NCT06931691Recruiting

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

Source: ClinicalTrials.gov · Last updated 1 Jun 2026

Brief summary (registry, in English)

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.

Status (registry): Recruiting
Study type: Observational
Enrollment: 80 (estimated)
First submitted: 14 Mar 2025
Start date: 10 Jun 2025
Primary completion date: 21 Apr 2027
Lead sponsor: Novartis Pharmaceuticals
NCT number: NCT06931691

What you commit to

Study type: Observational
Planned participants: 80
From 10 Jun 2025 to the primary completion date, 21 Apr 2027
10 sites in 1 country
Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Who the study is looking for

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.

Age in the registry: 18 years to 80 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (10)

Sites in registry order. Pick your city on the disease page to sort them by distance.

Site	City	Country	Site status
- Coordinator of this site? Confirm its status	Changchun	China	Recruiting
- Coordinator of this site? Confirm its status	Guangzhou	China	Recruiting
- Coordinator of this site? Confirm its status	Hefei	China	Recruiting
- Coordinator of this site? Confirm its status	Jinan	China	Recruiting
- Coordinator of this site? Confirm its status	Nanchang	China	Recruiting
- Coordinator of this site? Confirm its status	Nantong	China	Recruiting
- Coordinator of this site? Confirm its status	Shijiazhuang	China	Recruiting
- Coordinator of this site? Confirm its status	Tianjin	China	Recruiting
- Coordinator of this site? Confirm its status	Wuhan	China	Recruiting
- Coordinator of this site? Confirm its status	Zhengzhou	China	Recruiting

This page is information, not medical advice. Discuss any trial with your doctor.