Status: Finalised First registered on: 02/04/2015
Last updated on: 01/12/2021
1. Study identification
EU PAS Register NumberEUPAS9194
Official titleA Prospective Safety Sub-Registry to Assess Anaphylaxis and Severe Allergic Reactions, and Severe Cutaneous and Systemic Immune-Mediated Reactions with Alglucosidase Alfa Treatment
Study title acronymPompe Safety Sub-Registry
Study typeObservational study
Brief description of the studyThe objectives of this Safety Sub-Registry are to collect uniform and meaningful data on patients with Pompe disease who experience anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic immune-mediated reactions following treatment with alglucosidase alfa. This Safety Sub-Registry also will assess: • the symptoms, severity, outcome, and occurrence of those adverse events (AEs; anaphylaxis, severe allergic reactions, and signals of severe cutaneous and systemic immune mediated reactions); • the effect of antibody responses (both timing and pattern of responses) and cross reacting immunologic material (CRIM) status (in patients with age at symptom onset less than or equal to 12 months only) on the occurrence of such AEs.
Was this study requested by a regulator?Yes: United States
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 applicableLTS13930
2. Research centres and Investigator details
Coordinating study entity
Centre nameGenzyme, a Sanofi company
Centre locationCambridge, MA USA
Details of (Primary) lead investigator
Title Dr
Last name Trial Transparency Team
First name Trial Transparency Team
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

Belgium
Germany
Italy
Taiwan
United States
3. Study timelines: initial administrative steps, progress reports and final report
PlannedActual
Date when funding contract was signed08/12/201009/09/2015
Start date of data collection01/06/201120/03/2015
Start date of data analysis
Date of interim report, if expected
Date of final study report30/09/202202/11/2021
4. Sources of funding
Please provide estimates of the percentage of funding by source for this study
Names(s)Approximate % funding
Pharmaceutical companiesGenzyme, a Sanofi company100
Charities
Government body
Research councils
EU funding scheme
5. Contact details for enquiries
Scientific Enquiries
Title Dr
Last name Trial Transparency Team
First name Trial Transparency Team
Address line 11 Ave Pierre Brossolette
Address line 2 
Address line 3 
CityChilly-Mazarin cedex 
Postcode91385 
CountryFrance
Phone number (incl. country code)33160497777 
Alternative phone number 
Fax number (incl. country code) 
Email address Contact-US@sanofi.com
Public Enquiries
Title Dr 
Last name Trial Transparency Team 
First name Trial Transparency Team 
Address line 11 Ave Pierre Brossolette 
Address line 2 
Address line 3 
CityChilly-Mazarin cedex 
Postcode91385 
CountryFrance 
Phone number (incl. country code)33160497777 
Alternative phone number 
Fax number (incl. country code) 
Email address Contact-US@sanofi.com 
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