1. Study identification
EU PAS Register NumberEUPAS29462
Official titleA Prospective, Multicentre, Non-interventional, Observational, Post-authorisation Safety Study of Ropeginterferon alfa-2b in Polycythaemia Vera Patients
Study title acronymBesremi-PASS
Study typeObservational study
Brief description of the studyThe objective of the study is to provide further data to characterize the safety and tolerability of ropeginterferon alfa-2b by monitoring the hepatic and cardiovascular safety in patients with polycythaemia vera treated with ropeginterferon alfa-2b in routine post-authorisation use.
Was this study requested by a regulator?Yes: EMA
Is the study required by a Risk Management Plan (RMP)?
EU RMP category 3 (required)
Regulatory procedure number (RMP Category 1 and 2 studies only)
Other study registration identification numbers and URLs as applicable
2. Research centres and Investigator details
Coordinating study entity
Centre nameAOP Orphan Pharmaceuticals GmbH
Centre locationVienna, Austria
Details of (Primary) lead investigator
Title Dr
Last name AOP Orphan Pharmaceuticals GmbH
First name Clinical Trial Disclosure
Is this study being carried out with the collaboration of a research network?
No
Other centres where this study is being conducted
Multiple centres
In total how many centres are involved in this Study?39
Countries in which this study is being conducted
International study
Austria
Germany
Romania
3. Study timelines: initial administrative steps, progress reports and final report
PlannedActual
Date when funding contract was signed30/04/2019
Start date of data collection31/12/201917/12/2019
Start date of data analysis30/06/2025
Date of interim report, if expected31/12/2024
Date of final study report31/12/2025
4. Sources of funding
Please provide estimates of the percentage of funding by source for this study
Names(s)Approximate % funding
Pharmaceutical companiesAOP Orphan Pharmaceuticals GmbH100
Charities
Government body
Research councils
EU funding scheme
5. Contact details for enquiries
Scientific Enquiries
Title Dr
Last name AOP Orphan Pharmaceuticals GmbH
First name Clinical Trial Disclosure
Address line 1Leopold-Ungar-Platz 2
Address line 2
Address line 3
CityVienna
Postcode1190
CountryAustria
Phone number (incl. country code)4315037244
Alternative phone number
Fax number (incl. country code)
Public Enquiries
Title Dr
Last name Klade
First name Christoph
Address line 1Leopold-Ungar-Platz 2
Address line 2
Address line 3
CityVienna
Postcode1190
CountryAustria
Phone number (incl. country code)4315037244
Alternative phone number
Fax number (incl. country code)
6. Study drug(s) information
Substance class (ATC Code)L03AB15 (ropeginterferon alfa-2b)
7. Medical conditions to be studied
Medical condition(s)Yes
Polycythaemia vera
8. Population under study
Age
Adults (18 - 44 years)
Adults (45 - 64 years)
Adults (65 - 74 years)
Adults (75 years and over)
Sex
Male
Female
9. Number of subjects
Estimated total number of subjects228
10. Source of data
Is this study being carried out with an established data source?No
Sources of data
Prospective patient-based data collection
11. Scope of the study
What is the scope of the study?
Risk assessment
Safety and Tolerability
Primary scope : Risk assessment
12. Main objective(s)
What is the main objective of the study?
To assess the incidence rate of the important, identified risk “hepatotoxicity” in PV patients newly treated with ropeginterferon alfa-2b in routine post-authorization use.
Are there primary outcomes?Yes
Incidence of significant elevations of liver enzymes and/or bilirubin, and treatment-emergent hepatobiliary adverse drug reactions during the observational phase (i.e. first 6 months of treatment).
Are there secondary outcomes?Yes
Incidence of significant elevations of liver enzymes and/or bilirubin, and treatment-emergent hepatobiliary adverse drug reactions over the entire study period.
Incidence of cardiovascular adverse events (i.e. thromboembolic adverse events and Major Adverse Cardiac Events) during the observation phase and over the entire study period.
13. Study design
What is the design of the study?
Non interventional study
14. Follow-up of patients
Will patients be followed up?Yes
Please describe duration of follow up
Patients will be followed up after the observational phase (i.e. first 6 months of treatment) for an additional 12 months of ropeginterferon alfa-2b treatment for further evaluation of hepatotoxicity and major cardiovascular adverse events.
15. Data analysis plan
Please provide a brief summary of the analysis method
Absolute and relative frequencies of patients with treatment emergent adverse events, count and incidence rate (including two-sided 95% CIs) of events overall and by MedDRA primary System Organ Class and Preferred Term will be calculated.