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Terminated

NCT Number: NCT07036718

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

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.

Why the study stopped: Sponsor decision
Terminated

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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis Investigative Site, Syktyvkar, Komi, Russia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

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

Treatment and study plan

Primary outcomes

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

    Time frame: 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 outcomes

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

    Time frame: 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

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

    Time frame: 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

  3. Change from baseline in reticulocyte count (10⁹/L)

    Time frame: 12 months

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

  4. Change from baseline in LDH levels (U/L)

    Time frame: 12 months

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

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

    Time frame: 12 months

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

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

    Time frame: 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

  7. Number of breakthrough hemolysis (BTH)

    Time frame: 12 months

    Number of breakthrough hemolysis (BTH) cases 12 months following the initiation of iptacopan therapy

  8. Percentage of patients by BMI

    Time frame: 12 months

    Percentage of patients by Body mass index (BMI) (<25, 25.1-29.9, >30)

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Acronym: PRIORITY- PNH

Important dates

Study start
2025
Primary completion
2025
Study completion
2025
First posted
Jun 25, 2025
Registry last updated
Aug 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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