A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China

NCT06931691 | Recruiting FDA Drug

• 1 other identifier: CLNP023C1CN01
• Study Type: observational
• Target: 80
• Locations: 1 country
• Sites: 9

Brief Summary

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

Phase IV; Adult Patients; Paroxysmal Nocturnal Hemoglobinuria; LNP023

Outcome Measures

Primary Outcomes (1)

• Change from baseline in hemoglobin (Hb) levels at designated time points

  Assessment of the Hematological Response to iptacopan.

  Baseline, 12 months

Secondary Outcomes (23)

• Number of participants having Hb normalization (Hb level ≥ 12 g/dL) after iptacopan initiation.

  12 months

• Duration of Hb level ≥ 12 g/dL within a 12-month

  12 months

• Change from baseline in LDH Levels after iptacopan initiation

  Baseline, 12 Months

• Number of participants with LDH levels ≤ 1.5 x ULN before vs. after iptacopan initiation

  12 months

• Number of participants having LDH normalization before and after iptacopan initiation

  12 monhts

Study Arms (2)

Cohort1

PNH patients with a confirmed diagnosis of PNH in the medical record and who had never received complement inhibitor therapy (complement inhibitor naive patients)

Drug: LNP023

Cohort 2

PNH patients with a confirmed diagnosis of PNH in the medical record who had been treated with a stable dose of a C5 complement inhibitor (e.g., eculizumab) for at least 3 months (C5i-treated patients; this cohort will be only start enrollment after the indication for iptacopan is to be approved in China).

Drug: LNP023

Interventions

LNP023 DRUG

Capsules for oral administration

Also known as: Iptacopan Hydrochloride

Cohort 2; Cohort1

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adult PNH patients receiving iptacopan for the first time, including those who are complement inhibitor naive and treated

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (9)

Novartis Investigative Site

Guangzhou, Guangdong, 510000, China

RECRUITING

Novartis Investigative Site

Shijiazhuang, Hebei, 050000, China

RECRUITING

Novartis Investigative Site

Zhengzhou, Henan, 450008, China

RECRUITING

Novartis Investigative Site

Wuhan, Hubei, 430030, China

RECRUITING

Novartis Investigative Site

Nanchang, Jiangxi, 330006, China

RECRUITING

Novartis Investigative Site

Changchun, Jilin, 130021, China

RECRUITING

Novartis Investigative Site

Tianjin, 300020, China

RECRUITING

Novartis Investigative Site

Tianjin, 300052, China

RECRUITING

Novartis Investigative Site

Wuhan, 430022, China

RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 14, 2025
• First Posted: April 17, 2025
• Study Start: June 10, 2025
• Primary Completion (Estimated): April 20, 2027
• Study Completion (Estimated): April 20, 2027
• Last Updated: December 1, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (9)