A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China (PNH-NIS)

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.

Study Overview

• Status: Recruiting
• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Intervention / Treatment: Drug: LNP023

• Study Type: Observational
• Enrollment (Estimated): 80

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: +41613241111; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals

Study Locations

• China
  • Jinan, China, 250012
    • Recruiting
    • Novartis Investigative Site
  • Tianjin, China, 300052
    • Recruiting
    • Novartis Investigative Site
  • Tianjin, China, 300020
    • Recruiting
    • Novartis Investigative Site
  • Wuhan, China, 430022
    • Recruiting
    • Novartis Investigative Site
  • Anhui
    • Hefei, Anhui, China, 230022
      • Recruiting
      • Novartis Investigative Site
  • Guangdong
    • Guangzhou, Guangdong, China, 510080
      • Recruiting
      • Novartis Investigative Site
    • Guangzhou, Guangdong, China, 510000
      • Recruiting
      • Novartis Investigative Site
  • Hebei
    • Shijiazhuang, Hebei, China, 050000
      • Recruiting
      • Novartis Investigative Site
  • Henan
    • Zhengzhou, Henan, China, 450008
      • Recruiting
      • Novartis Investigative Site
    • Zhengzhou, Henan, China, 450003
      • Recruiting
      • Novartis Investigative Site
  • Hubei
    • Wuhan, Hubei, China, 430030
      • Recruiting
      • Novartis Investigative Site
  • Jiangsu
    • Nantong, Jiangsu, China, 226000
      • Recruiting
      • Novartis Investigative Site
  • Jiangxi
    • Nanchang, Jiangxi, China, 330006
      • Recruiting
      • Novartis Investigative Site
  • Jilin
    • Changchun, Jilin, China, 130021
      • Recruiting
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult PNH patients receiving iptacopan for the first time, including those who are complement inhibitor naive and treated

Description

Inclusion criteria For Cohort 1,

Patient who meets all the following criteria can be included in this study:

1. Age ≥ 18 years at the time of signing the ICF;
2. Patient with a documented diagnosis of PNH;
3. Patient who has never received complement inhibitor therapy;
4. Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
5. 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.
6. Patient who has signed the ICF.

For Cohort 2,

Patient who meets all the following criteria can be included in this study:

1. Age ≥ 18 years at the time of signing the ICF;
2. 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;
3. Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
4. 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;
5. 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.
6. 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:

1. Participating in an interventional PNH clinical study;
2. Have an active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to first dose;
3. Documented with a history of recurrent invasive infections, e.g. active systemic bacterial, viral or fungal infection within 14 days prior to first dose;
4. Documented with a history of HIV infection;
5. Women who are pregnant or breastfeeding or intending to conceive during the study period;
6. Existence of bone marrow failure (reticulocytes < 100 × 109/L, platelets < 30 × 109/L, and neutrophils < 0.5 × 109/L) determined by the investigator;
7. Other conditions that are not suitable for participating in the study, in the judgment of the investigator.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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)	Drug: LNP023; Capsules for oral administration; Other Names: Iptacopan Hydrochloride
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).	Drug: LNP023; Capsules for oral administration; Other Names: Iptacopan Hydrochloride

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in hemoglobin (Hb) levels at designated time points	Assessment of the Hematological Response to iptacopan.	Baseline, 12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants having Hb normalization (Hb level ≥ 12 g/dL) after iptacopan initiation.	Assessing the Impact of iptacopan on Treatment-Related Outcomes	12 months
Duration of Hb level ≥ 12 g/dL within a 12-month	Assessing the Impact of iptacopan on Treatment-Related Outcomes	12 months
Change from baseline in LDH Levels after iptacopan initiation	Assessing the Impact of iptacopan on Treatment-Related Outcomes	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	12 months
Number of participants having LDH normalization before and after iptacopan initiation	Assessing the Impact of iptacopan on Treatment-Related Outcomes	12 monhts
Duration of LDH level ≤ 1.5 x ULN within 12 months	Assessing the Impact of iptacopan on Treatment-Related Outcomes	12 months
Change from baseline in ARC Levels after iptacopan initiation	Assessing the Impact of iptacopan on Treatment-Related Outcomes	Baseline, 12 monhts
Number of participants having ARC normalization before and after iptacopan initiation	Assessing the Impact of iptacopan on Treatment-Related Outcomes	Baseline, 12 months
Change from baseline in bilirubin after iptacopan initiation	Assessing the Impact of iptacopan on Treatment-Related Outcomes	Baseline, 12 months
Number of participants with hepatosplenomegaly before and after iptacopan initiation	Assessing the Impact of iptacopan on Treatment-Related Outcomes	Baseline, 12 months
Number of participants with a positive coomb's test after iptacopan initiation	Assessing the Impact of iptacopan on Treatment-Related Outcomes	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	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.	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	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.	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.	Baseline, 12 months
Adverse events (AEs) and Serious adverse events (SAEs) occurring during treatment with iptacopan	Assessing the safety of iptacopan	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	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	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	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	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	12 monhts
Length of inpatient stay related to PNH	Assessing the impact of iptacopan on healthcare resource utilization	12 months

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): June 10, 2025
• Primary Completion (Estimated): April 21, 2027
• Study Completion (Estimated): April 21, 2027

Study Registration Dates

• First Submitted: March 14, 2025
• First Submitted That Met QC Criteria: April 16, 2025
• First Posted (Actual): April 17, 2025

Study Record Updates

• Last Update Posted (Actual): June 1, 2026
• Last Update Submitted That Met QC Criteria: May 28, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Phase IV; Paroxysmal Nocturnal Hemoglobinuria; Adult Patients; LNP023
• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Hemic and Lymphatic Diseases; Hemoglobinuria, Paroxysmal
• Other Study ID Numbers: CLNP023C1CN01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No