Paroxysmal Nocturnal Hemoglobinuria
Conditions
Keywords
Phase IV, Adult Patients, Paroxysmal Nocturnal Hemoglobinuria, LNP023
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.
Interventions
DRUGLNP023
Capsules for oral administration
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in hemoglobin (Hb) levels at designated time points | Baseline, 12 months | Assessment of the Hematological Response to iptacopan. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| 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 |
| 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 |
| 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. |
Countries
China
Contacts
Primary ContactNovartis Pharmaceuticals
Backup ContactNovartis Pharmaceuticals
Outcome results
None listed