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Status: Finalised First registered on: 10/02/2016

Last updated on: 16/06/2017

1. Study identification

EU PAS Register NumberEUPAS12387

Official titleMulticenter prospective open-label non-interventional uncontrolled Post-Authorisation Safety Study (PASS) to evaluate the safety profile of Polyoxidonium in daily practice

Study title acronym

Study typeObservational study

Brief description of the studyThis PASS aims to collect data on the safety of Polyoxidonium in patients, for whom Polyoxidonium is prescribed in routine practice in accordance with the terms of the marketing authorisation (MA). This is a local, multicenter, prospective, open-label, non-interventional, uncontrolled study. The decision to prescribe Polyoxidonium will be independent of the decision to enrol the subject into the study. Each subject will be observed for the duration of one cycle of Polyoxidonium treatment. In accordance with the SmPC, the treatment course consists of 5-10 injections depending on the disease. Thus, study duration for individual subject will take 7-23 days. There will be 5-10 study visits coinciding with routine visits to receive Polyoxidonium injections at the health care centre. Actual assessments undertaken at each visit will be determined by clinical practice. Subjects will not be administered any investigational medicinal products and/or medical procedures neither undergo any laboratory evaluations, diagnostic or monitoring procedures specifically for the purposes of this study.

Was this study requested by a regulator?No

Is the study required by a Risk Management Plan (RMP)? Not applicable

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 nameMEDIGROUP s.r.o.

Centre locationMlynské Nivy 54 821 05 Bratislava, Slovakia

Details of (Primary) lead investigator

Title Professor

Last name Pruzinec

First name Peter

Is this study being carried out with the collaboration of a research network?

Other centres where this study is being conducted

Multiple centres

In total how many centres are involved in this Study?15

Countries in which this study is being conducted

National study

Slovakia

3. Study timelines: initial administrative steps, progress reports and final report

PlannedActual

Date when funding contract was signed28/07/201528/07/2015

Start date of data collection16/05/201620/06/2016

Start date of data analysis02/01/201710/02/2017

Date of interim report, if expected

Date of final study report29/03/201708/05/2017

4. Sources of funding

Please provide estimates of the percentage of funding by source for this study

Names(s)Approximate % funding

Pharmaceutical companiesNPO PETROVAXPHARM100

Charities

Government body

Research councils

EU funding scheme

5. Contact details for enquiries

Scientific Enquiries

Title Professor

Last name Pruzinec

First name Peter

Address line 1Tabaková 1

Address line 2

Address line 3

CityBratislava

Postcode821 07

CountrySlovakia

Phone number (incl. country code)421905658777

Alternative phone number

Fax number (incl. country code)

Email address peter@bonusccs.sk

Public Enquiries

Title Ms

Last name Tutlyte

First name Indre

Address line 1Savanoriu 109

Address line 2

Address line 3

CityKaunas

Postcode44208

CountryLithuania

Phone number (incl. country code)37037366307

Alternative phone number

Fax number (incl. country code)

Email address indre.tutlyte@biomapas.eu

11. Scope of the study

What is the scope of the study?

This PASS aims to collect data on the safety of Polyoxidonium in patients, for whom Polyoxidonium is prescribed in routine practice in accordance with the terms of the marketing authorisation (MA).

Primary scope : This PASS aims to collect data on the safety of Polyoxidonium in patients, for whom Polyoxidonium is prescribed in routine practice in accordance with the terms of the marketing authorisation (MA).

12. Main objective(s)

What is the main objective of the study?

The primary objective is: (a) to assess the frequency of adverse drug reactions (b) to estimate the proportion of subjects, who develop signs and symptoms of adverse renal effects associated with the use of Polyoxidonium.

Are there primary outcomes?Yes

The primary outcome is a proportion of subjects with adverse renal effects.

Are there secondary outcomes?Yes

Proportion of subjects who experienced any AE, ADR, SAE, serious ADRs. Number of subjects who discontinued the study; Global assessment of tolerability by investigators and by subjects; Global assessment of improvement by subjects and investigators; Mean duration of primary treatment of disease; Days with fever >38°C/days with symptoms; Total and differential WBC count in blood and urine.

13. Study design

What is the design of the study?

This PASS is a local, multicentre, prospective, open-label, non-interventional, uncontrolled study.

14. Follow-up of patients

Will patients be followed up?Yes

Please describe duration of follow up

Each subject will be observed for the duration of one cycle of Polyoxidonium treatment. In accordance with the SmPC, the treatment course consists of 5-10 injections depending on the disease. Thus, study duration for individual subject will take 7-23 days. There will be 5-10 study visits coinciding with routine visits to receive Polyoxidonium injections at the health care centre.

15. Data analysis plan

Please provide a brief summary of the analysis method

This study focuses primarily on the safety profile of Polyoxidonium and no statistical hypothesis testing is intended. Statistical analysis will be merely descriptive in nature. Categorical data will be summarized in frequency tables. For continuous data, descriptive statistics will be calculated. For statistical comparison of categorical data, the chi-square test will be used. Parametric Student t-test or nonparametric Wilcoxon rank sum test will be used for comparison of continuous data between two independent samples. Parametric paired Student t-test or nonparametric Wilcoxon signed-rank test will be used for comparison of continuous data between two dependent samples. Proportions of two dependent samples will be compared using McNemar's test. Statistical tests will be interpreted at the 5% significance level (two-sided). Stratified analysis by indication will be performed to investigate safety profile and clinical benefit in different subjects subgroups.