ClinicalTrials.gov record NCT06931691
A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients 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
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This multi-center ambispective observational study collects real-world data on adults with documented PNH who are starting iptacopan (LNP023). It includes people who have not previously used complement inhibitors and those previously treated with C5 inhibitors. The study will look at blood test changes, symptoms, treatment patterns, safety events, and healthcare use around treatment start, with key comparisons at baseline and about 12 months.
What it is studying. The study aims to describe how starting iptacopan affects disease measures, treatment patterns, adverse events, and healthcare resource use in adults with PNH. It will track blood markers (for example hemoglobin and LDH), signs and symptoms of PNH, transfusions and hospital visits, patient-reported fatigue and work/activity measures, and reported adverse events over the study period.
What the researchers hope to learn. To evaluate the impact of iptacopan on hematologic outcomes (for example hemoglobin, LDH, absolute reticulocyte count), PNH clone measures, bilirubin, rates and reasons for breakthrough hemolysis, transfusion use, hepatosplenomegaly and Coombs test results, patient-reported fatigue and work/activity impairment, treatment patterns (duration, discontinuation, switching), healthcare resource use (hospitalizations, ER/outpatient/ICU visits, length of stay), and to collect adverse event and serious adverse event data.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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 being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- LNP023
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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.
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will start iptacopan as part of their care and be followed for about 12 months. The study collects medical-record information and routine laboratory results at baseline and follow-up, tracks transfusions, hospital/ER/ICU visits, treatment changes and adverse events, and asks participants to complete questionnaires (e.g., FACIT‑Fatigue and WPAI‑SHP). Vaccination records must be provided before starting iptacopan; if treatment is urgent, antibiotics may be used per the drug label until vaccinations are complete. Participants must provide informed consent and begin the first dose within 60 days of signing consent.
How the study is structured. A multi-center ambispective (using past records and prospective follow-up) observational cohort study. Participants are grouped into two cohorts by their prior complement-inhibitor treatment status: one cohort without prior complement inhibitor use and one cohort of patients previously treated with C5 inhibitors. The study collects clinical data, laboratory results, questionnaires, and safety reports around the time iptacopan is started, with key assessments at baseline and about 12 months. Cohort enrollment timing for previously treated patients is tied to the local approval status for iptacopan.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on paroxysmal nocturnal hemoglobinuria
Important dates
- Study start
- Jun 10, 2025
- Primary completion
- Apr 21, 2027 (estimated)
- Study completion
- Apr 21, 2027 (estimated)
- Target enrollment
- 80 participants (estimated)
- Last updated on registry
- Jun 1, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Novartis Pharmaceuticals
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Novartis Pharmaceuticals 📞 +41613241111 ✉ novartis.email@novartis.com
- Novartis Pharmaceuticals
Participating sites (14)
- Novartis Investigative SiteRecruiting
Hefei, Anhui, 230022 · China
- Novartis Investigative SiteRecruiting
Guangzhou, Guangdong, 510000 · China
- Novartis Investigative SiteRecruiting
Guangzhou, Guangdong, 510080 · China
- Novartis Investigative SiteRecruiting
Shijiazhuang, Hebei, 050000 · China
- Novartis Investigative SiteRecruiting
Zhengzhou, Henan, 450003 · China
- Novartis Investigative SiteRecruiting
Zhengzhou, Henan, 450008 · China
- Novartis Investigative SiteRecruiting
Wuhan, Hubei, 430030 · China
- Novartis Investigative SiteRecruiting
Nantong, Jiangsu, 226000 · China
- Novartis Investigative SiteRecruiting
Nanchang, Jiangxi, 330006 · China
- Novartis Investigative SiteRecruiting
Changchun, Jilin, 130021 · China
Terms used on this page · explanations by MedicalUpdates.ai
- Cohort
- A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
- ICF
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- interventional
- A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
- Inclusion criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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