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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
Led by Novartis Pharmaceuticals · Updated on 2026-06-01
80
Participants Needed
14
Research Sites
N/A
Total Duration
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AI-Summary
What this Trial Is About
Researchers are conducting a real-world study to evaluate the impact of iptacopan on adult patients with paroxysmal nocturnal hemoglobinuria (PNH) in China. The study aims to assess treatment-related outcomes, disease management, and healthcare resource use, considering new standards for PNH care and the introduction of iptacopan. It includes patients who are either new to complement inhibitor therapy or those stable on C5 complement inhibitors like eculizumab. The study involves two patient groups: one with PNH patients never treated with complement inhibitors who are starting iptacopan, and another with patients stable on C5 complement inhibitors who will switch to iptacopan after its approval in China. Treatments are oral capsules of LNP023 (iptacopan), and patients must start iptacopan within 60 days of consenting. Participants must have documented vaccinations against Neisseria meningitidis and Streptococcus pneumoniae before starting treatment. Participants will be followed for at least 12 months, with assessments including hemoglobin levels, lactate dehydrogenase (LDH), absolute reticulocyte count (ARC), bilirubin, PNH clone size, and signs or symptoms related to PNH. Researchers will monitor treatment effects, adverse events, medication use, transfusions, and healthcare visits. Patient fatigue and work productivity will be evaluated using specific questionnaires, with data collected at baseline and regular intervals to understand iptacopan's real-world impact on disease and quality of life.
CONDITIONS
Brief Title
A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older at the time of signing consent
- Documented diagnosis of paroxysmal nocturnal hemoglobinuria (PNH)
- For Cohort 1: No previous complement inhibitor therapy
- For Cohort 2: Receiving stable treatment with C5 complement inhibitors for at least 3 months prior to enrollment
- Initiating iptacopan therapy within 60 days of signing consent
- Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae at least 2 weeks before starting iptacopan
- Signed informed consent form
You will not qualify if you...
- Participating in another interventional PNH clinical study
- Active systemic bacterial, viral (including COVID-19), or fungal infection within 14 days before first dose
- History of recurrent invasive infections
- History of HIV infection
- Women who are pregnant, breastfeeding, or intending to conceive during the study
- Presence of bone marrow failure as determined by low reticulocytes, platelets, and neutrophils
- Other conditions deemed unsuitable for study participation by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 12 months
Participants are observed to evaluate the impact of iptacopan treatment on disease management and treatment-related outcomes over time.
Visits at baseline and 12 months
Trial Site Locations
Total: 14 locations
1
Novartis Investigative Site
Hefei, Anhui, China, 230022
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2
Novartis Investigative Site
Guangzhou, Guangdong, China, 510000
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3
Novartis Investigative Site
Guangzhou, Guangdong, China, 510080
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4
Novartis Investigative Site
Shijiazhuang, Hebei, China, 050000
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5
Novartis Investigative Site
Zhengzhou, Henan, China, 450003
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6
Novartis Investigative Site
Zhengzhou, Henan, China, 450008
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7
Novartis Investigative Site
Wuhan, Hubei, China, 430030
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8
Novartis Investigative Site
Nantong, Jiangsu, China, 226000
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9
Novartis Investigative Site
Nanchang, Jiangxi, China, 330006
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10
Novartis Investigative Site
Changchun, Jilin, China, 130021
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11
Novartis Investigative Site
Jinan, China, 250012
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12
Novartis Investigative Site
Tianjin, China, 300020
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13
Novartis Investigative Site
Tianjin, China, 300052
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14
Novartis Investigative Site
Wuhan, China, 430022
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Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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