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

Overview

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

• Drug LNP023
  Capsules for oral administration

Primary outcome measures

• Change from baseline in hemoglobin (Hb) levels at designated time points [Time frame: Baseline, 12 months]

Secondary outcome measures (12)

• Number of participants having Hb normalization (Hb level ≥ 12 g/dL) after iptacopan initiation. [Time frame: 12 months]
• Duration of Hb level ≥ 12 g/dL within a 12-month [Time frame: 12 months]
• Change from baseline in LDH Levels after iptacopan initiation [Time frame: Baseline, 12 Months]
• Number of participants with LDH levels ≤ 1.5 x ULN before vs. after iptacopan initiation [Time frame: 12 months]
• Number of participants having LDH normalization before and after iptacopan initiation [Time frame: 12 monhts]
• Duration of LDH level ≤ 1.5 x ULN within 12 months [Time frame: 12 months]
• Change from baseline in ARC Levels after iptacopan initiation [Time frame: Baseline, 12 monhts]
• Number of participants having ARC normalization before and after iptacopan initiation [Time frame: Baseline, 12 months]
• Change from baseline in bilirubin after iptacopan initiation [Time frame: Baseline, 12 months]
• Number of participants with hepatosplenomegaly before and after iptacopan initiation [Time frame: Baseline, 12 months]
• Number of participants with a positive coomb's test after iptacopan initiation [Time frame: 12 months]
• Number of participants with PNH related signs or symptoms before and after iptacopan treatment [Time frame: Baseline, 12 months]

Eligibility criteria

Inclusion criteria For Cohort 1,

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

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

For Cohort 2,

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

• Age ≥ 18 years at the time of signing the ICF;
• 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;
• Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
• 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.
• 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:

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

China · 14 centers

• Novartis Investigative Site — Hefei
• Novartis Investigative Site — Guangzhou
• Novartis Investigative Site — Guangzhou
• Novartis Investigative Site — Shijiazhuang
• Novartis Investigative Site — Zhengzhou
• Novartis Investigative Site — Zhengzhou
• Novartis Investigative Site — Wuhan
• Novartis Investigative Site — Nantong
• … and 6 more centers

Identifiers

NCT: NCT06931691 · CLNP023C1CN01

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗