Clinical trial NCT06931691: A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: Phase IV
Adult Patients
Paroxysmal Nocturnal Hemoglobinuria
LNP023
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2025-04-17
Enrollment: 80
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
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

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Change from baseline in hemoglobin (Hb) levels at designated time points
timeFrame: Baseline, 12 months
Secondary outcomes:
measure: Number of participants having Hb normalization (Hb level ≥ 12 g/dL) after iptacopan initiation.
timeFrame: 12 months
measure: Duration of Hb level ≥ 12 g/dL within a 12-month
timeFrame: 12 months
measure: Change from baseline in LDH Levels after iptacopan initiation
timeFrame: Baseline, 12 Months
measure: Number of participants with LDH levels ≤ 1.5 x ULN before vs. after iptacopan initiation
timeFrame: 12 months
measure: Number of participants having LDH normalization before and after iptacopan initiation
timeFrame: 12 monhts
measure: Duration of LDH level ≤ 1.5 x ULN within 12 months
timeFrame: 12 months
measure: Change from baseline in ARC Levels after iptacopan initiation
timeFrame: Baseline, 12 monhts
measure: Number of participants having ARC normalization before and after iptacopan initiation
timeFrame: Baseline, 12 months
measure: Change from baseline in bilirubin after iptacopan initiation
timeFrame: Baseline, 12 months
measure: Number of participants with hepatosplenomegaly before and after iptacopan initiation
timeFrame: Baseline, 12 months
measure: Number of participants with a positive coomb's test after iptacopan initiation
timeFrame: 12 months
measure: Number of participants with PNH related signs or symptoms before and after iptacopan treatment
timeFrame: Baseline, 12 months
measure: Change from baseline in PNH clone value
timeFrame: Baseline, 12 months
measure: Rate, reasons (complement activation conditions (CAC), missed doses, etc.), duration, and interventions for breakthrough hemolysis (BTH)
timeFrame: 12 months
measure: Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale after iptacopan initiation
timeFrame: Baseline, 12 months
measure: Work Productivity and Activity Impairment Questionnaire-Specific Health Problems (WPAI-SHP) at each visit, change from baseline in WPAI-SHP after iptacopan initiation
timeFrame: Baseline, 12 months
measure: Adverse events (AEs) and Serious adverse events (SAEs) occurring during treatment with iptacopan
timeFrame: 12 monhts
measure: Treatment regimen for PNH 12 months before iptacopan treatment; reasons of discontinuation of previous complement inhibitor;
timeFrame: Baseline
measure: 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;
timeFrame: Baseline, 12 months
measure: Duration of iptacopan treatment, reasons for discontinuation, switching to other treatments, proportion of patients who switch from iptacopan to other treatments;
timeFrame: 12 monhts
measure: Number of participants with blood transfusion before and after iptacopan treatment, duration and number of units of blood transfusion due to BTH
timeFrame: Baseline, 12 months
measure: Number of participants hospitalized, outpatient, emergency room and intensive care unit (ICU) visits related to PNH
timeFrame: 12 monhts
measure: Length of inpatient stay related to PNH
timeFrame: 12 months
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Novartis Investigative Site
Study officials:
References: