 months (C5i-treated patients; this cohort will be only start enrollment after the indication for iptacopan is to be approved in China). |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult PNH patients receiving iptacopan for the first time, including those who are complement inhibitor naive and treated
Key inclusion criteria (23 listed)
- Age ≥ 18 years at the time of signing the ICF;
- Patient with a documented diagnosis of PNH;
- Patient who has never received complement inhibitor therapy;
- Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
- Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation; If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LNP023Also: Iptacopan HydrochlorideCapsules for oral administration | Drug | Cohort 2, Cohort1 |
Primary outcome
Change from baseline in hemoglobin (Hb) levels at designated time points
Time frame: Baseline, 12 months
Assessment of the Hematological Response to iptacopan.
23 secondary outcomes
Number of participants having Hb normalization (Hb level ≥ 12 g/dL) after iptacopan initiation.
Time frame: 12 months
Duration of Hb level ≥ 12 g/dL within a 12-month
Time frame: 12 months
Change from baseline in LDH Levels after iptacopan initiation
Time frame: Baseline, 12 Months
Number of participants with LDH levels ≤ 1.5 x ULN before vs. after iptacopan initiation
Time frame: 12 months
Number of participants having LDH normalization before and after iptacopan initiation
Time frame: 12 monhts
Duration of LDH level ≤ 1.5 x ULN within 12 months
Time frame: 12 months
Change from baseline in ARC Levels after iptacopan initiation
Time frame: Baseline, 12 monhts
Number of participants having ARC normalization before and after iptacopan initiation
Time frame: Baseline, 12 months
Change from baseline in bilirubin after iptacopan initiation
Time frame: Baseline, 12 months
Number of participants with hepatosplenomegaly before and after iptacopan initiation
Time frame: Baseline, 12 months
Number of participants with a positive coomb's test after iptacopan initiation
Time frame: 12 months
Number of participants with PNH related signs or symptoms before and after iptacopan treatment
Time frame: Baseline, 12 months
Change from baseline in PNH clone value
Time frame: Baseline, 12 months
Rate, reasons (complement activation conditions (CAC), missed doses, etc.), duration, and interventions for breakthrough hemolysis (BTH)
Time frame: 12 months
Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale after iptacopan initiation
Time frame: Baseline, 12 months
Work Productivity and Activity Impairment Questionnaire-Specific Health Problems (WPAI-SHP) at each visit, change from baseline in WPAI-SHP after iptacopan initiation
Time frame: Baseline, 12 months
Adverse events (AEs) and Serious adverse events (SAEs) occurring during treatment with iptacopan
Time frame: 12 monhts
Treatment regimen for PNH 12 months before iptacopan treatment; reasons of discontinuation of previous complement inhibitor;
Time frame: Baseline
Use of concomitant medications (e.g., corticosteroids, androgen, immunosuppressants, anti-coagulants, etc.) for PNH and PNH-related complications (e.g. thrombosis, renal failure, etc.) before and after iptacopan treatment;
Time frame: Baseline, 12 months
Duration of iptacopan treatment, reasons for discontinuation, switching to other treatments, proportion of patients who switch from iptacopan to other treatments;
Time frame: 12 monhts
Number of participants with blood transfusion before and after iptacopan treatment, duration and number of units of blood transfusion due to BTH
Time frame: Baseline, 12 months
Number of participants hospitalized, outpatient, emergency room and intensive care unit (ICU) visits related to PNH
Time frame: 12 monhts
Length of inpatient stay related to PNH
Time frame: 12 months
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 1 Jun 2026.
- Individual participant dataSharing planned: No
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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
14 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, run by Novartis Pharmaceuticals at 14 sites in China.
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 23 inclusion and 0 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 1 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 14 sites in China. 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?
Change from baseline in hemoglobin (Hb) levels at designated time points (time frame: Baseline, 12 months). 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 1 Jun 2026.