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

A Prospective, Non-interventional Study to Evaluate the Efficacy and Safety of Iptacopan in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) in Routine Clinical Practice in the Russian Federation

Status

Terminated

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT07036718

Acronym

PRIORITY- PNH

Enrollment

Registered

2025-06-25

Start date

2025-07-29

Completion date

2025-12-25

Last updated

2026-08-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

PNH, Paroxysmal nocturnal hemoglobinuria, Iptacopan

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.

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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)

Countries

Russia

Contacts

STUDY_DIRECTORNovartis Pharmaceuticals

Novartis Pharmaceuticals

Outcome results