Clinical trial NCT06633536: Real-life Treatment Outcomes of Ravulizumab in PNH
Official title: Real-life Treatment Outcomes of Ravulizumab in Polish Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH). Prospective and Retrospective, Multicenter, Non-interventional Study.
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Paroxysmal Nocturnal Haemoglobinuria
Keywords: Paroxysmal nocturnal haemoglobinuria
PNH
ravulizumab
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
First posted: 2024-10-09
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult (aged ≥18) patients with PNH

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

Detailed description: 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Change in LDH levels from index date (first dose of ravulizumab) at every 6 month
timeFrame: up to 36 months
measure: Proportion of patients achieving or remaining at LDH<1.5 ULN at every 6 month
timeFrame: up to 36 months
Secondary outcomes:
measure: Proportion of transfusion-free patients from index date at every 6 month
timeFrame: up to 36 months
measure: Number of blood units used per patient-year
timeFrame: up to 36 months
measure: To present patients characteristics
timeFrame: Baseline
measure: To present patients characteristics
timeFrame: Baseline
measure: To present patients characteristics
timeFrame: Baseline
measure: To present patients characteristics
timeFrame: Baseline
measure: To present clinical characteristics
timeFrame: Baseline
measure: To present clinical characteristics
timeFrame: Baseline
measure: To present clinical characteristics
timeFrame: Baseline
measure: To present clinical characteristics
timeFrame: Baseline
Other outcomes:
Locations:
city: Bialystok
country: Poland
facility: Research Site
city: Brzozow
country: Poland
facility: Research Site
city: Bydgoszcz
country: Poland
facility: Research Site
city: Gdansk
country: Poland
facility: Research Site
city: Katowice
country: Poland
facility: Research Site
city: Krakow
country: Poland
facility: Research Site
city: Lodz
country: Poland
facility: Research Site
city: Lublin
country: Poland
facility: Research Site
city: Opole
country: Poland
facility: Research Site
city: Szczecin
country: Poland
facility: Research Site
city: Walbrzych
country: Poland
facility: Research Site
city: Warsaw
country: Poland
facility: Research Site
city: Wroclaw
country: Poland
facility: Research Site
Study officials:
References: