Iptacopan treatment for adults with PNH in China

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

This study will try iptacopan (LNP023) in adults with PNH in China to see if it improves clinical and laboratory outcomes and changes healthcare use.

Quick facts

• Study type: Observational
• Enrollment: 80 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Novartis (Industry-sponsored)
• Locations: 9 sites (Guangzhou, Guangdong and 8 other locations)
• Trial ID: NCT06931691 on ClinicalTrials.gov

What this trial studies

This is a multi-center, ambispective (retrospective + prospective) observational cohort study collecting real-world data on iptacopan (LNP023) use in adult Chinese patients with PNH. The study will include at least two cohorts, including adults initiating iptacopan and a broader adult PNH cohort, and will capture treatment patterns, lab results, transfusion needs, adverse events, and healthcare resource use. Vaccination against Neisseria meningitidis and Streptococcus pneumoniae and timing relative to iptacopan initiation will be recorded per label guidance. Data will be collected at Novartis investigative sites in Guangzhou, Shijiazhuang, and Zhengzhou to describe outcomes in routine clinical practice.

Who should consider this trial

Good fit: Adults (age ≥18) in China with a documented diagnosis of PNH, especially those who are treatment-naïve to complement inhibitors and are about to start iptacopan with required vaccinations, are ideal candidates.

Not a fit: Patients under 18, those already receiving other complement inhibitors, or those unable to complete recommended vaccinations may not be eligible and are less likely to benefit from this protocol's findings.

Why it matters

Potential benefit: If successful, the data could show that iptacopan improves patient-level outcomes and reduces transfusion or other healthcare needs for adults with PNH in China.

How similar studies have performed: Prior clinical trials of iptacopan in PNH have shown promising efficacy and safety signals, but comprehensive real-world evidence in Chinese patients is limited.

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.

Where this trial is running

Guangzhou, Guangdong and 8 other locations

• Novartis Investigative Site — Guangzhou, Guangdong, China (Recruiting)
• Novartis Investigative Site — Shijiazhuang, Hebei, China (Recruiting)
• Novartis Investigative Site — Zhengzhou, Henan, China (Recruiting)
• Novartis Investigative Site — Wuhan, Hubei, China (Recruiting)
• Novartis Investigative Site — Nanchang, Jiangxi, China (Recruiting)
• Novartis Investigative Site — Changchun, Jilin, China (Recruiting)
• Novartis Investigative Site — Tianjin, China (Recruiting)
• Novartis Investigative Site — Tianjin, China (Recruiting)
• Novartis Investigative Site — Wuhan, China (Recruiting)

Study contacts

• Study coordinator: Novartis Pharmaceuticals

Conditions Paroxysmal Nocturnal Hemoglobinuria; Phase IV; Adult Patients; LNP023