Iptacopan treatment for adults with PNH in China

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Novartis Investigative Site
  Hefei, Anhui, China Recruiting
• Novartis Investigative Site
  Guangzhou, Guangdong, China Recruiting
• Novartis Investigative Site
  Guangzhou, Guangdong, China Recruiting
• Novartis Investigative Site
  Shijiazhuang, Hebei, China Recruiting
• Novartis Investigative Site
  Zhengzhou, Henan, China Recruiting
• Novartis Investigative Site
  Zhengzhou, Henan, China Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: +41613241111

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Oral HS-10542 for paroxysmal nocturnal hemoglobinuria

  This study will test whether the oral drug HS-10542 can reduce hemolysis and raise hemoglobin in adults with PNH, including those not fully helped by C5 inhibitor therapy.

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• Danicopan added to ravulizumab or eculizumab for PNH in Korea

  This will see if adding Danicopan to ravulizumab or eculizumab is safe and helps people with PNH who are treated in routine care in Korea.

  Recruiting at 3 sites AstraZeneca · Busan and 3 other locations · NCT07457151

• Iptacopan use in Korean patients with PNH or C3G

  This post-marketing surveillance study tests how well iptacopan works and how safe it is for Korean adults with PNH or C3G in real-world care.

  Recruiting at 3 sites Novartis · Jeollanam and 2 other locations · NCT07416162

• Pozelimab + cemdisiran for adults with PNH whose current treatment isn't working

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• Evaluating MY008211A for treating anemia in PNH patients on anti-C5 therapy

  This study is testing a new treatment called MY008211A to see if it can help people with PNH who still have anemia even while on anti-C5 therapy.

  Recruiting Phase 3 Wuhan Createrna Science and Technology Co., Ltd · Beijing, Beijing Municipality · NCT06932471

• Long-term safety and tolerability of HSK39297 for patients with PNH

  This study is testing if continuing treatment with HSK39297 is safe and effective for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have already used this medication.

  Recruiting Phase 2 Haisco Pharmaceutical Group Co., Ltd. · Nanjing, Jiangsu · NCT06745622

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.