ClinicalTrials.gov record NCT07788105
Efficacy and Safety of Lanoracopan in the Treatment of PNH
Efficacy and Safety of Lanoracopan Hydrochloride in the Treatment of Paroxysmal Nocturnal Hemoglobinuria: A Prospective, Single-Center Real-World Study
AI summaryWhat this study is about, 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.
This prospective observational study will follow people with paroxysmal nocturnal hemoglobinuria (PNH) who receive lanoracopan. It will record adverse events and measure hemoglobin and hemolysis-related outcomes at about 12- and 24-week time points. The study aims to describe real-world safety, durability of hemoglobin response, transfusion independence, breakthrough hemolysis, and thrombotic events.
What it is studying. The study is collecting information on the safety of lanoracopan and how long hemoglobin improvements last in people with PNH.
What the researchers hope to learn. To describe the rate and pattern of adverse events during lanoracopan treatment; to measure how many people maintain hemoglobin ≥120 g/L and changes in hemoglobin over time; to determine the incidence of clinically significant hemolysis and major adverse vascular events (MAVE); and to provide real-world evidence to inform therapy switching and sequencing in clinical practice.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This study aimed to evaluate the efficacy and safety of Lanoracopan Hydrochloride in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Lanoracopan Hydrochloride
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Age 18-75 years.
- Diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) meeting any of the following conditions:
- Newly diagnosed PNH with active hemolysis (hemoglobin [Hb] <100 g/L, lactate dehydrogenase [LDH] >1.5×upper limit of normal [ULN]);
- Receiving a stable regimen of complement C5 monoclonal antibody (eculizumab) at standard dose and interval for ≥3 months, with Hb still <120 g/L despite treatment;
- Intolerant to eculizumab therapy;
- Receiving standard iptacopan therapy for ≥3 months, with Hb still <120 g/L;
- Intolerant to iptacopan therapy.
- Currently receiving standard lanoracopan therapy (patients who have completed lanoracopan clinical trials).
- Vaccination against meningococcal infection (quadrivalent conjugate vaccine, MenACWY) is required prior to the first dose of study drug (Day 1). If not vaccinated within 3 years prior to Day 1, vaccination must be administered at least 14 days before Day 1; if administered within 14 days before Day 1, antibiotic prophylaxis against meningococcal infection is required until 14 days post-vaccination.
- Vaccination against pneumococcal infection is required prior to Day 1. If not vaccinated within 5 years prior to Day 1, vaccination must be administered at least 14 days before Day 1; if administered within 14 days before Day 1, antibiotic prophylaxis against pneumococcal infection is required until 14 days post-vaccination.
- Willing and able to provide written informed consent and comply with study procedures.
Exclusion criteria
- Previous bone marrow or hematopoietic stem cell transplantation.
- Previous splenectomy.
- Known or suspected hereditary complement deficiency.
- History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
- A history of malignancy within 5 years before screening, except cured local basal cell carcinoma of the skin and carcinoma in situ of the cervix.
- Severe concurrent illness, including severe renal disease (e.g., dialysis), advanced cardiac disease (NYHA class IV), severe pulmonary hypertension (WHO class IV), or unstable thrombotic events, judged unsuitable for participation by the investigator.
- Any other condition that, in the investigator's opinion, may interfere with study conduct, increase subject risk, or preclude safe participation and completion, including concomitant disease, treatment, procedure, surgery, or clinically significant laboratory abnormality.
- Pregnant or breastfeeding women.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
In Chinese multicenter phase 3 trials, lanoracopan 400 mg BID was evaluated over 24 weeks. In the active-controlled study (N=66, complement-naïve), the proportion achieving Hb ≥120 g/L was 50.0% (lanoracopan) versus 9.09% (eculizumab). In the single-arm study (N=20, C5 inhibitor-inadequate responders), 70% reached Hb ≥120 g/L, and 100% had Hb increase ≥20 g/L from baseline. No treatment-discontinuation due to AEs, no severe breakthrough hemolysis, no major vascular events, no encapsulated bacterial infections, and no deaths were reported across both studies. These data demonstrate superior hemoglobin correction and favorable short-term safety. However, long-term real-world effectiveness and safety profiles stratified by prior treatment (C5 inhibitors vs. other factor B inhibitors) remain uncharacterized. This observational study will enroll a diverse cohort to capture durability of Hb response, transfusion independence rate, incidence of breakthrough hemolysis and thrombosis, and adverse event patterns over extended follow-up, providing evidence to guide therapy switching and sequencing in clinical practice.
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 be followed while receiving lanoracopan therapy and have assessments about every 12 weeks, with the main observation period up to 24 weeks. At visits they will have blood tests to check hemoglobin and markers of hemolysis, report any red blood cell transfusions, and be monitored for thrombosis and other adverse events. Before the first dose of study drug, vaccination against meningococcal and pneumococcal infection is required (antibiotic prophylaxis is required if vaccination was given within 14 days). Adverse events will be recorded throughout the treatment period.
How the study is structured. This is a prospective observational cohort study. People who are receiving lanoracopan hydrochloride (listed as 400 mg twice daily in the record) as part of routine care will be followed; the study will monitor adverse events and assess hemoglobin and hemolysis-related outcomes at roughly 12-week intervals, with safety described over about 24 weeks of treatment.
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 registry says recruitment has not started. When is it expected to open, and can I register interest now?
- No sites are listed on the registry yet. Where will the study take place?
- 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
- September 2026 (estimated)
- Primary completion
- December 2028 (estimated)
- Study completion
- December 2029 (estimated)
- Target enrollment
- 90 participants (estimated)
- Last updated on registry
- Aug 26, 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
- Peking Union Medical College Hospital
- Principal investigator
- Bing Han, Professor
Peking Union Medical College Hospital
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.
- Bing Han 📞 +86 13601059938 ✉ hanbing_li@sina.com.cn
Participating sites
Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.
Terms used on this page · explanations by MedicalUpdates.ai
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- stable regimen
- A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
- study drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- written informed consent
- 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.
- before screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- 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.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- phase 3
- A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
- active-controlled
- An existing treatment used as the comparison group instead of a placebo, so the study asks whether the new treatment does as well as or better than current care.
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- stratified
- Randomisation done separately within subgroups, such as age bands or disease severity, so that each group contains a similar mix.
- prior treatment
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- 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.
- adverse event
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
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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