NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice

Not yet recruiting

• Conditions: Paroxysmal Nocturnal Hemoglobinuria

• Registration Number: NCT07036718
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

This is a prospective, non-interventional, multicenter study to evaluate the efficacy and safety of iptacopan in patients with PNH in real-world settings in Russia.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-06
• Study First Submitted: 2025-06-17
• Study First Submitted QC: 2025-06-17
• Last Update Submitted: 2025-06-17
• Study First Posted: 2025-06-25
• Last Update Posted: 2025-06-25
• Study Start: 2025-07-31
• Primary Completion: 2028-01-31
• Study Completion: 2028-01-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

1. Age ≥18 years at the start of iptacopan therapy.
2. The patient is undergoing treatment with iptacopan.
3. The treating physician decided to prescribe iptacopan based on the Summary of Product Characteristics during routine clinical practice, regardless of study participation.
4. Provision of written informed consent.

Exclusion Criteria

1. Any situations where iptacopan is contraindicated in accordance with the Summary of Product Characteristics.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of patients with an increase in Hb level ≥ 20 g/l	6 months	Number of patients who achieved an increase in Hb level ≥ 20 g/l over 6 months after iptacopan treatment initiation and were Red Blood Cells (RBC) transfusion independent.

Secondary Outcome Measures

Name	Time	Method
Number of patients with an increase in Hb level ≥ 120 g/L	12 months	Number of patients who achieved an increase in Hb level ≥ 120 g/L at 12 months after initiation of iptacopan treatment and were RBC transfusion independent
Number of patients with an increase in Hb level ≥ 20 g/L	12 months	Number of patients who achieved an increase in Hb level ≥ 20 g/L at 12 months after initiation of iptacopan treatment and was RBC transfusion independent
Change from baseline in reticulocyte count (10⁹/L)	12 months	Change from baseline in reticulocyte count (10⁹/L) over 12 months after initiation of iptacopan treatment
Change from baseline in LDH levels (U/L)	12 months	Change from baseline in LDH levels (U/L) over 12 months after initiation of iptacopan treatment
Number of patients with an absence of administration of packed-RBC transfusions	12 months	Number of patients with an absence of administration of packed-RBC transfusions at 12 months after initiation of iptacopan treatment
Change from baseline in the Hb level (g/L)	Baseline, month 3, month 6 and month 12	Change from baseline in the Hb level (g/L) at 3, 6 and 12 months after initiation of iptacopan treatment
Number of breakthrough hemolysis (BTH)	12 months	Number of breakthrough hemolysis (BTH) cases 12 months following the initiation of iptacopan therapy
Percentage of patients by BMI	12 months	Percentage of patients by Body mass index (BMI) (\<25, 25.1-29.9, \>30)