Real-life Treatment Outcomes of Ravulizumab in PNH
PNH-RECORD
1 other identifier
observational
64
1 country
11
Brief Summary
The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Feb 2025
Typical duration for all trials
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 9, 2024
CompletedFirst Posted
Study publicly available on registry
October 9, 2024
CompletedStudy Start
First participant enrolled
February 6, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
August 1, 2028
April 1, 2026
March 1, 2026
3.5 years
August 9, 2024
March 31, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
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 Outcomes (10)
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
To present patients characteristics
Baseline
To present patients characteristics
Baseline
- +5 more secondary outcomes
Study Arms (1)
Ravulizumab group
Open-label
Interventions
open-label arm with ravulizumab administrated within the scope of routine clinical practice
Eligibility Criteria
Adult (aged ≥18) patients with PNH
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AstraZenecalead
Study Sites (11)
Research Site
Bialystok, Poland
Research Site
Brzozów, Poland
Research Site
Bydgoszcz, Poland
Research Site
Gdansk, Poland
Research Site
Krakow, Poland
Research Site
Lodz, Poland
Research Site
Lublin, Poland
Research Site
Opole, Poland
Research Site
Szczecin, Poland
Research Site
Warsaw, Poland
Research Site
Wałbrzych, Poland
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- RETROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 9, 2024
First Posted
October 9, 2024
Study Start
February 6, 2025
Primary Completion (Estimated)
August 1, 2028
Study Completion (Estimated)
August 1, 2028
Last Updated
April 1, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will share
- Time Frame
- AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
- Access Criteria
- When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.