Real-life Treatment Outcomes of Ravulizumab in PNH
- Registration Number
- NCT06633536
- Lead Sponsor
- AstraZeneca
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 40
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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Ravulizumab group Ravulizumab Open-label
- Primary Outcome Measures
Name Time Method Change in LDH levels from index date (first dose of ravulizumab) at every 6 month up to 36 months Proportion of patients achieving or remaining at LDH<1.5 ULN at every 6 month up to 36 months
- Secondary Outcome Measures
Name Time Method Proportion of transfusion-free patients from index date at every 6 month up to 36 months Number of blood units used per patient-year up to 36 months To present patients characteristics Baseline Mean body mass index (kg/m²)
To present clinical characteristics Baseline Proportion (%) of patients naïve to complement inhibitors
Trial Locations
- Locations (1)
Research Site
🇵🇱Wroclaw, Poland