Real-life Treatment Outcomes of Ravulizumab in PNH

N/AActive Not RecruitingNCT06633536

AstraZeneca64 enrolled

Overview

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

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Paroxysmal Nocturnal Haemoglobinuria

Interventions

RavulizumabDRUG

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

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Adult (aged ≥18) patients with PNH receiving ravulizumab treatment in the frames of NDP in Poland. 2. Patients willing to participate in the study and signed ICF. Exclusion Criteria: 1. 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. 2. Cognitive incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.

Locations (11)

Research Site

Bialystok, Poland

Research Site

Brzozów, Poland

Research Site

Bydgoszcz, Poland

Research Site

Gdansk, Poland

Outcomes

Primary Outcomes

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

Time frame: up to 36 months

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

Time frame: up to 36 months

Secondary Outcomes

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

Time frame: up to 36 months

Number of blood units used per patient-year

Time frame: up to 36 months

To present patients characteristics

Mean age (years) at PNH diagnosis

Time frame: Baseline

To present patients characteristics

Mean age (years) at index date

Time frame: Baseline

To present patients characteristics

Proportion (%) of men and women

Time frame: Baseline

To present patients characteristics

Mean body mass index (kg/m²)

Time frame: Baseline

To present clinical characteristics

Mean and median (years) duration of PNH

Time frame: Baseline

To present clinical characteristics

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

Data from ClinicalTrials.gov

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