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NCT Number: NCT06633536

Real-life Treatment Outcomes of Ravulizumab in PNH

The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Paroxysmal Nocturnal Haemoglobinuria 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

Research Site, Bialystok, Poland

Loading trial locations.

About this study

PNH-RECORD study aims to aims to collect data on the patients' characteristics and clinical outcomes of ravulizumab administered in the scope of routine clinical practice in PNH.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Adult (aged ≥18) patients with PNH receiving ravulizumab treatment in the frames of NDP in Poland.
  • Patients willing to participate in the study and signed ICF.

Exclusion criteria

  • Those who participated in ravulizumab clinical trial in the past, and/or those who participated/plans to participate in clinical trial on/after the date of first ravulizumab infusion through NDP.
  • Cognitive incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.

Treatment and study plan

Ravulizumab

Drug

open-label arm with ravulizumab administrated within the scope of routine clinical practice

Primary outcomes

  1. Change in LDH levels from index date (first dose of ravulizumab) at every 6 month

    Time frame: up to 36 months

  2. Proportion of patients achieving or remaining at LDH<1.5 ULN at every 6 month

    Time frame: up to 36 months

Secondary outcomes

  1. Proportion of transfusion-free patients from index date at every 6 month

    Time frame: up to 36 months

  2. Number of blood units used per patient-year

    Time frame: up to 36 months

  3. To present patients characteristics

    Time frame: Baseline

    Mean age (years) at PNH diagnosis

  4. To present patients characteristics

    Time frame: Baseline

    Mean age (years) at index date

  5. To present patients characteristics

    Time frame: Baseline

    Proportion (%) of men and women

  6. To present patients characteristics

    Time frame: Baseline

    Mean body mass index (kg/m²)

  7. To present clinical characteristics

    Time frame: Baseline

    Mean and median (years) duration of PNH

  8. To present clinical characteristics

    Time frame: Baseline

    Mean and median (months) time from diagnosis to complement inhibitors treatment initiation

  9. To present clinical characteristics

    Time frame: Baseline

    Proportion (%) of patients with complication of haemolysis, thrombosis, serious vascular event andaplastic anemia

  10. To present clinical characteristics

    Time frame: Baseline

    Proportion (%) of patients naïve to complement inhibitors

Sponsors and collaborators

Lead sponsor

AstraZeneca

Industry

Registry information

Official study title

Real-life Treatment Outcomes of Ravulizumab in Polish Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH). Prospective and Retrospective, Multicenter, Non-interventional Study.

Acronym: PNH-RECORD

Important dates

Study start
2025
Primary completion
2028
Study completion
2028
First posted
Oct 9, 2024
Registry last updated
Jun 12, 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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