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

Not yet recruiting

• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Interventions: Drug: LNP023

• Registration Number: NCT06931691
• Lead Sponsor: Novartis Pharmaceuticals

Brief 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.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-03-14
• Status Verified: 2025-04
• Study First Submitted QC: 2025-04-16
• Last Update Submitted: 2025-04-16
• Study First Posted: 2025-04-17
• Last Update Posted: 2025-04-17
• Study Start: 2025-04-30
• Primary Completion: 2027-04-20
• Study Completion: 2027-04-20

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 80

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Cohort1	LNP023	PNH 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	LNP023	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).

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Number of participants having Hb normalization (Hb level ≥ 12 g/dL) after iptacopan initiation.	12 months	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Duration of Hb level ≥ 12 g/dL within a 12-month	12 months	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Change from baseline in LDH Levels after iptacopan initiation	Baseline, 12 Months	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Number of participants with LDH levels ≤ 1.5 x ULN before vs. after iptacopan initiation	12 months	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Number of participants having LDH normalization before and after iptacopan initiation	12 monhts	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Duration of LDH level ≤ 1.5 x ULN within 12 months	12 months	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Change from baseline in ARC Levels after iptacopan initiation	Baseline, 12 monhts	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Number of participants having ARC normalization before and after iptacopan initiation	Baseline, 12 months	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Change from baseline in bilirubin after iptacopan initiation	Baseline, 12 months	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Number of participants with hepatosplenomegaly before and after iptacopan initiation	Baseline, 12 months	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Number of participants with a positive coomb's test after iptacopan initiation	12 months	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Number of participants with PNH related signs or symptoms before and after iptacopan treatment	Baseline, 12 months	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Change from baseline in PNH clone value	Baseline, 12 months	Change from baseline in flow cytometry results to assess the impact of iptacopan on Treatment-Related Outcomes.
Rate, reasons (complement activation conditions (CAC), missed doses, etc.), duration, and interventions for breakthrough hemolysis (BTH)	12 months	Assessing the Impact of iptacopan on Treatment-Related Outcomes
Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale after iptacopan initiation	Baseline, 12 months	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.
Work Productivity and Activity Impairment Questionnaire-Specific Health Problems (WPAI-SHP) at each visit, change from baseline in WPAI-SHP after iptacopan initiation	Baseline, 12 months	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.
Adverse events (AEs) and Serious adverse events (SAEs) occurring during treatment with iptacopan	12 monhts	Assessing the safety of iptacopan
Treatment regimen for PNH 12 months before iptacopan treatment; reasons of discontinuation of previous complement inhibitor;	Baseline	Assessing the impact of iptacopan on disease management
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;	Baseline, 12 months	Assessing the impact of iptacopan on disease management
Duration of iptacopan treatment, reasons for discontinuation, switching to other treatments, proportion of patients who switch from iptacopan to other treatments;	12 monhts	Assessing the impact of iptacopan on disease management
Number of participants with blood transfusion before and after iptacopan treatment, duration and number of units of blood transfusion due to BTH	Baseline, 12 months	Assessing the impact of iptacopan on disease management
Number of participants hospitalized, outpatient, emergency room and intensive care unit (ICU) visits related to PNH	12 monhts	Assessing the impact of iptacopan on healthcare resource utilization
Length of inpatient stay related to PNH	12 months	Assessing the impact of iptacopan on healthcare resource utilization