Status: Finalised First registered on: 15/07/2015
Last updated on: 24/05/2017
1. Study identification
EU PAS Register NumberEUPAS10301
Official titleMyozyme (alglucosidase alfa) Safety Information Packet effectiveness evaluation: a health care professional survey
Study title acronym
Study typeObservational study
Brief description of the studyThe Pharmacovigilance Risk Assessment Committee (PRAC) requested Genzyme, a Sanofi Company to further update the Myozyme Safety Information Packet (SIP) and to propose a study to evaluate the effectiveness of the updated version of the SIP. To comply with PRAC requirements, a health care professional survey that assesses the effectiveness of the updated SIP is proposed. The survey consists of two waves (wave 1 and wave 2) which are to be carried out pre- and post-implementation of the updated SIP, at least 18 months apart. The survey is intended to assess whether implementation of the updated SIP has led to increased awareness, usage, usefulness, readability, understanding, clinical knowledge and behavioural implementation of key safety messages compared with the previous version of the SIP. Distribution and opinion of HCPs about the SIP will also be evaluated.
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 to which the investigator belongsOXON
Department/Research group
Organisation/affiliationOxon Epidemiology Ltd.
Website/Homepagewww.oxonepi.com
Details of (Primary) lead investigator
Title Dr
Last name Qizilbash MBChB MRCP(UK) BSc MSc DPhil(Oxon.)
First name Nawab
Is this study being carried out with the collaboration of a research network?
No
Other centres where this study is being conducted
Not applicable (single centre)

Countries in which this study is being conducted
International study

France
Germany
Italy
Poland
Spain
United Kingdom
3. Study timelines: initial administrative steps, progress reports and final report
PlannedActual
Date when funding contract was signed09/12/201409/12/2014
Start date of data collection01/05/201517/06/2015
Start date of data analysis
Date of interim report, if expected20/09/2015
Date of final study report31/03/201731/03/2017
4. Sources of funding
Please provide estimates of the percentage of funding by source for this study
Names(s)Approximate % funding
Pharmaceutical companiesGenzyme Europe B.V., The Netherlands.100
Charities
Government body
Research councils
EU funding scheme
5. Contact details for enquiries
Scientific Enquiries
Title Dr
Last name Qizilbash MBChB MRCP(UK) BSc MSc DPhil(Oxon.)
First name Nawab
Address line 1The Euston Office
Address line 21 Euston Square 
Address line 340 Melton Street 
CityLondon 
PostcodeNW1 2FD 
CountryUnited Kingdom
Phone number (incl. country code)34-629846059 
Alternative phone number 
Fax number (incl. country code) 
Public Enquiries
Title Dr 
Last name Qizilbash MBChB MRCP(UK) BSc MSc DPhil(Oxon.) 
First name Nawab 
Address line 1The Euston Office 
Address line 21 Euston Square 
Address line 340 Melton Street 
CityLondon 
PostcodeNW1 2FD 
CountryUnited Kingdom 
Phone number (incl. country code)34-629846059 
Alternative phone number 
Fax number (incl. country code) 
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