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

Novartis Pharmaceuticals6 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (5)

Novartis Investigative Site

Syktyvkar, Komi, Russia

Novartis Investigative Site

Grozny, Russia

Novartis Investigative Site

Moscow, Russia

Novartis Investigative Site

Murmansk, Russia

Outcomes

Primary Outcomes

Number of patients with an increase in Hb level ≥ 20 g/l

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.

Time frame: 6 months

Secondary Outcomes

Number of patients with an increase in Hb level ≥ 120 g/L

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

Time frame: 12 months

Number of patients with an increase in Hb level ≥ 20 g/L

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

Time frame: 12 months

Change from baseline in reticulocyte count (10⁹/L)

Change from baseline in reticulocyte count (10⁹/L) over 12 months after initiation of iptacopan treatment

Time frame: 12 months

Change from baseline in LDH levels (U/L)

Change from baseline in LDH levels (U/L) over 12 months after initiation of iptacopan treatment

Time frame: 12 months

Number of patients with an absence of administration of packed-RBC transfusions

Number of patients with an absence of administration of packed-RBC transfusions at 12 months after initiation of iptacopan treatment

Time frame: 12 months

Change from baseline in the Hb level (g/L)

Change from baseline in the Hb level (g/L) at 3, 6 and 12 months after initiation of iptacopan treatment

Data from ClinicalTrials.gov

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